Prohibitive cost of treatment

The high cost of treating rare diseases, known as "orphan diseases," creates significant barriers to access. Due to small patient populations, drug manufacturers have little incentive to develop therapies, leading to extremely high prices. In countries like India, where most rare disease drugs are not produced domestically, treatment costs can exceed ₹1 crore annually per child. While measures like the Orphan Drug Act in the U.S. and Canada incentivize drug development, even in developed nations, the sustainability of funding and reimbursement programs is under pressure, leading to calls for greater transparency and price controls.