FDA providing $2 million in new grants for natural history studies in rare diseases

FDAThe U.S. Food and Drug Administration today announced the availability of $2 million in 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 so that knowledge can inform and support product development and approval. This will be the first time the FDA will provide funding through its Orphan Products Grants to conduct these types of studies for rare diseases.

Natural history is the course a disease takes in affected individualsfrom 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. This type of information is often not available, or incomplete, for many rare diseases.

"Rare diseases are often poorly understood," said Gayatri Rao, M.D., J.D., director of the FDA's Office of Orphan Products Development, within the Office of Special Medical Programs. "Not understanding how a rare disease progresses is often a major obstacle in the development of life-saving medical products. Information about a disease’s natural history can aid in clinical trial design, identify study end points and lead to faster, better trials - hopefully leading to new and effective diagnostics and treatments."

The goal of the 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.

"Despite their importance, it is very difficult for groups, such as patient advocacy organizations, to find funding to conduct natural history studies," said Dr. Rao. "As we observe Rare Disease Day today, we are excited to announce a program that will bridge some of this funding gap and complement existing efforts to help bring new and important therapies to rare disease patients."

Congress appropriates funding for the study of rare diseases. FDA is using the funds for this new Orphan Products Natural History Grants Program as well as the existing Orphan Products Grants Program for clinical trials, which has provided more than $350 million to fund more than 570 new clinical studies and has supported the marketing approval of more than 55 products since its creation in 1983.

Grant applications will be due 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.

The funding levels and durations 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).

Rare diseases, as defined in the Orphan Drug Act, are diseases or conditions 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.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

Therapy using dual immune system cells effectively…

A newly developed immunotherapy that simultaneously uses modified immune-fighting cells to home in on and attack two antigens, or foreign substances, on cancer cells was ...

Cleveland Clinic study suggests steroid nasal spra…

A recent Cleveland Clinic study found that patients who regularly use steroid nasal sprays are less likely to develop severe COVID-19-related disease, including a 20 to 2...

How to develop new drugs based on merged datasets

Polymorphs are molecules that have different molecular packing arrangements despite identical chemical compositions. In a recent paper, researchers at GlaxoSmithKline (GS...

New drug combination effective against SARS-CoV-2 …

More countries with greater resources are opening up for a more normal life. But COVID-19 and the SARS-CoV-2 virus are still a significant threat in large parts of the wo...

Sanofi to focus its COVID-19 development efforts o…

Recent positive interim results of Sanofi's mRNA-based COVID-19 vaccine candidate Phase 1/2 study confirm the company's platform robust capabilities and strategy in mRNA...

Discovery of mechanics of drug targets for COVID-1…

A team of international researchers, including McGill Professor Stéphane Laporte, have discovered the working mechanism of potential drug targets for various diseases suc...

Phase II/III trial shows Ronapreve™ (casirivimab a…

Roche (SIX: RO, ROG; OTCQX: RHHBY) today confirmed positive data from the phase II/III 2066 study, investigating Ronapreve™ (casirivimab and imdevimab) in patients hospit...

Pfizer and BioNTech receive first U.S. FDA Emergen…

Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) announced that the U.S. Food and Drug Administration (FDA) has authorized for emergency use a booster dose of the P...

AZD7442 request for Emergency Use Authorization fo…

AstraZeneca has submitted a request to the US Food and Drug Administration (FDA) for an Emergency Use Authorization (EUA) for AZD7442, its long-acting antibody (LAAB) com...

Pfizer and BioNTech receive CHMP positive opinion …

Pfizer Inc. (NYSE: PFE, "Pfizer") and BioNTech SE (Nasdaq: BNTX, "BioNTech") today announced that the Committee for Medicinal Products for Human Use (CHMP) of the Europea...

Boehringer Ingelheim acquires Abexxa Biologics to …

Boehringer Ingelheim announced the acquisition of Abexxa Biologics Inc., a biopharmaceutical company taking a new approach in the fields of immuno-oncology and oncology r...

GSK welcomes WHO recommendation for broad roll-out…

GlaxoSmithKline (GSK) plc welcomes and applauds the WHO recommendation for the broader deployment of GSK's RTS,S malaria vaccine to reduce childhood illness and deaths fr...