Rare Disease Treatment Market Trends

Rare Disease Treatment Market Trends

• The pharmaceutical industry is continuously involved in R&D activities for the development of novel drug therapies to treat rare diseases and commercialization of medicines for the treatment of uncommon diseases. Due to increase in funding for drug discovery and R&D activity, there are now significantly more novel molecular pharmaceuticals commercialized each year. More than 800 orphan drug indications have been approved from 1983 and 2019.
   
• Moreover, the target population base has also contributed to this success and has reinstated its capability to drive investment, inform research, and influence policymakers. For instance, as per the report published by U.S. FDA in January 2024, more than half (28 of 55, or 51%) of the novel drugs approved in 2023, these drugs were approved to prevent, diagnose, or treat a rare disease or condition, for which they received an orphan-drug designation. Therefore, the number of approved orphan indications is surging rapidly than the number of drugs, as some therapeutics are indicated for multiple conditions. Thus, increasing launch of novel drugs and treatment regimens for rare disease treatment will spur the market growth.