AYAs don’t fit into the current health system, which is set up for children and adults. Luckily, that is changing,” said Hilary Gan, director of hospital programs and services at Teen Cancer America, who spoke virtually from Los Angeles.īut difficulties remain and likely resonate among AYAs with any rare disease. “This age group had not seen improvement in survival of older and younger cancer patients in almost 30 years. One panel focused on the needs of AYAs with cancer. Several panels were devoted to the challenges and needs of adolescents and young adults (AYAs)-those 15-39 years old-living with rare diseases. “This idea will allow faster clinical development with more real-time FDA-sponsored communications, which can ultimately move products as fast as possible through the regulatory pipeline,” said Rutter. The Food and Drug Administration (FDA) is gearing up for a pilot program to authorize therapeutics that show promise. Rutter noted that NCATS is developing platform-based technologies to enable more treatments to reach patients faster. NCATS plays a leadership role in each of these initiatives. He outlined several initiatives-the Accelerating Medicines Partnership-Bespoke Gene Therapy Consortium, the Somatic Cell Genome Editing program and the Platform Vector Gene Therapy project-all underway at NIH to help speed development of new gene therapies. Lawrence Tabak, performing the duties of NIH director. Most rare diseases emerge from a single gene variant, “so gene therapy is potentially a powerful approach for treating a wide range of rare diseases, including those viewed as too rare for commercial development,” said Dr. Joni Rutter, “to celebrate the big and small wins, to support those who work tirelessly in caring for people with rare diseases, to honor all people with a rare disease and recognize that struggle, and to hear about new ideas and projects that spell hope.” Treatments on the HorizonĮvent participants gather to chat near the All of Us Research Program table. “This day is about pausing to remember loved ones we’ve lost,” said NCATS Director Dr. Several members of the Rare Disease Congressional Caucus delivered virtual remarks, voicing their support for continued research to find new therapies for rare ailments. Most rare diseases have few if any effective treatments.Ĭritical to finding new therapies is securing science funding, an effort driven by a cadre of bipartisan policymakers and legislators who support the research. There are more than 10,000 different rare diseases affecting more than 30 million people nationwide and more than 350 million people worldwide. It’s also a day when the heart renews hope and finds sources of new inspiration.”Īisha Dubose Campbell (l), director of resident services and family programming at the Children’s Inn, describes resources for young patients who stay at the inn, many of whom are adolescents and young adults.Ī rare disease is one that affects fewer than 200,000 people in the U.S. James Gilman said, “This is a day when the head acquires new information and knowledge. Several panels focused on an often-underserved population in the rare disease world-adolescents and young adults.ĭuring welcoming remarks, Clinical Center CEO Dr. The day’s sessions expounded on rare disease clinical research and resources as well as ways to improve the patient experience.
The Natcher Conference Center was abuzz with hundreds of in-person attendees while many more tuned in via videocast for the hybrid event, co-sponsored by the National Center for Advancing Translational Sciences (NCATS) and the Clinical Center. People with rare diseases and their loved ones, clinicians, researchers and patient advocates were among the more than 2,000 participants in this year’s Rare Disease Day at NIH on Feb. Joni Rutter (r) poses with Grace Moon of Mitre, a non-profit that supports NIH and other federal health projects.
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