10th Annual Rare Disease Scientific Workshop Conceptualizing a Rare Disease Center of Excellence at the FDA

The EveryLife Foundation convened its 10th annual Rare Disease Scientific Workshop, titled “Conceptualizing a Rare Disease Center of Excellence at the FDA,” in Washington, DC on Sept. 13, 2018. The goal of the workshop was to gather key thought-leaders from industry, the Food and Drug Administration (FDA), and patient organizations to discuss potential models, best practices and the pathway forward.

Senior-level officials from the FDA and National Institutes of Health (NIH) presented, along with representatives from industry and patient advocacy organizations. The video of this event, arranged in discrete, consecutive-speaker-order, is available online now.

FDA to Spend $2M on Rare Disease Research

The U.S. Food and Drug Administration (FDA) is making $2 million available in new research grants to fund natural history studies in rare diseases. The aim is to collect data on how specific rare diseases progress in individuals over time. This knowledge can inform and support drug product development and approval. This will be the first time the FDA will provide funding through its “Orphan Products Grants” to conduct natural history studies for rare diseases.

Natural history is the course a disease takes in affected individuals from the time immediately prior to its inception, progressing through a pre-symptomatic phase and different clinical stages, to a final outcome in the absence of treatment. This type of information is often not available, or incomplete, for many rare diseases, and this makes the drug-approval process additionally difficult.

The funding levels and duration of these grants will include:

● A maximum of $400,000 in total costs per year for up to five years for prospective (looking forward) natural history studies involving clinical examination of affected individuals.

● A maximum of $150,000 in total costs per year for up to two years for retrospective (looking back) natural history studies (i.e., chart review) or survey studies (i.e., questionnaire).

Grant applications are due not later than Oct. 14, 2016. All responsive applications will be reviewed and evaluated for scientific and technical merit by a panel of rare disease and natural-history experts. The anticipated start date of funding for grantees is March 2017.

Visit: FDA press release about $2m in new grants for natural history of rare diseases