{"id":85868,"date":"2026-09-29T18:17:07","date_gmt":"2026-09-29T12:47:07","guid":{"rendered":"https:\/\/www.nextias.com\/ca\/?p=85868"},"modified":"2026-09-29T18:18:33","modified_gmt":"2026-09-29T12:48:33","slug":"rare-disease-drugs-india","status":"publish","type":"post","link":"https:\/\/www.nextias.com\/ca\/current-affairs\/29-09-2026\/rare-disease-drugs-india","title":{"rendered":"India\u2019s Emerging Pharmaceutical Opportunity in Rare-Disease Drugs"},"content":{"rendered":"\n<p><strong>Syllabus: GS2\/ Health, GS3\/ Economy<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Context<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>The growing burden of rare diseases has highlighted the need to strengthen India\u2019s rare-disease pharmaceutical ecosystem and leverage its capabilities in affordable drug manufacturing.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>What are Rare Diseases?<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Rare diseases are conditions that occur infrequently within a population.\n<ul class=\"wp-block-list\">\n<li><strong>They are characterized by three key markers: <\/strong>Total number of people with the disease, Prevalence and Availability \/Non-availability of treatment options.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>The World Health Organization (WHO)<\/strong> defines a rare disease as a condition that affects a small percentage of the population, typically fewer than <strong>1 in 1,000<\/strong> to 2,000 people.\u00a0<\/li>\n\n\n\n<li>There are around <strong>8,000 rare diseases globally<\/strong>, while 1,004 rare genetic disorders have been reported in India.<\/li>\n\n\n\n<li>Drugs developed to treat rare diseases are called<strong> orphan drugs.<\/strong><\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>The Global Orphan Drug Market<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>The US Orphan Drug Act, 1983<\/strong>, introduced financial and regulatory incentives for developing drugs for rare diseases.<\/li>\n\n\n\n<li>The United States had approved only <strong>38 orphan drugs<\/strong> before the Act, compared with <strong>1,122 approvals by 2022.<\/strong><\/li>\n\n\n\n<li>Despite these incentives, the <strong>high cost of research, development and treatment <\/strong>continues to restrict patient access.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Challenges in Developing Rare-Disease Drugs<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Commercial incentives: <\/strong>The small number of patients suffering from individual rare diseases<strong> restricts the potential market for their medicines<\/strong>, reducing the commercial incentives for pharmaceutical companies to invest in research and development.<\/li>\n\n\n\n<li><strong>Clinical trials:<\/strong> Identifying sufficiently large patient cohorts is difficult because individual rare diseases affect relatively few people.<\/li>\n\n\n\n<li><strong>Regulatory constraints:<\/strong> Complex regulatory procedures and the need for additional clinical studies increase the time and cost involved in introducing orphan drugs in India.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Potential for India\u2019s Rare-Disease Pharmaceutical Sector<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Diverse patient population:<\/strong> India\u2019s large population and higher prevalence of certain genetic disorders due to endogamy provide opportunities for patient identification and clinical trials.<\/li>\n\n\n\n<li><strong>Manufacturing capabilities: <\/strong>India\u2019s expertise in generic medicines, biosimilars and advanced therapies, including<strong> CAR-T Cell Therapy<\/strong>, provides a strong foundation for orphan drug manufacturing.\n<ul class=\"wp-block-list\">\n<li><strong>Skilled professionals, established infrastructure and relatively low production costs<\/strong> can support affordable, small-scale production.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>International collaboration:<\/strong> Partnerships between foreign pharmaceutical companies, Indian research institutions and patient organisations can accelerate drug development and clinical trials.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Measures to Promote Rare-Disease Drug Development in India<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Under Rule 101 of the New Drugs and Clinical Trials Rules 2019<\/strong>, the Central Drugs Standard Control Organisation (CDSCO) has specified six countries \u2014 including the <strong>U.S. and the U.K<\/strong>.\u2014and if one of these countries approves a drug, the drug need not have a new clinical trial conducted in India before its maker seeking the Indian regulator\u2019s approval.<\/li>\n\n\n\n<li>The Department of Pharmaceuticals has launched the <strong>Production Linked Incentive (PLI) Scheme<\/strong> for Pharmaceuticals, offering financial incentives to selected manufacturers for <strong>domestic production of orphan drugs.<\/strong><\/li>\n<\/ul>\n\n\n\n<p><\/p>\n\n\n\n<div class=\"wp-block-group has-background\" style=\"background-color:#fff2cc\"><div class=\"wp-block-group__inner-container is-layout-constrained wp-block-group-is-layout-constrained\">\n<h3 class=\"wp-block-heading\"><strong>National Policy for Rare Diseases (NPRD), 2021&nbsp;<\/strong><\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>It was launched in <strong>2021,<\/strong> under which financial assistance up to Rs 50 lakh is provided to patients receiving treatment at an identified Centre of Excellence (CoE).<\/li>\n\n\n\n<li>In India, rare diseases are categorized into three groups based on the nature and complexity of available treatment options.\n<ul class=\"wp-block-list\">\n<li><strong>Group 1<\/strong> includes diseases that can be treated with a one-time curative procedure.\u00a0<\/li>\n\n\n\n<li><strong>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.\u00a0<\/li>\n\n\n\n<li><strong>Group 3<\/strong> diseases are those for which effective treatments are available, but they are expensive and must often continue lifelong.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>Initiatives Taken for Rare Disease Treatment<\/strong><\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>The Unique Methods for Management of Inherited Disorders (UMMID) initiative <\/strong>is designed to tackle newborn and rare genetic disorders through early screening, affordable molecular diagnostics, and clinical counselling.\n<ul class=\"wp-block-list\">\n<li>It establishes <strong>NIDAN Kendras (National Inherited Disorders Administration Kendras) <\/strong>to provide prenatal testing, genetic counselling, and multidisciplinary care in government hospitals.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<\/div><\/div>\n\n\n\n<p><\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>Way Ahead<\/strong><\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>India\u2019s established pharmaceutical manufacturing capabilities, diverse patient population and growing biotechnology sector provide opportunities to expand into rare-disease drug development.<\/li>\n\n\n\n<li>A coordinated approach combining research support, streamlined regulation, sustainable financing and assured patient access can help India develop a <strong>globally competitive orphan drug industry<\/strong> while<strong> addressing the unmet healthcare needs of its population.<\/strong><\/li>\n<\/ul>\n\n\n\n<p><strong>Source: <\/strong><a href=\"https:\/\/www.thehindu.com\/sci-tech\/science\/rare-disease-drugs-could-become-indias-next-pharma-frontier\/article71518560.ece\" target=\"_blank\" rel=\"noopener\"><strong>TH<\/strong><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p><strong> Context <\/strong><\/p>\n<li class=\"ms-5\"> The growing burden of rare diseases has highlighted the need to strengthen India\u2019s rare-disease pharmaceutical ecosystem and leverage its capabilities in affordable drug manufacturing. <\/li>\n<p><\/p>\n<p><strong> What are Rare Diseases? <\/strong><\/p>\n<li class=\"ms-5\"> \u00a0Rare diseases are conditions that occur infrequently within a population. <\/li>\n<li class=\"ms-5\"> They are characterized by three key markers: Total number of people with the disease, Prevalence and Availability \/Non-availability of treatment options. <\/li>\n<p><a href=\"https:\/\/www.nextias.com\/ca\/current-affairs\/29-09-2026\/rare-disease-drugs-india\" class=\"btn btn-primary btn-sm float-end\">Read More<\/a><\/p>\n","protected":false},"author":15,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[21],"tags":[],"class_list":["post-85868","post","type-post","status-publish","format-standard","hentry","category-current-affairs"],"acf":[],"jetpack_featured_media_url":"","_links":{"self":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/85868","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/users\/15"}],"replies":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/comments?post=85868"}],"version-history":[{"count":2,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/85868\/revisions"}],"predecessor-version":[{"id":85871,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/85868\/revisions\/85871"}],"wp:attachment":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/media?parent=85868"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/categories?post=85868"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/tags?post=85868"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}