DISCUSSION: The absence of specific regulations, shortage of healthcare resources, budget constraints, competing health priorities, lack of patient data, and insufficient research incentives discourage orphan drug development and global clinical trial inclusion, resulting in treatment inaccessibility and high costs. The Indian Government introduced the National Policy for Treatment of Rare Diseases (NPRD) to address these challenges. Several initiatives have been introduced to attract stakeholders with government-funded research, grants, incentives, and accelerated regulatory approvals of novel therapies that can ensure timely prevention and treatment of rare diseases. The National RD Registry by the Indian Council of Medical Research (ICMR) aims to provide prevalence data. Innovative approaches are required to improve rare disease management and promote orphan drug research. This will ensure the accessibility and affordability of life-saving therapeutics for India's rare disease patients.
CONCLUSION: An integrated RD management and orphan drug research framework focusing on robust data management, patient-oriented policies to improve the treatment landscape, flexible regulations, strengthening rare disease registry with clinical and diagnostic data, and a favorable research ecosystem to promote indigenous research catering to the Indian population, will improve the treatment landscape and orphan drug research and development in India. This will ensure timely availability of therapeutics at affordable prices.