Perform Patient Preference Studies
Engage with the FDA
Groups that have successfully traveled the therapy development path advise engaging with the FDA early in the process of developing and conducting patient preference studies. Establishing an ongoing conversation with the FDA can make certain you are asking your disease community the questions that are significant to the FDA throughout the clinical trial and marketing review process.
- Patient Affairs , available at +1-301-796-8460, works with patients and patient advocates to involve patients more effectively in regulatory decisions related to therapy safety and approval. The Patient Affairs Staff are the best resource to understand who to contact at the FDA and when.
- Medical Device Innovation Consortium (MDIC) Patient Centered Benefit-Risk Project Report (2015) emphasizes the opportunity to work with FDA Center for Devices and Radiological Health (CDRH) staff from early stages of medical device development. The report covers the value of patient-preference information and considerations that should be made when using this information. The Report has elements that cross over to drug and biologic development.
- FDA-led PFDD Public Meetings ran from 2012 to 2017 as an effort to collect the patient perspective on the burden of their specific disease and unmet medical needs. Meetings continue to be held periodically but tend to be focused on wider public health issues, such as chronic pain and opioid use disorder. You can learn more about the FDA-led PFDD Meetings using the following resources:
- FDA-led Patient-Focused Drug Development (PFDD) Public Meetings describes the purpose of the meetings and lists the 24 selected disease areas as well as links to the agenda, slides, recordings, transcripts, and meeting summaries.
- Enhancing Benefit-Risk Assessment in Regulatory Decision-Making provides history of the development of FDA-led PFDD meetings and the integration of patient preference into the benefit-risk assessment by the FDA during clinical trial and marketing review process through links to the legislation.
- NCATS Toolkit also has a section providing more information about the option of holding Externally-Led PFDD Meetings.