Rare Diseases Conference 2025: Pharma Secretary Calls for Inclusive Action

Shri Agrawal noted that while rare diseases may appear infrequent on an individual basis, collectively they affect nearly 5% of the population—about one in every 20 individuals.

The Rare Diseases Conference 2025, themed “Making Rare Care Possible: Availability, Accessibility, Awareness”, was inaugurated yesterday at the FICCI Auditorium in New Delhi with a strong call for collective action to improve care, access, and affordability for patients living with rare conditions.

Delivering a special address during the inaugural session, Shri Amit Agrawal, Secretary, Department of Pharmaceuticals, Ministry of Chemicals & Fertilizers, highlighted the urgency of addressing rare diseases as a public health challenge with far-reaching social consequences.

Rare Diseases: A Public Health Priority

Shri Agrawal noted that while rare diseases may appear infrequent on an individual basis, collectively they affect nearly 5% of the population—about one in every 20 individuals. This, he stressed, places rare diseases firmly within the realm of public health concern and national inclusion agendas.

“The rare disease challenge should be seen through a human lens and as a question of inclusion, not merely as a medical or technical problem,” he said.

Vision of Inclusion and R&D Investment

Drawing on the Prime Minister’s inclusive vision for Divyangjan, Shri Agrawal emphasised the need for government, industry, academia, and civil society to come together in addressing the multifaceted burden faced by patients and their caregivers.

He also echoed the Prime Minister’s Independence Day message: “We are known as the pharmacy of the world, but isn’t it the need of the hour to invest in research and development? Shouldn’t we be the ones providing the best and most affordable medicines for the welfare of humanity?”

Policy Milestones and Cost Reductions

Highlighting government interventions, the Secretary announced that Rare Diseases have been made a focus area under the Production Linked Incentive (PLI) Scheme for Pharmaceuticals.

As a result, support has been extended to eight drugs for rare conditions, including:

• Eliglustat for Gaucher’s Disease – treatment costs reduced from ₹1.8–3.6 crore annually to just ₹3–6 lakh.

• Trientine for Wilson’s Disease.

• Nitisinone for Tyrosinemia Type 1.

• Cannabidiol for Lennox–Gastaut Syndrome, among others.

“These dramatic reductions in treatment costs show the transformative potential of targeted policy interventions when backed by innovation and regulatory support,” Agrawal explained.

Corporate Responsibility and Inclusive Programmes

The Secretary called upon corporates and industry players to expand the scope of CSR initiatives and patient assistance programmes to include rare disease patients, who often face crippling financial and emotional burdens.

He also urged policymakers and regulators to re-examine existing frameworks through the lens of inclusivity, suggesting that special pathways or regulatory exemptions may be necessary to address the unique needs of the rare disease community.

Global Best Practices and the Way Forward

In his concluding remarks, Shri Agrawal expressed his intent to carefully consider the recommendations and policy insights emerging from the conference’s deliberations. He said that India must actively learn from global best practices to strengthen its rare disease policy framework and create a sustainable, inclusive care ecosystem.

Building Hope for the Rare Disease Community

The Rare Diseases Conference 2025 is seen as an important milestone in pushing forward the conversation on equity, access, and innovation for India’s rare disease patients. By aligning policy, research, and compassion, stakeholders hope to transform the narrative from isolation to inclusion, making “rare care” a reality for millions.