Heartening. That’s how I found last week’s PPMD PAAC Leadership and Advocacy Conference. Every time the whole community gets together, I leave feeling EMPOWERED. I like that word. Good word isn’t it? There is no limit to what I can do for my community. And I imagine I’m not the only one who feels like this. Or at least I hope you feel empowered too. And if you are, I say hold on to that feeling!
What are our PAAC meetings like? They’re part Justice League/Super Friends assembly, part spiritual retreat, part meeting of the Knights of the Round Table before adjourning to carry out more glorious deeds. It’s like heading into an armory and equipping ourselves for the spiritual combat of furthering our cause. We come out of these meetings energized, revitalized, ready to continue on with our duties and enthusiastic to begin new projects. The PAAC: Suit Up! That should be our motto. Though not all of us were able to physically attend the meeting, Alan, Ben Dupree, and Mo joined us by phone. And Matt Petrusko and Ben Cumbo were there in spirit, watching over us. Like Anakin and Obi Wan and Yoda at the end of Star Wars. Just like that.
Our friends Matt Petrusko and Ben Cumbo were there in spirit, watching over us.
Having been with the PAAC from its incarnation, nearly four years, I really feel that we are coming into our own at this point. We’re getting better at both delegating assignments and diversifying the kinds of projects we’re working on: clinical care, mental health, research, and outreach. And we can field questions from experts without having to look over at Annie in case we forgot something. Recently we made the decision to expand the PAAC. It was both inevitable and necessary. There’s work to be done! A lot of it!
I suppose after all of this, I owe you readers an idea of what went on in the hallowed halls of Justice League headquarters this weekend. (Justice League headquarters also happens to look a lot like The Mayflower Hotel as it happens.)
The PAAC - We're basically the Justice League!
We started out discussing recent business and developments. Much of the first day we received updates from the PPMD team about research, regulatory, and legislative activities. Outside of the PAAC, many of us also serve in leadership positions or capacities within the greater Duchenne community. Later in the afternoon, each of us provided brief updates to one another on our activities as appropriate. Right now I’m serving as patient representative for the C-Path D-RSC (Critical Pathways Institute’s Duchenne Research Science Consortium) to develop a mathematical model of the progression of Duchenne. The goal is to make sure that clinicians, researchers, patients, and government agencies like the FDA and CDC are all speaking the same language when it comes to Duchenne. (So we don’t run into the same kinds of issues that cropped up during the Eteplirsen Advisory Committee.) It will make it easier to compare similar sets of data. The next step is to use this model to predict and describe various milestones within the progression of the disease. The milestones will generally be characterized by the loss of particular functions. At this point, our work in defining the milestones is nearly done. The milestones will be ready to apply to the model once we have sufficient data to construct it. Now it is just a matter of “socializing,” these milestone definitions, running them by all groups involved. I’ll come back to this in a second. We also talked about our legislative agenda for the coming year, disability benefits, healthcare, and developments in research. But I’m sure none of this really comes as a surprise to you.
Let me skip ahead and give you an idea of some of the conversations that created a stir of excited energy among the PAAC members. Clinicians and researchers always talk a good deal about patient-centered/focused/developed outcomes. But what does that look like? And before we determine what it looks like, how do we collect data about what patients want and value? This will involve collecting data about the daily experiences of Duchenne and Becker patients. During the course of the meetings, we spent an hour with a tech company involved in medical research, brainstorming technology solutions, among them an app, that would do precisely this. A good portion of the conversation centered on determining exactly what kind of data these technologies would collect and how patients could input it on their own. The daily living data would in turn be aggregated in some sort of PPMD database, perhaps DuchenneConnect, but things are just in the planning stages now. The PAAC envisions this data giving medical practitioners and researchers an idea of what functions and abilities mean most to patients. This in turn could be brought to bear when the FDA for example reviews a drug for approval or asks for post-marketing data. For example, we could collect the data on improvement of patients who cannot perform the 6 Minute Walk Test by taking into account whether use of the drug results in improvement or maintenance of the ability to raise an arm to eat and drink independently. While the D-RSC works to identify milestones of progression, I think this real world data collection would allow PPMD to carry out a complementary project: assigning connotative, emotional, practical values to various functional abilities. To our great delight, when we convened with the Certified Clinical Care Center Directors, they were curious about the very same things!
In terms of other things in the works, we are considering developing a PAAC mentorship program in which PAAC members will get in touch with teens and young adults. Clinicians, researchers, and parents try as hard as then can to relate, but sometimes it’s easier to just talk peer to peer. Certain things can be left unsaid. Looking back, personally, I could have benefited from something like this. As I see it, our job on the PAAC is to make mistakes so YOU don’t have to. Many of us on the committee have achieved great success, but we’ve had our share of hardship too. We want to make things just a little easier for you. Let us be a resource. Even it’s something as simple as figuring out what kind of bed or shower chair is best.