NCHR Comments on FDA’s Draft Guidance on Patient-Focused Drug Development

National Center for Health Research: September 11, 2018

National Center for Health Research Public Comment on Patient-Focused Drug Development: Collecting Comprehensive and Representative Input; Draft Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders; Availability

Thank you for the opportunity to provide comments on FDA’s draft guidance on patient-focused drug development. The National Center for Health Research is a nonprofit research center staffed by scientists, medical professionals, and health experts focused on research, policies, and programs that affect public health. Our Center analyzes scientific and medical data and provides objective health information to patients, providers, and policymakers. We do not accept funding from companies that make medical products, so we have no conflicts of interest.

We support FDA’s efforts to increase patient engagement and input into medical product development and testing. However, we recognize how challenging it is to ensure that the information will reflect the views and experiences of a representative sample of patients. That challenge must be met in order to develop treatments that have a more meaningful impact on patients’ lives with risks that patients consider acceptable.

Patients for any given condition can have widely differing opinions, including opinions on meaningful improvement in symptoms or function, acceptance of risk, and acceptance of uncertainty. Therefore, it will be very important that information is collected from a diverse and representative population of patients – not just in terms of demographics but in terms of their medical needs and experiences. This is also true for information obtained from caregivers, advocates, or experts.

Recruitment of a participant sample that has a range of backgrounds and clinical characteristics is essential to obtain a diversity of opinions that adequately reflects the patient population. The method of recruitment could easily create bias in the patient sample, and so recruitment issues must be carefully addressed.

Some methods for recruiting participants, for example through clinical trials or sponsor-funded patient advocacy groups, could bias selection to people who have similar opinions. Those patients may be more interested in getting products on the market faster and may be more willing to accept lower safety standards than other patients, such as those who were previously harmed by an unsafe medical product. Sponsor-recruited patients are therefore not representative of the entire patient population, and the results of such studies would not represent what the entire patient population deems effective and safe. It is essential to recruit substantial numbers of patients who have no industry or commercial connections or support, and that will require the assistance of consumer organizations and patient safety organizations that are not funded by industry.

In addition, other aspects of the study could also bias results in ways that a sponsor desires but do not necessarily represent the views of the entire patient population. This includes many aspects of how data are collected, such as whether there is flexibility in the timing and location of collection; whether it is by phone, computer, or in person; how questions are phrased and their complexity; the availability of help with getting to an interview or having care for children or older adults for which the patient/caregiver is responsible; and length of questionnaire/interview. Thus, it will be important that FDA and sponsors actively select methods of recruitment and data collection that provide a true representation of the opinions of the whole patient population.

In cases where patients contribute to clinical trial design, it is necessary that they understand how design affect the meaningfulness of the results. Many patients do not realize, for example, that surrogate endpoints such as blood pressure are not direct indicators of meaningful medical outcomes. They may not realize that surrogate endpoints do not always independently predict longer life or better quality of life. Similarly, many patients may not realize that patients with certain traits (for example age, sex, race, or comorbidities) can have much higher levels of risk for specific medical products, even if the average risks for patients in general are relatively low.

In summary, we strongly urge that any recruitment by industry sponsors be balanced with recruitment by consumer groups and patient safety groups that are not supported by, trained by, or recruited by industry. A representative group of patients requires more than just demographic diversity – it requires diversity of medical and health needs and experiences. The FDA and sponsors must design recruitment and data collection strategies to obtain a truly representative sample of participants to create medical products that will address the safety and efficacy concerns of the patient population as a whole.

NCHR can be reached through Stephanie Fox-Rawlings at