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Rare diseases: National Policy for Rare Diseases, 2021 and orphan drugs

By CAShorts DeskPublished Sources: The Hindu; Ministry of Health and Family Welfare (NPRD 2021, rare diseases portal); PIB; CDSCO; Patents Act, 1970 (IP India)

Why in news: In October 2026 Keralam moved children with spinal muscular atrophy from branded risdiplam to a cheaper generic, and some parents objected.

Rare diseases: National Policy for Rare Diseases, 2021 and orphan drugs
Illustration (AI-generated)

The National Policy for Rare Diseases, 2021 sorts rare diseases into three groups. The Health Ministry says it funds treatment of up to ₹50 lakh a patient at notified Centres of Excellence. A crowdfunding portal helps with the rest. Keralam's October 2026 switch to a cheaper generic of risdiplam for spinal muscular atrophy has revived the branded-versus-generic debate.

Syllabus: Prelims · Economy & Social Development; GS2 · Government Policies; GS2 · Health, Education & Human Resources

Why this matters for UPSC

GS II health policy and GS III intellectual property. Prelims can ask the three groups of the National Policy for Rare Diseases, 2021, its ₹50 lakh ceiling and the CDSCO's role; Mains, how to make orphan drugs affordable.

In short

India's National Policy for Rare Diseases, 2021 defines rare diseases through three treatment-based groups and funds care at Centres of Excellence, balancing costly orphan drugs against wider health priorities.

Pillars of the 2021policyThree groups₹50 lakh ceilingCentres ofExcellenceCrowdfundingportalTax reliefPrevention first

India must keep the ₹50 lakh promise meaningful by cutting orphan drug prices through generics and pooled buying, while earning patients' trust in every switch.

Why it matters

A child with spinal muscular atrophy may need a drug every day for life, and a family cannot pay tens of lakhs a year for it. Public money stretches only so far, so the price of one medicine decides how many children a State can treat. When a cheaper copy is approved, a programme can cover many more patients, but only if families trust that the copy works as well. The larger issue is how a poorer country pays for medicines made for very few people, whose makers set high prices behind patents. The state must weigh that cost against wider public health needs.

Prelims pointers

  • The WHO, as cited in NPRD 2021, treats a disease as rare at a prevalence of 1 or less per 1,000 population.
  • Group 2 of NPRD 2021 covers disorders managed with special dietary formulae, such as phenylketonuria, or with hormones and specific drugs, such as growth hormone.
  • AIIMS, New Delhi is the nodal hospital of the consortium of Centres of Excellence; each Centre may get a one-time grant of up to ₹5 crore for equipment.
  • Nidan Kendras were set up by the Department of Biotechnology under the Unique Methods of Management and treatment of Inherited Disorders (UMMID) project.
  • The National Consortium for Research and Development on Therapeutics for Rare Diseases was set up by the Department of Health Research, with the ICMR as a member.
  • The digital crowdfunding portal lets individual and corporate donors, including under CSR, pay into a treating institution's earmarked rare disease fund.
  • Under section 84 of the Patents Act, 1970, any interested person may seek a compulsory licence three years after a patent's grant.
  • Section 53 of the Patents Act, 1970 sets a patent's term at twenty years from the date the application was filed.

Practise a UPSC-style question

With reference to the National Policy for Rare Diseases, 2021, consider the following statements:

1. Group 1 covers disorders amenable to one-time curative treatment, such as stem cell or organ transplantation.

2. Spinal muscular atrophy is listed under Group 2, the diseases needing lifelong but relatively low-cost treatment.

3. Disorders managed with special dietary formulae, such as phenylketonuria, are placed in Group 2.

Which of the statements given above is/are correct?

  1. (a) 1 and 2 only
  2. (b) 2 and 3 only
  3. (c) 1 and 3 only
  4. (d) 1, 2 and 3
Show the answer

Answer: (c) 1 and 3 only

Answer: (c) 1 and 3 only. Spinal muscular atrophy is a Group 3 disease, not Group 2.

Statement 1: correct. NPRD 2021 places disorders treatable once by haematopoietic stem cell or organ transplantation in Group 1.

Statement 2: incorrect. The policy lists spinal muscular atrophy, with antisense and gene therapy, in Group 3b, the very costly lifelong therapies.

Statement 3: correct. Group 2 includes disorders managed with special dietary formulae or Food for Special Medical Purposes, phenylketonuria among them.

Eliminate: Statement 2 is the surest: SMA therapy is among the costliest, so it cannot sit in the low-cost group. Out: (a), (b), (d).

Trap: Swapped attribution. A real disease is pinned on the wrong group of the policy, which reads plausibly because SMA also needs lifelong treatment.

Remember: Group 1 one-time cure, Group 2 lifelong low cost, Group 3 lifelong high cost (SMA, DMD).

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