This is about us engaging with the FDA, and them engaging with us.
Why do they care about our voices?
Because only we know what matters to us, because there’s variety in our opinions about that, because we have skin in the game, because we can report adverse events that might not get through, because we can carry information from them back to community as ambassadors and educators.
Slide up about history of patient engagement. It begins, natch, with the AIDS folk in 1988 and goes thru cancer & others until finally in 2011 they launched a patient network website. Between 2012 and 2014 the patient engagement thing was formalized by FDASIA 1137. And this year — 10 days ago — there was formed a patient engagement advisory committee, which is taking applications right now.
So there’s a patient representative program, which exists to get our voices into their ears. They recruit into their program based on need . . if they have patient reps, they contact those people. There are 200 patient reps in the program, but none of them seem to be us. What?!
Patient reps are trained and prepared through a well-developed protocol that includes webinars and an interactive website and an annual workshop.
What do patient reps do? They can be on advisory committees, serving as temporary voting members, sharing experiences and informing the agency. OR the y can be in consultation directly with the decision makers, behind closed doors.
They’re involved all along the drug process: basic research, translation, pre-IND, clinical trials, NDA/BLA review, and post-marketing.
For getting medical devices through the system, the process is quie similar. The idea is that a device or a drug has a life cycle. There are patient-focused drug development meetings that are disease specific. At those meetings patients get a chance to speak in-depth about what they care most about . . .
Each meeting results in a report; there’s video. The reports are used by industry and in the larger community.
Why aren’t we in this list?
Muscular dystrophy is an example of a community that wasn’t part of this list — but they created their own materials and gave them to the FDA. We could do that.
What should we be doing that we’re not? There’s a website, which I just checked and is quite functional. You can submit comments through the Federal Register, every one of which is given to every member of the committee that’s focused on your area. You can go to an FDA-sponsored public meeting or sign up for one of their webinars.
(Okay, slightly frustrated at the moment. Dudes, this is what we want to know:
- What is your job?
- Who are the people who are in charge of drugs and devices for spinal cord injury
- How do we get in touch with them?
- What is the best way for us to have an effect on what happens with respect to decisions that affect our situations?)
Instead he’s talking talking talking about what other groups have or haven’t done.
The bottom line that I take from here is that if you go to the website I linked to above, you’ll be put in touch with these people. Their job is to take your phone call, answer your email, respond to your letter, whatever. They can’t move the process themselves, but — THIS IS IMPORTANT — they can help you figure out how and where to push and who to talk to.
Q: Do you maybe have too much paperwork? Couldn’t things be a little more streamlined?
A: We’re getting better . . . one thing that’s going to stop everything in its tracks is the shutdown of the federal government. Call your congressmember on that one. I think there are about 3,000 new drug applications that arrive at the FDA every year, with maybe 35 or 40 getting through.
Q: What’s the threshold for the FDA to pay attention to our community? What do you need to hear? How much do you need to hear?
A: They’re looking at what gets submitted . . . the original patient meetings were set up as a basis. He’s recommending that we think about what the Duchenne Muscular Dystrophy group did, (so I better go see what that was.) They actually got things done more quickly than they could have through the FDA.
Q: Earlier someone suggested that the FDA is quicker to approve interventions in acute conditions. Not chronic. Why?
A: We just review the applications we get. We don’t control what comes through, we just regulate it. There’s a language that we speak, and patient groups are learning more and more what that is . . . they can only do that by showing up and being in the conversation. In terms of acute/chronic things — they do eventually weave together. But you who live with chronic conditions,
Q: Clarification from Lyn Jakeman: are we (FDA and NIH) really putting priorities on acutes? A lot of the basis that helps us decide whether a therapy is ready to try in people is testing in animals. So, what she’s saying is that it’s not a matter of priority, it’s a matter of it being so very difficult to get anything to work in chronic animals. There’s very little data to support the idea that something will work in people. In other words, scientists don’t know how.
(My thought on this is that we do know of one thing that has actually worked and been tested and peer-reviewed. And that’s epidural stimulation.)