FDA's Orphan Drug Program

Expedite Development of New Orphan Drugs 

The FDA’s Orphan Drug Program is designed to encourage the development of drugs and biologics for the treatment, diagnosis, or prevention of rare diseases or conditions, often referred to as orphan diseases. Here are the key details of the program: 

Definition of Orphan Diseases 

Orphan diseases, also known as rare diseases, are those that affect fewer than 200,000 people in the United States or diseases that affect more than 200,000 people but for which there is no expectation of recovering the costs of developing and marketing a treatment. The Orphan Drug Act of 1983 defines orphan diseases and provides incentives to encourage the development of treatments for them. 

Orphan Designation 

To qualify for the program, a drug or biologic product must be intended for the treatment, diagnosis, or prevention of a rare disease or condition. The sponsor of the product can apply for orphan designation with the FDA. If granted, orphan designation provides certain benefits, including tax credits for qualified clinical testing expenses, eligibility for orphan drug grants, and a period of marketing exclusivity upon approval. 

Marketing Exclusivity 

Orphan drug designation grants seven years of marketing exclusivity to the sponsor upon approval of the product for the orphan-designated indication. This means that during the exclusivity period, the FDA cannot approve another application for the same drug for the same rare disease or condition, except under limited circumstances. 

Orphan Drug Grants 

The FDA administers grants to support clinical research in rare diseases through its Orphan Products Grants Program. These grants fund clinical studies or trials, natural history studies, and other activities related to the development of orphan products. 

Fee Waivers and Reductions 

Sponsors of orphan drug applications may be eligible for fee waivers or reductions for certain FDA application fees, including those for new drug applications (NDAs), biologics license applications (BLAs), and annual establishment fees. 

Accelerated Approval 

Drugs granted orphan designation may also be eligible for accelerated approval, which allows for faster approval based on surrogate endpoints or intermediate clinical outcomes that are reasonably likely to predict clinical benefit. 

Post-Approval Commitments 

Despite the benefits provided by the Orphan Drug Program, sponsors are still required to fulfill post-approval commitments, such as conducting post-marketing studies or trials to gather additional safety or efficacy data. 

Conclusion 

Overall, the FDA’s Orphan Drug Program plays a crucial role in incentivizing the development of treatments for rare diseases by providing financial incentives, regulatory assistance, and marketing exclusivity to sponsors of orphan products. 

Links 

Read more about the program on the FDA website.