Patient focused drug development is a recent interest at the FDA following the adoption of the 21stCentury Cures Act. Sections 3001 to 3004 of this act add the definition of patient experience data to the FD&C Act and tells the FDA how to work with sponsors using patient experience data for drug and biological submissions. Patient experience data is defined in the 21st Century Cures Act as data that—

‘‘(1) are collected by any persons (including patients, family members and caregivers of patients, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers); and

(2) are intended to provide information about patients’ experiences with a disease or condition, including—

• (A) the impact of such disease or condition, or a related therapy, on patients’ lives; and
• (B) patient preferences with respect to treatment of such disease or condition.”

The agency was required by the Cures Act to issue guidance on the collection and use of such data and related information in drug development. The draft guidance was issued in December 2019, and its recommendations are presented in a Q&A format. The document proposes that sponsors may develop and submit their own proposed context sensitive guidance on handling patient experience data to the agency. These guidance documents would have a disease or methodological focus and would be particularly useful in areas where the agency has yet to issue guidance. The document also discusses other options for sharing patient experience data such as developing a patient data registry (such as the ALS registry), conducting natural history studies on a disease, coordinating work among patient groups and stakeholders, and other patient outreach activities. I think there is an opportunity to connect with the device community on software for collection, sharing, and reporting of patient experience data for any of these modes. As always, you may submit comments on the draft guidance through 21MAR2019 through its docket listed in the Federal Register.

Text Copyright © 2019 Katrina Rogers