Four new drugs for rare diseases on anvil, 5 medications for 7 ailments in mkt: VK Paul

Published On 2025-08-23 10:00 GMT | Update On 2025-08-23 10:00 GMT

New Delhi: India is making significant strides in the fight against rare diseases, with four new drugs currently in the pipeline and five already available in the market for the treatment of seven rare conditions, according to Niti Aayog member (health) Dr. VK Paul.

Speaking at an event here, Dr. Paul said that the launch of generic medications by the local companies has brought substantial relief to patients suffering from rare disorders.

Currently, 63 rare diseases are included under National Policy for Rare Diseases on recommendation of 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.

According to PTI, he noted that NITI Aayog had shortlisted 13 disorders as a priority in 2023.

Paul stated that rare diseases are estimated to collectively affect close to nine crore Indians, making them both a health and societal challenge. He emphasised that with only a fraction of the 7,000 rare diseases having approved therapies, indigenous manufacturing, stronger registries, and multi-ministerial coordination must be prioritised.

He underlined that collective resolve must ensure faster diagnosis, equitable treatment, and an improved quality of life.

Paul also highlighted the importance of collaboration across academia, industry, and government so that quality and affordable treatments can be developed and research can be translated into lasting cures.

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 area 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.

Article Source : with inputs

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Ruchika Sharma

Ruchika Sharma joined Medical Dialogue as an Correspondent for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751

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Four new drugs for rare diseases on anvil, 5 medications for 7 ailments in mkt: VK Paul

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