{"id":372903,"date":"2026-09-29T19:17:53","date_gmt":"2026-09-29T13:47:53","guid":{"rendered":"https:\/\/forumias.com\/blog\/?p=372903"},"modified":"2026-09-29T19:17:53","modified_gmt":"2026-09-29T13:47:53","slug":"tackling-rare-diseases-in-india","status":"publish","type":"post","link":"https:\/\/forumias.com\/blog\/tackling-rare-diseases-in-india\/","title":{"rendered":"Tackling Rare Diseases in India"},"content":{"rendered":"

Source: <\/strong>The post \u201cTackling Rare Diseases in India\u201d <\/strong>has been created based on “Tackling Rare Diseases in India\u201d<\/strong> published in \u201cThe Hindu<\/strong>\u201d on 29th September 2026.<\/p>\n

UPSC Syllabus: GS-3- Indian Economy<\/strong><\/p>\n

Context:<\/strong> Rare diseases individually affect a small number of people, but collectively affect hundreds of millions worldwide<\/strong>. India has reported 1,004 rare genetic disorders<\/strong>. The small market size has historically discouraged pharmaceutical companies from developing such medicines, leading to the term \u201corphan drugs.\u201d<\/strong><\/p>\n

About Rare Disease<\/strong><\/h2>\n
    \n
  1. The World Health Organization (WHO) defines rare diseases as debilitating, lifelong conditions that affect one or fewer persons per 1,000.<\/li>\n
  2. Some commonly recognised rare diseases include Haemophilia, Pompe disease, Thalassemia, Sickle-cell Anaemia, and Gaucher\u2019s disease<\/strong>.<\/li>\n<\/ol>\n

    Status of Rare Disease in India<\/strong><\/h2>\n
      \n
    1. In India, rare diseases are categorised into three groups based on the nature and complexity of available treatment options.\n
        \n
      1. Group 1<\/strong> includes diseases that can be treated with a one-time curative procedure.<\/li>\n
      2. Group 2<\/strong> diseases require long-term or lifelong treatment which are relatively less costly and have shown documented benefits, but patients need regular check-ups.<\/li>\n
      3. Group 3 diseases <\/strong>are those for which effective treatments are available, but they are expensive and must often continue lifelong. There are challenges in selecting the right beneficiaries for these treatments.<\/li>\n<\/ol>\n<\/li>\n
      4. It is estimated that around 6%\u20138% of India\u2019s population lives with a rare disease<\/strong>, translating to approximately 8.4 – 10 crore people.<\/li>\n
      5. Currently, 63 rare diseases are included under the National Policy for Rare Diseases, <\/strong>which was recommended by the Central Technical Committee for Rare Diseases (CTCRD).<\/li>\n
      6. Therapies are available for less than 5% of rare diseases,<\/strong> leading to less than 1 in 10 patients receiving disease-specific care.<\/li>\n
      7. Many medicines and therapies for rare diseases are patented,<\/strong> which makes them very expensive. <\/strong><\/li>\n
      8. The market for these drugs is small and the development costs are also high,<\/strong> so pharmaceutical companies often don\u2019t find it profitable to produce them, pushing up prices. Therefore, these drugs are called \u2018orphan drugs\u2019<\/strong><\/li>\n<\/ol>\n

        Challenges<\/strong><\/h2>\n
          \n
        1. Limited commercial incentive: <\/strong>The small patient population reduces the incentive for pharmaceutical companies to invest in research and development of orphan drugs.<\/li>\n
        2. High cost of medicines: <\/strong>Rare-disease medicines can cost around \u20b91 crore or more per patient annually<\/strong>, making them inaccessible without government support or insurance.<\/li>\n
        3. Insufficient financial support: <\/strong>Although the Government of India provides support of up to \u20b950 lakh<\/strong>, this may not cover the total treatment cost, forcing families to depend on crowdfunding.<\/li>\n
        4. Limited availability of drugs: <\/strong>Existing rare-disease drugs address only around 5% of rare diseases and associated conditions<\/strong>, leaving most patients without suitable treatment.<\/li>\n
        5. Difficulty in clinical trials: <\/strong>Identifying adequate patient cohorts and determining suitable clinical endpoints<\/strong> are major challenges in developing orphan drugs.<\/li>\n
        6. Limited participation of Indian patients: <\/strong>Despite India’s large patient population, Indian patients are generally underrepresented in international orphan-drug clinical trials<\/strong>.<\/li>\n
        7. Small-scale manufacturing: <\/strong>After approval, producing and distributing medicines in small volumes at affordable prices while maintaining quality<\/strong> remains difficult.<\/li>\n
        8. Regulatory challenges: <\/strong>Greater transparency and clearer guidance for drug developers and sponsors can improve the regulatory approval process.<\/li>\n
        9. Access versus benefit-sharing: <\/strong>Patients participating in research should be able to access the resulting medicines. Developing expensive drugs that remain unaffordable locally limits the social benefit of such research.<\/li>\n
        10. Global competition: <\/strong>Countries such as the U.S.A and China are investing heavily in rare-disease drug development, creating both competition and opportunities for international collaboration.<\/li>\n<\/ol>\n

          India’s Opportunities<\/strong><\/h2>\n
            \n
          1. Large patient pool: <\/strong>India’s population provides a potentially large cohort for clinical trials. <\/strong>Certain communities also have higher prevalence of particular genetic disorders because of endogamy<\/strong>.<\/li>\n
          2. Patient advocacy networks: <\/strong>Patient groups and disease-specific registries can help identify suitable participants for clinical research.<\/li>\n
          3. Unique genetic diversity: <\/strong>Indian and diaspora populations possess unique genetic mutations<\/strong>, providing opportunities for research into rare diseases.<\/li>\n
          4. Strong pharmaceutical manufacturing base: <\/strong>India has established capabilities in generics, biosimilars and newer therapies such as CAR-T<\/strong>, and can manufacture medicines at relatively low cost.<\/li>\n<\/ol>\n

            Way Forward<\/strong><\/h2>\n
              \n
            1. Promote global\u2013Indian collaboration: <\/strong>Encourage multinational pharmaceutical companies to establish rare-disease drug-development centres in India<\/strong> in collaboration with Indian organisations and patient groups.<\/li>\n
            2. Increase Indian participation in trials: <\/strong>Integrate Indian patients into international clinical trials while ensuring that participating communities can benefit from the resulting medicines.<\/li>\n
            3. Adopt benefit-sharing: <\/strong>Drug-development benefits should be shared fairly and equitably<\/strong>, including affordable access to medicines in India and the Global South.<\/li>\n
            4. Streamline regulation: <\/strong>Develop clearer regulatory guidance and reduce unnecessary duplication while maintaining bioequivalence, safety, toxicity, efficacy, pharmacovigilance and manufacturing standards<\/strong>.<\/li>\n
            5. Provide targeted incentives: <\/strong>Use production-linked incentives, tax incentives and investment support<\/strong> to encourage domestic orphan-drug manufacturing.<\/li>\n
            6. Support technology transfer: <\/strong>Facilitate technology transfer and process development through collaboration between pharmaceutical companies and national laboratories<\/strong>.<\/li>\n
            7. Use Advanced Market Commitments: <\/strong>The government can commit to purchasing a specified quantity at an agreed price, thereby providing manufacturers with predictable demand<\/strong>.<\/li>\n
            8. Strengthen public procurement and distribution: <\/strong>Medicines can be distributed through Centres of Excellence for Rare Diseases, CGHS and Pradhan Mantri Bhartiya Jan Aushadhi Kendras<\/strong>.<\/li>\n
            9. Promote exports: <\/strong>Incentives can encourage Indian manufacturers to supply affordable rare-disease medicines to low- and middle-income countries<\/strong>.<\/li>\n
            10. De-risk rather than merely subsidise: <\/strong>Government policy should reduce the initial financial risks sufficiently to make orphan-drug manufacturing commercially viable<\/strong>, rather than simply subsidising companies.<\/li>\n<\/ol>\n

              Conclusion:<\/strong> India already possesses the manufacturing capacity, large patient pool, scientific potential and pharmaceutical expertise<\/strong> required to enter the rare-disease drug sector. A combination of affordable access, patient participation, streamlined regulation, targeted incentives, government procurement and international collaboration<\/strong> can help India move from being the \u201cpharmacy of the world\u201d to becoming a significant producer of accessible rare-disease medicines<\/strong>.<\/p>\n

              Question:<\/strong> Rare diseases pose challenges of affordability, accessibility and limited research. Discuss how India can leverage its pharmaceutical capabilities to develop a globally competitive rare-disease drug industry.<\/p>\n

              Source: <\/strong>The Hindu<\/strong><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

              Source: The post \u201cTackling Rare Diseases in India\u201d has been created based on “Tackling Rare Diseases in India\u201d published in \u201cThe Hindu\u201d on 29th September 2026. UPSC Syllabus: GS-3- Indian Economy Context: Rare diseases individually affect a small number of people, but collectively affect hundreds of millions worldwide. India has reported 1,004 rare genetic disorders.… Continue reading Tackling Rare Diseases in India<\/span><\/a><\/p>\n","protected":false},"author":10320,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"jetpack_post_was_ever_published":false,"footnotes":""},"categories":[1230],"tags":[216,8184,10498],"class_list":["post-372903","post","type-post","status-publish","format-standard","hentry","category-9-pm-daily-articles","tag-gs-paper-3","tag-indian-economy","tag-the-hindu","entry"],"jetpack_featured_media_url":"","views":"","jetpack_sharing_enabled":true,"_links":{"self":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/372903","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/users\/10320"}],"replies":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/comments?post=372903"}],"version-history":[{"count":0,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/372903\/revisions"}],"wp:attachment":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/media?parent=372903"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/categories?post=372903"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/tags?post=372903"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}