On April 18, U.S. Food and Drug Administration (FDA) Commissioner Marty Makary announced plans to roll-out a new approval pathway for rare disease drugs. Commissioner Makary’s comments build on sentiments expressed across both the patient community and industry that rare disease drug development needs greater regulatory flexibility in order to…
Attendees at this year’s symposium were optimistic about the potential for progress, citing momentum from new FDA initiatives, growing legislative support, and increased global innovation in research and development. These efforts, alongside increased patient advocacy and a presidential administration focused on speeding patient access, could lead to significant advances in…
Goodwin’s Life Sciences team is excited to host its Annual Rare Disease Symposium in Boston on February 5, 2025. Participants are invited to join for an afternoon of engaging fireside chats, inspirational presentations, and networking with peers in the rare disease community. Please see the agenda below and register to…
On October 4, 2024, a US House version of the revised Promising Pathway Act (PPA) 2.0 was introduced, sponsored by Rep. Bruce Westerman (R-AR). The bill (H.R.9938) mirrors a US Senate version that was introduced in May 2024 (S.4426) that would authorize the US Food and Drug Administration (FDA) to…
Support Goodwin’s DC Life Sciences Team as they raise money for the National Organization for Rare Disorders (NORD) as part of its “Running for Rare” team at the annual 10K race held during the upcoming DC Marine Corps Marathon on October 27th. Assist NORD’s mission to drive public policy, accelerate…
In October, NORD will also hold its annual Breakthrough Summit in Washington, DC on October 20-22, 2024. This event draws over 1,000 attendees including patients/caregivers, patient advocacy organizations, and healthcare, biotech, and medical technology companies. Registration is available here. This year, Matt Wetzel will take part in a panel discussion…
Goodwin’s Life Sciences team will be hosting an upcoming event in our Boston office on March 13, 2024 to spotlight the critical work being done to address the 7,000+ rare diseases that impact more than 300 million people globally. Join us in person in our Boston office or attend virtually…
Last month, FDA issued a Request for Information (RFI) in the Federal Register seeking information and comments from interested stakeholders regarding “critical scientific challenges and opportunities to advance the development of individualized cellular and gene therapies (CGTs).” Individualized CGTs are therapies “developed for a single patient (or a very small…
Investigators interested in rare disease treatment development have the opportunity to approach drug and biologic developers to obtain investigational drug supply for trials in which the investigators, typically at academic institutions, act as sponsor-investigators. Similarly, companies open to extending their product development pipelines can look to investigator-initiated trials as a…
In global observance of Rare Disease Day, Goodwin invites you to join us for a special awareness event on March 1, 2023 in our Boston office or virtually for those attending remotely to spotlight the critical work being done to address over 7,000 rare diseases that impact more than 300…
