Saturday, October 27, 2012

Back to DC

This week I traveled to Washington DC (Well, Silver Springs, MD, more specifically) to deliver comments to the FDA in support of Pulmonary Hypertension becoming a part of the new Patient Focused Drug Development Initiative. In summary, the FDA has created an initial short list of 40 rare diseases to potentially study, and PH is on there. Now we are, in essence, competing to land on the final short list of 20 diseases. My task was to deliver remarks that would hopefully support an FDA decision to choose us. In short, they are looking to study these chosen diseases more in-depth from a patient perspective. When treating a rare disease there is significant cost/benefit analysis that must be undertaken with the drugs. They can be terribly toxic to our systems, and very difficult to manage, but to many people those risks are worth the benefit of being able to, let’s say… breathe. Or live. The FDA wants to take a closer look at the patient perspective during clinical trials and drug approval. A movement I personally applaud.

The process was very interesting. I arrived at the FDA with a PHA staffer and my friend Joshua Griffis, who is involved in the project from the Association end. We met up with Dane Christensen, formally my guide through the Congressional offices when I went to try and secure sponsorship for the Tom Lantos Pulmonary HypertensionResearch and Education Act this past May, and now one of my coaches and guides through this whole FDA thing. Cheering us on from the PHA office via the live webcast was Katie Kroner, Director of Advocacy and Awareness, also a key player in prepping me for this.

The FDA provided panel discussions from their experts that helped us better understand their initial selection process and what exactly they were looking for in this round. There was to be open commentary opportunity for the public, and because of the crowd, we had to kind of scramble to be sure I was signed up to speak. Dane rocked it, got there early, and got me on the list as #8.

We were originally told that I would have five minutes to speak, and I prepped for four in order to leave a little wiggle room. Then a few days before the event, when the significant public interest in the event became more evident, there was rumor that we might be cut down to two or three minutes. So, I ended up showing up with two speeches, one that lasted four minutes and one that lasted a little over two. It wasn’t until the event was under way, and they saw how many people had signed up, that we were finally told we would indeed have only two minutes to make our case. I had to make further cuts to be sure we were well within time limits. It was shaking in your stilettos nerve wracking. (Note to self: wear more solid shoes if I'm going to do public speaking and thus shake a little!)

I’ll include my commentary at the end of the blog. I’m hoping for an audio recording, but am not sure if one will end up being available or not. Before I get into that though, I have to say, this was an incredibly humbling and eye-opening experience. PH sucks, no doubt. But out of all the 7,000 rare diseases in the USA, we sure are amongst some other pretty serious stuff.

I learned a lot and am really glad I got to listen to the testimonies of people on conditions such as Chronic Fatigue Syndrome, ALS, Muscular Dystrophies… A fathers speaking of a disease that will kill him and leave behind young children because he has no treatments available, a 10 year old boy speaking so frankly from his wheelchair about how he and his brother will soon no longer be able to feed themselves and have a dramatically short life expectancy… I can only say that while we have a hell of a fight on our hands with PH, at least we have some good weapons. Others are not yet so blessed.

Now, we wait. Hopefully the FDA will come back with their decision before the end of the year, we shall see.

(Ignore any weird font. Blogger is being persnickety)

Hello, thank you for having me here today. Before I begin I would like to extend my sincerest thanks to the FDA for undertaking this tremendously important initiative. My name is Colleen Brunetti and I am a pulmonary hypertension patient.

I was diagnosed at the age of 28. I went from planning a life to worrying that I might not see my baby boy reach Kindergarten.

Well, that baby boy entered first grade this fall, and I was there. And with the help of the Pulmonary Hypertension Association, I’ve learned a lot since those first early months.

When you get a diagnosis of pulmonary hypertension, or PH for short, your life changes. For some patients, it means a sudden stop of activities once enjoyed, because you quickly find you no longer have the breath or endurance to sustain them. For other patients, it is finally the answer they were seeking, after too long of searching for an explanation to their symptoms. For me, I also found out I had an auto-immune condition as well as pulmonary hypertension, a common scenario with PH.

Because PH is often misdiagnosed, many who finally get a diagnosis are already in advanced stages, with quality of life severely impacted. Medications can help, but often with severe side effects. The only cure remains a lung and sometimes also heart transplant.

While medication can mitigate symptoms of PH and slow disease progress, that could be dramatic, or minor, or temporary, depending on the patient.

Even when therapy is successful, PH is still PH. It’s still progressive and the ultimate treatments are still extreme

The Pulmonary Hypertension Association, has blazed a trail of research, education and support. Patients, doctors, caregivers, allied health professionals, volunteers… all coming together to put hope in action, and to spur us forward until we do indeed reach a cure.

Please select “pulmonary arterial hypertension” and “organ transplantation” as disease areas for the initial 5-year pilot program of the Patient-focused Drug Development Initiative. PHA and the PH community are dedicated to working with FDA in a meaningful and constructive way to ensure this pilot program is a success. It is not only our goal to see that the patient experience better informs regulatory decision-making for PH, but that this effort grows into a sustained campaign of close collaboration between FDA and all patient communities, which continuously improves the system for reviewing and approving innovative therapies.     

Thank you.

1 comment:

  1. Oh my` a short list of 40. Then hopefully to another list of 20, with 7000 rare diseases . Seems like all should be priority. Frustrated that the funding resources are so limited. Keep going forward your words are being heard and remembered by 1000's... supporting others .... and pounding on the doors for more research for treatment and hopefully prevention. It is a huge web~ let your heart be heard for as long as it takes. Praying for your wisdom ~ networking and of course your health. Love to you and your family ~ love Andrea