Just a few years after the 1996 approval of the first treatment for pulmonary hypertension, Glaxosmithkline plc's epoprostenol, a two-line advertisement in The Chronicle of Philanthropy drew nonprofit consultant Rino Aldrighetti's attention: "Small rare disease association looking for part-time executive director."

Interested in the opportunity, he sent a letter of inquiry. The reply came in the form of an invitation to a board meeting of the then-nascent Pulmonary Hypertension (PH) Association, a group dedicated to supporting people fighting the condition, a rare disease in which tiny arteries in the lungs become narrowed or blocked, causing high blood pressure in the lungs.

At the board meeting, in a restaurant in Delaware, a serious conversation unfolded among a small group of women, each pulling tall oxygen tanks: Would they hire Aldrighetti to be their very first employee? "What I heard people say in different ways throughout the day was, 'This decision may not help me, but it will probably help the next generation of patients,'" he told BioWorld Today.

Little did Aldrighetti know, the decision would also have a tremendous impact on him, leading to a 17-year stint carrying the organization through an expansive period during which the number of drugs for PH would multiply from one to 14 approved therapies and the association's growth would significantly accelerate. What started as a group with a modest budget of $132,000 in 1998 was transformed during that period into an organization with $444,000 of income in the next year and $1.1 million of income the next. Aldrighetti, who has now helped shepherd the group to its 25th anniversary this year, will retire in June.

Prior to the relatively recent discovery of targeted therapies, patients diagnosed with the disorder averaged less than three years of post-diagnosis survival. With prompt diagnosis and adherence to newer therapeutics, some patients now live for 10 years or more. There are an estimated 30,000 PH patients in the U.S., though some believe that estimates are hard to pin down because PH is often misdiagnosed.

As the PHA has grown, it has become clear that organizing the community has led to concrete positive outcomes. For instance, the same scientists that the group recruited to its scientific advisory board were, in many cases, later recruited to lead trials of new drugs for the condition. New drugs launched during that period have included Actelion Ltd.'s selective prostacyclin IP receptor agonist, Uptravi (selexipag), United Therapeutics Corp.'s Remodulin and Orenitram (treprostinil) and Bayer AG's Adempas (riociguat). (See BioWorld Today, Oct. 24, 2012, and Dec. 23, 2015.)

The quarterly medical journal that the association started to raise awareness and share information about the area, Advances in Pulmonary Hypertension, has, despite an uncertain start, now published every quarter since 2002, reaching an estimated 50,000 cardiologists, pulmonologists and rheumatologists with new information about what was once a little known disease.

Aldrighetti has also helped guide the creation of the Pulmonary Hypertension Association Registry, a tool that PHA hopes will play a key role in developing new and better treatments and methods of care, eventually helping pave the way to a cure. It's now being piloted with 40 patients. Within five years, the group hopes to have 8,000 patients enrolled, making it the largest ever registry in PH.

Much of the association's progress has been made possible by a collaborative approach not only between patients' families and medical professionals, but also with a corporate committee that explores new avenues for therapy and education. But Aldrighetti has also been conscious of carefully handling those ties, he said.

"As we look at that, there's no question that we also have to build walls, so that industry does not influence medical education," said Aldrighetti. Frank and open conversations in times of industry overreach have "helped us to make sure that industry is supportive in an appropriate way," he said. "In 17 years, it has been a pretty honest relationship."

The connections the association has helped foster through 245 support groups across the U.S. have supported patients in not only training each other about what it means to live with the condition, but also supported them in mourning each other when they had a loss.

"We've grown because we've been able to pull people together," said Aldrighetti.

He views the PHA team, which now includes a staff of 50 people, as facilitators. The group's mantra, he said, is "any person whose life is touched by PH has the right to fight back as much or as little as health and interests allow. Our job is to make it possible."

As he prepares to retire on June 17, Aldrighetti's plans have gone through a series of changes. His wife was diagnosed with cancer several months ago, so that will become his primary focus. If time and health allow, he'd also like to set up a tropical fish tank. But he would like to find time to provide mentorship and guidance to some of the many individual leaders who take up the hard work of building a small rare disease organization association with hopes of making a dent in a disease impacting their lives.

As the former Foundation of the American Academy of Ophthalmology executive director, Brad Wong, prepares to take the reins as president and CEO of the association, Aldrighetti expects to see the PHA advance further down the path toward transforming the condition into one with which people can enjoy a normal life span with a good quality of life. The transition of that responsibility to Wong will coincide with the association's International PH Conference and Scientific Sessions, June 17-19, in Dallas.

No Comments