Govt to prioritise rare diseases and orphan drugs under new R&D push
New Delhi, Aug 21: The government is set to bring rare diseases and orphan drugs into sharper focus under its upcoming research and innovation framework, a senior official said on Wednesday. Speaking at the inaugural session of the Rare Diseases Conference 2025, held at the FICCI Auditorium, Amit Agrawal, Secretary, Department of Pharmaceuticals, Ministry of Chemicals & Fertilizers, said rare diseases. though individually uncommon, collectively affect nearly 5% of India’s population and must be treated as a pressing public health issue.
“Rare diseases are not just a medical or technical problem; they must be approached through a human lens and as a matter of inclusion,” Agrawal said, stressing that patients and caregivers face both financial and emotional burdens that require joint efforts from government, industry, academia, and civil society. He linked the government’s renewed focus to Prime Minister Narendra Modi’s call for greater investment in research and development in his Independence Day address, recalling that India, as the “pharmacy of the world,” must also lead in developing affordable therapies for rare conditions.
Underlining recent progress, Agrawal noted that rare diseases were included as a priority area under the Production Linked Incentive (PLI) Scheme for Pharmaceuticals.
As a result, eight drugs for rare conditions have been supported. These include Eliglustat for Gaucher’s Disease, where treatment costs have dropped from Rs1.8–3.6 crore annually to just Rs 3–6 lakh, as well as Trientine for Wilson’s Disease, Nitisinone for Tyrosinemia Type 1, and Cannabidiol for Lennox–Gastaut Syndrome. “These tangible reductions in treatment costs highlight the transformative potential of targeted interventions,” he said.
The official also urged corporates to consider rare disease patients in their CSR activities and patient assistance programmes. He suggested that policymakers explore dedicated regulatory pathways and exemptions tailored to the needs of this patient community.
Concluding his remarks, Agrawal said he looked forward to policy recommendations emerging from the conference and expressed interest in adopting global best practices to strengthen India’s rare disease framework. Organised around the theme “Making Rare Care Possible: Availability, Accessibility, Awareness,” the conference aims to bring together diverse stakeholders to build a more inclusive ecosystem for patients.
