Vol. 6, Issue 1, Part A (2024)

Regulatory authorities globally have established specific frameworks governing the approval of orphan drugs, crucial for addressing rare diseases that affect a small portion of the population. This comprehensive review article offers a detailed exploration of the distinct regulatory requirements outlined by five prominent bodies: CDSCO, USFDA, EMA, ANVISA, and SFDA. The Central Drugs Standard Control Organisation (CDSCO) in India requires a thorough process for orphan drug designation. Companies requesting permission are required to provide proof of disease prevalence, unmet medical needs, and strong safety and efficacy information. The USFDA, which focuses on illnesses that affect less than 200,000 people, provides tax benefits and expedited reviews for orphan medications in an effort to hasten their development. In Europe, approval of orphan drugs that affect fewer than 5 out of 10,000 people is governed by the European Medicines Agency (EMA). In order to promote innovation in the treatment of rare diseases, EMA has established a framework that offers fee reductions, protocol support, and ten years of market exclusivity. Brazil's National Health Surveillance Agency (ANVISA) requires comprehensive documentation for orphan drug approval. Companies must demonstrate evidence of disease rarity, safety, efficacy, and, in some cases, ongoing clinical trials. Similarly, the Saudi Food and Drug Authority (SFDA) in Saudi Arabia oversees drugs for conditions impacting less than 5 in 10,000 individuals, necessitating detailed justifications and post-designation compliance.
Every regulatory agency upholds specific standards for the designation of drugs as orphans, diverse application processes, and a range of incentives to encourage the development of treatments for uncommon diseases. Pharmaceutical companies navigating the complex orphan medication approval process must have a thorough understanding of various regulatory environments.