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. 

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)	Rating FYE 12/2011
Overall	69.03
Financial	68.64
Accountability & Transparency	70.00

Onward to make an even greater difference in 2014!

You and your Specialty Pharmacy...an important new tool for you

This guest blog marks the first announcement of an important new PHA service, launched in partnership with Caring Voice Coalition. You will be hearing much more about it in Pathlight and through other channels in coming months. As with many programs and activities at PHA, this one began with one person's story and need and grew from there to involve many others.  In this case, Colleen Brunetti was that person. Here is her story...

Drum roll, please... after years of hard work, the Pulmonary Hypertension Association and Caring Voice Coalition are rolling out something that will address a challenge that has been at the forefront of patient concerns for far too long.

Introducing: The Specialty Pharmacy Feedback Form.

What is this thing and where did it come from? Well, for me, it started back in October of 2010. At that time, I was in a deep battle with my specialty pharmacy company. They were having serious issues with getting my life-sustaining medication to me as promised. And then there was the pivotal moment I will probably never forget.

At the time, I had to sign for delivery for my medications. When the meds didn't show up one day and I had to wait at home again the next day, I missed an event at my son's preschool. I was livid. I was on the phone with a department manager (having long since given up on working with the call center reps) and sputtering out my frustration. I'm usually articulate – I was too upset to be at the moment.

Then the manager said, "I understand your frustration, I'm a mom too." And in that moment I knew how much she didn't get it, and clarity returned. I replied something to the effect of, "Yes, but you are pretty well promised you will see your children grow up. I'm not promised that because with this disease I don't know if I'll live long enough! This mistake made me miss a moment in his life, and I can't get that back."

Then I hung up the phone and sobbed. To express that fear aloud was more painful than anything else I had to deal with regarding PH or the pharmacy company. It still is.

Well, we eventually got the delivery issues straightened out, and while I still lived in slight unease as I had completely ceased to trust the company, things seemed okay.

Then a new mess surfaced. I started a new medication and had an adverse reaction. The way you track an adverse reaction is simple: get the lot number and report it to the manufacturer, which is exactly what was requested by the drug maker. But as it turned out, the specialty pharmacy's distribution protocol at the time was ineffective in that they did not track such things. In other words, I had no way of reporting my adverse reaction to the manufacturer, and thus there was no way to track a potentially dangerous situation for other patients.

To be fair, tracking lot numbers at the point of pharmacy distribution is not an FDA requirement. But I would argue it should be considered best practice and done anyway – especially when the medication in question has the power to save someone's life or quickly kill them if something goes wrong. And as I knew the pharmacy's competitors were tracking lot numbers, I saw it as industry standard that absolutely should have been practiced.

Every time I tried to talk to someone to deal with this issue, I got vastly conflicting information. In short, I felt I was either being lied to (probably not the case), or literally no one had a clue what they were talking about... although I do believe they thought they did and had good intentions, there was clear disconnect between information I was being given, and what was actually happening.

It would take me pages to explain what this particular battle entailed. In short, I ended up on the phone with everyone as high up in the company as I could stalk, my doctor's office wrote a strongly worded letter of protest, I filed complaints with HR for my husband's company urging them to drop this specialty pharmacy from their insurance plan, contacted the biomedical company that distributes the drug and complained, and so on.

Still, I felt I was getting nowhere. The misinformation persisted, and I never did get to report my adverse reaction in an effective way.

And then the next pivotal moment. There I was sputtering on the phone again - this time with people like corporate pharmacy managers and the head of global patient safety for a drug company. And I was repeatedly told, "You have my phone number, you call me if you have more issues." And I finally replied, "That's all well and good, but what is the next patient with problems going to do? They don't have your number. And I don't want your number. I want your company to do their job."

And I knew - even if I somehow got my own issues straightened out, odds it would help anyone else were slim. And the idea that other patients were going through this same mess was unacceptable to me.

As luck would have it, this was around the time of a PH-related conference in Boston in 2011. I was in attendance, along with the PHA president, Rino Aldrighetti. I told him what was going on, and that I was having trouble getting a certain key person to return my call. He picked up his cell phone and made the call himself - and he lit that person's voicemail on fire. My jaw hit the ground, as I had never heard a sharp word from him before, and this was an entirely new side of the PHA leader.

Rino then asked me to begin to track the time I was spending on these issues, and to write a letter to the Corporate Committee for PHA and express my concerns. This is a committee made up of representatives from many of the corporations involved with PH care, including drug companies and the specialty pharmacies that distribute their medications. I did so, and what became known as THE LETTER went out. I guess it caused a stir... or so I am told.

We have continued to do hard work on this issue in in the two years since. I have flown to PHA headquarters twice and, along with PHA staff, a doctor, and members of the Board of Trustees (dialed in by phone) met directly with leaders from one of the pharmacies. Countless emails and phone calls have gone on.

A Specialty Pharmacy Advisory Board has been launched, comprised of: a patient (me), a caregiver, representatives from specialty pharmacies, representatives from drug companies, the Pulmonary Hypertension Association, and the Caring Voice Coalition. We've discussed in detail the issues at hand, and I have been forwarding individual patient concerns to those directly involved for months.

We see issues and trends. We see areas of strength. And now, we want to hear from YOU.

Please, please, use this form to offer feedback to the specialty pharmacies. When you have an issue, be it small or large, report it. When you have a praise or accolade, we sure want to hear about that too. I continue to believe that change is best made when we build on strengths.

Your comments will be accessed regularly by both PHA and the specialty pharmacy for whom it is intended, and all entries will be tracked carefully for trends and areas of ongoing concern. I have worked really closely with these people over the last several months. I am entirely confident that those on the ground care a great deal about these issues and are making sure changes are made. The Advisory Board will continue to meet and discuss as well.

And if anything, I want you to remember... PH has dealt us a really lousy hand. But we are never victims unless we allow ourselves to be.

Three years ago as I sobbed in my driveway after slamming off my phone, I never would have dreamed that such progress and change could happen. But it has.

And now you have to use it to make it work.

 View the Specialty Pharmacy Feedback Form