The Patient Voice in 21st Century (Rare Disease) Drug Development

September 24, 2015 YourEncore Marketing



Two weeks ago, I covered “The Top 10 Regulatory Issues for Today’s Drug Developers.” One of the 10 regulatory policy topics that keeps me up at night is patient focused drug development – specifically, how can patients help drive development? This topic is even more relevant and complex in the rare disease space.

PDUFA V introduced incentives for rare disease development but other key questions were unaddressed: how to include the patient voice and what is the proper role of advocacy groups, agencies, and industry? In the recent past, the patient voice was limited to teary anecdotes. Parent Project Muscular Dystrophy (PPMD), however, has made groundbreaking strides introducing the patient voice into the Duchenne muscular dystrophy development process – and giving other disease communities a blue print to follow.

I had the opportunity to moderate a panel discussion with Pat Furlong, the Founding President and CEO of PPMD, about this topic and I’d like to share some of this exchange.

Peter: What are your feelings on how PDUFA V moved incentives forward?

Pat: I think PDUFA V crystalized the need for and importance of patient focused drug development, but it gave no definitions or metrics on how it should be done. How do we include a patient voice? What does it mean? How do we incorporate it in a sufficiently rigorous way? This led to PPMD’s interest in benefit/risk and the need to quantify benefit/risk (caregiver/patient preferences) for our patient community. We did a pilot in an effort to explore this with our community and I am hopeful that it set the stage for the broader rare disease community to begin exploring the equation within their communities.

I feel like PDUFA V was an open door to develop models for engagement. Clearly this is new and the FDA is exploring opportunities to include patients/caregivers to better understand their preferences and what matters to patients. The FDA is driven by data and in that, patients have an extraordinary opportunity to engage, to inform, and to develop models and systems in which the patient preferences are known and understood and considered within the review process.

Peter: Is there a shift among the patient community from being social support groups to becoming a source for clinical trial recruitment, outcomes research and benefit/risk social science?

Pat: I think it depends on where you are in the rare disease community. Social networking encourages people to start their own foundation to do something, but for regulatory agencies, it may not be enough to tell your story. We have to be good partners and scientific partners. We who have been here a bit are looking at how to provide data in terms of benefit/risk, how to look at these patient reported outcomes in a rigorous way, and the value of engaging with the Critical Path Institute to develop a disease progression model. We believe good partnerships are critically important for Duchenne and the rare disease community, and we are able to provide the scientific data that’s going to be important and useful for the approval process.

Peter: What’s your biggest ask for PDUFA VI?

Pat: The big ask for PDUFA VI would be more transparency in the review process. Obviously the FDA is not allowed to give a confidential report of what was discussed, but I would like to see them inform the disease community about what was included in that review. Was benefit/risk data available? How did the benefit/risk inform their thinking? Were PRO (patient reported outcomes) available and if so, considered in the review process? For the patient community, it is important to understand what was included in the review process and how that informed/contributed to the regulatory decision.

PDUFA VI discussions are underway and here’s my take on how patient-focused drug development (PFDD) may evolve under the new act:

  1. What happens after the agency has held a disease-specific conversation? Will the agency devise a way to train patient organizations to create draft guidances for treatment pathways?
  2. How early in the development process should the patient voice be heard? Can PFDD mean more than just a variable in the benefit/risk conversation?
  3. What about adaptive licensing? Will the patient voice be a potent one when it comes to Phase IV requirements?

Next week, I’ll highlight other experts from this panel discussion and talk about expanded access and Right to Try Legislation. You won’t want to miss it!

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Authored By: Peter J. Pitts is an authority on global regulatory issues and an Executive Partner at YourEncore. He is a former FDA Associate Commissioner, the Chief Regulatory Officer for Adherent Health Strategies, and the President of the Center for Medicine in the Public Interest, a policy institute he founded in 2004.

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