Sunday, May 18, 2014

A Chat with the FDA

FDA 2012 - Round 1
Back in 2012 I visited the FDA in a bid to have PH chosen as one of several rare diseases that would be studied over the next five years under the PDUFA V mandate. This mandate basically directed the FDA to study at least 20 rare diseases in-depth, looking specifically at patient reported outcomes and view points during the clinical trial process.

While my testimony that day played a part in our selection, as usual, it was a team effort from PHA that got the job done. Amongst the 7,000 known rare diseases, we got "short listed" to 40, and then finalized as part of the 20 to be focused on. This past week, we got our first chance to talk directly to the FDA about what it is like to be a PH patient and live under the medications they approve... or don't end up approving.

Lunch at PHA
 This was an historic opportunity, to say the least. And quite a bit of fun. Many people gathered at PHA prior to the meeting to catch up and have lunch. Then we loaded on to the bus to be brought over to the FDA Campus in Silver Spring, MD.

Prior to the meeting, the FDA put out requests for people who wanted to be panelists and speak
directly at the event. I threw my hat in the ring and was honored to be one of 10 chosen. So I had four (!) minutes to present my comments. I have to say, every single panelist was just outstanding. They were honest, they were raw (some of it was hard to hear), they did an amazing job sharing what living what this disease is like and how desperately we need better medications and a cure.

Along with the panelists, they frequently opened up the room for commentary, and even integrated comments online. Overall, they did a really nice job integrating as many people's viewpoints as possible.

I left feeling we were well heard, but that perhaps this was just the first of what are hopefully many conversations. Because we had to focus on such a select set of questions, the really big picture of living with PH wasn't fully covered. I hope they want to hear more from us in the coming months (and I believe they do).

Above all, I feel like this day served one really key (although perhaps unintended) purpose. As people gave live updates of the day online, and discussed it after, the excitement was palatable. What an incredible boost for our community. You could feel the sense of morale, ownership, and excitement build as people interacted around the experience.

I am very proud of my community. The state of this disease isn't really going to change because of the FDA, or the NIH, or just about anything else. It will really change because although we are small, we sure are loud and focused... in very good ways. As long as we keep our message clear, and continue to be honest about our experiences with each other and with those who can make a difference, it is us, the patients, and PHA, who will lead the charge to our better tomorrow.

The Whole Crew

My commentary to the FDA appears below. If you'd like to add your own, it isn't too late! Open public comment is available until July here:!documentDetail;D=FDA-2012-N-0967-0592


Hello. Before I begin, I would like to take a moment to thank the FDA for having us here today. As you well know, in a rare disease state like pulmonary hypertension, it can be a challenge to feel heard. We really appreciate you taking the time to listen.

I was diagnosed on January 2, 2008. What I thought was shortness of breath due to being deconditioned from having a baby was in fact pulmonary hypertension. From the night I went to the ER for an unrelated illness to the point of diagnosis was actually just two weeks. I was very lucky.

I currently take three medications: Adcirica, Letaris, and Tyvaso.  I am also on nocturnal oxygen. For the first couple years after diagnosis I needed digoxin, but do not any longer.

To begin,  I wanted to try one treatment at a time so that I could gauge which might be helping me and how I might be impacted by side effects. We started in the beginning with Revatio, which later was switched to Adcirica for convenience. We added Letaris about eight months after the Revatio. I felt okay on these two medications, but there were definitely moments my PH stopped me short, particularly when it came to keeping up with my son.

I caught Swine Flu when it came through in 2009 and I was unable to recover fully. It was at that point that we added Tyvaso, which has worked quite well, and it was soon after I started it that my activity levels started to come back up.

In addition to all these treatments, I have practiced integrative medicine almost from the beginning, believing that the PH medications alone are not enough. I could see how they helped a great deal, and I could also see how they would never work as well as I wanted them to if I used them as a stand-alone approach.

I take supplements which are carefully selected with the help of my naturopath and the go ahead from my PH specialist, I exercise several times a week, and I try to have a spiritual practice that keeps me in balance.  I also overhauled my diet a few years after diagnosis and am now a vegetarian. After studying nutrition for some time, I felt that a vegetarian diet was the most heart-healthy and conscientious decision I could make. I do think I fare better eating and living this way because fighting disease is a mind/body/soul effort.

My current treatments (in conjunction with lifestyle practice) control the shortness of breath and heart function quite well. Nothing has been able to touch the fatigue that comes and goes though.

The medications have definitely improved quality of life. However, they also kind of run my life because every four hours I am taking something.

My quality of life is also impacted by the specialty pharmacy system. I have personally experienced quite a few barriers to getting my medications in a timely fashion, primarily due to issues with the order and delivery process, and I know many other patients have experienced the same, some even being forced to go without treatments, or getting dangerously low on a vital medication because the system does not always work as smoothly as it should.

I have taken an active roll in addressing these issues with the help of the Pulmonary Hypertension Association. In fact, we now have an entire system in place where many of our key players in the pharmacy industry are taking a direct roll in gathering and analyzing patient feedback and working with PHA to build on best practices, as well as improve in areas where there is need.

The downsides to the treatments themselves include a nagging cough from the nebulizer and the acute nasal dilatation, which makes it a little uncomfortable to breath on a good day, and nearly impossible if I have even a simple cold. The equipment can be somewhat cumbersome.

One key area of impact for me is actually psychologically related. I understand that it is an FDA mandate that I must use two forms of birth control, get monthly pregnancy testing AND monthly pregnancy counseling because of one my medications.  I cant stress enough the emotional toll this takes on so many women just like me. We do not want to have to re-live every month the way this disease has taken one of lifes most precious gifts. And yet we are forced to… as if we could ever forget for a minute what this disease has done not only to our lungs and heart, but to our lives and families as well.

Assuming there is no complete cure, my dream is to have treatments where the side effects dont impact us just as badly as the disease.

I want equipment that doesnt get us treated like potential criminals at the airport just because we carry high-caliber medical devices… or better yet, no need to carry equipment at all!

If there were more ways to titrate up and down on oral medications more carefully, that would be wonderful.

Finally, I want FDA approved medications for children with pulmonary hypertension.  This is a pretty tough disease to handle as an adult, but we do have lots of options. I would love to see so much more research and opportunity for the kids. Right now we know that the outcome of PH for children is likely worse than it is for adults, and that any treatments that might be selected are prescribed off label. Additional research and trials that will get our kids appropriate treatment options would be priceless.

Again, thank you for taking the time to hear us today, and to weigh our thoughts and ideas moving forward. I feel confident speaking for the PH community and saying we very much appreciate it.


  1. When I first got diagnosed with PAH, I found your blog to be one of the few places where it seemed like there was hope. Three years later, my symptoms are under control and I feel strong enough to talk about my experiences. I did that by speaking on Panel 1 at the FDA public meeting last Tuesday (May 13). Imagine my delight to see that you were there continuing the fight as well. To hear your comments live was great. I truly appreciate all the crusading you do on behalf of those of us suffering with PAH.

  2. Oh, wow... you have just completely made my day (week? month?). Thank you, thank you for sharing that with me. Are you going to Conference? I would very much like to connect.