Four new drugs for rare diseases on anvil; five medications for 7 ailments in market: VK Paul

New Delhi: Four new drugs for rare diseases are in the pipeline expected to be launched soon while five medications for seven illnesses have already been introduced in the market alleviating suffering of patients, Niti Aayog member (health) Dr VK Paul said on Wednesday.

Speaking at an event here, he noted that generic medications launched by the local companies have led to immense savings for the people suffering from rare diseases.

Currently, 63 rare diseases are included under National Policy for Rare Diseases on recommendation of the Central Technical Committee for Rare Diseases (CTCRD).

“For seven diseases, we have made significant progress. These seven diseases include Thalassemia, Wilson’s disease and Cystic Fibrosis… there are five drugs which are now available in the market,” Paul stated.

Four other drugs are on the anvil, he added.

He noted that NITI Aayog had shortlisted 13 disorders as a priority in 2023.

Department of Pharmaceuticals Secretary Amit Agrawal said the government under the PLI scheme for pharmaceuticals has been able to support production of drugs to treat eight rare diseases.

He noted that the government aims to treat rare diseases and orphan drugs as a strategic priority among the priority areas with higher policy support.

Novartis MD & Country President Amitabh Dube said rare diseases affect over 300 million globally, with 70-90 million in India.

Acknowledging the progress made, he stressed the need for continued efforts to address the challenges faced by patients in the rare disease management.

“We have to create an ecosystem that encourages and enables R&D, reduces regulatory delays and values innovation so treatments can reach Indian patients faster,” he added.

He also advocated sustainable financing solutions to ensure access to emerging, advanced therapies that exceed Rs 50 lakh.

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