FDA to Fund Natural History Studies for Rare Diseases

Rare Disease Day 2016: FDA Announces Grants for Natural History Studies in Rare Diseases

FDA's Office of Orphan Products Development Funds Research for Rare Diseases

On Monday, February 29th, FDA celebrated the ninth annual Rare Disease Day by announcing its new grant program, which will award $2 million in research grants to fund natural history studies for rare diseases.

What is Rare Disease Day?

The Orphan Drug Act defines a rare disease as a disease or condition “with a prevalence of less than 200,000 persons in the United States. Though the diseases are individually rare, together, the 7,000 known rare diseases affect approximately 30 million Americans.”

Rare Disease Day is held every year on the last day of February in an attempt to raise awareness about rare diseases and their impact on patients’ lives around the world. Although it initially began as a European event in 2008, Rare Disease Day quickly became a worldwide phenomenon with the U.S. joining the event in 2009.

Natural History Grants Program

With the launch of a new grant program to fund research for rare diseases in the U.S., the FDA made Rare Disease Day 2016 a date to remember for many. The program was developed “to collect data on how specific rare diseases progress in individuals over time so that knowledge can inform and support product development and approval.” This will be the first time the FDA has provided funding for these types of studies for rare diseases, and it will do so through its Orphan Products Grants Program.

In a recent press release, FDA defined natural history as “the course a disease takes in affected individuals from the time immediately prior to its inception, progressing through a presymptomatic phase and different clinical stages, to a final outcome in the absence of treatment.” For most rare diseases, natural history data and information is either not available or incomplete.

Natural history studies, defined as studies “that follow a group of people over time who have, or are at risk of developing, a specific medical condition or disease,” generally require a large amount of time and resources to conduct. As such, despite their significance, it can be very difficult for patient advocacy organizations and other [similar] groups to receive funding and allocate time for these types of activities.

The goal of FDA’s grant program is “to help characterize the natural history of rare diseases, identify subpopulations, and develop and/or validate clinical outcome measures, biomarkers and companion diagnostics.” Furthermore, Gayatri Rao, MD, JD, director of the FDA’s Office of Orphan Products Development (OOPD) stated that the Agency is very excited about this program, which will hopefully bridge the funding gap and supplement recent efforts to develop new therapies for patients suffering from rare diseases for which no treatments are currently available.

Organizations interested in applying for the grant program must submit applications to the FDA by October 14, 2016. All “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.” Additionally, FDA’s recent press release provides funding levels and durations of the grants, which 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).”
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