Wednesday, April 22, 2015

4 Stars... Building Longer, Better Lives

Shortly after I came to PHA as its first employee in 1999, I was invited to attend an annual meeting that the National Heart, Lung, and Blood Institute of the National Institutes of Health organized for nonprofit organizations in NHLBI’s area of medical interest.

I was pretty excited to attend. It was my first chance to connect with other disease associations. I was hoping to learn something from them … and I did. During the breakout sessions, conversation was dominated by one disease that had four separate organizations. They were all shouting at each other and not accomplishing much. To my knowledge, that’s still the case today.

The lesson of the day for me was that PHA needed to be built as much more than an organization. It needed to be a community, a community that reaches across all constituencies whether they be patient, caregiver or medical. It was important that PHA be managed to its mission by people who could work in solidarity across the many issues that will make life better for PH patients. Fortunately, that lesson was consistent with our founders’ vision.

Does that approach of solidarity in community-building work? Well, there’s a lot that could be written about what PHA has accomplished over the years, but that will be for other blogs. This one is about people looking at this organization from the outside.

According to its Wikipedia profile, Charity Navigator was launched in the spring of 2001, with the mission of helping "donors make informed giving decisions and enabling well-run charities to demonstrate their commitment to proper stewardship" of donor dollars. Initially, Charity Navigator provided financial ratings for 1,100 charities. Charity Navigator currently evaluates more than 5,400 charities in the United States.

Due to their comprehensive review process, Charity Navigator quickly emerged as the most respected of the nonprofit evaluators. PHA became one of those evaluated charities in January of 2004, receiving their highest rating – 4 stars.

In April of this year, PHA received our 12th consecutive 4-star rating. As Ken Berger, Charity Navigator’s CEO, writes, “Less than 1% of the charities we rate have received at least 12 consecutive 4-star ratings, indicating that Pulmonary Hypertension Association outperforms most other charities in America….”

Our score this year, at 98.55 (out of 100), was our third highest ever.

PHA has also been recognized for excellence by many other organizations – including the Better Business Bureau, GuideStar Exchange, Independent Charities of America, Great Nonprofits, National Organization for Rare Disorders (NORD) and the Center for Nonprofit Advancement – based on a variety of standards.

We have accepted these awards as the validation of a system that is essential to success in a small rare disease state. That is, we move forward because of the strength and support of a single and powerful community: people like you, bringing the “power of one” to the table for the good of many.

Wednesday, April 8, 2015

Four Days in March...

Usually, we see things in pieces. Once in a while, we are privileged to see those pieces come together… to understand the greater whole.

The PHPN meeting on Thursday
During the course of four days beginning on Thursday, March 26, I had that opportunity. We began on Thursday morning with a daylong meeting of the PH Professional Network (PHPN) executive committee led by Melisa Wilson, a nurse practitioner from Orlando, Florida. PHPN, founded in 2000, is a structure within PHA to involve nurses, nurse practitioners, physician assistants, respiratory therapists, pharmacists and other non-MD healthcare professionals working in PH. It has become an essential and highly valued body for education and networking in the field. There was much discussion at the meeting about the upcoming PHPN Symposium. This event, which PHPN organizes every two years, has become the largest PH meeting of non-MD healthcare professionals in North America. Among many other topics, PHPN leadership also devoted considerable time to discussing the development of publications and supporting the quality and accuracy of PHA publications.

As PHPN continued its work in the afternoon, the PH Care Centers (PHCC) leadership began their meeting to discuss the new registry PHA is currently building. The registry is based on an understanding that PHCC accreditations can only achieve full value for the PH community when we look across all centers to create and share data to better understand what works best for patients. One of the compelling reasons for PHA to take the significant financial risk of building this registry was a review of the cystic fibrosis registry results. Over a 24-year period (1986-2010) during which there were no disease-specific treatments, patient survivability increased by over 11 years. While other factors, such as earlier diagnosis contributed to this advance, there is no question that shared knowledge of what works, flowing through the work of the registry, was an essential pillar of this success.

The Registry Committee of the PHCC, led by Dr. Steve Kawut of the University of Pennsylvania, is working to build a similar pillar in PH. It was gratifying to see that leaders of five registries working in PH came to this meeting to discuss creating a consistency of structure that would allow patient data to be used in multiple registries. If successful, this will allow more rapid development of knowledge in the field.

So that was our first day…

We began Friday with an all-day meeting of PHA’s Scientific Leadership Council (SLC). This international and globally-regarded group of medical experts in the field oversees PHA’s entire medical structure. This includes research, our multiple medical education programs, the development of our accreditation and registry structures, and the accuracy and relevance of the medical information we provide to patients and their families.

The SLC meeting had much discussion of the rapid progress we are making on the new PH Care Centers accreditation program. With a target of 60 accreditation reviews by the end of 2015, we have already completed 20, with another 20 submitted and being processed for more information or site-visits, and an additional 17 online applications in process of completion. A little over three months into the year, we are ahead of schedule.

Research updates related to PVDOMICS
A highlight of our research discussion was PHA’s new research partnership with the National Institutes of Health. This relates to the PVDOMICS program, the importance of which was described in a guest blog by Michael Gray.

While the SLC meeting continued, PHA’s Board of Trustees Committees began meeting in the early afternoon. The Development Committee discussed how to fulfill their obligations to help raise the funds for PHA to fulfill its mission. Harry Rozakis, a CTEPH patient, chairs the Development Committee.  

The Governance Committee also met. They are chaired by Laura D'Anna, a former PHA Board chair who lost her sister to PH. This Committee reviews PHA's bylaws - our organizational rules - and works to make sure the Board functions well to oversee all of the many issues in which PHA is involved.

The Strategic Planning Committee, chaired by John Hess, the parent of a child with PH, is charged with overseeing the development of our multi-year plan that tells us where we want to go so we have a direction to steer the organization.

A new committee also met: the Search Committee. They are charged with finding my successor as President/CEO when I retire after PHA's International PH Conference in 2016. Their first task will be to find a search firm that fits well with PHA's goals for the position. PHA's Board chair-elect, Roger Towle, the father of a PH patient, is leading this important effort.

To be clear on those goals, PHA's Board has to have strong focus. To assure that focus, they had an all-day session on Saturday with a consultant from Board Source. Board Source is the most highly regarded nonprofit organization dedicated to nonprofit management and governance issues. PHA is a pretty complex organization these days, much more so than when I started as the first staff person 17 years ago. Does the Board seek someone with a medical background, one in organizational development, corporate or nonprofit management expertise, or the ability to raise funds? Those (and more) are the kinds of questions that will define the next stage of PHA's growth.

The Saturday session was very productive in defining the Board conversation and led us into an abbreviated Board meeting on Sunday where many of PHA's medical and patient-serving programs where presented and discussed. The Board is led by Steve White, an Episcopal priest with a doctorate in health management. Steve lost his daughter to PH.

I should also mention that the Board had the opportunity to meet with over 100 Texas support group leaders and members who had come to the Dallas Omni where PHA will have our upcoming 2016 International PH Conference and Scientific Sessions. It was a great opportunity for the Board to meet our local hosts and share our common excitement about the Conference that will take place in PHA's 25th anniversary year!

In closing, I've always felt the all-volunteer PHA Board is a pretty good reflection of the PH community of patients, caregivers and medical professionals. The members put in a lot of time dealing with the many complex issues a rapidly growing organization must face. Seeing all the meetings I described flow from one to the other during our recent four-day span and understanding the interlocks between those meetings, my view is that PHA is not a series of activities but a single organization that connects those activities for maximum impact for the good of patients. It has always been my privilege to work for PHA.


WATCH for Rino's next blog: PHA's 12th straight Charity Navigator 4-Star rating.  

Wednesday, March 25, 2015

Make the Halls of Congress Ring on National PH Call-In Day

This week, PHA’s Advocacy and Awareness Director, Katie Kroner, is our guest blogger. Katie describes an important – and easy – way we can make a real difference in the lives of PH patients (and their families).

PHA staff make calls during
National PH Call-In Day 2014
April 16 is National PH Call-In Day – a day to make the halls of Congress ring with the PH community’s voice. You can take part in less than 10 minutes, without leaving your home.

Why me?
A friend of mine was talking with a staffer in Rep. Chris Van Hollen’s (D-MD) office last week. When she mentioned PH, the staffer jumped in and started to tell the story of a young woman who visited his office almost a year ago and shared her experience of being diagnosed while working in Afghanistan. He told me that he'll never forget the way she described her pain from having pulmonary hypertension: "I could breathe in, but my lungs didn't feel like they were getting any oxygen." He remembered her words a year later and said his boss, Rep. Van Hollen, remembers them too.  He was telling PHer Jessica Armstrong's story.

Everyone whose life has been touched by PH has a story to tell and your story can make a difference. Join PHers across the country on April 16 and make sure that every Member of Congress knows about PH.

How can I help?
PHers Jessica Armstrong (left) and Katie Tobias
advocate to the Food and Drug Administration
On April 16, PHers across the country will call their senators and representatives and ask them to co-sponsor the Patients’ Access to Treatments Act.

The bill aims to limit patients’ out-of-pocket costs for complex, life-saving medications by capping possible co-insurance. For PHers, that means a limit on what your insurance could require you to pay for infused and inhaled PH drugs. The bill is specific to commercial insurance, but its success would likely impact Medicare and Medicaid too.

Learn more and take action.

What Should I Do on April 16?
Follow these steps to add your voice on April 16:

  1. Use PHA’s tools to look up phone numbers for your senators and representatives and view a call script. All you need are your street address and zip code: www.PHAssociation.org/ActionAlert/PHNationalCallInDay 
  2. Make the calls! Ask your senators and representatives to co-sponsor the Patients’ Access to Treatments Act.
  3. Tell PHA … and your friends! Don’t forget to fill out the call feedback form that appears after the talking points. PHA will use this information to help hold your Members of Congress accountable. Then, post the alert to social media or email it to your friends. Anyone can join the “phight” on National PH Call-In Day!
  4. Congratulate yourself! Thank you for adding your voice to this critical issue!

For more information, or to join PHA’s grassroots advocacy network, contact Angelia at 301-565-3004 x753 or Advocacy@PHAssociation.org.

Wednesday, March 11, 2015

PVDOMICS and What It Means for the PH Community

Since 2000, PHA has created five distinct research programs through which we have committed almost $16,000,000. Each year, through those programs, we support about a dozen research projects. Each year, through our partnerships, our donors' research dollars are doubled.  

PHA's most recent research partnership - our second with NIH - will help expand a new program that is generating more excitement among researchers than anything we've seen before. PHA's $500,000 commitment, while adding 2 percent to the overall budget, is allowing the program to expand by 20 percent. We feel that's a great investment!

This week, I'm pleased to introduce PHA's Senior Director of Medical Services, Michael Gray, as our guest blogger. He explains the new PVDOMICS program and what it means to the PH community.  

As I turn this page over to Michael, I should tell you that PH is the first disease that will be studied in this way. NIH has told us we are preceding the diseases that will follow because of the strength and structure of the PH community. As part of this community, you are making this important advance possible.

"The only simple truth is that there is nothing simple in this complex universe. Everything relates. Everything connects"
-Johnny Rich, The Human Script

For centuries, scientists have been fascinated with how traits are passed from one generation to another. Significant progress has been made in this understanding, from Gregor Mendel’s discovery of the laws of inheritance with pea plants in the mid-nineteenth century to Dr. Marshall Nirenberg decoding the language of DNA at the National Institutes of Health (NIH) in the mid-twentieth century. Despite our increased understanding, we still have not solved how genes and changes to your genetic code called “mutations” affect most diseases, including pulmonary hypertension. A new initiative through the NIH, the “Redefining Pulmonary Hypertension through Pulmonary Vascular Disease Phenomics” (PVDOMICS) program, seeks to change that for this particularly devastating disease state.

Mutations, or accidental changes in the DNA code, can occur frequently as cells duplicate, and can even be passed onto the next generation when this mutation occurs in a sperm or egg cell. Mutations sometimes will cause no changes to the individual, sometimes will cause harm to the individual, and sometimes can even provide a benefit. Genetic changes like this have been directly implicated in a number of diseases, including cystic fibrosis, sickle cell anemia, Tay-Sachs disease, and phenylketonuria. Genetic changes have also been implicated in PH, most famously with the discovery of the “bone morphogenetic protein receptor, type II” (BMPR2) gene. However, we don’t know exactly how changes in the BMPR2 “code” lead to PH. We know that BMPR2 genetic mutations are present in more than 70 percent of people with hereditary PAH and 25 percent of people with idiopathic PAH; but having the genetic mutation does not mean you eventually have PAH.

Think of DNA like letters in a book. Individually, these letters have no meaning; together, they tell the story that makes you, you. This story can be changed in a number of ways. Certainly letters and even entire words can be added, removed or changed, which might or might not change the meaning of the story. But direct changes to the letters on the page is only one modification that can have an impact on how you read the story. Maybe your book has a sentence that has been highlighted, or bolded; the words and the meaning of this sentence have not actually changed with this modification, but it has been emphasized in a way that has consequences in context. Maybe your book has a stain on one of the pages masking several words; the words are still there, but hiding these words could change the entire meaning. These different types of changes to how the “story” of your DNA is told make up a web of interactions that can have consequences nearly as drastic as finding one disease causing “gene.” This web includes the molecules that take chapters of your DNA “story” from the nucleus to your entire cell (called RNA), the proteins that are made by this RNA and used by your entire body, and how your cell uses energy to name just three strands of this web.

The NIH in Bethesda, Md., is the world’s largest biomedical research institute, with more than 5,000 scientists working on a number of scientific advancements with basic scientists, physician scientists, patient advocacy organizations, pharmaceutical companies and, of course, patients and family members themselves. The NIH has a long history working in pulmonary hypertension, beginning more than 35 years ago with an early, important registry characterizing the devastating nature of PAH. Since then—with significant advances in the field including 12 PAH-targeted therapies, a successful surgical therapy for CTEPH, and a medical therapy for a specific form of CTEPH—the NIH continues to invest in successful PH research. The most recent demonstration of this is the PVDOMICS program.

PVDOMICS seeks to ask research questions about PH in a way that will lead to a better, more complete understanding of the disease and to developing therapeutics that are more precise by building a clinical research network of seven centers across the United States. They will be performing a comprehensive analysis on around 1,500 patients with all types of PH (WHO Groups 1-5), collecting a range of clinical information; blood for analysis of genetics and the molecules that turn on and off expression of these genes; and imaging studies normally used in the clinic to better understand the difference between normal and diseased lung vascular tissue. There are many benefits from analyzing these very different types of information together, according to the NIH. If the investigators are able to develop this robust, more complete understanding of why some people develop PH and others with similar risk factors do not, more sensitive measures of diagnosis, more uniform responses to medications, and new biomarkers of disease worsening could be discovered. It is anticipated that most WHO Group 1-5 pulmonary hypertension patients who want to participate in this research study may be able to enroll.

The Pulmonary Hypertension Association is a proud partner with the NIH in this important study, and in 2014 awarded a five-year grant of $500,000 to the program to support one of the clinical centers in collecting data from 250-300 patients. For more information on the PVDOMICS program, please see the Pathlight article on PVDOMICS from the winter 2015 issue.

Wednesday, February 25, 2015

Cybercondria and Pulmonary Hypertension Awareness

For this second guest blog post in our series of guest posts from PHA staff members, Jessica Armstrong, PHA’s Early Diagnosis Campaign Manager and a CTEPH patient, looks at an interesting Internet phenomenon called cyberchondria and what it could mean for pulmonary hypertension patients.

Last summer I came across a new term that I'd never heard before: Cyberchondria. As you might guess, it is defined by Wikipedia as “the unfounded escalation of concerns about common symptomology based on review of search results and literature online.” Put simply, a cyberchondriac is that person we all know who gets a cold, googles their symptoms, and becomes convinced they’ve contracted the bubonic plague. Although funny to think about, what's interesting is the sheer number of articles and guides that have become available geared toward advising healthcare providers on how to calm the cyberchondriac patient.

For patients with pulmonary hypertension, this phenomenon of the Internet age comes with a challenge: How does a rare disease patient differentiate him or herself from the cyberchondriacs of the world? The answer is to be informed.

We've already learned the value of educating ourselves, of being good investigators, and in being knowledgeable about our health. We’ve used this knowledge in advocating for ourselves, and frankly, that's how many of us arrived at the point of being diagnosed with pulmonary hypertension. By now most of us have learned that the need for self-advocacy doesn’t stop at diagnosis, which is why so many of us have continued our efforts to learn as much about pulmonary hypertension as we possibly can. This includes learning our WHO group classification, being an active participant in our own health, and hopefully staying organized via the Empowered Patient Toolkit.

Today, with 12 PH therapies available, it becomes more and more likely that we’ll have to deal with other health conditions and concerns in addition to our pulmonary hypertension, and it remains important that we continue to be good self-advocates and good stewards for health, while at the same time not falling into the trap of jumping to extreme conclusions (which is easy to do when you already have one rare disease).

Over the next few weeks, PHA will be launching the newest addition to the Early Diagnosis Campaign, our Patient Self-Advocacy Toolkit at www.SometimesItsPH.org/PatientSelfAdvocacyToolkit (not live yet). Unlike PHA’s existing Empowered Patient Toolkit, this resource is intended for any patient experiencing unexplained symptoms.

The goal of the Patient Self-Advocacy Toolkit is to provide information and tools for patients who are at that point that many of us were at shortly before we were diagnosed with PH - experiencing unexplained symptoms, uncertain of what’s happening, full of unresolved questions and concerns. Some of these patients may have pulmonary hypertension and some may not, but the goal is really to equip them to figure out what the problem is and to help them be organized and prepared as they embark on the journey toward diagnosis. We hope it will be something you’ll be proud to tell your friends and family about.

And for those of us who have already received our PH diagnosis, we hope this resource will prove a valuable tool as we approach other health concerns along the way. In particular, I’m excited that the Toolkit will provide a guide to finding credible health information online. This resource, which will be found under the ‘Be Informed’ section, will offer tips that will hopefully make it easier to identify good quality resources on the web. We hope that this resource will be something that you continue to utilize not only in regard to diagnosis of new concerns, but also as you continue to learn about your pulmonary hypertension.

And the good news is, if you’ve suffered from cyberchondria in the past, there will soon be a cure.

Wednesday, February 11, 2015

Let's Work Together to Advance the PH Cause

Over the next several weeks, I have invited PHA staff to offer guest blog posts about the work they are doing on behalf of the PH community. This first guest post in the series comes from Kim Lamon-Loperfido, PHA’s Patient & Caregiver Services Manager. Kim coordinates the Tom Lantos Innovation in Community Service Awards, and since its launch six years ago, the program has held true to the goals of supporting good ideas that can make a difference to the PH community. The Lantos Grants help patients and family members bring those ideas to reality for the betterment of all​.

Carmen Lozada-Bruno (right) received a
Lantos grant to distribute Spanish language
posters in Puerto Rico with information on
symptoms, diagnosis and the causes of PH.
I’m looking forward to another successful year with the Tom Lantos Innovation in Community Service Awards program this spring. This program allows members of the PH Community to fund and bring into reality their ideas. Last year we had 25 applicants and 13 funded projects.

Some fabulous projects came from 2014 like Brittany Evans, who along with the Generation Hope advisory board, created notepads printed with PHA’s most important web links for both patients and healthcare professionals. Her first distribution of this project took place at one of the healthcare professional meetings at PHA’s 2014 International PH Conference. Another project by Carmen Lozada-Bruno of Puerto Rico involved distributing Spanish language posters with information on symptoms, diagnosis and the cause of PH across Puerto Rico’s pharmacies, emergency rooms, hospitals and government offices. She received nothing but gratitude and positive feedback throughout the process.

I have had conversations with several community members who have experienced the spark of inspiration, but aren’t quite sure how to turn that spark into a full project for their application. Through these conversations, we have explored the primary goal they are trying to work toward, and from there, we’ve filled in details on how they can reach that goal. I’ve encouraged them and I encourage you to watch our helpful webinar on the process, which takes you from brainstorm to application and, finally, to successful project implementation. But I’m also here to help through any part of the process, and I can connect you with previous winners who are more than happy to mentor you in the process.

This program is now in its sixth year of funding projects to advance our shared cause. It provides grants of up to $5,000 each, and both domestic and international applications are welcome.

Projects over the past five years have raised awareness of PH, created PH resources, and educated others about PH. Those wishing to apply for funding should know their projects can also replicate the efforts from previous Lantos Awards, for example introducing a project to a new audience or adding a new component.

The grant program is named for the late Rep. Tom Lantos, a longtime congressman from California who was the original sponsor of our bill in Congress after his granddaughter was diagnosed with PH.

For more information or for help with your application, please reach out to Lantos@PHAssociation.org. I am so excited to work with you as you turn that spark of inspiration into a full-blown project to advance the PH cause. Although applications are due by March 25, 2015 – let’s chat today!

Wednesday, January 28, 2015

PHA Accredits 11 New Pulmonary Hypertension Care Centers

When I came to PHA in 1999, there were about 100 doctors in the U.S. treating pulmonary hypertension. Today there are more than 10,000. While that growth is good news, it’s also a call for clear standards to improve quality of care for patients. That is the purpose of PHA’s new PH Care Centers (PHCC) accreditation program. The program was launched on Sept. 27, 2014, after three years of organization and development by committees of leading medical professionals.

Today's news is that PHA recently accredited 11 Pulmonary Hypertension Care Centers, bringing the total number of PHCCs to 17, in a program that seeks to improve overall quality of care and ultimately improve outcomes of patients with pulmonary hypertension. This includes promoting improved diagnostic and treatment standards in the field of PH, while creating unique partnerships between accredited centers and PHA.

PHA is still a relatively small disease organization. For us to now be accrediting PHCCs is a great accomplishment. I believe this comes from our unique relationships with medical professionals and patient populations working together to find a cure for this devastating disease. We have come a long way in changing the trajectory of this disease and improving the lives of patients in the race for a cure.

These first phases of accreditation are open to centers applying as Comprehensive Care Centers (CCC). We expect that to result in more than 90 applicant Centers. (We are currently over 35.) In 2016, applications for Regional Clinical Programs (RCP) will begin to be accepted as RCP criteria requires collaboration with regional CCCs. By linking accredited CCC and RCP centers together, the program will improve knowledge among professionals and patients about where to seek quality care.

PHCC accreditation signifies that a PH program has demonstrated a dedication to making a proper diagnosis and has the capacity to appropriately and comprehensively manage PH patients through a set of criteria. Both CCCs and RCPs will be accredited by PHA after undergoing a thorough evaluation and satisfactorily meeting the respective standards set forth in the PHCC Criteria. In accordance with the program’s vision, all PHA-accredited PHCCs will be expected to uphold the principles of delivering appropriate and effective care, offering research participation opportunities and providing appropriate support to PH patients.

To see the full list of nationwide accredited PHCC Centers of Comprehensive Care, please visit our website.  In looking at this information, it's important for everyone to understand that this process is just beginning. Many excellent Centers have not yet been reviewed or begun the complex application process.  It will take well over a year for PHA to work through national-level accreditations, after which Regional Center accreditations will begin.

The PHCC online application portal was also launched this month with high interest in the program resulting in a steady increase of application submissions. Many site reviews are being scheduled for the coming months. Centers interested in applying can access the online application at the PH Care Centers Site Application Portal.


For additional information on PHCC and the application process, go to: www.PHCareCenters.org or email PHCC@PHAssociation.org.

Please join me in congratulating the newly accredited centers. With the hard work and dedication of these Centers, the diligence of the medical community and patient population to get the best care possible, and the work of our staff at PHA to coordinate initiatives and raise awareness about PH, we are closer to a cure than we have ever been.