The Rare Disease Congressional Caucus hosted a briefing during Rare Disease Week on Capitol Hill which focused on Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform.
FasterCures, a non-profit think tank with the goal of speeding and improving the medical research system, recently debuted a new resource to track implementation of the 21st Century Cures Act.
PDUFA enables FDA to collect user fees from biopharmaceutical companies in order to enable the Agency to review the safety and efficacy of new medicines more quickly. It is important for PDUFA to be reauthorized by the end of July or FDA will not be able to accept the user fees and would need to send furlough notices to staff.
More than 600 rare disease patients, caregivers, researchers and other advocates joined us during Rare Disease Week on Capitol Hill in Washington, DC from Monday, February 27th, through Thursday, March 2nd.
There are a few things you can do to prepare to ensure a productive experience at Rare Disease Week at Capitol Hill and minimize last-minute stress.
On January 23rd, President Trump instituted an immediate hiring freeze that would affect many federal agencies including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Unfortunately, this freeze could significantly hamper research into rare disease treatments as well as the review and approval of new medicines. The EveryLife Foundation for […]
Kids v Cancer has put together a yearbook summarizing the achievements of pediatric cancer advocacy organizations in 2016 and outlining steps that pediatric cancer organizations plan to take in 2017.
If you are not able to join us and advocates from across the country at Rare Disease Week on Capitol Hill on February 27th through March 2nd, you can still make your voice heard on Capitol Hill and participate in some of the programming.
The webinar will feature Michael Werner, Executive Director for ARM, as well as a representative from the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research. Questions will be welcome.
As we noted in a news brief last month, the law firm of Hyman, Phelps & McNamara P.C. has featured several blogposts on different provisions of 21st Century Cures Act. They will host two free webinars this month to further examine the Act, which will be good opportunities for advocates to learn more and ask […]