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Engaging with FDA: A guide for foundation funders of research

04/18/2012

Mary Dwight of the Cystic Fibrosis Foundation (CFF) and Cynthia Rice of JDRF shared their views about why it’s important for groups like theirs to engage with regulators, as well as how to get started and how to be effective, during a recent FasterCures Webinar, “Engaging with FDA: A Guide for Foundation Funders of Research.”

The panelists recommended that foundations ask themselves the following questions before approaching the FDA :

  • What do you want to accomplish?
  • What do you bring to the table as a nonprofit foundation and patient representative?
  • What are your strengths and weaknesses? What tools do you have — and what tools do you need to develop?
  • What are your internal rules of engagement? For example, what is your relationship with industry trial sponsors? Are you comfortable advocating for a specific product? How do you gauge when it’s appropriate?

Based on their experiences, Dwight and Rice said that organizations should understand the following about the FDA:

  • Know who you need to deal with at FDA and how they work
  • Know how different centers operate within FDA and understand their
  • complex decision making processes
  • Know what the FDA can and can’t do


Both panelists discussed how valuable medical research foundations can be to the FDA. They shared some case examples of their own experiences working with FDA on issues such as patient-reported outcomes measures, defining endpoints, and creating a regulatory pathway for a novel medical device.

Dwight emphasized that patient groups bring to the process “the voice of the patient, and the clinical need for a potential new therapy or class of therapies. The story is told differently” by patients. “We are the experts on the patient experience. How do you strategically contribute to the reviewer’s knowledge about the drug they are looking at?”

Rice added that “FDA has the ability to impact not only the approval of products once they come to the agency – they’re impacting the entire pipeline. We find it incredibly important to engage the agency about an entire class of therapies. They don’t see the scope in the field the way we do. We can bring a lot of knowledge about what is happening in the whole space to provide context to the agency that’s very important.”

Bringing the voice of the patient front and center is a vital role of medical research foundations. At FasterCures, we are dedicated to amplifying these best practices so that nonprofit funders of research can benefit from the experiences of their peers.