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

Leadership, Face to Face...

I had an interesting call from Sean Wyman the other day. Sean is an energetic young man who is active in our community, a patient who is currently attending medical school.

As we spoke about a number of issues, we got onto the subject of PHA’s recent Board meeting. Sean found it interesting and suggested I share it with the broader community. Thanks for a good idea, Sean.

"When you can get these leaders together, face to face, that’s when you’ll see real change."
– Bruce Brundage, MD (2001)
Chair of PHA’s Scientific Leadership Council (SLC)

When we talk about a Board meeting at PHA, we’re really talking about a lot more.

Our most recent set of meetings offers a good example.

On Wednesday, March 12, I flew to Orlando and headed over to the Marriott Renaissance. After checking sites in Florida and Texas, our staff picked this venue because the hotel had the meeting room availability we needed for our various sessions and activities, airfare is cheaper because there are so many direct flights, and we were able to get a great room price.

For more fluid planning, PHA usually holds our leadership meetings back to back, since our Board, SLC and PH Professional Network (PHPN) structures are interlocked. Plus, holding them together keeps costs down. This time was no exception.

We began with the executive committee of PH Professional Network on Thursday morning at 7:30 a.m. This is leadership of PHA’s membership group for nurses, nurse practitioners, physicians’ assistants, pharmacists, respiratory therapists and other non-MD medical professionals. Much of their conversation is always about different projects they are creating or reviewing for patients and families. (Take a look at our new School Resource Guide for an example.) There was also a lot of conversation at this meeting about the restructuring of their membership newsletter, development of online medical education programs for their peers working in PH and their members’ involvement in supporting our International PH Conference.

 

When they broke at about 4:00 p.m., I headed over to a combined meeting of the four leadership committees of the PH Care Centers (PHCC). This effort to create an accreditation system for PH Centers is important for a number of reasons.  

• PHA has always publically proclaimed that it is important for patients to see physicians who are experts in PH. However, when people contact us, we have no standard by which to make referrals.
• PH has grown from about 100 treating physicians in 2001 to more than 10,000 today. Most of those physicians see two or three PH patients and are not attached to the latest research in this fast-moving field.
• About three years ago, one of the nation’s largest insurers put out notice that they would no longer be covering combination therapy for PH patients in North Carolina and that this was the pilot for that policy being spread across the U.S. As this limitation on access to treatment moved toward reality, PH doctors were frightened for their patients. PHA’s Scientific Leadership Council (SLC) joined with PH Centers in that state to begin conversations. They made a case for the insurer to defer their decision. They also learned that a major concern for the insurer was that many PH patients had been diagnosed without the essential right heart catheterization, and they claimed to have no objection to providing approval for combination therapy where the prescribing physician was expert in the field. The only problem is that there were no expert standards in the field.

The committees of doctors, other medical professionals and patient liaisons were reviewing current progress and next steps. The PHCC program is now in the midst of its pilot phase (six accreditations), and medical leaders will be holding a webinar on April 30 to explain the program to patients and caregivers. It was a productive and intense meeting that went on until 10:30 p.m.

The next morning, Friday, at 8:00 a.m., it was time for PHA’s SLC to begin. The SLC is a body of world-class PH physicians who – among other things – help PHA develop strong medical education activities for medical professionals, patients and families, oversee our various research programs and make sure that all of our medical information is correct.

Discussions were held around the work of a number of active SLC committees. The Insurance and Advocacy Committee (chaired by Dr. Ron Oudiz) works on making it easier for PH patients to get approval for Social Security Disability and coordinates various state efforts where the voice of medical professionals is needed to increase the value of these programs for PH patients. The Research Committee (chaired by Dr. Serpil Erzurum) reported on an upcoming review of our research programs, which have, to date, committed more than $13,000,000 to research grants. The Education Committee (chaired by Dr. Bob Schilz) has been working on fact sheets for the three new treatments approved for PH by the FDA. All three of those new treatments came in a 73-day period between October and December of 2013. 

With 12 treatments now available – all since 1996, 11 since 2001 – PAH has as many or more treatments than all but two of the 7,000 rare diseases identified in the U.S. The FDA has told us that, unless you count all the cancers as a single disease, they have never seen so many treatments approved in so short a time for any disease, rare or common. This speaks to the collaborative work of our PH medical community and the power of working in a community that does not separate medical professionals, patients and families. We may be a rare disease with a rare model of operation, but PHA’s approach certainly is working.

The other SLC committees also moved forward on many fronts. I was touched that so many of the SLC members donated their travel expenses in honor of Dr. Richard Channick, who is completing a very productive term as SLC Chair. The suggestion had been made by his successor, Dr. Karen Fagan. The SLC meeting went on through mid-afternoon, but I had to leave at 2:00 p.m. as PHA Board Committees began their sessions.

PHA’s Development Committee was first up, followed by the Strategic Planning Committee and the Governance Committee and, finally, the Conference Committee. So what do these folks do?

I think the most succinct description I’ve ever heard of the job of a development committee came from a nun who was president of a non-profit hospital in Rochester, Minn. She said to Dr. Mike McGoon, “No money, no mission.” It’s true. Unlike government, organizations like PHA don’t get money through the power of taxation; unlike businesses, we don’t have a product to sell. We keep our doors open to do the things we are asked to do because people vote with their pocketbook. PHA’s basic dues have remained at $15 per year for well over a decade and a half, and while our membership numbers have grown considerably, if every patient in the U.S. joined PHA, dues would only bring in about 3 percent of our budget. Our Board throughout the years has directed us to build a structure that will not be limited by our numbers. After all, it is just as expensive to fix a rare disease like PH with 20,000 to 30,000 patients in the U.S., as it is to fix a more common one like diabetes with 26,000,000. So the Development Committee works with staff to make sure our fight is never limited by the size of our disease.

The Strategic Planning Committee has similar simplicity to its mission. If you haven’t decided where you want to go, you’ll never get there. PHA’s Strategic Planning Committee works with staff and stakeholders (various segments of the community we serve) to plan our future directions and evaluate whether we are progressing toward those targets. Our plans are usually developed for three-year time periods and evaluated annually.

The Governance Committee proposes the rules that the Board will live by. This runs anywhere from conflict of interest policies to nominating future officers… and a lot in between. While PHA is a community rather than a business, we also have a strong responsibility to manage well the resources that our members and friends provide. Thoughtful governance provides direction for us to do that.

Then there’s the Conference Committee. PHA’s bi-annual International PH Conference has grown to become the largest PH meeting in the world. The 2012 Conference drew well over 1,500 registrants from 30 nations. Pre-Conference includes Scientific Sessions, the International PH Association Leaders' Summit (PHA has played a central role in expanding the number of global PH associations from three in 2000 to 68 today), support group leader and other training sessions and patient and family meet-ups. And that’s just before Conference opens. Conference itself is a complex agenda of patient and medical education, individual and group connections and plenary sessions designed to display the present and the future.

On Saturday at 8:00 a.m., we moved on to PHA’s Board of Trustees meeting. PHA’s Board is a volunteer group of patients, family members and medical professionals. This blend helps us to get the best from each constituency to benefit our mission: To find ways to prevent and cure pulmonary hypertension, and to provide hope for the pulmonary hypertension community through support, education, research, advocacy and awareness.

 

After a difficult 2012, we were able to report on stronger financials for 2013. This was especially important considering the major new initiatives PHA has been asked to take on. There was considerable discussion about the PH Care Centers since our Board provides oversight and ultimate governance for that important new program. Also, the Board reviewed progress on our new Specialty Pharmacy Advisory Board, which emerged from the frustration of patients and medical professionals in a field that is undergoing major changes. PHA has recently hired a staff person (Eva LaManna) to manage this program and to help move it rapidly to its next stage, evaluation of pharmacy response time and comparing the patient/medical professional and company view of the success in positively closing cases. You will be hearing much more about this program as we complete Phase 2 of the feedback system.  

The five-year Early Diagnosis Campaign has accelerated with Jessica Armstrong as our new staffer on the project. Jessica began to show symptoms of PH at 17,000 feet in Afghanistan… and was described as a malingerer. Her story appears in the winter 2014 issue of Pathlight. She understands the importance of early diagnosis and has been successfully moving our three committees forward.

We spent considerable time discussing the Chapter structure begun in January 2013. This was something the Board came to after investigating many options. The goal was to assure PHA’s ability to sustain its programs into the future. The Chapters’ goal is to create new funding opportunities in communities to support the programs we are asked to begin and maintain. Progress is good although not instant and the Board must carefully evaluate our investment and movement toward stability and success. Between our grassroots and Chapter events, PHA is on track for more than 100 events in 2014. Board members are among the many in our community who organize and host these events.

Well, there was a lot more, but the Board meeting ended on Sunday afternoon. A number of us were stranded for a while due East Coast weather conditions, leading to flight cancellations… but that’s the nature of service on the Board.