Current Affairs · · GS2 · Social Justice

Rare-disease drugs: can India make specialised treatments affordable?

Small patient groups and costly research make many orphan drugs expensive or unavailable. India could use its patient registries, manufacturing strength and growing biotechnology skills to improve access. Success will need investment, careful regulation and policies that link innovation to affordable treatment.

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REq1
  1. Context1 / 6

    A rare disease affects relatively few people. There are around 8,000 rare diseases worldwide, and more than 1,000 rare genetic disorders have been reported in India. Each condition may affect a small group, but together they create a large health burden.

    India's New Drugs and Clinical Trials Rules, 2019 define an orphan drug as a medicine for a condition affecting not more than five lakh people in India.

    Developing a medicine involves research and development (R&D), testing and production costs. With few patients, a company has fewer buyers over whom to spread those costs. Researchers may also struggle to find enough people with the same condition for a clinical trial.

    Few patients → a small market → high development costs per patient → costly or unavailable medicines.

    Imagine spreading the cost of developing a medicine across one crore patients. Now imagine spreading that cost across only a few hundred. The second market may not give a company enough reason to invest.

    The United States responded with the Orphan Drug Act, 1983. It offered incentives such as tax benefits, research grants and market exclusivity to encourage development.

  2. Key highlights2 / 6

    A treatment can exist and still be out of reach. Some rare-disease therapies cost over ₹1 crore per patient each year. Advanced gene therapies can cost several crores.

    India's National Policy for Rare Diseases, 2021 (NPRD) supports diagnosis, research and the development of medicines in India. Under its current support framework, eligible patients can receive up to ₹50 lakh for treatment of 63 identified rare diseases through 15 designated Centres of Excellence.

    For an expensive treatment needed throughout life, ₹50 lakh may cover only part of the bill. Developing a treatment and making it affordable are therefore separate challenges. Public policy must address both.

  3. Key concepts3 / 6

    India has three strengths to build on.

    A large, diverse patient population: For some rare conditions, India's size can help researchers find enough patients for studies. A patient registry records who has a condition, where patients live and how the disease changes over time. It can also help identify people who may be suitable for trials. India has a National Registry for Rare and Other Inherited Disorders. Databases for individual diseases and biobanks, which store biological samples for research, add to this capacity.

    Manufacturing experience: Indian firms have experience with generics, biosimilars, small batches and production at lower cost. Generics are versions of existing medicines; biosimilars are closely comparable versions of biological medicines. These skills could help lower orphan-drug production costs.

    Advanced treatments: Firms and research institutions are working on biologics, gene therapies, cell therapies and personalised medicine. The opportunity is to build on affordable generics, develop more complex medicines and make specialised rare-disease treatments affordable.

  4. Way forward4 / 6

    Rule 101 and possible local-trial waivers

    Under Rule 101 of the New Drugs and Clinical Trials Rules, 2019, the government can name jurisdictions whose drug approvals may be considered when deciding whether to waive a local clinical trial.

    In 2024, the specified jurisdictions were the United States, United Kingdom, Japan, Australia, Canada and European Union. The specified categories cover orphan drugs, gene and cellular therapies, new drugs for pandemic situations, medicines for special defence purposes and drugs that offer a significant advance over existing care.

    For a condition with only a few hundred Indian patients, repeating a large trial may be difficult, costly and slow. Reliable evidence from abroad can help avoid unnecessary duplication.

    Foreign approval does not automatically grant Indian approval. The Central Drugs Standard Control Organization (CDSCO) still reviews safety and effectiveness. It considers possible differences in Indian patients, pharmacokinetics (how the body handles a drug) and pharmacodynamics (how the drug affects the body). Conditions can include studies after approval to check safety and effectiveness.

    The aim is to remove avoidable delays while keeping patient safeguards.

  5. Note5 / 6

    Making orphan-drug development viable

    Because the market is small, public support can reduce the risk of investing in a new treatment.

    Production-linked incentives (PLI): Support can encourage domestic production, investment and technology transfer. Policy can link assistance to affordable patient access. In 2026, the government said the active pharmaceutical ingredient (API) for risdiplam, a medicine used for spinal muscular atrophy, was being made in India under the pharmaceutical PLI scheme. An API is the ingredient that produces a medicine's intended effect.

    Advance market commitments: A government can commit funds to buy a successfully developed medicine if it meets agreed conditions. A farmer may be more willing to plant an unusual crop when a buyer commits in advance to purchasing the harvest. A future purchase commitment can similarly reduce the risk of developing a medicine for a small group.

    Public procurement: Centres of Excellence, public hospitals and government health programmes can purchase and supply affordable therapies.

    Research partnerships: Manufacturers, laboratories, hospitals and global pharmaceutical firms can combine new technology, Indian patient data and lower-cost production.

  6. Note6 / 6

    Building India's rare-disease drug capacity

    1. Improve registries. Reliable patient data helps plan trials, research and treatment.
    2. Support Indian R&D. Back biologics, gene therapies and other advanced medicines.
    3. Reduce avoidable delays. Use reliable foreign evidence where the rules allow, with continued safety monitoring.
    4. Tie incentives to access. Public funding should support innovation, domestic production, reasonable prices and treatment for patients.
    5. Encourage technology transfer. Global partnerships can bring expertise that Indian firms combine with manufacturing capacity.
    6. Protect research participants. People who share data or join studies should have meaningful access to successful treatments.

    India needs research, production and access to develop together. Its experience supplying affordable generics offers a starting point. Making highly specialised medicines affordable will also require biotechnology, suitable regulation and sustained investment. Success could help other Global South countries facing similar costs.

Sources

Syllabus

PaperSubjectSub-topic
GS2Social JusticeHealth and access to affordable treatment
GS2GovernancePublic health policy and regulatory reform
GS3Science & TechnologyBiotechnology, clinical trials, gene and cell therapies
GS3EconomyPharmaceutical manufacturing, R&D and innovation
PrelimsScience & TechnologyOrphan drugs, CDSCO, Rule 101 and the National Policy for Rare Diseases

Topics

IndustryGovernanceBiotechnologyDiseasesHealth

Related previous-year questions

Asked in earlier UPSC Prelims papers on this topic. Answer, then check.

  1. UPSC Prelims 2026 · Science and Technology · Biotechnology

    Which of the following statements with regard to genetic medicine is/are correct? 1. Genetic medicines correct/compensate for the faulty genes responsible for disease. 2. Engineered viruses and lipid nanoparticles are used as carriers of the genetic medicine. 3. Genetic medicines alter the entire DNA sequence. Select the answer using the code given below:

    1. 1 only
    2. 2 and 3 only
    3. 1 and 2 only
    4. 1, 2 and 3
    Show answer

    Answer: C. Genetic medicines are therapeutic agents that work by correcting, compensating for, or silencing faulty genes responsible for diseases. Examples include gene therapy, antisense oligonucleotides, and RNA interference therapies. Statement 1 is correct: Genetic medicines correct or compensate for the faulty genes responsible for disease. This is the fundamental principle of genetic medicine, which aims to fix or compensate for defective genes causing diseases. Statement 2 is correct: Engineered viruses (viral vectors like adeno-associated viruses and lentivirus) and lipid nanoparticles, used in mRNA vaccines and therapies, serve as delivery vehicles or carriers for genetic medicines. Statement 3 is incorrect: Genetic medicines do not alter the entire DNA sequence. They target specific genes or gene segments that are responsible for a disease, making precise and targeted modifications rather than changing the whole genome. Technologies like CRISPR make precise edits at targeted locations. Statements 1 and 2 are correct, so the answer is Option (c).

    Difficulty: medium · statement

    Open this question on its own page, with the full explanation →

  2. UPSC Prelims 2025 · Science and Technology · Biotechnology

    With reference to monoclonal antibodies, often mentioned in news, consider the following statements: I. They are man-made proteins. II. They stimulate immunological function due to their ability to bind to specific antigens. III. They are used in treating viral infections like that of Nipah virus. Which of the statements given above are correct?

    1. I and II only
    2. II and III only
    3. I and III only
    4. I, II and III
    Show answer

    Answer: D. Statement I – CORRECT: Monoclonal antibodies (mAbs) are laboratory-engineered proteins that mimic the immune system's natural ability to fight harmful pathogens. They are man-made proteins. Statement II – CORRECT: Produced from a single clone of B-cells, they are designed to bind to specific antigens such as those found on viruses, bacteria, or cancer cells. This targeted action allows them to neutralize threats, mark infected or malignant cells for destruction, or block harmful biological processes. Statement III – CORRECT: mAbs are widely used in treating cancers, autoimmune disorders, and viral infections like COVID-19, Ebola, and Nipah. In India, the monoclonal antibody from Australia has been used on some individuals around the world for Nipah and its use was found to be safe. All three statements are correct → Option (d).

    Difficulty: medium · statement

    Open this question on its own page, with the full explanation →

  3. UPSC Prelims 2019 · Science and Technology · Biotechnology

    RNA interference (RNAi)' technology has gained popularity in the last few years. Why? 1. It is used in developing gene silencing therapies. 2. It can be used in developing therapies for the treatment of cancer. 3. It can be used to develop hormone replacement therapies. 4. It can be used to produce crop plants that are resistant to viral pathogens. Select the correct answer using the code given below.

    1. 1, 2 and 4
    2. 2 and 3
    3. 1 and 3
    4. 1 and 4 only
    Show answer

    Answer: A. RNAi is a gene silencing technology that inhibits protein synthesis in target cells using double-stranded RNA. Statement 1 – CORRECT: RNAi is used in developing gene silencing therapies. Statement 2 – CORRECT: RNAi is used in functional genomics (systematic analysis of loss-of-function phenotypes induced by RNAi triggers) and developing therapies for the treatment of viral infection, dominant disorders, neurological disorders, and many types of cancers. Statement 3 – INCORRECT: RNAi is not specifically used to develop hormone replacement therapies. It is not a recognized application of RNAi technology. Statement 4 – CORRECT: RNAi has huge significance within the Indian context, considering the deep-seated resistance over the years to Bt cotton and other genetically modified seeds. It can be used to produce crop plants that are resistant to viral pathogens. Correct statements: 1, 2 and 4 → Option (a).

    Difficulty: hard · statement

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  4. UPSC Prelims 2020 · Science and Technology · Biotechnology

    Consider the following statements: 1. Genetic changes can be introduced in the cells that produce eggs or sperms of a prospective parent. 2. A person's genome can be edited before birth at the early embryonic stage. 3. Human induced pluripotent stem cells can be injected into the embryo of a pig. Which of the statements given above is/are correct?

    1. 1 only
    2. 2 and 3 only
    3. 2 only
    4. 1, 2 and 3
    Show answer

    Answer: D. Statement 1 – CORRECT: Germline gene therapy targets the reproductive cells, meaning any changes made to the DNA will be passed on to the next generation. DNA is transferred into the cells that produce reproductive cells — eggs or sperm — in the body. Hence statement 1 is correct. Statement 2 – CORRECT: Although germline gene therapy is illegal, embryonic genetic editing is different and it is possible to make genome changes to eliminate the vertical transfer (through inheritance) of certain diseases. Hence statement 2 is correct. Statement 3 – CORRECT: Human-animal chimeras are animals bearing human cells and organs. Researchers have experimented by injecting several different forms of human stem cells into pig embryos. The cells that survived longest were intermediate human pluripotent stem cells. Hence statement 3 is correct. Correct statements: 1, 2 and 3 → Option (d).

    Difficulty: medium · statement

    Open this question on its own page, with the full explanation →

  5. UPSC Prelims 2024 · Science and Technology · Biotechnology

    In which of the following are hydrogels used? 1. Controlled drug delivery in patients 2. Mobile air-conditioning systems 3. Preparation of industrial lubricants Select the correct answer using the code given below:

    1. 1 only
    2. 1 and 2 only
    3. 2 and 3 only
    4. 1, 2 and 3
    Show answer

    Answer: D. Hydrogels are cross-linked polymeric networks swollen in biological fluid. They are widely used in the field of medicine. Their uses are varied. Use 1 – CORRECT: Hydrophilic polymeric networks capable of imbibing huge volumes of water and undergoing swelling and shrinkage suitably to facilitate controlled drug-release are called hydrogels. Their porosity and compatibility with aqueous environments make them highly attractive bio-compatible drug delivery vehicles. Use 2 – CORRECT: Due to its water-absorbing properties, hydrogels may be used to cool down electronic items (mobile air-conditioning systems). Use 3 – CORRECT: Researches show hydrogels can be used as lubricants because of their excellent anti-friction and anti-wear properties. All three uses are correct → Option (d).

    Difficulty: hard · direct

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Practice questions

  1. Under India's New Drugs and Clinical Trials Rules, 2019, an orphan drug treats a condition affecting how many people in India?

    1. Not more than 50,000
    2. Not more than 1 lakh
    3. Not more than 5 lakh
    4. Not more than 10 lakh
    Show answer

    Answer: C. The Indian rules set the threshold at not more than five lakh people in India.

    Difficulty: easy · statement

  2. Consider these statements about Rule 101: 1. It can help enable a local-trial waiver for certain orphan drugs. 2. Approval in a specified foreign jurisdiction automatically grants Indian approval. 3. Gene and cellular therapies are among the specified categories. Which statements are correct?

    1. 1 and 3 only
    2. 1 and 2 only
    3. 2 and 3 only
    4. 1, 2 and 3
    Show answer

    Answer: A. Statements 1 and 3 are correct. Statement 2 is wrong: CDSCO must still review the medicine, and a local-trial waiver is conditional.

    Difficulty: medium · statement

  3. What is the main purpose of an advance market commitment?

    1. Extend drug patents indefinitely
    2. Provide a future market for a successful product and reduce investment risk
    3. Replace clinical trials
    4. Ban imported medicines
    Show answer

    Answer: B. An advance commitment to fund purchases, subject to agreed conditions, gives developers greater confidence that a successful product will find buyers.

    Difficulty: medium · statement

Mains practice

Answer-writing practice on this article. Attempt it first, then open the hints.

  1. GS2 · 150 words

    Why do rare diseases create a distinctive challenge for affordable healthcare in India?

    Show hints
    1. Explain small patient groups and limited commercial incentives.
    2. Connect research costs and trial difficulties to high prices.
    3. Assess the ₹50-lakh support limit against expensive lifelong care.
    4. Suggest public purchasing and affordable domestic development.
  2. GS3 · 250 words

    Examine India's potential to become a global centre for developing and manufacturing affordable orphan drugs.

    Show hints
    1. Discuss the patient base and the role of registries.
    2. Assess manufacturing and biotechnology strengths.
    3. Explain conditional local-trial waivers and safety safeguards.
    4. Consider incentives, purchase commitments and research partnerships.
    5. Link innovation to affordability and access for patients.