{"id":126675,"date":"2026-09-29T11:38:52","date_gmt":"2026-09-29T06:08:52","guid":{"rendered":"https:\/\/vajiramandravi.com\/current-affairs\/?p=126675"},"modified":"2026-09-29T11:38:52","modified_gmt":"2026-09-29T06:08:52","slug":"rare-disease-drugs","status":"publish","type":"post","link":"https:\/\/vajiramandravi.com\/current-affairs\/rare-disease-drugs\/","title":{"rendered":"Rare-Disease Drugs: Can India Become a Global Drug Hub?"},"content":{"rendered":"<h2><b>Rare-Disease Drugs Latest News<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">India transformed global healthcare by making generic drugs affordable. Experts argue that the next frontier for India is to make rare-disease drugs accessible.\u00a0<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">With a streamlined regulatory pathway and government procurement, India could build a globally competitive rare-disease pharmaceutical sector.<\/span><\/li>\n<\/ul>\n<h2><b>Understanding the Scale of the Problem<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Each rare disease affects very few people, but collectively the numbers are staggering.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">There are around <\/span><b>8,000 rare diseases worldwide<\/b><span style=\"font-weight: 400;\">, cumulatively affecting hundreds of millions of people.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">In India, 1,004 rare genetic disorders have been reported so far.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Because patient numbers for any single disease are tiny, pharmaceutical interest has historically been limited \u2014 hence the term &#8220;<\/span><b>orphan drugs<\/b><span style=\"font-weight: 400;\">.&#8221;<\/span><\/li>\n<\/ul>\n<h2><b>How Other Countries Solved This: The Orphan Drug Act<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">To overcome industry hesitancy, several governments introduced incentives.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">The <\/span><b>US Orphan Drug Act<\/b><span style=\"font-weight: 400;\"> (1983) offered tax incentives, seven years of market exclusivity, grants, and regulatory fee waivers.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Impact:<\/b><span style=\"font-weight: 400;\"> Before the Act, only 38 relevant drugs had been approved in the US. By 2022, this rose to 1,122 approvals.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Japan, Australia, and the European Union later adopted similar legislation.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>The catch:<\/b><span style=\"font-weight: 400;\"> Because markets remain small, companies set very high prices. A year&#8217;s treatment can cost $100,000 (\u20b91 crore) or more \u2014 putting it out of reach without insurance or government support.<\/span><\/li>\n<\/ul>\n<h2><b>India&#8217;s Current Support System \u2014 and Its Limits<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">The Indian government supports rare disease patients with up to <\/span><b>\u20b950 lakh<\/b><span style=\"font-weight: 400;\"> total for treatment.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">This is often insufficient to cover even a year&#8217;s costs, forcing patients toward crowdfunding.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Example:<\/b><span style=\"font-weight: 400;\"> In mid-2025, \u20b99 crore was crowdfunded for gene therapy for a 16-month-old child in Kolkata.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Nearly 4,000 children are listed on a Government of India crowdfunding platform, with cumulative annual treatment costs exceeding \u20b99,000 crore.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Existing drugs cover only about 5% of rare diseases \u2014 most patients have no suitable drug at all, even at high cost.<\/span><\/li>\n<\/ul>\n<h2><b>The Two Core Challenges in Drug Development<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Finding a patient cohort large enough for clinical trials.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Determining trial endpoints \u2014 what &#8220;success&#8221; means for a very rare condition.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Beyond approval, affordable, high-quality small-scale manufacturing and distribution remain major hurdles.<\/span><\/li>\n<\/ul>\n<h2><b>India&#8217;s Advantage: A Natural Trial Ground<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">India&#8217;s demographics make it uniquely suited for rare-disease research.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">With a population exceeding 1.4 billion, India has <\/span><b>large absolute numbers of patients<\/b><span style=\"font-weight: 400;\"> even for very rare conditions.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>High rates of endogamy<\/b><span style=\"font-weight: 400;\"> in certain communities raise disease prevalence further. For instance, beta-thalassemia prevalence is 10.5% in specific ethnic groups, versus 3.7% in India&#8217;s general population (2023 review).<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">The Indian diaspora&#8217;s gene pool carries unique mutations across ethnicities, adding further research value.<\/span><\/li>\n<\/ul>\n<h3><b>The Gap<\/b><\/h3>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Indian patients rarely participate in international orphan-drug trials, despite this potential.\u00a0<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Analysts suggest government-fostered collaboration between foreign companies and local organisations \u2014 similar to India&#8217;s approach in chip manufacturing \u2014 and inviting multinational firms to set up rare-disease drug-development centres with patient groups.<\/span><\/li>\n<\/ul>\n<h2><b>A Benefit-Sharing Model for Pricing<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Experts propose a key principle: Indian patients participating in drug development should be <\/span><b>beneficiaries of the resulting drugs<\/b><span style=\"font-weight: 400;\">, not just data sources.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Proposal: India should be <\/span><b>allowed to decide the price of drugs<\/b><span style=\"font-weight: 400;\"> sold domestically or in the Global South, while companies remain free to set prices elsewhere.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">This echoes the benefit-sharing concept discussed under the WHO&#8217;s Pandemic Agreement, where pathogen information shared globally must lead to fair, equitable access to resulting drugs and vaccines.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">US and Chinese firms are already racing on rare-disease drugs, with China leveraging patient access and low-cost manufacturing. Western companies may find India an attractive collaboration partner too.<\/span><\/li>\n<\/ul>\n<h2><b>India&#8217;s Manufacturing Strength<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">India is already the <a href=\"https:\/\/vajiramandravi.com\/current-affairs\/pharma-sector-in-india\/\" target=\"_blank\">&#8220;<\/a><\/span><a href=\"https:\/\/vajiramandravi.com\/current-affairs\/pharma-sector-in-india\/\" target=\"_blank\"><b>pharmacy of the world<\/b><\/a><span style=\"font-weight: 400;\"><a href=\"https:\/\/vajiramandravi.com\/current-affairs\/pharma-sector-in-india\/\" target=\"_blank\">&#8220;<\/a> for generics.<\/span>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">India fills 47% of US generic prescriptions.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">It has the largest number of US-regulator-approved manufacturing sites outside the US.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Indian firms supply 15% of all biosimilars sold in the US.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Indian facilities now produce CAR-T therapy \u2014 a personalised cancer treatment \u2014 to international standards.<\/span><\/li>\n<\/ul>\n<\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Regulatory shortcut: <\/span><b>Under Rule 101<\/b><span style=\"font-weight: 400;\"> of the <\/span><i><span style=\"font-weight: 400;\">New Drugs and Clinical Trials Rules, 2019<\/span><\/i><span style=\"font-weight: 400;\">, the CDSCO allows drugs already approved in six specified countries (including the US and UK) to skip a fresh Indian clinical trial before regulatory approval.\u00a0<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">This is a strong incentive for manufacturing such drugs locally \u2014 though the authors call for greater transparency and guidance documents to ease the application process.\u00a0<\/span><\/li>\n<\/ul>\n<h2><b>What the Government Should Do<\/b><\/h2>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><span style=\"font-weight: 400;\">Analysts propose a policy package modelled on Production-Linked Incentive (PLI) schemes:<\/span>\n<ul>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Tax incentives for investing in orphan drug manufacturing.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Production-linked incentives for approved orphan drugs.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Support for technology transfer with national laboratories.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Advance Market Commitments (AMCs) \u2014 government pre-committing to buy a certain output at a set price.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Distribution through Centres of Excellence on Rare Diseases, the Central Government Health Scheme, and Pradhan Mantri Bhartiya Jan Aushadhi Kendras.<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"2\"><span style=\"font-weight: 400;\">Incentives for exporting to low- and middle-income countries.<\/span><\/li>\n<\/ul>\n<\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>The Underlying Philosophy<\/b><span style=\"font-weight: 400;\">: The goal shouldn&#8217;t be simply subsidising companies, but de-risking initial investment enough to make orphan drug manufacturing commercially viable on its own.<\/span><\/li>\n<\/ul>\n<p><strong>Source: <a href=\"https:\/\/www.thehindu.com\/sci-tech\/health\/what-hinders-indian-pharma-companies-from-making-drugs-for-rare-diseases\/article70371582.ece\" target=\"_blank\" rel=\"nofollow noopener\">TH<\/a> | <a href=\"https:\/\/www.rarediseases.in\/made-in-india-missing-in-india-the-orphan-drug-access-challenge\/\" target=\"_blank\" rel=\"nofollow noopener\">RD<\/a><\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Rare-disease drugs could be India&#8217;s next pharmaceutical frontier, using manufacturing strength, regulatory reforms and government incentives to improve access and affordability.<\/p>\n","protected":false},"author":18,"featured_media":126712,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[18],"tags":[60,10549,22,59],"class_list":["post-126675","post","type-post","status-publish","format-standard","has-post-thumbnail","category-upsc-mains-current-affairs","tag-mains-articles","tag-rare-disease-drugs","tag-upsc-current-affairs","tag-upsc-mains-current-affairs","no-featured-image-padding"],"acf":[],"_links":{"self":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/126675","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/users\/18"}],"replies":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/comments?post=126675"}],"version-history":[{"count":4,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/126675\/revisions"}],"predecessor-version":[{"id":126729,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/126675\/revisions\/126729"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/media\/126712"}],"wp:attachment":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/media?parent=126675"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/categories?post=126675"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/tags?post=126675"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}