#NORD – Pulmonary Hypertension on Agenda of NORD’s Oct. 16-17 Rare Disease Summit

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by Larry Luxner |

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A top pulmonary hypertension expert will be among more than 80 speakers at this month’s Rare Disease & Orphan Products Breakthrough Summit in Washington.

The two-day event Oct. 16-17, which is in its sixth year, will be hosted by the nonprofit National Organization for Rare Disorders (NORD). It is expected to draw 550 to 600 participants to the Marriott Wardman Park Hotel.

Michael Patrick Gray, senior director of medical services at the Pulmonary Hypertension Association (PHA), will be one of four panelists in a breakout session whose subject is promoting earlier diagnosis of the disease.

Gray oversees his organization’s professional medical membership groups — PH Professional Network (PHPN) and PH Clinicians & Researchers (PHCR). He also works closely with PHA’s Scientific Leadership Council — which provides leadership and guidance for the Silver Spring, Maryland-based nonprofit group — and helps develop its medical programming.

“One of the most wonderful things about this conference — and the thing that makes it unique — is the fact that it brings together the entire rare disease community,” Mary Dunkle, NORD’s vice president of educational initiatives, told Pulmonary Hypertension News in a phone interview. “Roughly one-third of our participants represent patient organizations. Some of these are very well-established, have existed for many years and have a high degree of sophistication about the research process.”

But others, she said, “are brand-new organizations, and this will be the first time their representatives have come to a major conference of this type. Many of these are kitchen-table groups run entirely by volunteers.”

The topics on this year’s agenda will include rising healthcare costs, patient assistance programs, orphan drug development and controversial right-to-try legislation. These are laws that some states are passing that allow patients to try unapproved drugs.

Of the nearly 600 people who attended last year’s NORD summit, 32 percent represented advocacy or patient groups. The conference also attracted consultants (23 percent of the total), pharmaceutical executives (23 percent); government officials (13 percent); journalists (5 percent), scientists and researchers (3 percent) and investors (1 percent).

Dunkle, who’s been with NORD since 1999, said this year’s speakers include Dr. Scott Gottlieb, commissioner of the U.S. Food and Drug Administration, and Dr. Janet Woodcock, director of the agency’s Center for Drug Evaluation and Research.

Several speakers will also be coming from the National Institutes of Health, led by Dr. Petra Kauffmann, director of clinical innovation at the NIH’s National Center for Advancing Translational Studies, and Dr. Carrie Wolinetz, the agency’s associate director for science policy.

“This is a really great opportunity for the patient organizations to learn what these agencies do, and how they can interact with them,” Dunkle said. She added that she’s especially interested in hearing what Gottlieb — who became head of the FDA this year — has to say.

“Everybody’s looking forward to Dr. Gottlieb’s presentation,” she said. “He’s really hit the ground running. He’s shown great leadership and an interest in addressing issues and challenges, and, I think, a great interest in the needs of the rare-disease community.”

Another highlight of the NORD summit, she said, will be a talk by Dr. David Lebwohl, a senior vice president at Novartis, on the promise of gene therapy. His speech comes a little over a month after the FDA’s historic approval of Kymriah (tisagenlecleucel) to treat children and young adults with a form of acute lymphoblastic leukemia — making it the first gene therapy available in the United States.

Other company presentations will include one on advances in genetic testing by Dr. Robert Nussbaum, chief medical officer of Invitae, and a roundtable discussion on patient access programs led by Jenica Stroock, director of corporate responsibility at Pfizer.

Lending an international perspective to the conference will be a talk by Matthieu Boudes, operations and projects manager at the Paris-based European Organisation for Rare Diseases (Eurordis). In addition, a Chinese scientist working in Hong Kong will present her poster, “The Unbearable Weight of Being Rare: Results from the First National Survey Among People Living with Rare Diseases in China.” And Dr. Ramaiah Muthyala of the University of Minnesota will discuss the state of rare diseases in India.

The lineup will also include a talk by Dr. Marshall Summar, director of the Children’s National Rare Disease Institute in Washington. The institute houses NORD’s first Center of Excellence. The centers will be focusing exclusively on advancing the care and treatment of people with one of the world’s 7,000 or so rare genetic diseases.

“One of the chief challenges of 21st-century pediatric medicine is our continued inability to provide more help to children born with rare genetic diseases,” Summar said in a press release to mark the center’s inauguration in January 2017. “Collectively, these conditions impact one out of every 10 Americans and account for up to 40 percent of pediatric medical expenses.”

One controversial subject on the NORD agenda will be the right-to-try debate. It will be the focus of a panel moderated by Kate Rawson, contributing editor at Provision Policy.

“Some states are introducing right-to-try legislation, which has to do with giving patients access to therapies that are not yet approved,” Dunkle explained. “NORD generally does not support such legislation, because we feel it kind of cuts the FDA out of the process, and the FDA does have a program we feel works pretty well in giving people access to investigational therapies.”