patient journey clinical trial

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Understanding the patient journey

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The patient journey — from expressing an interest to trial participation

Learn about what patients may experience while taking part in a clinical trial.

Prescreening  Before a person can take part in a clinical trial, they are usually prescreened. The prescreening process enables the clinical investigator to check a patient’s eligibility to participate in the trial as described in the inclusion and exclusion criteria of the protocol. Thus, the prescreening stage facilitates an effective selection of the trial participants before any trial-related screening procedure is performed, and consequently reduces the number of screen failures.

The most frequent methods of prescreening are over the telephone and online, but it can be conducted in person if potential participants find out about the trial during a medical appointment. The prescreening interview includes general questions about patients’ interest in joining the trial, their willingness and capability to attend visits at the trial site, and the collection of information that may help to preliminarily assess trial eligibility, if necessary (e.g. patient height and weight).

Informed consent Once a potential trial participant is identified, and they are willing to consider participating, the next step is to obtain their informed consent. To help potential participants make an informed decision about their participation in the trial, they are provided with a Participant Information Sheet (PIS). This document has ethical approval and contains details about the trial rationale and aim, the intervention being tested, potential risks and benefits, and trial-related procedures. The PIS covers the information that needs to be explained and discussed with potential participants and their legal representatives or parents, if applicable, before signing the Informed Consent Form (ICF). No trial-related procedure can be performed prior to obtaining the patient’s informed consent.

Screening  After the ICF is signed, the trial-related screening and any other procedure mentioned in the protocol can be performed. The information collected and procedures undertaken at screening vary from one trial to another, and may include: a review of the participant’s medical history, physical exam, measurement of vital signs, and specific laboratory analyses. When the screening results are available, potential participants will be informed if they are eligible to continue in the trial.

Participant allocation to trial groups Participants may be assigned to trial groups ( controlled trials ), which most often include the treatment group, in which participants receive the trial intervention, and the control group, in which participants receive the standard-of-care treatment, placebo, or no treatment. Frequently, participants are assigned at random to trial groups ( randomized trials ).

In most trials, participants and/or research staff are not informed of the participants’ assigned trial groups, to prevent investigator bias when reporting participants’ results ( blinding ). The information may be disclosed to trial participants and research staff when the trial has been completed or anytime during the trial for safety reasons. In an open-label trial, the participants and research staff know what treatment the participants receive.

Does it cost money to participate in clinical trials?

In most clinical trials, participants do not have to pay for any drug tested in the trial or any procedure involved. Throughout the trial, all the necessary assessments are provided at no cost.

Some trials may require the insurance company to cover the costs related to drugs, devices, or procedures. Information related to trial reimbursement is detailed in the Participant Information Sheet.

Who are the people patients might meet?

Conducting a clinical trial requires a multidisciplinary team typically consisting of physicians, research nurses, and other healthcare professionals. Collectively, their role is to perform any trial-related activities, such as continuously monitoring the participant’s health and condition throughout the trial, administering the trial drug as stipulated in the protocol, performing any trial-related procedures, reporting any side effects and outcomes, and maintaining contact with the Sponsor. During their participation in the trial, patient volunteers would mostly interact with the Principal Investigator, Sub-Investigator, and Clinical Research Coordinator.

• Principal Investigator : responsible for running the trial in accordance with the trial protocol, keeping accurate records on implementation of the trial-related procedures and reporting any side effects of the tested intervention.
• Sub-Investigator(s) : medical doctors that work in a trial under the principal investigator’s supervision.
• Clinical Research Coordinator (CRC) : involved in monitoring and evaluating trial-related activities and trial set-up at site level.
• Clinical Research Associate (CRA) : involved in running a trial at the site level to help ensure the rights and safety of participants, and that the trial follows the protocol provisions closely. (Often, the Principal Investigator is the primary point of contact for CRAs).
• Regulatory Coordinator : reviews trial compliance with regulations and ethical standards.
• Data Coordinator : works with trial team to collect and collate all relevant clinical research data according to the protocol.
• Research Nurse(s) : offers support for trial management and may be involved in the screening of potential participants, consenting, randomization, data collection, reporting of adverse events, and preparing trial documents and reports.
• Research Pharmacist : works to support the provision of trial medication at certain visits and documents the returns of trial medication.
• Trial Assistant : provides support to the Principal Investigator for trial monitoring and reporting, data management, and audits.

How much time will it take?

The time commitment for participants in a clinical trial varies according to the trial phase and protocol. Usually, trial participants are invited to come to the trial site for a screening visit, followed by several evaluation visits. Once the testing of the intervention reaches its end, a follow-up visit may be required.

Some clinical trials require just a follow-up visit or a follow-up call, while others have longer follow-up periods with several visits at certain intervals. Details like the number of visits, their duration, and their purpose are explained in the Participant Information Sheet.

Other activities may include filling in questionnaires, keeping a research diary, and attending additional visits due to experiencing side effects.

How will patients get to the trial site?

Participation in clinical trials does not normally involve extensive traveling. When it does, trial-related travel costs for participants, legal representatives, and caregivers may be covered by the Sponsor. Supporting documents, e.g. receipts and tickets, may need to be provided. Details and conditions for the reimbursement are explained in the Participant Information Sheet.

What happens to patients after a clinical trial ends?

Usually, a participant leaves the trial once their participation has ended. The trial itself may continue for longer. Patients may return to the care of their regular doctor and continue to receive the standard medical care for their condition.

What happens to the data after a clinical trial ends?

At the end of a trial, the data collected throughout is collated and analyzed. The analysis aims to answer key questions related to the scope of the trial. The main findings may discuss drug safety, side effects, and efficacy of the tested drug, device, or procedure in comparison with the current treatment or placebo. The final results should be made available to everyone who took part in the trial by providing a lay language summary. The results are usually published in medical journals, presented at conferences, and/or issued as a press release by the Sponsor.

If a clinical trial phase has a positive outcome, the Sponsor may recommend progressing to the next phase, following consultations with regulatory bodies. When the results of several trials suggest that a potential new drug, device, or procedure works and has an acceptable safety profile, the Sponsor usually applies for marketing authorization (product license) to the competent regulatory agency (e.g. the FDA in the United States, EMA in Europe). Cost-effectiveness analysis is considered when deciding whether to grant marketing authorization. Following marketing authorization, national healthcare authorities run a cost-effectiveness analysis, at the country level, to recommend market access (product reimbursement). The cost-effectiveness analysis usually compares the new drug, device, or procedure with the current standard of care in terms of costs and benefits. It also considers physicians’ and patients’ preferences.

• Deng CQ. Pre-screening procedures in clinical trials. On Biostatistics and Clinical Trials blog. Published 15 July 2014.
• European Medicines Agency. Authorisation of medicines.
• European Medicines Agency. The European regulatory system for medicines: a consistent approach to medicines regulation across the European Union. EMA/716925/2016.
• Moreira Lima Gamboa M, Tesainer Brunetto A, Ferreira Dos Santos ME, et al. The pharmacists' role in clinical research.   Farm Hosp . 2011;35:341-342.
• Motor Neurone Disease Association. Information sheet D: clinical trials.
• National Cancer Institute. Clinical trials information. Research team members.
• National Health Service, NHS Choices. How do I take part in a clinical trial?
• National Institute for Health Research, UK Clinical Trials Gateway. What happens during and after a clinical trial?
• Pick A, Liu A, Drew VL, et al. The role of the research nurse.   Nurs Times . Epub 2011 April 26.
• Rico-Villademoros F, Hernando T, Sanz JL, et al. The role of the clinical research coordinator – data manager – in oncology clinical trials.   BMC Med Res Methodol . 2004;4:6.
• Scottish Parliament Information Centre (SPICe).  The licensing of new medicines in the UK and approving their use in NHS Scotland.  SPICe Briefing (SB) 12-51. 23 August 2012.
• US Food and Drug Administration. Development & approval process (drugs).
• US Food and Drug Administration. The drug development process. Step 3: clinical research.

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The Patient Journey in Clinical Research: Insights & Observations to Scale Engagement

• Posted on April 25, 2022
• By ciscrp_admin
• In News , Press & Media

Authored by Lindsey Elliott, Marketing & Communications Manager and Joan Chambers, Senior Director of Marketing & Outreach

Understanding the patient journey in clinical research is critical for increasing participation and diversity in clinical trials. A survey on patient perspectives on clinical trial participation was conducted by Rare Patient Voice and Informa Pharma Intelligence. Panelists discussed the findings in a February 2022 webinar, which can be accessed here .

Co-moderator Mike Wenger, Global Head of Patient Engagement at Informa Pharma Intelligence, is a software developer with over a decade of experience in clinical trials. He is also a patient, having been diagnosed with a brain tumor when he was in his 20s. “I had a very fortunate prognosis, but it is something that has opened my eyes to the importance of clinical research and moving science forward,” says Mike.

Co-moderator Wes Michael is a longtime market researcher who founded Rare Patient Voice and serves as President. “I wanted to help patients take part in surveys as well as clinical trials,” says Wes. “This survey let patients share their experiences, as well as their desires when it comes to clinical research.”

Respondents who had taken part in a clinical study presented a variety of perspectives in the survey. About half of those who took part in clinical trials were told about them by their doctors, while the other half learned about them from patient advocacy groups.

Panelist Steve Jones, President of EU-IPFF (The European Idiopathic Pulmonary Fibrosis and Related Disorders Federation), a pulmonary fibrosis patient and patient advocate, says, “The main source of information is undoubtedly physicians.” However, widespread education in the medical community is needed to assist in increasing participation in clinical trials. The process of finding a trial must be simplified.”

When it comes to physicians sharing information about clinical trials, panelist Ashley Smock, a woman living with ovarian cancer, says, “I think it comes down to patient-physician relationships. Had my own physician brought up a clinical trial as an option, I probably would have been more interested.” When Ashley sought a second opinion about her cancer diagnosis, that doctor mentioned clinical trials as an option. Ashley was less open to the information the physician shared, explaining, “He didn’t have the best bedside manner. I did not feel valued as a patient during our conversation, so I didn’t follow up. There was no relationship and there wasn’t a lot about the visit that was positive.”

There is a need to raise awareness of clinical trials, the value of participation, and provide an easier way to access clinical trials, as mentioned in the webinar discussion. Steve is part of a collaborative effort where he lives in the United Kingdom to develop a European clinical research database that is easier to use than clinicaltrials.gov . “The website [clinicaltrials.gov] is almost impossible to use for ordinary people,” says Steve. “Information needs to be made simple to understand and in plain language wherever possible, to make it easier for patients to access.” Steve cited CISCRP’s Search Clinical Trials database as a streamlined option to review.

“There have been many recent initiatives to improve the patient experience with clinicaltrials.gov,” says Mike, and despite complexities, “…it is still an important entry point for patients to learn about clinical trials.” 

Other initiatives to help with the clinical trial participant’s experience include the use of technology that offers flexibility and convenience. “I’m a big proponent of decentralized clinical trials, when they’re possible,” says Wes.

The majority of clinical trial participants questioned (80%) said their recruitment process was “great or good.” Within a week of starting the process, two-thirds said they were enrolled in a clinical trial.   Participants value communications when they are adequately explained throughout the life cycle of the clinical trial and when they are timely and relevant at each stage of the process. Conversations with the physician investigator and clinical research site staff can take place via phone calls, emails, text messages, telehealth appointments, and in-person visits.

Patients also want to know the results of clinical trials. Sixty-four percent of those who had participated in clinical trials said it was important to hear results, while 88% of those who had not participated in a clinical trial said it was important. “Simple charts and summaries of the salient points of the trial would be very helpful going in, to know that at the end of the day, I will have that information and be able to access the results,” says panelist Sarah Nechama Frisch, who lives with narcolepsy and cataplexy. “Email is probably the most practical way to disseminate information for most people.”

“The more everyone in the United States is exposed to clinical trials, particularly with what is in the headlines currently about what clinical research is, why we do it, and how it’s run, that awareness will go a long way,” notes Mike.

“As patients, we want medications to be effective, approved, and on the market, because our population has a lot of living to do and we want to be part of the development of things that are going to keep us here for a long time,” says Ashley. She advises biopharma companies conducting clinical trials to “ask us for our feedback about clinical research, because we want this to move forward.”

You can learn more by accessing the webinar recording here .

View additional companion content:

• Infographic
• Q&A Follow-up

To search for medical conditions in a specific location, visit our  Search Clinical Trials  page.

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About Informa Pharma Intelligence

Informa Pharma Intelligence powers a full suite of analysis products—Datamonitor Healthcare™, Sitetrove™, Trialtrove™, Pharmaprojects™, Medtrack™, Biomedtracker™, Scrip™, Pink Sheet™, and In Vivo™—to deliver the data needed by the pharmaceutical and biomedical industry to make decisions and create real-world opportunities for growth. For more information visit pharmaintelligence.informa.com.

About Rare Patient Voice

Rare Patient Voice, LLC provides patients and caregivers an opportunity to participate in all types of research including market research, health economics outcomes and real-world evidence, user experience/human factors studies, and clinical trials. The RPV panel has over 100,000 patients and caregivers across more than 700 diseases, both rare and non-rare, in the United States, Canada, the United Kingdom, Germany, France, Italy, Spain, Australia, and New Zealand. Visit us at www.rarepatientvoice.com.

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The (True) Patient Journey

How Anticipating Patient Needs Facilitates Trial Participation

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From the outset, clinical trial patients must grapple with logistical, physical, and mental challenges: hurdles which begin before they walk through the clinic door for their first visit.  

By truly understanding this journey, sites, sponsors, and CROs can provide improved support and reduce patient burden. This eases the patient's journey, enhances engagement, elevates the trial experience, and significantly reduces drop-out rates.  

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Home > Blog > Inside Clinical Trials > Clinical Trial Participation: The Patient’s Journey

Clinical Trial Participation: The Patient’s Journey

Published on January 31, 2023

Last Modified on September 27, 2023

• Clinicalresearch.com: The Patient Journey in Clinical Trials
• Great Western Hospitals: The Patient Journey During Clinical Trials
• Clinical Leader: A Patient’s Journey In Immunotherapy Trials
• Metastasis Research Society (Video): MBC Webinar Including Clinical Trial Experiences From the Patient Perspective

Tags: Clinical Trial Navigation Living With MBC Navigating Cancer Care Patient Stories Inside Clinical Trials

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Beyond Science: The Humanization of Clinical Trials through Patient Journey Mapping

We’re witnessing a paradigm shift. The trial landscape is changing rapidly, and a significant part of this transformation stems from an increased focus on the patient experience. A prime example of this shift is the innovative approach of Patient Journey Mapping, a tool that helps researchers to understand the patient’s path through the healthcare system. The recent Decentralized Trials Research Alliance (DTRA) journey maps have marked an exciting development in this domain. They perfectly illustrate how harmonizing the patient journey with the clinical trial protocol can exponentially enhance patient engagement, retention, and the overall trial experience.

Unveiling Patient Journey Mapping

Patient Journey Mapping is a strategic approach to optimize clinical trials. It’s a comprehensive visual representation of a patient’s experience – from their initial symptoms or diagnosis, through treatment, and onto post-therapy follow-up. It captures the thoughts, emotions, and challenges that patients face at every stage.

This holistic model allows researchers to identify potential pain points, unmet needs, and areas for improvement. It’s about providing a patient-centric experience rather than a researcher-centric one. In doing so, it’s transforming clinical trials from a purely scientific endeavor to an empathetic, patient-focused process.

The DTRA Journey Maps

The recently unveiled DTRA journey maps are a beacon in the realm of Patient Journey Mapping. They employ a detailed, empathetic approach to delineate the patient’s journey, resulting in highly personalized, engaging, and streamlined clinical trials. The DTRA maps not only outline the clinical stages but also capture the psychological and emotional aspects of the patient experience.

These maps have been instrumental in redefining the understanding of a patient’s perspective, hence making it easier for researchers to create a trial design that resonates with the patient’s expectations and needs. This alignment fosters a smoother experience, enhancing patient engagement and retention in trials.

The Impact on Engagement and Retention

Patient Journey Mapping can profoundly affect the levels of patient engagement and retention in clinical trials. By empathetically charting the patient’s journey, researchers can better understand the obstacles faced by participants and proactively work on eliminating them. This not only enhances patient engagement but also makes them feel valued and understood, thereby improving retention rates.

Datacubed Health aligns content and engagement with the patient journey based on each individual protocol. When patients find the process simple and easy to navigate, they are more likely to stay engaged and complete the trial, directly improving retention rates.

Enhancing the Overall Experience

Patient Journey Mapping is about the democratization of the clinical trial process. It empowers the patient, gives them a voice, and makes them active participants rather than passive subjects. It is a shift from a ‘doing to’ to a ‘working with’ mentality, which significantly enhances the patient’s overall experience.

In the age of patient-centricity, journey mapping is critical. At Datacubed Health, we’ve been aligning content and engagement strategies to the patient journey since our inception in 2016. If you’re interested in how we do this and how we’re different, request your complimentary patient journey map today. 

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Clinical Trials: An End to End Patient Journey Overview

Nov 6, 2022

Clinical trials are essential for the development of new medical treatments and to establish their safety and efficacy. They play a crucial role in advancing healthcare and improving patient outcomes. But what exactly is involved in a clinical trial from a patient's perspective? In this article, we will provide an overview of the end-to-end patient journey in a clinical trial. 

What are Clinical Trials?

Clinical trials are research studies that aim to evaluate the safety and efficacy of new medical treatments. These trials are conducted to ensure that new treatments meet the necessary standards of quality, safety, and efficacy before they are made available to the public. 

Why Participate in a Clinical Trial?

There are several reasons why a patient may choose to participate in a clinical trial. For some, it may offer access to new and potentially life-saving treatments before they are widely available. Others may participate in clinical trials as a way to contribute to scientific research and help advance healthcare. Additionally, participating in a clinical trial can provide patients with access to specialized medical care and close monitoring of their health, which can be beneficial in certain conditions. 

The Clinical Trial Process

The clinical trial process can be divided into several stages, each with its own set of objectives and requirements. 

Screening and Enrollment

The first stage of the clinical trial process is screening and enrollment. During this stage, potential participants are screened to ensure that they meet the eligibility criteria for the trial. This typically involves a review of their medical history and a physical examination. If a patient is eligible to participate, they will be asked to sign a consent form indicating their willingness to participate. 

Treatment and Monitoring

Once a patient has been enrolled in a clinical trial, they will receive the treatment being studied. This may involve taking medication, receiving an experimental treatment, or undergoing a medical procedure. During this stage, patients are closely monitored to ensure their safety and to track the effects of the treatment. 

Follow-Up and Data Collection

After the treatment stage, patients will continue to be monitored during a follow-up period to assess the long-term effects of the treatment. Data from the trial is also collected during this stage to evaluate the safety and efficacy of the treatment. 

Potential Risks and Benefits of Clinical Trials

While participating in a clinical trial can offer many benefits, it is important to understand that there are also potential risks involved. For example, the experimental treatment may not work or may cause side effects. It is important for patients to weigh the potential risks and benefits of participating in a clinical trial before making a decision.

Clinical trials are an important part of the medical research process and offer patients the opportunity to access new and potentially life-saving treatments. By providing an end-to-end overview of the patient journey in a clinical trial, we hope to give patients a better understanding of what is involved and to help them make informed decisions about participating in clinical trials. 

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Mapping the Patient Journey

Visualizing the decentralized clinical trials patient experience to appreciate the journey of the research participant.

Description of the Patient Journey Map

These patient journey maps are designed to enhance the clinical research experience for stakeholders such as sponsors, CROs, and sites involved in global decentralized clinical trials (DCTs). These visual representations depict the patient's journey across various research phases and indications. This offers a more comprehensive, patient-focused understanding of the decentralized process. By incorporating the patient's perspective, our maps aim to minimize barriers and patient burden. This increases the likelihood of completing studies on time and within budget, with increased patient retention and protocol compliance.

Study team members can use the foundation of these visualizations to create tailored DCT patient journeys. These are examples to help support you as you develop your trial’s ideal patient journey, using the templates provided. Keep in mind that each patient's experience is unique, as demonstrated by example journey maps from oncology and rare disease trials.

Research Methods

The journeys depicted here are a result of conversations with multiple patients representative of the disease states being depicted, as well as lessons learned from industry leaders in decentralized and hybrid clinical trial conduct.

These visualizations will make it easier to understand and contextualize the decentralized patient journey across multiple phases of research and indications of interest. In the near future, you will be able to use the work behind these outputs to map out your own DCT patient journeys.

Download the Patient Journey Map Template

Oncology, rare disease, and rsv vaccine studies.

These therapeutic areas were chosen because oncology represents the largest proportion of clinical trials being conducted, rare disease is a rapidly expanding area of development, and RSV vaccines are pushing new frontiers in preventive healthcare.

While DCT isn't right for every trial, by thoughtfully incorporating elements as appropriate, we have the power to democratize research and bring clinical trials to members of the population typically underrepresented in trials.

These journey maps—the Oncology Journey Map, the Rare Disease Journey Map, and the RSV Vaccine Journey Map—are available for download below. Review these journey maps and incorporate the lessons learned into your own patient journey-mapping initiatives.

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Patient Journey Map Feedback

Download the  full initiative charter, relevant resources.

December 10, 2020 DECENTRALIZED TRIALS & RESEARCH ALLIANCE (DTRA) LAUNCHES TO DEMOCRATIZE AND ACCELERATE CLINICAL TRIALS

December 1, 2021 One Year In, DTRA Meets Goals & Looks To The Future

April 26, 2021 Decentralized Trials & Research Alliance (DTRA) Reaches Milestone of 100 Member Organizations

February 24, 2023 Decentralized Trials & Research Alliance Release Best Practices Rubric to Provide Framework to Evaluate DCT Processes

November 10, 2022 Decentralized Trials & Research Alliance (DTRA) Shares Key Findings from Annual Meeting

August 29, 2022 Decentralized Trials & Research Alliance Releases Glossary to Improve Adoption of More Accessible Research Approaches

May 17, 2021 Decentralized Trials & Research Alliance (DTRA) Announces Priorities and Initial Initiatives

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Patient Journey Mapping: Clinical Study Participants

Written by Sabine Hutchison

May 18, 2021.

Recruiting (the right) patients, onboarding them, and keeping them on board is one of the most critical factors determining the success of a clinical trial. Let’s talk about patient journey mapping.

This is a good news, bad news situation. Let’s start with the bad news: even before the COVID-19 pandemic, this was no easy feat. But there’s also good news: there are ways to make this whole process run more smoothly. It’s called patient journey mapping.

Before we get into the nitty gritty of what that is and how to do it, let’s take a closer look at the recruitment situation as it stands – i.e., the situation you likely find yourself in now, if you are a CRO or sponsor testing new pharmaceuticals or medical devices.

Recruiting Woes

Finding, recruiting, engaging, and retaining study participants might be the hardest part of running a successful clinical trial. To complicate matters even further, you’re usually not looking for just any study participants, but for the right patients to participate in your study.

You’re not alone in this struggle. One study of 114 trials in the UK found that only 31% were able to meet their patient-recruitment goals. Another study found that a staggering 80% of clinical trials fail to recruit the number of participants needed within their timeline targets. A fact that leads to around one-third of publicly funded trials needing a timeline extension . Worse yet, another study found that 19% of clinical trials needed to be halted altogether due to participant recruitment issues.

Add to this the extra complication of recruiting during a global pandemic, where travel restrictions and extra social-distancing measures must be taken into account, and things begin to look grim.

The Consequences Of Not Finding Your Study Participants

Not being able to meet your recruitment goals within your set timelines not only leads to study delays, not to mention delays in your ability to actually treat the patients who need the drugs or devices you are testing, but also a loss in revenue. This can translate to up to $8 million in lost revenue for each day you are delayed.

Why is this happening? COVID-19 aside, there has always been tight competition for eligible participants. This is partly because the recruitment pool is made smaller by an unappealing recruitment process, one too often planned without the user experience in mind. Or at least not tailored to your different groups of participants – patients and control group participants generally join a study for different reasons, and thus need to be approached differently as well.

Yes, this means you might need to rethink the entire participant process, from first contact through to the end of the study. In fact, we urge you to do this. But you don’t have to do it alone – let’s walk through all the details, starting at the beginning.

Patient Journey Mapping

Connecting with potential participants. 

How will you reach your participants, the patients vs. control group? What motivates each to want to participate, and what publications or websites might that interest drive them to read or visit? 

As Tesheia Johnson, executive director of the Yale Center for Clinical Investigation, put it:

“It’s confirmed that there is a lack of understanding about what’s involved in clinical research and why it’s important to participate.”

How can you overcome this gap?

Next, thinking again about why potential participants would be interested in your study, you need to plan how you will address each group, separately. What message will catch their attention and engage them? What do you have that they need? Do they have a specific concern? Are they looking to make an impact? What phrasing and language will appeal to them most?

We suggest some good target group mapping to uncover each group’s “pain points,” what’s driving them, and how you can make them aware of the potential solution you are offering.

The 7 most important questions you should be asking

When analyzing your patient journey to improve your participant-recruitment process, you should start by asking yourself seven important questions:

Plan, plan, plan While you are uncovering who your potential participants are and what they want and need, also consider which suppliers you can work with to best meet those unique needs. Build those participant desires and needs into your governance and oversight model for the whole study.

Plan your timelines as realistically as possible, padding them and setting expectations and budgeting appropriate to the high level of risk and possible delays. Think through all of your risks and your back-up plans.

Don’t forget that your participants will need ongoing support throughout the trial to maintain their motivation and commitment. The more attention you can offer, the lower the drop-out rate.

And, different groups of participants will need different types of support. Think about your participants and your study and consider age, the physical demands of your study, how easy it is to access the study location, or send updates remotely. How will the needs of your study fit into your desired participants’ lives, and how can you make them fit better?

Getting Help

Yep, there are a lot of things you need to think about when doing patient journey mapping  – and keep thinking about a new throughout your entire clinical trial. You might benefit from some help from someone experienced in this kind of planning and tracking.

Specialized consultancies like Seuss+ can work with you to draw out roadmaps for each of your key stakeholders together. Let’s all sit down and put our heads together to crank this out – it will even be fun! We promise.

After that, we can support you in setting up a more participant-friendly clinical trial, built from the participants’ perspective using innovative new processes and technology.

We’ll also guide you through letting your would-be participants know that, by joining your trial, they’re in for a better experience. Where they’re treated more like clients than patients. And in offering extended support throughout the trial.

The result: dramatically lower drop-out rates, leading to more valuable and successful outcomes.

Get in touch for a free consultation to find out how we can help.

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What is the patient journey of people actively looking for clinical trials?

“85% of clinical trials fail to recruit and retain enough patients in order to meet their enrollment timeline ” – a number which we see quite often in analyses, publications, or events presentations within the clinical research industry. With one-third of study budgets going into patient recruitment and hundreds of experienced patient recruitment vendors and experts working on solving enrolment challenges on a global level, why does this trend still persist and continue to prevent clinical trials from being feasible and efficient?

We’ve been hearing a lot about patient centricity and embedding the patient’s voice into every stage of the drug development process, but what exactly does this mean?

At FindMeCure we have always been close to people living with chronic illnesses thanks to our global clinical trial portal and the awareness work which FindMeCure Foundation has been doing together with Patient Advocacy Groups worldwide. Our experience revealed how many hurdles clinical research hides mainly because the real-life patient journey has not been taken into account or partially considered in a very late stage. This prevents patients from being able to access a relevant clinical trial and at the same time delays clinical trial timelines at a huge cost.

In the last few months, we have been mapping the experience of people with chronic and life-threatening conditions that are actively looking for clinical trials on FindMeCure in order to identify the key moments to be considered by those developing new therapies. If you are interested in all the findings, take a look at the study we have produced.

From symptoms to diagnosis

Getting diagnosed seems to be one of the biggest obstacles for many people out there. When it comes to rare diseases, for instance, the length of time from symptom onset to an accurate diagnosis is around 5-7 years depending on the country. A similar issue happens to people with some autoimmune conditions.

Thanks to the wide variety of technologies and tools that have been involved in the diagnostic process in recent years and the better knowledge doctors have gained on many indications, we enjoy a better picture in terms of statistics, i.e 43.55% have been diagnosed within 6 months of symptoms onset.

But how is the patient journey during this period important for clinical research professionals such as feasibility and patient recruitment experts? 

Critical moments to understand the experience of a patient with a particular condition and potential touchpoints include:

• What is the point of entry into the healthcare system?
• Which tests and procedures are usually performed in different countries?
• Which healthcare providers (HCPs) will be involved in conducting these assessments?
• Where do undiagnosed and just diagnosed patients look for information and support?

From diagnosis through treatment pathway

The treatment journey is a crucial stage to pay attention to in order to identify whether patients would be motivated to join a clinical trial and if so – when the right moment is for them to become aware of such an opportunity.

46.7% of the respondents in our survey report they have tried different treatment options within the standard of care in their respective country. The majority of people ( 61.5% ) are not satisfied with their current treatment and this explains their motivation to look for an investigational therapy in clinical trials.

Questions to be taken into consideration at this stage are also crucial for finding out more about the experience of patients while being treated and how different it is from country to country.

• Which specialists are usually treating patients with such a condition? 
• Who may be involved in providing the study treatments?
• Which are all the available treatment options that the patient may consider? 
• Does the patient have access to reimbursed therapies?
• Who may influence the treatment decision-making?
• Where else the patient might seek information about different treatment options?
• What is the disease and treatment burden for patients during their treatment pathway? Has their quality of life improved thanks to available therapies or do they need a better alternative?

This is a key moment to also identify what types of investigators might be involved who have knowledge of and interaction with such patients.

Why clinical trials?

As we expected, most of the respondents in our survey had not been referred and advised by their doctor to look for a clinical trial option. 77.9% say they have not even discussed this step during their hospital visits. One of the reported reasons is unawareness among doctors in the country which is a critical moment in the patient journey to be considered when planning clinical trials.

Also, assessing potential reasons why patients would be interested in taking part in a clinical trial is something that requires attention. “ My treatment doesn’t work/stopped working ”, “ No available treatments ”, “ I can’t afford to pay for a treatment ” or “ Side effects of my current treatment ” are some of the reported factors that motivated our respondents to actively look for an alternative in research.

The questions to be answered and dived into include:

• Would patients in this country be motivated to take part in a clinical study?
• What obstacles might prevent them from taking part, e.g. reimbursed available treatment options in place, no clinical trial awareness, patients not aware of their diagnosis, strong patient-doctor relationship, etc.?
• Where are all of the places, venues, and times the patient might be able to learn about the trial opportunities?
• Are there any Patient Advocacy Groups to help raise awareness?

It is also important to examine the patient-doctor relationship , as well as the patient-to-patient interactions and the interactions patients have with patient support organisations in order to gain a holistic picture of what the patient feels, experiences, thinks and how he or she behaves as a result.

To conclude

Looking at a chronic disease through the eyes of a patient is one of the most insightful ways of determining the patient’s most pressing challenges and needs and how to best implement them within the drug development process. The patient journey is always unique and might be different than what doctors initially think it is or might differ due to cultural factors from country-to-country.

How a patient (prospective research subject) navigates through the healthcare system and the touchpoints where they might learn about and encounter opportunities to get involved in a given clinical trial is the key to precision when it comes to planning patient recruitment efforts.

If you are interested in learning more about all patient insights and survey findings, you can download our study in just a few clicks. 

We help CROs and sponsors to stop fixing patient recruitment challenges and start preventing them.

Country feasibility

Site feasibility, patient feasibility, privacy policy, terms of use.

The opportunity of patient-journey studies for academic clinical research in oncology

Affiliations.

• 1 Clinical Trial Unit, Istituto Nazionale Tumori IRCCS Fondazione Pascale, Napoli, Italy [email protected].
• 2 Clinical Trial Unit, Istituto Nazionale Tumori IRCCS Fondazione Pascale, Napoli, Italy.
• 3 Medical Statistics Unit, Università degli Studi della Campania Luigi Vanvitelli, Napoli, Italy.
• 4 Human Medicines, European Medicines Agency, Amsterdam, Netherlands.
• PMID: 34551954
• PMCID: PMC8461282
• DOI: 10.1136/bmjopen-2021-052871

A wave of new treatments and treatment combinations are becoming available for solid tumours. Trials performed to obtain registration establish a positive benefit-risk but unavoidably leave many questions unanswered on place-in-therapy and the relative efficacy of different treatment sequences. Such limitations create problems in terms of strength of treatment guidelines and reimbursement (in countries where a public payer exists). Data on new drugs arriving during the last 10 years for the treatment of hepatocellular carcinoma and renal cancer are reported as an example of how the fortunate condition of having new effective treatments may translate into uncertainty regarding the optimal treatment plan. We suggest that academic research should react to such limitations and propose a model of patient-journey study (PJS), where patients are followed from the initial diagnosis across subsequent lines of treatment. A PJS master protocol might include at each node of clinical decision either the possibility of choosing treatment according to guidelines (generating prospective real-world evidence) or the possibility to randomise where uncertainty exists (generating comparative effectiveness data). PJS protocols might be adaptively modified every time a new drug arrives on the market. Overall, methodologically sound analyses of PJS will produce knowledge on the efficacy and the effectiveness of different treatment pathways and might significantly optimise treatment of patients in clinical practice. PJS would represent a jump from a few snapshots (trials performed to get regulatory approval) to a full movie (evidence on the relative value of treatment pathways).

Keywords: clinical trials; statistics & research methods; therapeutics.

© Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

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Trailblazers talk, living labs, alan s. louie research director, idc health insights, subhro mallik senior vice president and head - life sciences, infosys.

Empowering the Patient Journey in Clinical Trials

With cross-industry technological advances supporting consumer engagement, there is a wealth of new data becoming available that can provide direct insights into the patient’s journey from sickness to health. Combined with the life sciences and health care industry’s embrace of digital transformation, it is becoming increasingly possible to gain an in-depth understanding of both disease progression at the individual patient level and the patient’s journey through the life sciences and health care process ecosystems. From the International Data Corporation (IDC) perspective, digital transformation is defined as the development of integrated platforms infused with data with a focus on delivering actionable insights.

Within the life sciences, two key areas highlight the role of patients and digital transformation in advancing new therapeutics to market in the industry today. These areas are:

• Participation in clinical trials.
• Contribution of real-world evidence data (e.g., electronic medical records) to support population health-based approvals and commercialization of safer and more effective drugs and treatments.

Clinical trial hurdles

One of the largest hurdles that must be overcome in the development of a new therapeutic is the recruitment and retention of patients for clinical trials. Failure to recruit enough patients to conduct a trial stops a trial in its tracks and prevents key data collection required for regulatory approval. Historically, patients have been undervalued for their role in clinical trials, disrespected for their contributions, and neglected once their contributions to the trial have ended. It is not surprising that patient recruitment for trials remains a primary hurdle, an issue further exacerbated by the continued segmentation of diseases that further shrinks the pools of patients available to participate.

Patient engagement has become a major focus for leading innovators in the life sciences industry. From a data perspective, life sciences companies are finding several opportunities to improve, including:

• More timely and effective identification and recruitment of prospective clinical trial participants.
• Improved real-time monitoring of trial participants to minimize dropout.
• More targeted and effective trial and drug messaging for maximum impact.
• Identification of biomarkers to better characterize patients likely to benefit from treatments while simultaneously avoiding patients likely to experience adverse events.
• Collecting population-based insights in support of drug dossiers supporting formulary access.

Expanding clinical trials

In the short term, digital technology has the potential to transform the clinical trials process. Digital patient registries and electronic medical records are simplifying the identification and recruitment of patients eligible for trials. Using electronic communications, smartphones, local doctors, local labs and remote sensors, virtual clinical trials are becoming reality. That inevitably expands the number of patients eligible to participate in trials. Wearables and other remote sensor technologies are expanding the potential data that can be collected from patients as part of clinical trials. And finally, available patient data can be mined in real time during the trial to more efficiently determine trial performance, enhance patient impacts and ensure regulatory compliance.

Infosys sees a greater number of organizations using digital tools to enhance the patient journey and understand treatment patterns across therapeutic areas. Real-world evidence and data are gained by analyzing line of progression, treatment and timeline to identify intervention points and enhance efficacy and patient experience.

In the long term, digital transformation promises to significantly improve the patient journey from both the patient and the trial sponsor perspectives. With some pilots in place already, patients should be able to personally monitor their own progress in trials and directly communicate with trial sponsors (and vice versa).

Advances in the internet of things and remote sensor technologies will enable new patient-generated insights above and beyond data currently being collected. And, of course, ongoing growth of knowledge of human and disease biology promises to enable and empower personalized medicine through practical understanding of diseases at a molecular level.

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The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making

Michael wang.

1 University of Colorado Anschutz Medical Campus, Aurora, CO, USA

Claude Negrier

2 National Reference Center for Haemophilia, Louis Pradel Cardiology Hospital, University of Lyon, Lyon, France

Frank Driessler

3 Bayer, Basel, Switzerland

Clifford Goodman

4 The Lewin Group, Falls Church, VA, USA

Mark W Skinner

5 Institute for Policy Advancement Ltd, Washington, DC, USA

6 McMaster University, Hamilton, ON, Canada

The anticipated emergence of hemophilia gene therapy will present people with hemophilia (PWH) and treating clinicians with increasingly complex treatment options. It will be critical that PWH and their families be empowered to participate fully in decision-making through transparent communication and the development of targeted educational resources.

The Council of Hemophilia Community (CHC) convened across a series of roundtable meetings to define the patient journey for hemophilia gene therapy, and to develop a question-and-answer style resource to guide discussion between healthcare professionals (HCPs) and their patients. Patient groups were also consulted during the development of this tool.

The CHC defined 5 key stages in the hemophilia gene therapy patient journey: pre-gene therapy (information-seeking and decision-making), treatment initiation, short- and long-term post-gene therapy follow-up. PWH will have different questions and concerns at each stage of their journey, which should be discussed with their HCP to aid decision-making. The resulting patient journey infographic and Q&A resource (see Supplementary Materials ) has been developed for HCPs and PWH to provide a novel and practical roadmap of key issues and considerations throughout all stages.

These resources support a collaborative, patient-centric, shared decision-making approach to inform treatment decision discussions between HCPs and PWH. The value of such discussions will be influenced by the language adopted; health literacy is a particularly important consideration, and these discussions should be accessible and tailored to PWH. HCPs and PWH can benefit from awareness of the common questions and uncertainties as they progress together along the patient journey. While the contents of this article are specific to hemophilia gene therapy, the concepts developed here could be adapted to aid patients in other disease states.

Introduction

Limited literature is available that combines the concepts of the patient journey in hemophilia gene therapy with corresponding life stages. As clinical developments to address the unmet need in hemophilia advance there is also a great need to address the educational and clinical decision-making challenges that will follow market authorization. Herein, we provide a new, interactive, question and answer (Q&A) PDF resource (see Supplementary Materials ) for shared decision-making along the patient journey.

The Unmet Need in Hemophilia

Hemophilia is a rare X-linked recessive genetic disease, with an estimated prevalence of 17.1 and 3.8 cases per 100,000 males worldwide for hemophilia A and B, respectively. 1 The current standard of care for severe hemophilia, in countries where it is available, is prophylaxis with factor or non-factor replacement therapy, which aims to prevent bleeds and greatly improves patient outcomes and quality of life. 2 However, people with hemophilia (PWH) remain at increased risk of bleeds (including joint and post-surgical) as well as hematomas (and ensuing synovitis and joint disease) 3 and life-threatening intracerebral hemorrhage. 4 , 5

As single-gene disorders, hemophilia A and B are promising candidates for gene therapy. Introduction of the missing F8 (hemophilia A) or F9 (hemophilia B) gene aims to deliver a potentially long-term, functionally curative treatment by restoring coagulation factor production to clinically effective levels. Simultaneous goals include ensuring no vector- or transgene-related toxicity and broad patient eligibility across age groups and in patients with inhibitors. 6–8 Gene therapy can therefore offer a new therapeutic option within this treatment landscape, to fit the needs of each individual.

Different gene therapy products are likely to meet varied unmet needs of PWH. While several hemophilia gene therapy clinical trials are in progress, no product had been approved at the time of publication. 8 Among the approaches in development, adeno-associated viral (AAV) gene transfer solutions are the most advanced in clinical trials, though these products have different constructs, serotypes, promoter/enhancer regions, and manufacturing methods that will affect patient eligibility and choice. 8 , 9

Defining the Patient Journey

The gene therapy patient journey extends throughout all life stages for PWH–from birth/diagnosis through to later adulthood–with different checkpoints corresponding to patient life stages. The constantly evolving treatment landscape involves numerous decisions that require guidance and support from healthcare professionals (HCPs). The patient journey for PWH undergoing gene therapy in particular can raise common concerns and experiences, eg, understanding benefits, risks, adverse events, impact on quality of life, and eligibility. 10 Therefore, full transparency and personalized information about the existing knowledge, therapeutic options, and unmet needs are essential to empower PWH to make informed decisions before, during, and after treatment. 7 , 10–12

Patient Centricity and Shared Decision-Making in Clinical Practice

Current gaps and limitations.

Patient engagement and personalized care are gaining attention in medication development, validation, and adoption, beyond the more traditional emphases on safety, efficacy, regulatory aspects, and communication with HCPs. PWH are typically instructed about their current and future treatments, rather than engaged in conversations and decision-making on treatment options. 13 Often, healthcare materials are written at a technical level in what may be the patient’s second language. 14 This has made it difficult for them to access, interpret, and apply health information to their decisions, contributing to inadequate health literacy. PWH and families are also exposed to a multitude of information sources via social media and traditional news channels, which often present incomplete, inaccurate, and contradictory content. 15 , 16 Furthermore, in some countries, direct-to-patient marketing may present an additional confounder to patient comprehension, particularly in vulnerable groups. 17 In addition to inadequate patient educational materials, a discordance often exists between the physician’s perception of their patients’ understanding and the patient’s actual comprehension. 14 , 18 This communication failure leads to postponement of treatment decision-making, non-adherence, and poorer health outcomes. 14

Appropriate, individualized communication is necessary to facilitate targeted treatment approaches and disease prevention. 19 This issue extends beyond patient understanding. Particularly, with emerging therapies and novel mechanisms of action, it is inappropriate to assume that HCPs have adequate understanding of gene therapy.

Proposed Solutions

A complementary approach includes patient centricity, defined as engaging with patients to achieve the best experience and outcomes for the individual and their family. 13 The process of shared decision-making can reduce the information and knowledge imbalance, and lead to more personalized treatment decisions, better medical outcomes, treatment adherence, improved quality of life, and decreased costs of treatment and absenteeism. 20–22 The introduction of a new option, such as gene therapy, provides opportunities for shared decision-making to ensure patients understand their best, individualized option. 22 This type of patient-centric decision-making is relevant to clinical practice and is particularly important for AAV gene therapy due to current limitations on re-treatment via that modality. Importantly, shared decision-making is distinct from informed consent, with a primary difference being the timing and extent of patient involvement. 23 Shared decision-making is more than a one-size-fits-all description of the risks and benefits associated with each treatment option in order to meet a legal requirement; patients, and family members where appropriate, should be actively involved in an individually tailored, two-way discussion with their care provider, with patient preferences and concerns at the forefront. 23

Appropriate Language in Patient Communication

A key element in a patient-centric approach is dialogue, which must be accessible to PWH and their families according to widely diverse levels of health and medical comprehension, as well as personal circumstances. 13 , 18

PWH who are diagnosed and treated, and families, tend to be well informed about their condition and are committed to its management, including keeping abreast of treatment options. 24 However, the evolving treatment landscape poses an array of questions, so it is imperative that PWH have access to health information that they can easily find, understand, and use to inform their decisions.

General recommendations to improve physician–patient communication include: avoiding assumptions of low or high patient health literacy, avoiding jargon and unnecessary detail, implementing the “teach-back” method (reiterating in your own words what was learned) to ensure full patient understanding, and preparing written materials, with only key points and graphics or other visuals where relevant. 18 A recent review identified additional variables relevant to shared decision-making in hemophilia treatment, including bleeding phenotype, musculoskeletal status, treatment adherence, venous access, and lifestyle. 3

When considering health literacy, it is also important to understand the issues facing PWH enrolling in clinical trials, and the clinical and technical terminologies involved. Physicians and the surrounding care team can play an important role in ensuring that patients achieve clinical trial literacy and are fully informed when making decisions about participation. 25 This is also necessary when communicating with patients who have already started their gene therapy journey in clinical practice, 11 who may still have outstanding questions or concerns.

The Q&A resource presented here will help to tailor communications and support a patient-centric shared decision-making process.

The Council of the Hemophilia Community (CHC) convened with the aim of filling an information gap through the development of a resource to support discussions between HCPs and PWH about gene therapy. Here, we summarize the results of focus group discussions conducted by the CHC, which aimed to define the patient journey in hemophilia gene therapy within the different life stages of PWH and create an accompanying Q&A resource for HCPs and PWH. The intention is to support shared decision-making in real-world practice by giving HCPs insight into likely patient questions regarding gene therapy and clinical trial participation.

Resource Development Strategy

The resources described here are based on expert discussions involving HCPs and patients. The CHC was established in 2019 as a multidisciplinary expert group, formed of independent advisors, HCPs, industry leads, and patient representatives with experience in medicine, research, advocacy, patient support, health economics, data, and healthcare access. The CHC collaborate on behalf of PWH to provide guidance for: 1) overcoming clinical and access challenges in the hemophilia environment; 2) perceived gaps in programs and initiatives; and 3) developing initiatives based on emerging insights.

Over the course of 3 roundtable meetings held between November 2020 and May 2021, the CHC first conducted a process of insight identification to understand the elements of the specific patient journey that could best guide the hemophilia community and facilitate treatment decision-making. The life cycle of PWH was aligned to patient journey stages, based on exchanges of opinion within the interdisciplinary working group. The CHC then identified and refined key questions based on those defined in an early patient questionnaire and by the National Hemophilia Foundation (NHF). 26 The selection of these source materials was based on a consensus of expert opinion; no formal literature search was conducted.

Patient questions were then selected and assigned to the patient journey stages and refined by the CHC members during iterative CHC expert workstream discussions using a focus group format. Answers to the questions were refined separately by the authors following multiple rounds of review. The target audience for the developed questions and answers were hematologists and their patients.

Collaboration with Patients

This collaborative development and feedback process engaged all members of the CHC, patient groups, patient representatives, and nurses. In addition, both resources were reviewed by groups of European and US-based patients, caregivers, and parents of PWH (Bayer Global and European Patient Councils for Hemophilia) and the Bayer Hemophilia Employee Council, to seek their insight on the relevance and accessibility of the text, as well as their opinions on the need for any additional questions and answers. All members of the Bayer Global and European Patient Councils groups are PWH or a caregiver of a person with hemophilia. Members of the Bayer Hemophilia Council include Bayer employees with hemophilia. The resultant patient journey roadmap and Q&A resource were shared with the entire CHC for feedback. The authors met regularly to discuss this feedback, and to decide on the content and placement of key questions along the patient journey.

The Patient Journey: Key Stages and Aims

As an initial step in developing the Q&A resource, the CHC undertook defining the patient journey through gene therapy for hemophilia. The CHC has categorized 5 pillars of the patient decision-making journey in the current treatment landscape:

• Pre-gene therapy information seeking
• Pre-gene therapy decision-making
• Treatment initiation
• Short-term post-gene therapy follow-up (≤1 year)
• Long-term post-gene therapy follow-up (>1 year)

The patient journey as mapped onto these 5 pillars is shown in Figure 1 .

The patient decision-making journey.

Key Themes of the Q&A Resource

The Q&A development process revealed several areas contributing to patient decision-making in hemophilia gene therapy. First, expectations of PWH need to be managed, as the potential benefits and risks of gene therapy are likely to vary among PWH. In addition, the need for a commitment to participating in a long-term registry 21 , 27 should be communicated before gene therapy is initiated. Further explanations of eligibility and effects should be clarified, notably that any gene expression occurs only in somatic cells (not germline cells), hence any effect is not conveyed to future generations. 21 The questions should also cover the current unknowns of treatment, including long-term durability, whether the achieved level of FVIII/FIX is sufficient to effectively prevent bleeds, and treatment options in the event that gene therapy does not achieve its therapeutic goals.

Summary of the Q&A Resource

The CHC agreed that questions from PWH concerning gene therapy tend to fall under the following broad categories: treatment regimen/adherence requirements, treatment predictability and variability, treatment durability, and the risk:benefit profile. The full, collated list of questions and answers is presented in the Supplementary Materials . The resource is organized from initial information-seeking to decision-making, treatment initiation and trial information, and short- and long-term follow-up. The reading level of the document is a grade 10–11 (high school) level, based on a Flesch-Kincaid readability test. It is fully referenced, as appropriate, for further context and can serve as a practical tool for the treating physician in shared decision-making. Information to facilitate discussion regarding participation in clinical trials is also included, from eligibility through follow-up. Some of the questions deal with complex queries, such as explaining what gene therapy is and how it works in the body, as well as efficacy and safety.

Additional online resources to support patients considering gene therapy are summarized at the end of the Supplementary Materials .

Hemophilia Gene Therapy Awareness

The CHC-defined patient journey and Q&A resource reported here are designed to help to inform HCPs regarding the questions and considerations likely to be encountered by PWH at different life stages, and when discussing gene therapy options. Each patient’s journey will encounter the emergence of new therapeutic options, growing evidence and evolving individual preferences, goals, aspirations, and risk tolerance.

By identifying and providing key information to aid these discussions on gene therapy, this tool will help to address knowledge gaps of HCPs and PWH. A recent global survey sent to patient organizations reported that although most (68%) PWH express that they have a basic understanding of gene therapy, only a small proportion (6%) consider themselves to have an advanced understanding. 28 A similar pattern was observed for HCPs, with 44% recording a basic or intermediate understanding, and only 12% reporting an advanced understanding. 28 A different international survey of physicians and scientists echoed these results, with 40% of physicians directly in hemophilia care claiming limited ability to discuss gene therapy with their patients. 29 As gene therapy emerges, it will be critical to meet the knowledge gaps of HCPs and PWH. Educating physicians, nurses, and the surrounding care teams who care for PWH will better enable them to meet the needs of their patients through constructive and informative discussions. 29 Although the majority of PWH report positive opinions of gene therapy, including with respect to such considerations as annual bleeding rates, factor levels, long-term impacts, use of factor replacement, and treatment costs, 30–32 many believe that they will not benefit from new products (eg, gene therapy and new factor/non-factor replacement therapies). 31 , 32 Therefore, HCPs will need to educate PWH about all available treatment options as they engage them in shared decision-making. 31

HCP-Patient Communication on Gene Therapy Advances

The resources will be shareable during in-person or virtual consultations and available for reference afterwards. In order to maximize the impact of this tool, HCPs should become familiar with appropriate language and educational materials to answer these questions. Results from a qualitative study across a variety of stakeholders suggest that consistency in the language and terminology used when discussing hemophilia can reduce miscommunication and facilitate conversations, promoting informed decision-making. In particular, the need for appropriate terminology to describe gene therapy has been previously highlighted and that it is preferable to discuss “what gene therapy is,” rather than what current treatments “are not.” 24 As HCPs will likely become increasingly engaged in the use of gene therapy, their adoption and comfort with the preferred terminology will enhance their communications. The usefulness of the resource provided here should be evaluated by an assessment of publication metrics as well as focus group discussions to understand the uptake of the tool by the hemophilia community.

Lessons on communicating with patients may be learned from past and ongoing miscommunications between the scientific community and the public, 33 eg, the harmful misconceptions around antimicrobial resistance or those that arose from the flawed and ultimately retracted report of an association of the measles, mumps and rubella vaccine with autism development. 34 , 35 Poorly delivered or incorrect information, including in clinical trials, can result in a lack of confidence in treatment, unnecessary costs, and discontinuity of care. Transparency is an essential criterion for gaining trust. 36 In particular, the need for adequate education of HCPs will be vital for facilitating these conversations, alongside the need to adjust the information and method of delivery to particular care contexts.

Strengths and Limitations

The Q&A resource provided here is a novel, readily available educational tool for HCPs and their patients. Its strengths include an easy-to-follow format, ready for use as a comprehensive shared decision-making resource in the clinic. Moreover, the development of the resource was heavily guided by its intended audience, therefore we hope it proves immediately useful.

The key limitation of the Q&A resource is its reading level (grade 10–11; high school), which is higher than the grade 5–8 level (10–14 years old) at which the average patient comfortably comprehends patient educational materials. 37 , 38 However, this resource is intended for use by HCPs to tailor their delivery of the material appropriately to different patients (for example, by omitting or describing content based on patient knowledge and/or age) and thus the reading level is targeted higher to include relevant details. In addition, while the Q&A resource is intended to guide conversations with PWH, the patient journey starts in infancy and hence will also involve discussions with parents or other caregivers of pediatric patients, who may have additional specific questions. While gene therapy is not currently available to children, advice to parents of pediatric PWH should be provided to ensure full awareness of future considerations. 39 The Q&A resource therefore functions as a starting point from which HCPs can individualize discussions. Other concerns, including those of reimbursement and financing, while important, will vary by region and are beyond the scope of this article and Q&A resource. Clinical evaluations to date have been conducted predominantly in high-income countries, 40 and therefore, this communication tool is currently limited to patients in those regions. Finally, despite good levels of engagement across key stakeholder groups, the relatively small size and composition of the CHC group could limit the generalizability of these conclusions to the global hemophilia community.

Future Directions

Ideally, the resource would be updated and published as needed to provide the latest relevant information and address any feedback from the community. This work would benefit from even wider stakeholder engagement including surveying a larger, more diverse group of PWH. It would incorporate findings of clinical trials and other research (such as from registries) that would be of potential relevance to decision-making (eg, probability of expressing FVIII, levels of FVIII expression, duration of expression, probability of side effects) and shared and reviewed with patients as it becomes available. 41–43

Furthermore, while this resource provides an educational and decision support tool to aid discussions between HCPs and patients, barriers to providing requisite biomedical and clinical education about gene therapy for HCPs (eg, time and travel costs) should also be identified and addressed. 29

Conclusions and Key Takeaways

Gene therapy offers a potential alternative to lifelong infusions, with the promise of transformative changes in the lives of PWH 7 , 44 and a major shift within the treatment paradigm. 8 The emergence of gene therapy will also add to the complexity of treatment decisions faced by PWH and their families. HCPs will have a crucial role in providing the appropriate, personalized guidance and support to ensure that PWH fully understand the options available to them.

The educational and decision support resources described herein recognize that each patient’s decision journey will evolve throughout their lifetime with their individual preferences at different life stages, and with the emergence of new therapies and a growing evidence base.

The Q&A resource provides HCPs and PWH with timely, relevant information, facilitates discussions, and empowers PWH to engage in shared decision-making. As gene therapy products enter the market, the themes and questions mapped here should stimulate discussion and aid interactions among HCPs, PWH, and family members, to ensure that they are fully informed and realize the clinical potential of this treatment. While the issues discussed here pertain to hemophilia, they could also be applied to other hereditary diseases with multiple treatment options.

Acknowledgments

The authors would like to acknowledge all other members of the Council of the Hemophilia Community for their contributions to this manuscript, particularly in the development of the Q&A resource: Erik Berntorp, Alfonso Iorio, Christoph Königs, Keiji Nogami, Johannes Oldenburg, Brian O’Mahony, Mark Reding, and Dawn Rotellini. This independent group was funded by Bayer. They would also like to thank members of the Bayer Global and European Patient Councils for Hemophilia, as well as Sophie Babicz and Alex Mehuys, and the Bayer Hemophilia Employee Council (particularly Gregory LeCleir) for their insight into the Q&A resource. The authors would like to thank Alexandra Wissle for her support in facilitating the survey. Editorial support (in the form of writing assistance, including development of the initial draft based on author direction, assembling tables and figures, collating authors’ comments, grammatical editing, and referencing) was provided by Ewa Kilinska, of Fishawack Communications Limited, part of Fishawack Health, UK, and was funded by Bayer. The authors would also like to acknowledge Sharon Eastwood, of Bayer, for editorial support during the development of the manuscript.

MW received honoraria for advisory boards from Bayer, BioMarin, Bioverativ, Catalyst Biosciences, CSL Behring, Genentech, Novo Nordisk, and Takeda; CN has received grants/research support/honoraria or consultation fees from Bayer, BioMarin, Catalyst Biosciences, CSL Behring, Freeline, Novo Nordisk, Pfizer, Roche, Sanofi, Sobi, Spark Therapeutics, Takeda, Uniqure; FD is an employee of Bayer; CG is a full-time employee of The Lewin Group, and has been assigned to projects with various life sciences companies, including AbbVie, Alkermes, Amgen, Bayer, BioMarin, bluebird bio, Genentech, GlaxoSmithKline, Janssen, Medtronic, Merck, Mitsubishi Tanabe, and Roche, as well as not-for-profit organizations including Hemophilia Alliance and National Hemophilia Foundation; MS reports grants and personal fees from Bayer, BioMarin, Roche/Genentech, Takeda, Novo Nordisk, and Sanofi; grants from Freeline, UniQure, and Sobi; other from Spark and Pfizer, outside the submitted work; and is a Member of the Institute for Clinical and Economic Review (ICER) Governing Board, Blue Cross Blue Shield Medical Advisory Panel, Consultant for National Hemophilia Foundation and the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) Member. The authors report no other conflicts of interest in this work.

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Integrating Clinical Trials and Practice : A New JAMA Series and Call for Papers

• 1 Executive Editor, JAMA
• Special Communication The Integration of Clinical Trials With the Practice of Medicine Derek C. Angus, MD, MPH; Alison J. Huang, MD, MAS; Roger J. Lewis, MD, PhD; Amy P. Abernethy, MD, PhD; Robert M. Califf, MD; Martin Landray, PhD; Nancy Kass, ScD; Kirsten Bibbins-Domingo, PhD, MD, MAS; JAMA Summit on Clinical Trials Participants; Ali B Abbasi; Kaleab Z Abebe; Amy P Abernethy; Stacey J. Adam; Derek C Angus; Jamy Ard; Rachel A Bender Ignacio ; Scott M Berry; Deepak L. Bhatt; Kirsten Bibbins-Domingo; Robert O. Bonow; Marc Bonten; Sharon A. Brangman; John Brownstein; Melinda J. B. Buntin; Atul J Butte; Robert M. Califf; Marion K Campbell; Anne R. Cappola; Anne C Chiang; Deborah Cook; Steven R Cummings; Gregory Curfman; Laura J Esserman; Lee A Fleisher; Joseph B Franklin; Ralph Gonzalez; Cynthia I Grossman; Tufia C. Haddad; Roy S. Herbst; Adrian F. Hernandez; Diane P Holder; Leora Horn; Grant D. Huang; Alison Huang; Nancy Kass; Rohan Khera; Walter J. Koroshetz; Harlan M. Krumholz; Martin Landray; Roger J. Lewis; Tracy A Lieu; Preeti N. Malani; Christa Lese Martin; Mark McClellan; Mary M. McDermott; Stephanie R. Morain; Susan A Murphy; Stuart G Nicholls; Stephen J Nicholls; Peter J. O'Dwyer; Bhakti K Patel; Eric Peterson; Sheila A. Prindiville; Joseph S. Ross; Kathryn M Rowan; Gordon Rubenfeld; Christopher W. Seymour; Rod S Taylor; Joanne Waldstreicher; Tracy Y. Wang JAMA
• Viewpoint Why Should the FDA Focus on Pragmatic Clinical Research? Ali B. Abbasi, MD; Lesley H. Curtis, PhD; Robert M. Califf, MD JAMA

Randomized clinical trials remain the cornerstone of evidence-based medicine. As a leading medical journal publishing the science that advances the clinical care of patients and the health of the public, JAMA is committed to the publication of clinical trials, as well as promoting the discourse on how clinical trial evidence can best serve the needs of patients and clinicians.

In autumn of 2023, JAMA hosted its inaugural in-person JAMA Summit 1 on the topic of randomized clinical trials and the challenges and opportunities to improve their design and conduct to be most responsive to the needs of clinical practice. The Special Communication published in JAMA, 2 “The Integration of Clinical Trials With the Practice of Medicine: Repairing a House Divided,” was written on behalf of the participants in the JAMA Summit and outlines many of the themes discussed during this provocative 2-day meeting.

A central theme of the JAMA Summit, which is elaborated on in the Special Communication in this issue, is how best to integrate clinical trials with clinical practice. Traditionally, clinical trialists and clinicians have worked independently, and, as the authors of the Special Communication underscore, their separate missions, incentives, and infrastructures have been siloed. The result is inefficiency in the performance of trials and limitations in their scope and impact. The authors offer solutions, including advances in implementation science, novel approaches to statistical analysis of trial data, and integration of trials with the electronic health record as a very rich source of clinical data. The clinical trial and health care delivery disciplines have functioned as a house divided. The authors proffer an approach to bridging the gap.

The future of clinical medicine will be greatly influenced by the data derived from randomized clinical trials, and trials will need to be accomplished more quickly, efficiently, and cheaply than they are today. A recent example is the platform trials 3 that were conducted during the COVID-19 pandemic. 4 - 6 These trials were embedded within clinical care and permitted the study of multiple therapeutic interventions simultaneously, with the results applicable to patient care in short order. The platform trials are exemplary of the types of novel approaches to clinical trials that are needed to realign the house divided.

With this Special Communication and an accompanying Viewpoint, 7 we launch a new series in JAMA— Integrating Clinical Trials and Practice. JAMA invites submissions that explore innovations in clinical trial design, implementation, funding, regulation, education, and application that may bring randomized trial evidence to more effectively address the needs in clinical practice. Our goal is to engage the research community in a significant publishing project to advance clinical trials and better align them with clinical practice. We welcome articles of any type, and we are happy to hear from you with presubmission inquiries. We are eager to chart a new path for randomized clinical trials and the delivery of health care, and we hope you will participate in navigating the road ahead.

Published Online: June 3, 2024. doi:10.1001/jama.2024.10266

Corresponding Author: Greg Curfman, MD, JAMA, Editorial Office, 330 N Wabash Ave, Chicago, IL 60611 ( [email protected] ).

Conflict of Interest Disclosures: None reported.

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Curfman G. Integrating Clinical Trials and Practice : A New JAMA Series and Call for Papers . JAMA. Published online June 03, 2024. doi:10.1001/jama.2024.10266

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Deepening Diversity: LGBTQIA+ Diversity in Clinical Research

Current efforts to improve diversity in clinical research fall short for the lgtbqia+ population..

Date: July 10, 2024 Time: 12:00 – 1:00 PM ET

In this live webinar, expert panelists will discuss how sponsors can smooth the LGBTQIA+ patient journey for clinical trials, starting with gender-neutral protocols, how to make trial communication more inclusive, the FDA’s draft guidance on diversity plans and data collection, and the roles that sites/investigators can play.

Upon completion of this continuing education program, participants should be able to:

• Know the basics — and limitations — of the FDA’s diversity plan requirements
• Understand the importance of including LGBTQIA+ trial participants
• Discover ways technology can capture SOGI (sexual orientation and gender identity) data

Fenwick Eckhardt is a solutions consultant lead for clinical new product development at Citeline. She brings several years of experience working in the pharmaceutical and healthcare industry, working with small and large clients globally. Before joining Citeline, Fenwick worked at a boutique healthcare consultancy in London. In this role, she gained invaluable quantitative and qualitative experience conducting secondary market research for various major and rare diseases, proposal and report development, client relations, patient and KOL interviews, and behavioral analysis. Since joining the Citeline solution consulting team, Fenwick has focused on clients’ needs in the areas of new product development strategy, opportunity assessment, competitive intelligence, and product management. This work has covered projects across therapeutic areas, including oncology, autoimmune and inflammation, ophthalmology as well as rare diseases. Fenwick holds a bachelor of science degree in human physiology from Boston University and a master of science degree in international health management from Imperial College London.

Liam Paschall is a proud transgender man and a dedicated advocate and voice for the transgender community. With a passion deeply rooted in fostering understanding and inclusion, he has devoted his career to educating others on the nuances of trans experiences. Liam has been particularly active in the clinical research field, providing speaking engagements, training, and sharing his own experiences to increase awareness and improve healthcare for the transgender community. Through public speaking engagements, comprehensive workshops, and tailored training sessions, Liam works tirelessly to bridge gaps in knowledge and empathy. He has presented at various organizations, schools, medical facilities, and events, including the Drug Information Association’s annual event, CISCRP, National Medical Fellowships, Inc., Cisco, Red Hat, and more. Beyond educating, Liam is committed to actionable change, offering insights into how institutions and individuals can evolve toward genuine allyship. His work empowers his audience to become proactive participants in the journey toward equity and acceptance for transgender people everywhere.

Shir Netanel is an accomplished leader in the field of healthcare advocacy and clinical trial management, currently serving as the Associate Director of Patient Advocacy and Clinical Trial Advocacy within the Global Medical Affairs Oncology division at Johnson & Johnson. With a background in biological sciences and a deep commitment to social justice, Shir has dedicated her career to promoting diversity, equity, and inclusion (DEI) to advance beyond industry standards when it comes to representation in clinical trials. At Johnson & Johnson, Shir leverages her lived experience as a queer individual to spearheaded initiatives that advocate for the inclusion of LGBTQIA+ folx in clinical research, determinedly working to break down barriers and promote understanding. In this role, she challenges traditional approaches to caring for queer communities by dispelling the stigma that associates the community solely with infectious diseases and mental health. Instead, Shir encourages people to view the work through a lens that recognizes how the SDoH impacts intersecting marginalized communities within sexual orientation, gender identity (SOGI) groups, and how they can profoundly impact health outcomes. A direct outcome of her efforts is the initiation of the LIBERTAS study – which is one of the first degendered and transgender inclusive prostate cancer clinical studies and serves as a foundational model for SGM inclusion in research.

Garo Kiledjian is the Founder and CEO of SGM Alliance, a 501c3 nonprofit that focuses on educating and advocating for inclusive Sexual and Gender Minority (SGM) participation in clinical research, life sciences, and the evolution of healthcare. As someone passionate about people living with HIV, he also provides consulting for Trialogic, a Site Network for one of the largest global HIV healthcare providers. Having served in multilevel roles within administration, research, pharmacy, operations, and finance, Garo brings a versatile skill set to his leadership role. He also serves on the Membership Planning Committee for Society for Clinical Research Sites (SCRS) and is on the Executive Committee for the National LGBTQ Task Force—one of the oldest social advocacy groups that focuses on legislative and policy changes at the grassroots level in Washington, DC. He is passionate about DEI and offers a unique perspective on how the intersectionality of race and ethnicity overlap with gender and sexuality. Garo earned his Bachelor’s from UCLA and studied Leadership and Global Strategy at Woodbury’s Graduate School of Business.

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