Patient Experience Moves to Center Stage in Medical Product Development


Applied Clinical Trials

Applied Clinical TrialsApplied Clinical Trials-06-01-2018
Volume 27
Issue 6

Jill Wechsler details the continued rise of incorporating the “patient experience” in drug development measures.

The Patient-Focused Drug Development (PFDD) program at FDA has progressed over the last decade to achieve a more systematic approach to incorporating patients’ experiences and priorities into the clinical testing process.

Jill Wechsler

Efforts to identify what is most important to patients regarding burden of disease and treatment preferences have become important in designing protocols and research strategies most likely to address medical needs and risks.

Congress has signaled strong support for such initiatives: the latest Prescription Drug User Fee Act (PDUFA VI) authorizes additional strategies for utilizing the “patient’s voice” in assessing the burden of disease and treatment most important to patients. And the 21st Century Cures Act calls on FDA to develop guidance on methods for collecting “relevant, objective, accurate, representative, and meaningful patient experience data” throughout drug development. In finalizing FDA’s budget for 2019, the House Appropriations Committee specifies the importance of considering patient experience information in the review of new medical products and relevant product labeling to inform treatment decisions and “payer determinations.”

FDA is responding with initiatives to better coordinate patient input activities for drugs, biologics, and medical devices. A new Patient Affairs Staff (PAS) in the Office of Medical Products and Tobacco (OMPT) is collaborating with the National Organization for Rare Disorders (NORD) to hold a series of listening sessions to further incorporate patient experience with rare diseases into regulatory decision-making. PAS also has launched the Patient Engagement Collaborative with the Clinical Trials Transformation Initiative (CTTI) to establish an outside group able to provide regular patient views to FDA. The CTTI collaborative is similar to the Consumers’ Working Party of the European Medicines Agency (EMA). Separately, FDA and EMA formed a Patient Engagement Cluster in 2016 to discuss best practices and experiences in this area on a more formal, regular basis.

PDUFA and the Cures legislation both instruct FDA to issue multiple guidances on collecting and submitting patient experience data to FDA to enhance how the agency uses it to inform decision-making. The advisories will assist sponsors in assessing burden of disease and burden of treatment most important to patients, how best to identify measures in clinical trials that matter most to patients, and how to use such measures as endpoints significant for regulatory decision-making. These and other topics will be discussed at FDA public workshops in the coming months.

Seeking new strategies

The new PAS initiatives reflect a shift at FDA from expanding on the 24 PFDD meetings conducted since 2014 to strategies to incorporate more clearly the wide range of patients’ perspectives gained from these meetings into credible evidence that can support product development and regulatory decision-making. The aim is to identify best practices for identifying meaningful patient data and new programs and policies to utilize this information.

Insight and advice for achieving this goal may arise from an initiative to advance the science of patient input to achieve more rigorous, credible evidence for use in medical product R&D. At a workshop in May, organized by the Forum on Drug Discovery, Development and Translation of the National Academies of Sciences, Engineering and Medicine (NASEM), experts from industry, disease organizations, academic research centers, and government agencies discussed optimal methods for collecting patient experience reports, barriers to this process, and how these initiatives relate to the drive to tap real-world evidence and other information sources useful to decision makers (see

Theresa Mullin, associate director for strategic initiatives in the Center for Drug Evaluation and Research (CDER) and head of the PFDD program, noted that many drug development programs do not ask patients early enough about setting eligibility criteria, avoiding barriers to trial participation, and what endpoints are most meaningful in dealing with a disease. Mullin outlined how FDA seeks to better understand those aspects of a disease that matter most to patients and whether such attitudes vary by age, culture, or severity of disease. A related question is which commonly measured endpoints are most relevant to different patient subgroups. FDA seeks ideas for dealing with clinical trials that exclude patients who want to participate and with study protocols that are intolerable or unworkable for some individuals. And the agency wants information on measures that may increase the likelihood of patient enrollment and retention in a study and what challenges patients may face in trying to adhere to a prescribed drug regimen.

Workshop participants discussed a range of strategies for improving the measurement and collection of patient experience data, including the use of registries to identify in advance those patients who meet enrollment criteria. The goal is to outline in a report where further research may advance methods for soliciting patient input in research processes that reflect the needs and preferences of affected populations.


Jill Wechsler is the Washington Correspondent for Applied Clinical Trials.


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