Dr Mark Fleury Highlights ACS CAN's Involvement in the FDA's Patient-Focused Drug Development Initiative

The latest version of FDA's patient-focused drug development initiative is trying to gather patient perspectives in a systematic way, but the effort faces the challenge of understanding the spectrum of those perspectives, said Mark Fleury, PhD, MS, principal of Policy Development and Emerging Science at the American Cancer Society Cancer Action Network (ACS CAN).

How is the American Cancer Society Cancer Action Network partnering with FDA’s patient-focused drug development initiative?
ACS CAN is quite interested in the patient-focused drug development, this is an initiative that has evolved over the years with FDA. It started in 2012 with PDUFA [Prescription Drug User Fee Act] V and in that agreement, there was an agreement for FDA to hold 20 different disease-specific meetings. And, basically what they did was, they invited individuals or caregivers with a specific disease to come in and share their experiences with the disease with FDA.

Traditionally, what we’ve had, is we’ve had an individual patient talk to FDA about their individual experience. And, obviously, one person’s experience can’t be extrapolated to everyone, so these meetings were an attempt to get a broader spectrum of viewpoints. But, what the patient community and FDA quickly learned is that you’re still only able to gather the input of the people who can make it to the meeting. FDA tried to facilitate remote attendance and things like that, but we think of this as patient-focused drug development as 1.0.

Version 2.0, which we are moving into as a result of the 21st Century Cures [Act] and the latest PDUFA VI agreement is FDA is required to come up with a series of guidances that inform how we can incorporate the patient perspective but to also gather in a very systematic way so it’s not anecdotal, it’s not a handful of people providing their experience of a disease, but really gathering data in a way that we can be scientifically comfortable that we really captured the full spectrum of patient participation.

So, ACS CAN will be very involved in those meetings and helping to develop those guidance and making sure that they really are patient focused.

What could be the biggest challenges that the initiative faces in the real world?
Well the goal of patient-focused drug development is really to formally insert the patient perspective into the regulatory decision-making process. So, FDA is charged with making a benefit–risk assessment of any drug or device, and really the balance of that benefit and risk is dependent on each individual patient and each individual disease. So, cancer patients, for example, may be willing to take a much greater risk for the opportunity of a benefit than, say, maybe somebody with diabetes would. And, to try to quantify how the patient perspective from disease to disease is going to be really challenging because you have a spectrum of patients—one individual has a different risk–benefit tolerance than another.

So, really, the challenge for this is to sort of create that standardized patient perspective and to be able to capture both what is the average and what is the spectrum of those perspectives.
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