FDA official: Patients play an increasing role in rare disease drug development
Patients have a critical role in drug development, from identifying unmet medical needs to assessing meaningful clinical benefits, according to Kerry Jo Lee, MD, associate director of rare diseases in the US Food and Drug Administration’s (FDA) Office of New Drugs (OND).“Patients are increasingly recognized by all stakeholders as experts,” Lee said during a webinar on rare disease drug development hosted by the National Organization for Rare Disorders (NORD) on 27 July. “If you look at the lifecycle of drug development, there are many aspects in which patient input should be sought early in the process.”
Patient-focused drug development
She pointed to the agency’s recent push to incorporate the patient perspective into drug development and evaluation through patient-focused drug development (PFDD). FDA is developing a series of PFDD guidance documents to address how to systematically collect patient experience data that can aid in medical product development and regulatory decision making. The first guidance document, which was finalized in 2020, is focused on how to collect patient input, including sampling methods and defining a target population. (RELATED: FDA’s first patient-focused drug development guidance now final, Regulatory Focus 17 June 2020)
In February 2022, the agency finalized its second PFDD guidance, which focuses on methods to identify what is important to patients, including best practices for qualitative research and how to avoid misleading results. (RELATED: PFDD: FDA finalizes guidance on identifying what’s important to patients, Regulatory Focus 28 February 2022)
Most recently, in June 2022, FDA released draft guidance on the third part of its PFDD effort, focusing on clinical outcomes assessments to measure outcomes of interest to patients in clinical trials. (RELATED: PFDD: Draft guidance seeks to demystify clinical outcomes assessments, Regulatory Focus 30 June 2022)
Patients provide “a tremendous amount of robust input” into the development of clinical outcomes assessments, which is an instrument to measure treatment benefit, Lee said.
The final guidance document in the series, which has yet to be published, will focus on methodologies, standards, and technologies used in collecting clinical outcome assessment data and how to incorporate this data into endpoints. FDA held a public workshop on this topic in December 2019.
Role of registries
Participation in disease registries is another way that patients can impact drug development, especially for rare diseases, according to Lee. Disease registries are often used to help researchers select clinical trial endpoints, plan the duration of a clinical trial, learn more about the presentation of disease, and identify patient subpopulations that could be candidates for research.
However, Lee cautioned that registry data, like all forms of real-world data, must be carefully collected to ensure data integrity. For groups considering setting up disease registries, she said it is critical to consider how the data will be used from the beginning of the process to ensure collection of the appropriate information.
“Collecting it in a manner in which you can trust the data and validate the data and that the data integrity is not compromised, is just as important as all of the uses that you can get out of it,” Lee said.
NORD webinar
