Thoughts on last week’s FDA Draft Guidance for Clinical Trials representation

Postmarketing Approaches to Obtain Data on Populations Underrepresented in Clinical Trials for Drugs and Biological Products: https://www.fda.gov/media/170899/download

Last week the FDA shared draft guidance to address underrepresentation of diverse populations in medicine. The guidance was related to a postmarketing setting – meaning that it’s for treatments that have already been given an initial commercial approval. While I personally have an untrained eye for all of the nuances and implications of the draft guidance, I have thoughts based on what I think I’ve read and how it impacts the work for organizations like Leapcure and the various stakeholders in our space.

1. It’s great for everyone that the the FDA is further demonstrating that we need to institutionalize diversity of race, gender, age and more in our processes for developing new treatments. At last months Chief Patient Officer Summit, it was shared how Black and Hispanic populations make up about 6% of clinical trial participants combined compared to their 33% actual combined population in the US. The FDA is channeling some Kobe Bryant “Jobs Not Finished” energy around our need to continue to test treatments after approval, and I couldn’t agree more.
   
2. The guidance touched not just on who participates in clinical trials, but also who runs them – the clinical research site staff. The FDA gets it on this point. It’s an important win-win-win when research site staff are diverse from an approachability and education perspective. Staff representation = better trust = better quality treatments.
   
3. …But there’s an elephant in the room. The burden that needs to be done in postmarketing must be handled significantly more during premarketing / clinical trials. To be fair, even if I had all of the data in front of me for what risks are considered when a treatment is approved, I wouldn’t know enough about how to make the largest impact. Still, I have a lot of feelings on this, mostly concern related to the lack of clarity that would need to be addressed. I’m concerned about how we’re disclosing and educating populations. How many underrepresented people are receiving a newly approved treatment where similar people have demonstrated safety and effectiveness? Are we doing enough to prepare education to patients and physicians around the true unknown risks? What approval tolerance levels are needed for postmarketing monitoring to be able to effectively support the patient and physician education? Can we resource advocacy groups to play a bigger role in education and monitoring gaps? I’d imagine a broad brush policy has to be really difficult (think of rare diseases we often find ourselves with severe data limitations). However, I would rather be more explicit about where we make exceptions with an eye on how we maintain controls related to health information.

Thank you for coming to my TED Talk.