Tuesday, January 21, 2014

Brushing aside the shadows…

In January of 1991, PHA’s four founders came together to end isolation – their own and that of all others whose lives have been touched by PH.  Their message to others living with PH was, “You are not alone!”
Today, twenty four years later, that message continues.  It has become a rallying cry not only for patients and families but medical professionals and researchers, as well.  It speaks to the reality that we are stronger when we stand together.

This week, I found myself thinking about the globalization of that unity.

The story goes back to 2000…

In Chicago in June of that year, people from seven nations came together at PHA’s Fourth International Pulmonary Hypertension Conference.  At the time, there were three PH associations in the world, the U.S.http://www.phassociation.org (1991), France (1996) and Germany (1996).  At the end of the Conference, our board invited all those from other nations to meet with them.  About a dozen people came. We stayed in touch over the internet and before the end of the year, PHA of the United Kingdom and PHA Israel had both formed.  Now there were five!

As we prepared for PHA’s 2002 Conference in Irvine, California, we planned ahead for an International
session.  When we walked in the door, I will never forget the thrill of seeing 80 people from throughout the world pack the room.

We asked a simple question, “How can we help?”  After a good amount of discussion, a patient from Mexico said, “You are the oldest and the biggest association.  We need to learn from you and from each other.  We need you to help.” 

At the end of the meeting, PHA agreed to try to raise funds for an International Coordinator.  In 2004, I introduced that person, Allison Marian to our international guests at the 2004 Conference in Miami.  That was ten years ago.  Allison was followed by Angie Knott (today back in her home country as National Manager of PHA Canada), others and today, Julia Friederich.  These PHA staffers have maintained daily contact with our peers around the globe.  They have used connections to our staff of over 40 to solve problems and create value and opportunity for our partners.

When we began this international work, a board member asked an important question, “Why?”  The answer was clear and immediately accepted by our leadership.  We are all in this together and, as we end isolation – wherever in the world it may be – we all become stronger in the fight against PH. 

Put more simply, it’s the right and the smart thing to do.

By sharing our resources and building communications networks, we all grow more rapidly.  We minimize mistakes and encourage those who are isolated to take the first steps toward unifying and organizing.  We make sure that any good idea – no matter where in the world it emerges – is available to and used for the good of all.  We accelerate the fight against PH.  We maximize its effectiveness.

Today, there are 68 national PH associations across the globe. Umbrella structures are in place in Europe (PHA Europe) and Latin America (Sociedad Latina de HipertensiĆ³n Pulmonar).  Associations are active in Asia, the Middle and Far East, Africa and North America.  We continue to grow together.

At PHA’s 2010 Conference and Scientific Sessions, we introduced the International Leadership Summit as an opportunity for association leaders around the world to get to know each other personally.  The friendships and direct exchanges that emerged proved the value of the session.  The second Summit brought together leaders from 23 nations (30 nations were represented at conference overall).  We just got word this week that our funding is in place for the Third International Leadership Summit.  It will take place as part of the 2014 PHA International Conference in Indianapolis.  

It is one of the important activities – patient, family and medical education, scientific sessions, networking opportunities and research being among the others – that make the International PH Conference the largest and most essential PH meeting in the world. 

It's also a place where none of us are alone and all of us are stronger.  No matter where in the world we live, we are one...brushing aside the shadows, sharing the flame.

Tuesday, January 7, 2014

Moving into 2014...

Just before we ended 2013, I wrote to PHA’s Board of Trustees and other leaders about what an amazing year it had been…and where we were headed in 2014.  I’d like to open the year by sharing some of those thoughts with you…

As we enter each new year, it’s important to assess what took place during the past twelve months and determine whether and how we continue to bring value to our cause.  In 2013, we brought to reality four new initiatives that will mature in 2014 in important ways. We believe each, in its own way, will set a new path for all those whose lives are touched by PH. The approval of a record number of new treatments for PH during the fourth quarter of the year adds to our march forward in the fight against PH.

PHA-accredited Centers of Care evolved over the past two years from an idea and a wish to a complex and functioning mechanism designed to increase the quality of patient care…and help create order in this rapidly growing field.  During the past 13 years, the number of PH treating physicians has grown at an extraordinary pace – from about 100 to over 10,000.  While there are now a good number of experts in the field, there have been no standards - until now - by which to judge expertise.  Following a testing phase in the first half of the year, the program will begin to roll out accreditations of PH Centers during the second half of 2014.  Webinars have been held for medical professionals and industry and, very soon, we’ll be announcing a webinar to explain the Centers concept to patients and their families.

The Specialty Pharmacy Advisory Board was launched in December and is now hearing from members of our community who are seeking a voice for what needs to once again be a guarantee – the timely delivery of their medication from supportive specialty pharmacies.   This program is a model of how many of PHA’s programs have been started…out of one person’s well-articulated concern.  As we prove success and show value for our own community, our hope is that this will be a model for other disease organizations, as well.

The PHA Chapter Initiative was launched in January of 2013, with the creation of our first three Chapters.San Francisco Bay area, Chicago-Midwest and New York Tri-State and will be expanding in 2014 to include Houston and, we hope, another city to be named later in the year.
This was a new path for us, designed to deliver economic sustainability for PHA’s programs and activities.  Whether it be research, medical education, patient support programs or a host of other activities, each year we are asked to organize and manage many more important services and opportunities for patients, families and medical professionals.  Yet, we are a small disease state…about 10% (20,000 to 30,000 patients) of the upper limit of 200,000 or fewer patients that categorizes a disease as rare.   The Chapters are fundraising structures to help us reach out to the broader community we need to support our work.  It will be a long path but we’ve taken the first steps in 2013, with Chapters in the

PHA’s five Research Programs continued to grow thanks to support from our community.  The addition in 2013 was special. The Robyn Barst Pediatric Research and Mentoring Fund is the world’s first pediatric PH research fund and is just now making its first grants. It is designed to expand pediatric research and clinical expertise in treating children with PH. Besides the rapid inflow of donations to build the quasi-endowment for this fund, it has been amazing to see families jump in to fund grants named for their loved ones under the giving rules of the Barst Fund. As someone who in 1999 had to tell a father who wanted to raise funds for pediatric PH research that there was no such specialized field, I find this launch particularly gratifying…and important.  We will never again have to say there is no such thing as pediatric PH research. The first two awards through the Barst Fund were recently made to Dr. Melanie Nies at Johns Hopkins University and Dr. Mehdi Fini at the University of Colorado Denver.

We did these things in a difficult economic environment and we did them without giving up programs that have meaning and value for our community.  We did it because of all of the volunteers – patient, family and medical – who see value in our work and our staff who facilitate what they do.  We did it because of a strong community that believes in itself.  Thank you.

Treatments are expanding.  On October 8, riociguat (Adempas) was approved by the FDA for PAH and Chronic Thromboembolic PH. Ten days later, on October 18, macitentan (Opsumit) was approved. Then, on December 20, oral treprostinil (Orenitram).  So, during 74 days – 2½ months – three new treatments have been approved by the FDA.  When PHA contacted the FDA information office to learn whether there has ever been such an introduction of new treatments over such a short period of time…for any disease, much less a rare disease like PAH,   they referred us to the Director of the Health Professional Liaison Program. She told us, after checking with a colleague from the Office of Orphan Product Development that, unless you group together all cancers which account for 30% of approvals, it is very unlikely.

Whether or not this has ever happened before, it is an extraordinary achievement for the good of patients.  The continuing investment and effort of each of the pharmaceutical companies in our Corporate Committee, combined with the collaborative work of our medical community, driven by the needs of our patient and family community is a privilege to observe and participate in.  PAH began 2013 with 9 approved treatments and closed the year with 12….as many or more than all but two of the 7,000 identified rare diseases. Only about 400 of those rare diseases have any treatment at all.

I’ll close with some outside evaluations of our role.  We were the most recent recipient of the National Organization for Rare Diseases Leadership Award.   This honor was not something that PHA applied for.  It was a recognition by our peers in the rare disease community of the value and effectiveness of the work that we do. And, in 2013 PHA received our 10th consecutive four-star rating from Charity Navigator (America’s most highly regarded charity evaluator).  Only 1% of the charities they evaluate have received this distinction.

Here are some of the rating numbers. Note the yellow circle which is where we fit in the chart among four star charities…

Charity Navigator Rating
Score (out of 70)
FYE 12/2011
4 stars
4 stars
  Accountability & Transparency
4 stars

Onward to make an even greater difference in 2014!