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Medical products for rare diseases and conditions

Rare diseases affect patients and their families. Over 7,000 rare diseases affect more than 30 million people in the United States. Many rare conditions are life threatening and most do not have treatments.

The FDA works to enhance to the availability of treatments for rare diseases by evaluating information from product sponsors to determine if drugs meet the criteria for certain incentives and administering grants to provide funding for research on rare diseases.

Incentive programs for rare disease medical products

The Orphan Drug Designation program provides orphan status to drugs and biologics for rare diseases that meet certain criteria. Orphan drug designation provides incentives including:

  • Tax credits for qualified clinical trials
  • Exemption from user fees
  • Potential for seven years of market exclusivity after approval

Under the Rare Pediatric Disease Priority Review Voucher program, FDA determines if drugs or biologics meet the definition for treating or preventing a rare pediatric disease to grant rare pediatric disease designations which may provide the opportunity for products to qualify for a priority review voucher.

The Humanitarian Use Device (HUD) program designates medical devices that are intended to treat or diagnose a disease or condition that affects 8,000 individuals or fewer in the U.S. The HUD program creates an alternative pathway for getting marketing approval for medical devices that may help people with rare diseases or conditions.

Grant programs to support medical product development for rare diseases

FDA also administers grant programs to provide funding for research on rare diseases. These grant programs include:

The Orphan Products Grants program provides funding for clinical trials and natural history studies to help advance rare disease medical product development.

The Pediatric Device Consortia (PDC) Grants program provides funding to develop nonprofit consortia to facilitate pediatric medical device development.

The Rare Neurodegenerative Disease Grant program awards grants and contracts to public and private entities to cover costs of research and development for interventions intended to prevent, diagnose, mitigate, treat, or cure ALS and other rare neurodegenerative diseases in adults and children.


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