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.

Sunday, October 21, 2012

Confessions of a Reluctant Fundraiser

It's here, it's here!

The 2nd annual Zumbathon for Pulmonary Hypertension!

Or, as I've affectionately dubbed it "Shake it for PH!"

It will be Friday, November 9th at the Valley Ballet in Canton, CT. Doors open at 6:30, Zumba starts at 7:00. Full information and how to register or donate is HERE.

Can I let you in on a little secret? I hate doing fundraising. I really really do. In fact, there was a time that I wrote on this blog that fundraising was not for me. I still kind of feel that way, but the drive to move us forward in this disease has trumped that. Truth be told, there is so much good coming from pushing myself out of my comfort zone, that it is really worth it. But, first, my confessions.

I hate asking for help. I am really uncomfortable saying "I need this!" I think it's mostly because I'd much rather be giving the help. It's hard for me to be on the other side. It's hard for me still to admit that PH is so freaking serious that massive action has to be taken if I, or anyone, am to survive it.

But I am indeed here. Until someone hands me a cure, I'm here, and I'm in it deep. Since PH is a rare disease, it pretty much stands to reason that nobody else is going to step up, unless those of us most directly involved in the community do it ourselves (hint-hint).

As I approach people and have to explain it for the first time it starts with,

"I'm doing a fundraiser for Pulmonary Hypertension. Have you heard of it? No? Well, PH is a rare disease of the pulmonary arteries. The hypertension is in there, not your whole body, and it backs up pressure to the heart. Which has to work too hard, then it enlarges, and can ultimately fail. It is progressive and there is no cure."

Pause. Watch them process.

Then say, "I have it."

Watch them process that.

If you want to have a little fun, lighten the mood if you will, you can always throw in, "AND, I take Viagra!" (Actually, I take Cialis, but same thing... more or less.)

With people who already know about the disease it's a little different. It's asking for help of people who already know you're sick. And here's my other dirty little secret. I don't like doing that either. I wish they'd just magically help on their own. But... life isn't like that. Not because they don't care though. Everyone is pulled in a zillion directions, with a zillion possible things to tug on heart strings, demand attention, request or require support. Let's face it, our world is kind of messed up. So it's up to me, it's up to you if it's important enough, to lead the charge.

Okay, I can do that.  And what I can DEFINITELY do is throw a party!! So, in just a couple of short weeks, we will "Shake it for PH", and we will do so in style.

Five instructors. 50+ participants. One very very fun night, for a great cause!

And... I would be so remiss not to mention that doing this makes me appreciate the goodness in people too. And that TOTALLY wins. First, PHA makes this so easy, with awesome help from their event staff. My husband will take the day off from work and, I can already tell you now, knock himself out doing whatever is needed. His direct co-workers have all donated. They don't even know me. A classmate of mine donated. He's spoken to me once. A friend from the dance studio is working so hard creating the best event t-shirts ever. My Zumba instructor is coordinating music and instructors. The owner of the dance studio is donating her space. Several friends are stepping up in big ways to secure donations, sell tickets, etc. Two friends and one sister will travel from out of state just to be by my side. All of this humbles me, and makes me so grateful. Big time.

As I've said before, if I have to be here, it's worth being here.