Thursday, August 8, 2013

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 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
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. 


Rino Aldrighetti
President and CEO

Wednesday, August 7, 2013

The Power of Teamwork: the PHPN Symposium

Ten years ago, PHA hosted the first PH Professional Network (PHPN) Symposium.

PHPN was a young group then.  Members were mostly nurses working in the PH field and they wanted to build professionalism in what was then a much smaller field.   They wanted to share their experiences and knowledge and help integrate young medical professionals entering the field more rapidly into a community that could do its best for patients.

So, in 2003 – on very short notice – PHA helped to organize a two day Symposium.  Sixty members attended and were excited about what took place.  Nurses and other medical professionals ran workshops.  I remember a very engaging medical debate in which leading physicians discussed major treatment issues.  And, of course, there was great networking where medical professionals broke down the isolation that still existed then in that smaller field.

The positive reaction led to a second Symposium two years later. Preparations for 2005 had a different feel and reality.  The Symposium moved from an event being done for the medical community to one being created by the medical community.  That made all the difference. A planning committee from PHPN took the reins, listened to their members and implemented.  Attendance grew by 400% to 250.

Each PHPN Symposium since then has continued to grow and add value.  Now the Symposium is a 3 day meeting that opens with an optional training and lobbying activity in Washington, DC.  As members visit Capitol Hill, they not only build their own skills in an area becoming more critical to health care but they are gaining knowledge they can take back home to share with their colleagues and patients.

As PHPN itself has grown to over 1,400 nurses, nurse practitioners, physician assistants, pharmacists, respiratory therapists, social workers and other non-MD medical professionals working in the PH field, new tracks have been added to create value for the broader group of medical professionals who now attend.

The 2013 PH Professional Network Symposium  (September 26-28 in Arlington, VA) remains a unique opportunity to learn from, and with, PH-treating colleagues.   If you are a non-MD medical professional working in this field, you won’t want to miss this upcoming opportunity!

Here a few of the 2013 Symposium Highlights:
·       28 diverse educational sessions featuring new topics, including exercise right heart catheterization, transitioning pediatric patients to adulthood and patient adherence.
·       Optional session tracks for respiratory therapists, pharmacists, social workers, and pediatric professionals.
·       Opportunity to earn up to 10.25 hours of continuing education credit.
·       Daily networking at meet-ups, receptions and in the Exhibit Hall with other PH-treating healthcare professionals from across the country.
·       Latest research presentations in the Poster Hall.
·       A visit to Capitol Hill to advocate for PH patients.

PHA is offering a discounted registration rate of $100 (a $150 savings!) to the first 250 PH-treating healthcare professionals who register for Symposium*. Don't delay — this special reduced pricing is going quickly!  Register now to take advantage of this special rate.