Rare diseases were included as a focus area under the production-linked incentive (PLI) scheme for pharmaceuticals, which has considerably brought down the treatment cost, an official statement from Ministry of Chemicals and Fertilisers said on Thursday.

Eight drugs for rare conditions have been supported under the PLI scheme, including Eliglustat for Gaucher’s Disease, where the cost of treatment has come down from Rs 1.8–3.6 crore annually to Rs 3–6 lakh, according to Amit Agrawal, Secretary, Department of Pharmaceuticals.

Other supported treatments include Trientine for Wilson’s Disease, Nitisinone for Tyrosinemia Type 1 and Cannabidiol for Lennox–Gastaut Syndrome, among others. He noted that such tangible reductions in treatment costs demonstrate the transformative potential of targeted policy interventions.

Agrawal delivered a special address during the inaugural session of the ‘Rare Diseases Conference 2025’ at FICCI auditorium.

He commended the organisers for bringing focus to an issue of growing significance that has historically not received adequate attention.

He underlined that although rare diseases may appear infrequent individually, collectively they affect nearly one in every twenty individuals — around 5 per cent of the population — making them a major public health concern.

Agrawal also emphasised that the rare disease challenge should be seen through a human lens and as a question of inclusion, not merely a medical or technical problem.

Citing the Prime Minister Narendra Modi’s inclusive vision of ‘Divyangjan’, Agrawal called for a response from government, industry, academia and civil society to address the multifaceted burden faced by patients and caregivers.

Referring to the Prime Minister’s Independence Day address, he recalled: “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?”

The Secretary also encouraged corporates to factor in rare disease patients under their CSR initiatives and patient assistance programmes, considering the heavy financial and emotional burden on affected families.

He urged all stakeholders to evaluate their policies, regulations, funding models and programme designs through the lens of inclusivity. He suggested exploring special pathways or regulatory exemptions to address the unique needs of the rare disease community.