Advancing Treatment: New Hope for Rare Disease Patients

Four new drugs for rare diseases are set for launch while five are already available, potentially alleviating patient suffering. The National Policy includes 63 diseases, with emphasis on affordable treatments and collaboration. Government initiatives aim to support rare disease therapies as a core strategic priority.

Four novel treatments for rare diseases are nearing market release, complementing five newly launched medications for seven illnesses. These developments, announced by Niti Aayog member (health) Dr. VK Paul, promise to alleviate patient suffering significantly.

At a recent event, Dr. Paul highlighted that the introduction of generic medications by local companies has resulted in noteworthy savings for rare disease patients. Currently, the National Policy for Rare Diseases lists 63 conditions based on recommendations from the Central Technical Committee for Rare Diseases.

With an estimated nine crore Indians affected by rare diseases, Dr. Paul emphasized the need for indigenous manufacturing, stronger registries, and coordinated efforts to ensure faster diagnosis and equitable treatment. Government officials reaffirmed commitment to this goal through initiatives like the PLI scheme and strategic prioritization of rare diseases and orphan drugs.

(With inputs from agencies.)