Developing Products for Rare Diseases & Conditions
NEW: FDA’s Overview of Catalyst Pharms., Inc. v. Becerra. Please click here to read more.
During this public health emergency associated with the COVID-19 pandemic, the Office of Orphan Products Development (OOPD) is providing sponsors with increased flexibility for submission of orphan drug designation requests and related submissions (amendments, annual reports, etc.). During this public health emergency, orphan drug designation, humanitarian use device designation, and rare pediatric disease designation requests and submissions may be submitted electronically by email to the OOPD.
When transmitting information to the Orphan Drug Designation Program via email, please utilize the mailbox [email protected]. The use of automated read receipt is recommended to avoid the need to call to verify receipt of the email. Sponsors and others who plan to email information to FDA that is considered to be private, sensitive, proprietary, or commercial confidential are strongly encouraged to send it from an FDA secured email address so the transmission is encrypted. The OOPD will assume that the addresses of emails received or email addresses provided as a point of contact are FDA secure when responding to those email addresses.
Sponsors and others can establish a secure email address link to FDA by sending a request to [email protected]. There may be a fee to a commercial enterprise for establishing a digital certificate as part of the set-up process before emails can be sent to FDA encrypted.
On this page:
About OOPD
OOPD Programs
Resources
OOPD Contact Information
About OOPD
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. The office also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.
OOPD provides incentives for sponsors to develop products for rare diseases. The program has successfully enabled the development and marketing of over 600 drugs and biologic products for rare diseases since 1983. In contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983. The Orphan Grants Program has been used to bring more than 60 products to marketing approval. The Humanitarian Use Device Program has been the first step in approval of 70 Humanitarian Device Exemption approvals.
OOPD Programs
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.
The Rare Pediatric Disease Priority Review Voucher Program says that a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
The Humanitarian Use Device (HUD) program designates medical devices that are intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year as eligible for Humanitarian Device Exemption.
The Orphan Products Grants Program provides funding for clinical trials and natural history studies that 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.
Resources
- FDA’s Overview of Catalyst Pharms., Inc. v. Becerra
- Recommended Tips for Creating an Orphan Drug Designation Application (slides and script)
- FDA Guidance on Clinical Trial Conduct during COVID-19
- FDA awards 6 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
- Draft Guidance for Rare Pediatric Disease Priority Review Vouchers
- FDA Orphan Drug Designation Request Form
- Tutorial: Recommended Tips for Creating an Orphan Drug Designation Application
- Orphan Drug Modernization Plan and Updates
- Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations
- Draft Guidance for Rare Pediatric Disease Priority Review Vouchers
- Guidance for Industry Clarification of Orphan Designation of Drugs and Biologics for Pediatrics
- Rare Diseases: Common Issues in Drug Development Guidance for Industry
- Interpreting Sameness of Monoclonal Antibody Products Under the Orphan Drug Regulations
- Guidance for Industry, Researchers, Patient Groups and FDA Staff on Meetings with OOPD
- Guidance for Industry and FDA Staff - Humanitarian Use Device (HUD) Designations
OOPD Contact Information
For more information on any of our programs, please visit the programs’ web pages.
Office of Orphan Products Development
Food and Drug Administration
WO32-5295
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
Main Telephone Number: 301-796-8660
Fax Number: 301-847-8621
Email: [email protected]
