"About 10-12 orphan drugs are available for patients in India"

Prasanna Kumar Shirol, Co-founder & Executive Director, The Organisation for Rare Diseases India (ORDI), in an interaction with BioSpectrum India, shared his views about the efforts of ORDI in reducing inequities and ensuring that persons with rare diseases have access to the same resources as the general community. Edited excerpts;

- Narayan Kulkarni

There are 443 rare diseases which ORDI has listed. How many of these rare diseases are being treated in India?

These are the conditions reported by patients who have registered with ORDI through helpline, online and referrals. As per National Policy for Treatment of Rare Diseases (NPTRD), 2017, the Government of India mentions about 450 conditions have been identified in India, but the list of diseases being treated is not available. Lysosomal Storage Disorders (LSDs), a group of genetic metabolic disorders where enzymes responsible for breaking down complex molecules within lysosomes are deficient or absent such as Gaucher, Pompe, MPS and Neuro Muscular Diseases like DMD, SMA and IEMs like PKU, MSUD, Organic Acidemias, Cystic Fibrosis, Osteogenesis Imperfecta, Primary Immuno Deficiencies, are predominantly visible across India.

The National Policy for Rare Diseases (NPRD) has listed only 67 diseases under Group 3. How is ORDI pushing the government to include other rare diseases under NPRD?