Join PHA for a Rare Opportunity … A Conversation with FDA

This is a guest blog by Katie Kroner, PHA’s Director of Advocacy and Awareness. Katie is working to fill the room (and the Internet) with PAH patients for a unique meeting at the FDA White Oak, Maryland, campus. PAH has been selected from thousands of diseases to be one of 16 to talk about what patients want from the FDA approval process. Now all we have to do is show up to be heard. It is truly a rare opportunity. If you haven't registered yet, do so now. If you have registered and are willing to speak on a panel, make sure you send an outline of your comments to FDA. You should have received an email from FDA with the details.

We count on the U.S. Food and Drug Administration (FDA) to ensure that the medications we buy are safe.

FDA is also responsible for making sure that drugs do what the company selling them says they will do and that each new drug does something at least a little different from previous ones. In other words, that those drugs are effective and offer new options.

Every drug comes with some risks. How does FDA decide that a drug is safe and effective enough to be sold? In the past, they’ve based these decisions on specific, measurable criteria such as improving performance on the six-minute-walk test.

What they haven’t done much of is talk with people who will actually be taking the drugs they approve. In fact, some disease communities have had to protest in front of the FDA building just to get a meeting.

That’s what makes the May 13 meeting between FDA and the PAH community so unique. FDA has decided to make some changes in how they measure safety and effectiveness, and this time they are asking patients for advice. When they announced their new program, they got requests for meetings from hundreds of groups. Rino wrote comments, and PHA Board Member Colleen Brunetti traveled to DC to present to the FDA. In the end, FDA decided to start small, they will meet with 16 disease communities over the next three years, and the PAH community is one of them.

That meeting will take place on Tuesday, May 13, and it’s important that you take part. The room holds 250 and the Internet holds a whole lot more. I invite you to come in person if you can, but if you can’t make the trip, you can still respond to poll questions and submit comments online.

We know from FDA’s meetings with other disease communities that the conversation will be informal. After kick-off comments from a few pre-selected panelists, anyone in the room who is living with PAH and the parents of children with the disease will be invited to speak to questions like: 

• What symptoms of PAH most impact your daily life?
• Are there activities that are important to you that you can’t do the way you would like because of your PAH?
• What do you do to treat your PAH?
• Are there downsides to your treatments? How do these impact your life?

Those who participate online can respond to similar questions via poll. 

It’s about time a federal agency is asking PAH patients these important questions. PHA has worked hard to secure this meeting. Now it’s your turn to make your voice heard.

• Register with FDA. Whether you are attending in person or by webcast, you must register on FDA’s website. All members of the PH community are welcome, but PAH patients and the parents of young patients will do the talking.
• Claim your seat on the bus. For those who would prefer not to drive to the FDA campus, buses are available from pick up points in New York, Philadelphia and Maryland. Complete this form to claim your seat on the bus

We continue to advance..

This blog was originally posted on August 8, two days after an FDA advisory panel voted their opinion on Riociguat.  It is now updated following the publication of an August 29 New England Journal of Medicine article  and September 2 presentation of new data on Macitentan at the European Society of Cardiology Conference in the Netherlands.  If approved, both drugs may offer additional treatment options for PAH (and Riociguat also for CTEPH). FDA decisions are expected by mid-October.


Tuesday, August 6 was a rare and important day...

A new drug for PAH and CTEPH was being reviewed by the Cardiovascular and Renal Drugs Advisory Committee of the FDA.  This is generally the final step before the FDA releases a decision on whether a drug is approved for release to market.

Thirteen years ago, I attended an FDA Advisory Committee meeting for the first time.  Actually, I attended two on two consecutive days.  The hearings were for what became the second and third drugs to be approved for PAH -  Tracleer and Remodulin.  It was an exciting two days.  The first treatment for PH - Flolan - had been approved in 1996 and five years later we were looking at a tripling of options for patients.

That was only the beginning.  Over the next decade, we would see another tripling...to nine treatments.  And, this week, we were looking at the strong possibility of a tenth.

Other changes, have taken place as well.  Unlike in those early hearings, three of the 11 panelists on the Committee - Drs. Stuart Rich, John Newman and Steven Kawut - are recognized experts i n the field.  Their contributions to the Committee conversation helped the entire panel understand the nuances and needs of this complex area of medicine.

During the public comments section of the meeting, I had the opportunity to read a letter that had been reviewed and approved by leadership of PHA's Scientific Leadership Council and our Board of Trustees.

While PHA NEVER directly recommends the approval of a drug - we believe it is the FDA's role to determine the safety and effectiveness of a treatment - we ALWAYS speak to the need of additional treatments for their individual and combination value to patients.

By 3:00pm following presentations by the new drug's sponsor (Bayer) and the FDA staff and many questions for both and much discussion, the Advisory Committee took their votes.  On the first, they recommended Riociguat's approval for treatment of pulmonary arterial hypertension. On the second they voted to recommend approval for Riociguat as the first treatment for Chronic Thromboembolic Pulmonary Hypertension (CTEPH) for patients who cannot undergo a pulmonary endarterectomy surgery to remove clots in their lungs, or for those who still have complications after having the procedure.

With nine treatments, PAH has as many or more treatments than all but two of the 7,000 rare diseases identified in the U.S.  Now, we will wait for the FDA to make the final decision on a tenth treatment by early October.


Here's a copy of the letter we sent to the FDA and delivered to the Advisory Committee:

August 2, 2013

Kristina A. Toliver, PharmD
Center for Drug Evalulation and Research
Food and Drug Administration
10903 New Hampshire Avenue
WO31-2417
Silver Spring, Maryland 20993-0002

Dear Dr. Toliver:

While there have been great advances in research and expanded treatment options for pulmonary arterial hypertension in recent years, I want to assure you that it is not enough.  Each week, we continue to send an average of 20 condolence cards to families of our members.  We continue to lose too many patients with this disease.  Different treatments work for different patients.

It is up to the FDA to judge the safety and efficacy of riociguat.  However, we at the Pulmonary Hypertension Association want you to know that, should this drug be deemed effective, the need is there for PAH where it will be a valuable addition in the arsenal of therapies. Additionally, for our patients with CTEPH who are not candidates for surgery or have post-operative pulmonary hypertension, we are excited that, if approved, riociguat will be a valuable therapeutic option. 

Sincerely,

Rino Aldrighetti
President and CEO

Yes we can...yes we did!

In 2012, the FDA began an initiative to involve patients in the drug development process.  They identified 39 potential diseases of which PAH was one.  Through their public process, the candidate diseases grew to 90.  For each of the selected 20 disease areas during the five year course of this effort, the FDA will conduct a public meeting to discusss the disease and its impact on patients' daily lives, the types of treatment benefit that matter most to patients, and patients' perpectives on the adequacy of available therapies. Besides patients, the meetings will include participation of FDA review divisions.

Today, we got the great news that PAH is one of the 20 selected diseases for participation!

After our Washington rep, Dane Christiansen notified us, here's what PHA's Advocacy and Awareness Director, Katie Kroner, wrote in her announcement...

In October, PH patient and PHA Board Member, Colleen Brunetti, represented the PH community before the Food and Drug Administration (FDA). In her testimony, Colleen argued for the inclusion of PAH in FDA’s new patient focused drug development initiative.

The goal of this initiative is to engage those living with a wide variety of diseases in giving input to the FDA about how the drug and device approval processes can be adapted to better meet patients’ needs. For example, FDA will seek input on the way they assess risk versus benefit during the approval process for new drugs and devices.

FDA selected 20 disease communities to work with over the next three years and the PAH community is one of them! Several diseases associated with PAH are also among the 20 selected, including sickle cell, HIV and idiopathic pulmonary fibrosis.

PHA plans to use this opportunity to build a long-term, collaborative relationship with FDA that will benefit the entire PH community well beyond the term of the initial project. We look forward to updating you as we learn more.


So, PHA was successful and, as always, that success emerged from the "power of one" - an individual willing to step forward and unite with other individuals in the fight against PH.

Like Colleen, you can make a difference, too.  PHA's 435 Campaign matches patients and families with your own Member of Congress.  Over time - and with PHA's help - you can develop a relationship with your Congressman or Congresswoman.  The late Speaker of the House, Tip O'Neill often said "all politics is personal".  He was so right...and it's also true that all politics is local.  Your House Member or Senators want to hear from you and they will respond.  We can only win the battles we must continue to fight if we work together.  We very much need you.   If you would like to find out more about how you can help with your Member of Congress through the 435 Campaign (or make a connection with your local media through PHA's PHAware Campaign), contact Elisabeth Williams at ElisabethW@PHAssociation.org.

I'll close this blog with Colleen's testimony. She made a difference...and so can you.  Drop Elizabeth an email today!

Colleen's Testimony

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 Patient-focused Drug Development initiative. My name is Colleen Brunetti and I am a pulmonary hypertension patient, looking to my coming 5-year anniversary of diagnosis.

I was diagnosed at the age of 28. I went from a young mom and wife, quickly climbing the ladder in a successful career, to suddenly facing down my own mortality with a disease that I was originally led to believe might kill me in less than five years. 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 I’ve learned a lot.

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.

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.

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

I have responded well to therapy, although this was not always the case. But I am always acutely aware that this improvement could be temporary. I’m doing great. But 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.

A new step forward in pediatric medical education...and a growing concern

Last week, I wrote that Caitlin Flewellen on our Medical Services staff and I would be going to San Francisco for the Fourth International Neonatal and Childhood Pulmonary Vascular Disease Conference.

I had attended last year's meeting in Banff and was impressed at how much of the programming focused on pediatric PAH.  It struck me then that  the pediatric field was progressing rapidly, much as the broader PAH field was developing two decades ago.

As we thought about how we could help with this acceleration, the idea of filming those sessions where speakers were agreeable and posting them with educational credit on PHA Online University emerged.

Thanks to the active support of Dr. Jeffrey Fineman, Conference organizer, Matt Trojnar and PHA SLC members, PAH pediatricians Dunbar Ivy and Erka Berman Rosenzweig, we were able to work out an agreement and get word out to the 31 speakers.  We were also able to work out an arrangement with the University of California at San Francisco to make it financially feasible to re-purpose the talks for educational credit.

We contracted with Fleetwood the same company that did such a great job filming sessions at the PHA International Conference last June.  I love their presentation tool which syncs slides and speaker video.  Here's a sample from Conference.

By the time we had made these arrangements, we were within a week or so of the Conference start.  Our e-mails to the speakers generated 11 positive responses.  So, we went to the Conference with some nervousness.

Caitlin was invited to sit at the registration desk and connected with speakers as they arrived.  By the time she was done, speakers agreed to be filmed in 28 of the 31 sessions!

We believe this success, besides making a great deal of pediatric medical education available online and publicizing the value of this Conference, will be groundbreaking as a model for the filming of other valuable events.

Because we have given the physicians review rights on their filmings and because we have to go through CME review, the presentations won't appear immediately but we're hoping to have them up within four months.

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As a cautionary aside, one disturbing factor in this march toward the development of pediatric medical education is a new FDA rule which requires that pharmaceutical industry support can only be provided for pediatric medical education if the supporting company has an approved indication for the pediatric use of their product.  In the case of PH (and we are not alone, given the FDA's caution in allowing pediatric trials), all approved drugs are being used off-label for children. 

So a question must be asked of the FDA... 

If the pharmaceutical industry is being regulated out of providing support and we already know that neither government nor academia are willing or able to provide such support, are physicians to be condemned to restricted knowledge in the name of purity?

As this Conference's funding is being threatened, PHA has offered to do our best to help but the problem is a rapidly growing one and our own resources are limited.

We will do our best to help and fulfill our mission in the face of a regulatory system that is more and more ignoring the (unanticipated and damaging) consequences of its actions.

We are simple people, trying to do a simple thing...and I'm convinced it is the right thing.

A striking point...

On June 29, the following appeared in an article titled FDA Weighs Rare Disease Drug Regulation But Agency Nixes New Division in the publication, FDA Week.

“Experts suggest that there are roughly 200 FDA-approved treatments for the nearly 7,000 rare diseases that exist globally. So while there have been many successes, the unmet needs are huge,"

I read these words and read them again. 

Why were they so striking to me?  Do the math and consider this...

With 8 treatments for PH, 4% of the approved treatments for the 7,000 rare diseases are for PH.

While PH is a rare disease, our patients' and families' focus on putting a spotlight on this disease has brought it out of the dark corners and into the attention of our dedicated researchers and clinicians and the pharmaceutical companies considering new treatments to pursue.

We are not yet where we need to be...at this point there is not a cure.  However, these numbers  are the result of hard work toweards a clear target.  They.are one indicator that we are moving forward rapidly thanks to the dedication of our community of hope.