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AMWA Participates in first White House Rare Disease Forum

More than 80 advocates for patients with rare diseases gathered yesterday, February 28, for the first White House Rare Disease Forum. Attendees included families, community leaders, advocacy organizations and innovators, all committed to delivering progress to address the nearly 10,000 known rare diseases that impact up to 30 million Americans.

AMWA was an invited guest due to our particular interest in rare diseases. Women often face delayed diagnoses for a variety of diseases.  The mean number of years that it takes to get a diagnosis of a rare disease is six. “Put these two together and they cause women with rare diseases to face even longer delays to inaccurate diagnoses,” said Lisa M.. Tate, AMWA, Director of Partnerships & Development, who attended the forum.

One announcement brought much applause from the audience. The Advanced Research Projects for Health (ARPA-H) is launching a new $48 million project that invests in a novel, AI-driven platform to repurpose existing drugs to address rare and other diseases currently lacking treatment options. There are 9,000 rare diseases without any approved treatment.

Dr. Monica Bertagnolli, Director of the National Institutes for Health (NIH), reiterated NIH’s commitment to advancing understanding and approaches to rare diseases and made it clear that NIH’s role is not complete until new treatments are having an impact on people’s lives.

The speaker lineup also included the President’s chief advisor on science and technology Arati Prabhakar, who spoke about the need for progress against rare diseases so that all Americans can be part of the President’s vision for an America defined by possibilities.

Danielle Carnival, Deputy Director for Health Outcomes at the White House Office of Science and Technology Policy, noted that, “We celebrate the triumphs and progress, we learn from the experiences and expertise of those living with a rare disease, and we face head-on the many challenges that still remain: namely, to shorten the time to an accurate diagnosis, to reach more people with the tools we already have to diagnose and treat some rare diseases, and to drive new discovery and the development of effective therapies.”

L-R: Dr. Monica Bertagnolli (Director, NIH), Lisa Tate

Dr. Monica Bertagnolli, NIH Director, Lisa Tate (R)

L-R: Lisa Tate with Tamar Thompson (Head, Global Corp Affairs) and Christina Hochul (Head, Strategi Alliance Dev) of Alexion

L-R: Lisa Tate with Tamar Thompson (Head, Global Corp Affairs) & Christina Hochul (Head, Strategic Alliance Dev), Alexion (AstraZeneca Rare Disease)

L-R: Kimberly McClellan (Recurrent Resp Papillomatosis Fdn), Emily Milligan (Barth Synd Fdn), Lisa Tate


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