The government will soon firm up a roadmap to encourage research and development of orphan drugs – medicines used for the treatment of rare conditions which are often serious or life threatening.

Incentives could be offered to the private sector in the form of faster approvals and an extended period of exclusivity for orphan drugs. Since only a small population of patients is affected by these rare conditions and so needs such drugs, it is often not profitable enough for the private sector to venture into them without government support.

The government wants to make it profitable for the private sector to invest money and manpower for the development of such drugs, a senior government official told ET.

Worldwide 300 million people are affected from more than 7,000 rare diseases, with 90 million in India alone.

Niti Aayog member VK Paul has held several rounds of consultations with stakeholders, including department of pharmaceuticals, clinicians and active pharmaceutical ingredient manufacturers, to consider identification of a set of priority disorders or indications and their corresponding treatments that can be enabled for domestic manufacturing of orphan drugs.

The government is of the view that the area of rare diseases is complex and heterogeneous. While the number of disorders with known molecular basis is rapidly rising, the number of approved therapies lags far behind.

“Keeping this in view, the Aayog has kick-started discussions and will soon lay out a detailed policy prescription to encourage research and development of orphan drugs,” the official added.

As per the National Policy on Rare Disease 2021, so far only a limited number of diseases requiring such drugs has been recorded in India from tertiary care hospitals. Some of these include primary immunodeficiency disorders, lysosomal storage disorders (Gaucher‘s disease, Mucopolysaccharidoses, Pompe disease, Fabry disease, etc.), small molecule inborn errors of metabolism (Maple Syrup urine disease, organic acidemias, etc.), Cystic Fibrosis, osteogenesis imperfecta, certain forms of muscular dystrophies and spinal muscular atrophy.