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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
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)
|
Rating
FYE 12/2011 |
Overall
|
69.03
|
 |
Financial
|
68.64
|
|
Accountability &
Transparency
|
70.00
|
|
Onward to make an even greater difference in 2014!
