Key Points

With few targeted treatments, and average drug costs exceeding USD 100,000 a year, rare diseases are a burgeoning area of study with enormous potential for drug development.

There are an astounding ~350 million cases of rare diseases worldwide. India alone accounts for approximately 72-96 million patients, which means one in every 20 Indians suffers from a rare disorder. Of these, only one in 10 receives targeted treatment.

In 2017, the Indian Council of Medical Research (ICMR) launched the National Registry for Rare Diseases (NRRD) to fill the major gaps in epidemiological data on these serious, chronic and life-threatening conditions. Furthermore, the comprehensive National Policy for Rare Diseases 2021 addresses challenges around diagnosis and treatment. In May 2022, the Health Ministry increased the financial support for patients undergoing rare disease treatment from ~USD 24,000 to ~USD 60,000 under the umbrella scheme of Rashtriya Arogya Nidhi.

The most immediate need is to launch orphan drugs to address vital treatment needs. This presents a significant opportunity for pharmaceutical companies. However, launching orphan drugs in India can be challenging due to the complex regulatory landscape, limited healthcare infrastructure and high cost of treatment.

Strategic partners can provide invaluable support in navigating the complex regulatory landscape, developing pricing and reimbursement strategies, and creating market access strategies for orphan drugs. By working with the right consulting firms, pharmaceutical companies can increase their footprint in the Indian healthcare market while addressing the unmet needs of patients with rare diseases.

Download our full paper to explore India’s rare disease landscape and commercial opportunities with orphan drugs.