Intro: Welcome to the Director's Corner, an audio podcast series, featuring the Director of FDA's Center for Drug Evaluation and Research.
Anne Rowzee: Hi, I'm Anne Rowzee from the Office of Communications and in this session of the Director's Corner Dr. Woodcock and I will be talking about a somewhat new experience for CDER and that is regulatory guidances drafted and submitted to CDER by patient groups. Dr. Woodcock thanks for joining me again.
Dr. Janet Woodcock: Most welcome.
Anne Rowzee: Okay, so let's get started. In 2014, CDER received a draft regulatory guidance from a patient advocacy group. Can you tell me more about this submission and how CDER used it?
Dr. Janet Woodcock: Yes. A group called Parent Project Muscular Dystrophy, which involves parents and related advocates for children with Duchenne muscular dystrophy had been working with FDA for some time and they took up the challenge of writing their own guidance to submit to us. And what is a guidance first of all? Well, that's kind of an explanation to those who might try to develop drugs for treating muscular dystrophy in this case of what the parameters are that they might consider in testing the drug in people. For example, what endpoints could you use? In other words, how would you tell whether the drug is working? How long should the study be? And in whom should the new drug be tested and so forth? They submitted a very long draft that they had received input from researchers, from clinicians who take care of Duchenne patients, as well as many families and Duchenne patients themselves about what they thought was most important to the patients, what should be measured and, so forth. And then we took that and turned it into an FDA guidance and put it out there as a draft guidance that also is for drug developers.
Anne Rowzee: So how should industry or other advocacy groups and academics now use FDA's version of the draft guidance?
Dr. Janet Woodcock: Well, right now it's out for comment and so we're seeking the whole community that's involved in this -- the drug developers, patient groups, because they are many, researchers, clinicians who take care of Duchenne patients -- to comment and try to refine this. Eventually, we'll put out a final guidance but even now developers can look into that guidance and think, you know: Who should we study? What stage of disease would be reasonable? What end points? For example, the patients pointed out that even for patients confined to a wheelchair there are many things you can measure that are meaningful and important to those patients. The typical outcome measure that had been used was a 6-minute walk test but clearly very young children or children who could no longer walk would not be eligible for those trials, and yet they also could stand to benefit potentially from a new drug that was developed. So, the parents and the patients felt it was very important that the range of disease would be studied. That's just one example of their contribution, but they can also tell us a great deal about what study design works for patients and families, some work better than others, some things are very onerous for them to undergo, and we need to know about that.
Anne Rowzee: Okay, so really a jumping off point for the next level. Turning back to the patient advocacy aspect of this story, do you see this type of input from groups playing a role in patient focused drug development going forward?
Dr. Janet Woodcock: Yes. We are very interested in elevating the patient voice during drug development. We have, during the last several years, had many of these patient focused drug development meetings. These meetings were part of our agreement under our user fee negotiations that we do and what we said we'd have 20 meetings about patient point of view for specific diseases and these meetings have been very moving and they have really showed us that people with chronic diseases are really expert in their disease and we need to hear their input and understand their point of view. The patient focused drug development meetings though are not that structured to lead to a guidance and so, we are hoping that moving forward we can set up a framework so that patient groups themselves, especially people with rare diseases, can gather the input from a comprehensive representative group of people with the disease, can write that down, and then can go through a process to perhaps turn that into a guidance or at least tell us outcome measures that should be studied.
Anne Rowzee: Okay, so I'm sure in hearing this there are many patient advocacy groups out there interested in pursuing a similar route. What are your recommendations to these groups now?
Dr. Janet Woodcock: Well, many patient groups are at different stages of sophistication. For a lot of patient groups their orientation has been to raise money for research and also perhaps to support patients with the disease and so they've been focusing their energies and resources on that and so this would be a different step. Other patient groups are very sophisticated and organized and they've been in the game a long time providing research dollars and so forth and many of them have moved to a place of having more direct input into how drugs are developed and studied because they really care about the end game; the goal, getting treatment available for patients. So, I see a spectrum of patient groups in their ability to dive right into this, but we are seeing other patient groups getting involved in these sorts of things. And to start with, I think they really need to assess, you know, where are we as an organization, what percentage of our resources are we willing to commit to something like this. For some diseases there are very good outcome measures, there just aren't any drugs and so that's a different kind of problem. There they might focus on setting up clinical trial networks or other enabling activities, but where the disease isn't very well understood we've been encouraging natural history studies so to understand the progression of the disease and then this would be another step.
Anne Rowzee: Oh, wonderful! Thank you so much for sharing your thoughts with me today and taking your time to talk to our listeners.
Dr. Janet Woodcock: Thank you.
Exit: Thanks for listening. For more information about what you heard today, please visit our web site at fda.gov/drugs.