ORPHAN DRUGS

Orphan drugs are synthetic pharmaceuticals which are commercially underdeveloped and are mainly used for rare diseases which affect only small number of individuals.

Example: Haem arginate used to treat acute intermittent porphyria, variegate porphyria and hereditary coproporphyria is used as an orphan drug.

Ireland has proposed a unique process for assessing orphan drug reimbursement with 2018 ‘Pricing and Supply of Medical Goods’ amendment.

An initiative by CDSCO is – Companies developing orphan drugs will get a boost from Indian drug regulator through partial and full waiver of clinical trials depending on specific drugs.

Re-purposing of drugs provides an opportunity for Not-For-Profit organizations to develop and market affordable orphan drugs.

So, research is needed in pricing strategy and in creative economic modeling techniques.

Kajal Shah