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CBER Rare Disease Program Frequently Asked Questions

  • What are the CBER development and approval processes for medical products including those for rare diseases?
    • CBER reviews Investigational New Drug Applications (INDs) and Biologics License Applications (BLAs) for biological products used in the treatment of rare diseases in accordance with federal laws including the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act. CBER’s development and approval process webpage provides a description of the various regulatory approval and notification processes, including BLAs, Premarket Approvals (PMAs), New Drug Applications (NDAs) and 510(k)s.
  • How can patients and caregivers contribute to the development and regulatory review of products for rare diseases?
    • Patient input is a critical part of CBER’s understanding of diseases and conditions. Engaging with patients and caregivers, and listening to their perspectives on their diseases and treatments, may help us advance patient-focused medical product development. CBER’s patient engagement program is committed to incorporating patient involvement in the center’s regulatory work in accordance with relevant provisions in the 21st Century Cures Act of 2016 and the Food and Drug Omnibus Reform Act of 2022 (FDORA), as well as in commitments under PDUFA VI, authorized under the Food and Drug Administration Re-authorization Act (FDARA) in 2017, and PDUFA VII, authorized in part by the FDA User Fee Reauthorization Act of 2022.  

      Furthermore, FDA offers the patient community a web platform, Patients Ask FDA, which is designed to encourage patients, caregivers, patient groups, patient advocates, and health care professionals to ask a question, request a meeting, or request a Patient Listening Session for all the FDA medical product Centers (biologics, drugs, and devices), and provide effective and efficient responses. Note: This webform is not for industry stakeholders.

      Learn about FDA’s Patient Engagement activities and resources at: https://www.fda.gov/patients/learn-about-fda-patient-engagement.
  • How can sponsors include patients and their advocates in regulatory meetings with the FDA?
    • Patients and their advocates possess the unique, first-hand perspective of what it is like to live with or care for an individual with a disease, and the impact of available treatments on daily life. Patients can provide valuable input into the discussion of investigational therapies by describing their experience with a disease or condition, and by defining meaningful change in terms of their specific disease and the risks they are willing to accept. CBER welcomes the participation of patients and their advocates in formal meetings related to the development of investigational products. CBER encourages sponsors to consider including patients or their advocates in their meetings with CBER and may do so by directly inviting and coordinating participation for such guests.
  • Where can I find information about clinical trial design for rare diseases?
    • Clinical trials are voluntary human research studies designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments. It is important to know that the FDA does not conduct Clinical Trials. You can learn more about clinical trials and how to search for a clinical trial that might be right for you at our Clinical Trials: What Patients Need to Know webpage.

      CBER also hosts an educational series, RegenMedEd: An FDA CBER OTP Series on Regenerative Medicine. The RegenMedEd series aims to bring together important stakeholders and FDA staff to discuss foundational information about regenerative medicine therapies, such as gene therapy and cell therapy products, and explore opportunities for patients, caregivers, and advocates to engage with FDA to help advance drug development. The topics for many of these events have focused on how patient and their advocates can get involved to support development of products for rare diseases.

      Additional resource: Patient Matters Video Series at https://www.fda.gov/patients/learn-about-fda-patient-engagement/patients-matter-video-series
  • What is an Orphan Product Designation?
    • An Orphan Product Designation is for a drug intended to treat a rare disease or condition and qualifies sponsors for various incentives to support rare disease drug development. Orphan Product Designation is granted by FDA after receipt and review of a request submitted by a sponsor to FDA’s Office of Orphan Products Development (OOPD).
  • Where can I find FDA funding to help advance rare disease product development?
    • CBER encourages the development and adoption of advanced technologies to modernize biopharmaceutical manufacturing. CBER has awarded several grants and contracts to support research projects to promote the development and adoption of innovative approaches for the manufacture of CBER-regulated products, including products for rare diseases. To learn more about CBER’s awarded grants and contracts visit the CBER Advanced Technologies Program Extramural Research Funding webpage. CBER solicits applications for extramural funding through the FDA Broad Agency Announcement (BAA) (SAM.gov) and the NIH Grants & Funding portal (grants.nih.gov).

      FDA’s Orphan Products Grants Program awards grants to clinical investigators to support the development of safe and effective medical products for patients with rare diseases. The program has supported clinical research since 1983 and has funded clinical trials that have facilitated the approval of more than 80 products. For more information about OOPD grant programs, visit the OOPD Grants Program webpage.
  • How does the standard for approval of rare disease products differ from other medical products?
    • FDA applies the same standards in reviewing and approving drugs, including biologics for rare diseases, as it does for drugs for other diseases and conditions. In reviewing a marketing application for a new drug or biologic, FDA determines whether the drug is safe and effective for its intended use. Evidence that the drug will benefit the target population should outweigh any risks and uncertainties. FDA considers flexible approaches while ensuring that statutory standards are met in the review of submissions for rare diseases. For additional information read FDA’s Draft Guidance Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products. The draft guidance complements and expands on the 1998 guidance entitled “Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products.” When finalized, the draft guidance will represent FDA’s current thinking on “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products.”
  • What is a Rare Pediatric Disease Priority Review Voucher (RPD PRV)?
    • Under Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C Act), FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. Under this program, 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.

      For general questions concerning pediatric rare diseases or RPD PRVs, please contact the Office of Orphan Products Development at orphan@fda.hhs.gov. For questions concerning RPD PRV eligibility of biological products, contact the Center for Biologics Evaluation and Research (CBER) at industry.biologics@fda.hhs.gov.

      For additional information about RPD PRVs click here.
  • How do I request a meeting with CBER and my rare disease advocacy organization?
    • Meetings between patient organizations and CBER staff present opportunities to enhance CBER’s understanding of disease burden and the impact of treatment on patients. These meetings may also help to inform efforts by patient organizations working to advance drug development such as developing or conducting natural history studies or patient input studies to support medical product development.

      To request a meeting with CBER staff, a representative from a patient organization may email CBERPatientEngagement@fda.hhs.gov. CBER will review requests and make every effort to hear directly from patient organizations according to staff availability and resources.

      For a meeting concerning a topic that applies to more than one medical product type, the request should be submitted through the Patients: Ask FDA page, which can also be used when a patient organization is unsure about which FDA center or program to contact to request a meeting with FDA.
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