Wednesday, December 11, 2013

You and your Specialty important new tool for you

Colleen Brunetti
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

Wednesday, November 27, 2013

Celebrating Life with the Breathe PHree Gala

As we conclude Awareness Month, PHA staffer Megan Mallory writes about a leader, a moment and the PH community...

Five and a half years ago, I joined PHA’s staff in the publications department, my primary responsibility being to put Pathlight together each quarter. About six months into my new position, a young woman named Katie Tobias contacted me about writing for the newsletter. Katie was 23 at the time. As with so many of our community members, Katie had a remarkable story to tell.

“I have always loved art: painting, drawing, you name it. Everyone knew me as the ‘creative one.’ … Then, in a heartbeat, everything changed. I found out I was in the ‘severe’ stage of PH and was ‘knocking on death’s door.’ When I first got out of the hospital, I lost everything, including my desire to do so much as pick up a pencil,” Katie wrote in her first Pathlight article, published in the spring of 2009.

Katie had a way with words, and lucky for us, she found her desire to pick up a pencil again. After that first article, she contributed several more pieces to the newsletter, even helping us to establish the Breath of Fresh Air column in Pathlight for Generation Hope, PHA’s community for PH patients in their teens, 20s and 30s. But then her health declined, and for more than a year, Katie was unable to contribute to Pathlight.

“At this moment, disease progression is inevitable. But this moment is all we have, so make the most of it. Allow yourself to feel the emotions but also know that no matter what life’s path has in store for you, there is ALWAYS a ray of hope illuminating every step,” Katie wrote in the winter 2011 issue of Pathlight just before taking the time she needed to devote to her health.

Katie (pictured left) and me at
the Breathe PHree Gala
That’s why this past Saturday, Nov. 23, was so special. Katie’s health has greatly improved, she has transitioned off IV medication, and she felt well enough to host the Breathe PHree Gala in Wilkes Barre, Pa., with more than 150 people in attendance. As PHA’s final special event of PH Awareness Month this November, I felt honored to represent PHA at this stellar event.

When I arrived at the hotel where the event took place, I walked into a banquet room filled with dinner tables, a spot for a DJ, a stage for a live band, and a table brimming with raffle items. It was quite an impressive site. And Katie was perfectly suited to the role of hostess for the evening. She greeted everyone as they arrived and introduced me to other patients in attendance. Shortly after dinner, she took the stage to explain to audience members what pulmonary hypertension means and why their attendance Saturday night was so important to the cause. She also acknowledged each patient in the room and dedicated the song “Keep Breathing” by Kerrie Roberts to, as she explained, “all PH patients – here and around the world – but also a very dear friend of mine, Jessa, who had her second lung transplant last night.”

Katie’s spirit is a perfect example of the hope that thrives in our community. We have countless stories of patients and their families facing this disease head on and inspiring others with their courage and determination. Katie’s event wraps up a month of these amazing stories, and we’ve been chronicling all of them on the PHA Daily Beat. I invite you to see what our amazing community has been up to this month.

And as we look ahead to 2014, the spirit of hope and determination just keeps growing. In June 2014, PHA will host its largest International PH Conference and Scientific Sessions to date, bringing together patients, caregivers and medical professionals from all over the globe to learn, share and connect.

I look forward to capturing all of this energy in the pages of Pathlight, and I’m excited to share that Katie’s first article in more than a year will be published this January. As she writes in her forthcoming article, “I am a PHighter. I am a Survivor. I am PHenomenal. We ALL are.” Be on the lookout for more from Katie and all of our amazing community members. And if you have stories to share, let us know! Contact me, Megan Mallory, at You are the energy that drives everything we do at PHA. Thank you for all that you do and for making November 2013 such an outstanding Awareness Month.

Wednesday, November 13, 2013

The Awareness Month marathon...

The Awareness Month marathon continues…

Last Thursday evening, I was at PHA’s first N.Y. Chapter Gala, which honored the memory of Dr. Robyn Barst.  That was followed on Friday, speaking to the patient session at the always great Duke/University of North Carolina PHSymposium.  On Saturday, I visited the windy north country of Rochester, Minn., for the Reach for the Stars Gala organized by Bonnie McGoon and her great team of volunteers.

With all that’s happening in our small but active community of PHers, I wasn’t the only one from PHA on the road.  Staff and Board members joined our community at the above events but also at our San Francisco Bay Area Chapter Gala on Friday and the Omaha 8th Annual Phenomenal Hope for a Cure Brunch & Auction (one of 8 events on Saturday (PHkids for a Cure 5K in Houston, 4th Annual Breathe Easy 5K in Albuquerque,  "Walk for a Breath" Sentara Heart PH Awareness Walk in Virginia Beach, VA, Jude's PHamily of PHaith 5K PHun Run in Denton, Texas, South Florida PH Fun Walk in Pompano Beach, Florida, and in Toronto Canada “Let Me Breathe” – A Vegas Night Fundraiser for PH)!

This Tuesday, we hosted PHA’s Annual Congressional Luncheon (and Congressional Office visits).  It was an important event with our leadership – patients, family members and medical professionals – making the case for the PH Research and DiagnosisAct of 2013 and the importance of government support for NIH research.

So why do we do these activities and events?  The answer for our Galas and Walks and other funding events is that PHA is not like government which is supported by our taxes or business which has a product to sell.  We do what we do for PH research and patient-serving programs based on the voluntary support we receive.    And then there are the education events – like the Duke UNC PH Symposium where we offer information and connect with our community and the Congressional Luncheon and lobby day where we make our case to decision makers on issues that are critical to our community’s future.

In the process, we also present new information and media and meet extraordinary people.  

Here's a new video we premiered at the New York Gala (thank you for helping to make this possible Actelion) and which two days later was already being used in Rochester, will be on the PHA website soon. 

Also, at the NY Gala, two patients performed heroically, Jennifer Lombardi was captain and choreographer of her dance team at Villanova when she was diagnosed with advanced PH in 2006.  She and her partner performed a salsa dance after which she spoke about her faith and finding her plan for the future. She was followed by Chloe Temtchine, the Grand Prize recipient of the 2011 AVON National Songwriting Competition who also performed at our Congressional luncheon 

There were many great speakers these events, including Joanne Sperando Schmidt , who spoke at both our NYC Gala and told the story of her own and her family member's PH at the NY Gala and the Congressional Luncheon, 11 year old Lucas Van Wormer who told his PH story at the San Francisco Gala and Juliet Skuldt who spoke about her family's PH journey with their daughter, Cordelia.

There's a lot more but I'm heading out to the Cleveland Clinic tomorrow for their annual PH Symposium on Friday and Saturday. If you're in the area, I hope to see you there.

Tuesday, October 29, 2013

Racing Toward ... PH Awareness Month

Welcome to Pulmonary Hypertension Awareness Month! This November, the PH community is Racing Toward a Cure. In fact, PHers have been racing toward Awareness Month for weeks now, getting an early start on everything from special events to media coverage. As I write, November is still a few days off, but take a look at just a few of the things that are already underway.

Hear ye, hear ye, PH Awareness Month proclamations are coming in from every part of the country.

Golf, Galas and 5Ks! PHers and their loved ones are hosting more than a dozen special event fundraisers during November alone. Check out PHA’s event calendar for one near you.
The annual Sarah Smiles PHun-Raiser kicks off our Awareness Month event lineup this year.

In the news, Joy and Vernon Gore, Janet Mabe, Diane Ramirez and others used PH awareness events taking place in October to garner news coverage announcing that November is PH Awareness Month. Read Joy’s story | Read Diane’s and Janet’s stories
Diane Ramirez (left) and Janet Mabe created a unique Unity Miles event in solidarity with Team PHenomenal Hope, the all-women's cycling team racing across America in 2014. Together, Diane and Janet walked more than 100 miles in 30 days.

Awareness of every shape and size. Whether it’s an awareness table, a class presentation, a personal fundraising page or Facebook posts, PHers are finding unique ways to put their talents to work for PH awareness.
The Bowling family raises PH awareness.

Not long ago, PH Awareness Month didn’t exist. Instead, we had an awareness week focused on setting up information tables in hospitals. Thanks to the work of those first advocates, and all the advocates who have followed, the power of the PH community has grown.

Today, everything you do represents more that we can do. Your awareness-raisers and fund-raisers will translate into better treatments, earlier diagnosis and eventually a cure. Thank you.

There’s still time to get involved this November. Nov. 1 is PH Online Awareness Day, and there’s still time to participate in the Congressional Luncheon and National Advocacy Day on Nov. 12.

We’ll share opportunities and success stories throughout the month. Don’t forget to share your stories with us!

Wednesday, October 16, 2013

Eleven busy days … building better tomorrows

At PHA, we’re always busy, but recently, we hit a streak that’s worth writing about. In an 11-day period from Sept. 25 to Oct. 6, PHA hosted nine PHA-managed meetings and activities. Here’s a snapshot of what we’ve been doing.

We started out with the PH Centers of Care meeting that Wednesday. Four committees that have been developing an accreditation system to help improve PH knowledge and quality of care met under the leadership of Dr. Murali Chakinala (University of Washington). It was an exciting meeting as we get close to piloting the system in early 2014.

The next day PHA’s Scientific Leadership Council (SLC) meeting brought together our medical leadership to discuss medical education initiatives, PHA’s research program and medical programming for PHA’s upcoming International PH Conference and Scientific Sessions. SLC meetings take place twice each year. Dr. Richard Channick (Massachusetts General Hospital) chairs the SLC.

The same day about 100 medical professionals traveled to Capitol Hill to meet with their legislators and discuss the Pulmonary Hypertension Research and Diagnosis Act which will bring together various government agencies like the National Institutes of Health, the Centers for Disease Control and the Department of Health and Human Services to meet regularly to coordinate their work and discuss progress on pulmonary hypertension. Many of the medical professionals are planning to take what they learned about lobbying back to their own communities where they will share it with patients and their families.

The SLC meeting led directly into the three-day PH Professional Network Symposium, The Power of Teamwork: 10 Years of Professional Collaboration in PAH. The PH Professional Network (PHPN) is PHA’s membership network for 1,300 nurses, nurse practitioners, physician assistants, pharmacists, respiratory therapists, social workers and other allied healthcare professionals. With educational sessions featuring everything from echo interpretation to exercise right heart catheterization, this year’s Symposium – a major medical conference – celebrated a decade of growth and continued cooperation among PH-treating healthcare professionals. More than 400 medical professionals working in PH attended this meeting which only takes place every two years. 

Lillian Hansen, NP-C, MSN, MS, MEd, (pictured right) and
Franz Rischard, DO, MS, both from the University of
Arizona Medical Center in Tucson, Ariz., present during the
Symposium session entitled Exercise Right Heart Catheterization.

During Symposium, the editorial committee for PHA’s medical journal, Advances in Pulmonary Hypertension, met to begin planning for the future. Dr. Myung Park began her new role as the journal’s medical editor, and she and the editorial team mapped out the next four issues of the quarterly journal. The upcoming topics will be:
The journal is mailed to more than 40,000 medical professionals around the globe and is also available online at

As the PHPN Symposium came to a close, the PHPN executive committee members met to plan their upcoming projects. This group spends a great portion of its time planning the development of educational material for PH patients and their families. One of the executive committee’s most recent projects includes updates to the School Resource Handbook, a tool for PH parents to share with their child’s teacher. The updated version of the Handbook will be available soon on PHA’s website.

These first six meetings and activities wrapped up on Sept. 29, and a new round began on Oct. 1 when the executive directors and their staff from PHA’s three chapters came to PHA headquarters in Silver Spring, Md., for two days of training, conversations with headquarters staff, and 2014 goals and budget planning. Throughout 2013, our chapters – located in the N.Y. Tri-state area, the Midwest and the San Francisco Bay area – have been hosting fundraising and awareness-raising events across the country and laying the groundwork for future growth. You can follow their activities at

As our chapter staff meetings concluded, PHA’s Corporate Committee meetings began. This committee, comprised of 14 pharmaceutical companies in the PH field, meets with PHA three times a year to discuss how to best support the PH community and our efforts. October’s meeting focused on support for a number of initiatives, including PH advocacy, PH Awareness Month and PHA’s early diagnosis campaign.

Finally, on Oct. 4 through Oct. 5, we hosted a meeting of PHA’s Board of Trustees. This all-volunteer Board meets twice a year, and this meeting focused on a variety of topics, including PHA’s International Conference and Scientific Sessions taking place in Indianapolis, Ind., on June 22-24, 2014.

And if this sounds like a lot, it doesn’t stop there. Throughout this period of time and continuing during October and into PH Awareness Month in November, we have a large number of special events taking place across the country. On Oct. 6, I was able to attend the Baltimore Walk for Hope, which tripled in size from last year. And this past Saturday, Oct. 12, was a big event day for PHA with the Dallas/Fort Worth Walk, the Long Island Walk, the Baltimore Pub Crawl, our first Color PHor a Cure event (watch the video!), and the launch of the PHA Midwest Chapter’s Blue Lips Campaign. I was fortunate to attend the Color PHor a Cure event in little Jennings, La., which drew more than 2,200 registrants.

We celebrated the end of the race with color explosions every five minutes.

We have so much going on, and as November draws near, we will continue to get busier and busier, spreading awareness of pulmonary hypertension and engaging the entire community in our quest for a cure. I invite you to check out our events website to see what’s going on in your area, and I will continue to blog as we go. I hope you will join us on the journey.

Wednesday, October 2, 2013

Football, Pumpkins and PH Advocacy!

Here’s a guest blog from Katie Kroner, PHA’s Advocacy and Awareness Director. The PHPN Symposium was an important and exciting event – with many other activities built around it – and I look forward to writing more about it after our Board of Trustees and other meetings this week.

Here at PHA headquarters in Maryland, we get just as excited about fantasy football and pumpkin carving as the next person, but nothing says fall at PHA quite like the way we ramp up our face-to-face advocacy efforts.

Lauren Johnson, RN (left), with
Rep. Lois Capps, a sponsor of the
Pulmonary Hypertension
Research and Diagnosis Act.
Last week, more than 400 allied health professionals from across the country traveled to the D.C. area for our 2013 PH Professional Network Symposium, The Power of Teamwork: 10 Years of Professional Collaboration in PAH. To kick off the Symposium, nearly 80 PH Professional Network members visited 60 senators from 30 states and asked them to co-sponsor the Pulmonary Hypertension Research and Diagnosis Act. Advocates received a warm welcome and lots of positive feedback from legislative staff about the new PH bill. In particular, the fact that the new bill doesn’t call for any additional government spending made it something more offices were willing to consider co-sponsoring.

PHPN Advocacy Day was also an empowering experience to participants who had never visited their Members of Congress before. One first-time advocate described the day as “a great way to stand up for patients that I take care of in the hospital setting and to let people know about the disease.”

What’s next for PH advocacy? I’m glad you asked!

PHA’s annual Congressional Luncheon is less than six weeks away on Tuesday, Nov. 12 and you’re invited!

PHA’s Congressional Luncheon & National Advocacy Day
Tuesday, Nov. 12
Cannon House Office Bldg, Room 345 Washington, D.C.

The luncheon is free to attend, and transportation is provided from PHA's office.
RSVP to us by email or by calling 301-565-3004 x753
The Congressional Luncheon is an opportunity to make a powerful impact in Congress, advancing our work for accurate diagnosis and a cure for PH. Even if you can’t attend this free event, I hope you’ll join me in inviting your Members of Congress. Then, share this post with ten friends and ask them to either attend the Luncheon or invite their Members of Congress.

Next week we’ll be posting sample messages on PHA’s website that you can use to invite your senators and representative to the event. Learn more about participating in National PH Advocacy Day.

Stay tuned for Congressional Luncheon updates in PHANews and this blog for more about the PH Professional Network Symposium, the strength of teamwork within the PH community, and the amazing examples of all we can accomplish together.

Wednesday, September 18, 2013

Dr. Hudak's Delayed Diagnosis Story...

Sometimes it's PHDuring the past year, PHA has been developing an Early Diagnosis Campaign.  Titled "Sometimes It's PH", the campaign emerged from recent publications showing that over the past 20 years, despite the increased visibility for PH, there has been little if any reduction in the time from onset of symptoms to point of diagnosis.  With 9 FDA-approved treatments available - all over the past 17 years, eight during the past 12 years - earlier diagnosis is an essential pathway to improving patient care and extending life.  Dr. Lynn Brown at the University of Utah leads the campaign and writes a column that circulates quarterly to 40,000 physicians through PHA's medical journal, Advances in Pulmonary Hypertension.  Here is her newest column.  It is the compelling diagnosis story of Dr. Bonnie Hudak, a pulmonologist living with PH... 

Even a Physician with PH Can Be Misdiagnosed
By Lynn Brown, MD, Sometimes It's PH Campaign Chair

Dr. Hudak
Diagnosing PH is often so tricky that even a patient practicing pulmonology can experience delayed diagnosis.  That’s what happened to Bonnie Hudak, MD, a new member of the Sometimes it’s PH early diagnosis campaign’s Education Committee.  

Dr. Hudak is a pediatric pulmonologist at Nemours Children’s Clinic in Jacksonville, Fla., where she often treats asthma and cystic fibrosis but not PH.  Yet her path to diagnosis parallels that of many other PH patients, particularly middle-age women.

Dr. Hudak had long been treated for scleroderma and Reynaud’s disease.  Her rheumatologist knew of the association between PH and scleroderma.  Dr. Hudak maintained a healthy weight, exercising regularly while practicing medicine and raising children.  In her 40s, exercising became more difficult, but with her busy life, she says she paid this little attention. Then while hiking in 2004, Dr. Hudak discovered that at altitude she could not walk uphill.  

In Jacksonville she underwent an echo, an EKG and a chest x-ray.  Her doctor called the results “maybe slightly abnormal.” He was reassured and attributed her symptoms to perimenopause and deconditioning.  He reported that the cardiologist had considered her echo normal. “They were happy with normal, and I was, too,” Dr. Hudak says. 

Still, Saturday morning tennis games left her tired all weekend.  Once, at a neighborhood party, she was chatting with a cardiologist friend.  He told her firmly, “anyone with scleroderma and shortness of breath with exercise has PH unless proven otherwise.”  Two weeks later she was diagnosed by right-heart catheterization and referred to a PH specialty center.

Dr. Hudak’s experience at Mayo Clinic in Jacksonville under the care of Charles Burger, MD, highlights the importance of referral to specialty centers, a key element of the Sometimes it’s PH campaign. In a single day she received comprehensive testing including a more detailed echo which successfully measured tricuspid regurgitation velocity. Those administering these tests pursued results doggedly.  

Dr. Burger also admitted Dr. Hudak to the hospital for a right-heart catheterization that included a vasodilator challenge. Without that thorough procedure and all of the necessary testing, Dr. Hudak’s vasoreactive type of PH would not have been discovered. Dr. Hudak has stayed on nifedipine as her sole PH treatment and has improved from Class III to Class I.  She has also participated in a clinical trial.

In her practice Dr. Hudak now looks for a few more zebras among the horses.  She also looks more carefully at the data used to interpret studies. She would advise other physicians to be more vigilant with a patient who has an underlying condition associated with PH and to work up minimal symptoms that may be due to PH. She also suggests further evaluation if existing results don’t make sense in the clinical setting. 

Dr. Hudak’s experience illustrates that both patients and professionals must be more active in questioning the data and the decisions that drive diagnosis.  Her unique insights will be an asset as PHA works to enhance primary and specialty care professionals’ ability to diagnose and treat PH promptly and correctly.

To find out more about Sometimes it’s PH, visit the website.

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.

Tuesday, July 23, 2013

Ripples in the stream...

It's nice when recognition for PHA comes from others who recognize the work this organization does.

It's very special when that recognition comes from our own community.

Recently, I got the following e-mail message with the magic words, "your supporters have spoken"...

This message is brought to you by

Your supporters have spoken, and you've earned a spot on the 2013 Top Rated List! ""Pulmonary Hypertension Association"" is one of the first nonprofits to be honored this year and you are now listed as a winner on our leaderboard here. You'll be featured in our 2013 Holiday Giving Guide and on our 2013 Top-Rated List, distributed to media and corporate foundations.

All of us at PHA appreciated that people had taken - and are taking! - the time to tell Great NonProfits about what PHA has meant to them.  

But that wasn't all...  On the same day, Jaclyne Franciscone of LaRue PR wrote to our New York
Chapter executive director, Gina Parziale, to let her know that PHA has been included in the Charity Finds section of People StyleWatch. 

That's a big awareness-builder...with a great fundraising bonus.  People StyleWatch features a shiny gold “mirror” tote bag whose manufacturer, Galian, will donate 20% of proceeds to PHA on bags ordered at by Aug. 16.  And, get a discount on the bag, too, by typing in PEOPLESTYLE20 at online checkout. 

So, this blog is not really about the honors, it's about the people who make them, and the awareness of PH they bring, happen.  People like those who initiated the Great NonProfit listing with their stories.  And, people like Jaclyne Franciscone who - when I wrote to say thank you - responded,
Absolutely! I’m thrilled it made it into the issue. Last February, after a three year struggle, I lost my mother to PH. I, along with my sister & dad, would do anything in our reach to help raise awareness for Pulmonary Hypertension. So happy I was able to make a small impact!     
Ripples in the stream.  Each of us makes a choice to act.  Each action makes a difference. 


Wednesday, July 10, 2013

The Bill is it's up to you

Did you know that there is a bill in Congress dedicated to making life better for people with PH? It’s called the Pulmonary Hypertension Research and Diagnosis Act and was introduced by our friends Reps. Kevin Brady (R-TX) and Lois Capps (D-CA). Sen. Bob Casey (D-PA) is working on a Senate version to be introduced this month.

Some of you may be familiar with PH legislation from previous years, but our new bill is completely different. What's important about it in the current legislative environment is that it’s designed to make a big impact without asking the government to spend any new money.

The bill may be new, but its success still depends on you. Take a look at the guest post below from PHA’s Grassroots Campaigns Manager, Elisabeth Williams, to learn more.

PHers Educate Congress on New PH Legislation
Taking a stand together to advocate to Congress about PH is standing together to save a life.
We need more early diagnosis, more education in the medical field, more affordable treatments.”
                                                                                        --Nicole Cooper, PH patient

This spring, Reps. Kevin Brady (R-TX) and Lois Capps (D-CA) introduced a new bill called the PulmonaryHypertension Research and Diagnosis Act of 2013 (H.R. 2073) in the House of Representatives (H.R. 2073). Since then PHers have been emailing, calling and scheduling visits with their Members of Congress to educate them about how this bill will improve life for those living with pulmonary hypertension.

This budget neutral bill calls for the creation of a committee within the federal government focused on giving people living with PH longer, better lives. The group, including representatives from NIH, the Centers for Disease Control and the Department of Health and Human Services would work together to:
  • Advance the full spectrum of PH research from basic science to clinical trials
  • Increase early and accurate diagnosis of PH
  • Educate medical professionals and the public
So far, these efforts have resulted in several Members of Congress co-sponsoring the bill, including:

Rep. Timothy Bishop (D-NY)

Rep. Jim Costa (D-CA)

Rep. Peter King (R-NY)

Rep. Richard Neal (D-MA)

Del. Eleanor Holmes Norton (D-DC)

Rep. Devin Nunes (R-CA)

Rep. Peter Roskam (R-IL)

Stand Together and Advocate!

The success of this bill depends on you! Join other PHers who are standing together to push this bill through Congress. Here’s how:

1)      Contact Your Members of Congress! Educate your own Member of Congress and ask him or her to co-sponsor the newly introduced Pulmonary Hypertension Research and Diagnosis Act of 2013. It’s easy! Simply send an email using PHA’s new online advocacy tool. All you have to do is add your name and zip code and click on Take Action.  That will show you your Member of Congress. Then just take a few minutes to personalize the sample letter with your PH experience. With that brief effort you'll be maki9ng a big difference in helpong your Member of Congress understand the need for more treatments and early diagnosis.  (Oh, and if your Member of Congress is one of those listed above...change the sample letter to just say Thank You for supporting the Bill!)

2)      Sign up for the 435 Campaign! Stand with other PHers who are working to ensure that all 435 Members of Congress support legislation critical to the PH community. We’ll give you the tools to help you easily share your story and make PH more visible in Congress. Email Elisabeth at to join the 435 Campaign.

3)      Stay in the know! Stay up-to-date on late-breaking Congressional news and opportunities to advocate for PH legislation. Sign up for PHA’s monthly Advocacy in Action Alert emails

Wednesday, June 26, 2013

From fantasy to reality…and only 359 days, 4 hours and 42 minutes away

Once upon a time, a man came to a meeting and said, “We should organize an International PH Conference.”

That man was Ed Simpson, the husband of one of the four founders of PHA, and the year was 1992.  He spoke those words at the organization’s annual meeting which drew a total of 10 people.  He spoke them at a time when the entire treasury was $853.31.

Those words and the unlikely success of the meeting he proposed have changed the lives of thousands of people living with PH.  They have taken down the barriers between patients, caregivers and medical professionals and created knowledge, fellowship and partnerships that have made those fighting this rare disease stronger than could ever have been expected when they were spoken.

PHA’s International PH Conference and Scientific Sessions have grown to become the largest and most unique PH meeting in the world.  Like the disease, Conference is rare … being offered only once every two years.

We have now just crossed the midpoint between our 2012 Conference and PHA’s upcoming 2014 Conference.  As I write this, our Conference timer tells me we are 359 days, 4 hours and 42 minutes from banging the gavel to open Conference 2014.

Here are the basic facts…
              Theme: Racing Toward a Cure
              Dates: June 20 to 22, 2014
              Place: J.W. Marriott inIndianapolis Indiana

But Conference is so much more than a date and place.  If you’re a patient or family member, you can expect presentations from well over 100 medical professionals and many more from patients, caregivers and other experts. Many deep and continuing friendships are formed at Conference … you’ll have the opportunity to meet and connect with people living the same experience from throughout the U.S. and around the world.  Thirty nations were represented at Conference 2012.

If you’re a medical professional, you’ll have the chance to teach, learn and connect.  Virtually all the PH experts in the U.S. and many from other nations attend Conference for its stellar Scientific Sessions, its medical track and the opportunity to connect with patients and their families in very different ways.

You’ll be hearing much more about Conference in coming months - program, registration, special hotel rates and more - but here’s a summary video from a few years back.

Feel free to browse the  PHA You Tube Channel to find other Conference videos (and other interesting stuff) or check out a newsletter from a past Conference.