{"id":30880,"date":"2024-10-15T18:29:41","date_gmt":"2024-10-15T12:59:41","guid":{"rendered":"https:\/\/www.nextias.com\/ca\/?p=30880"},"modified":"2024-10-15T18:29:43","modified_gmt":"2024-10-15T12:59:43","slug":"treatment-of-rare-diseases","status":"publish","type":"post","link":"https:\/\/www.nextias.com\/ca\/current-affairs\/15-10-2024\/treatment-of-rare-diseases","title":{"rendered":"Treatment of Rare Diseases"},"content":{"rendered":"\n<p><strong>Syllabus: GS2\/ Health<\/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 Delhi High Court issued directions aimed at improving the availability of orphan drugs, which are medications used to treat rare diseases.<\/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, also known as <strong>orphan diseases,<\/strong> 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 1 in 1,000 to 2,000 people.\u00a0<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Status of rare diseases in India<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Around<strong> 55 medical conditions<\/strong>, including Gaucher\u2019s disease, Lysosomal Storage Disorders (LSDs), and certain forms of muscular dystrophy are classified as rare diseases in India.\u00a0<\/li>\n\n\n\n<li><strong>The National Registry for Rare and Other Inherited Disorders (NRROID) <\/strong>started by the Indian Council of Medical Research (ICMR) has the records of 14,472 rare disease patients in the country.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Challenges in the Treatment of Rare Diseases\u00a0<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Limited Availability:<\/strong> Less than 5% of rare diseases have available therapies, leaving fewer than 1 in 10 patients with access to disease-specific care.<\/li>\n\n\n\n<li><strong>High Cost: <\/strong>Many rare disease treatments are patented, leading to high prices due to limited market size and high development costs.\n<ul class=\"wp-block-list\">\n<li>Pharmaceutical companies find it unprofitable to produce these drugs, further driving up costs.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>Delays in approval processes<\/strong>: the National Rare Diseases Committee discussed delays in the Drug Controller General of India (DCGI) approving Sarepta Therapeutics&#8217; medicines, leaving patients without timely access.<\/li>\n\n\n\n<li><strong>Unequal Treatment Across Groups:<\/strong> While limited assistance is available for Group 1 and Group 2 diseases, Group 3 patients face significant financial and healthcare barriers.\u00a0<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>National Policy for Rare Diseases (NPRD), 2021\u00a0<\/strong><\/h2>\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<h2 class=\"wp-block-heading\"><strong>Other initiatives taken in India<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>The Health Ministry has opened a <strong>Digital Portal for Crowdfunding &amp; Voluntary Donations <\/strong>with information about patients and their rare diseases.\n<ul class=\"wp-block-list\">\n<li>Donors can choose the CoE and patient treatments they wish to support.\u00a0<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Each CoE also has its own <strong>Rare Disease Fund<\/strong>, which is used with approval from its governing authority.<\/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<h2 class=\"wp-block-heading\"><strong>Way Ahead<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Domestic Manufacturing:<\/strong> Developing and manufacturing orphan drugs within India can significantly reduce costs.\n<ul class=\"wp-block-list\">\n<li>The government should offer incentives such as <strong>tax breaks and subsidies<\/strong> to encourage pharmaceutical companies to invest in research and production of rare disease treatments.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>Leveraging the Patents Act of 1970:<\/strong> If treatments for rare diseases are unavailable or unaffordable, the government can use provisions under the Patents Act, 1970, to enable third-party manufacturing of patented drugs.\u00a0<\/li>\n\n\n\n<li><strong>Faster approval<\/strong> processes for life-saving therapies will ensure that patients get quicker access to essential treatments.<\/li>\n\n\n\n<li><strong>A sustainable, long-term funding mechanism<\/strong> needs to be established, especially for Group 3 rare diseases, to cover both immediate and lifelong treatment costs.\u00a0<\/li>\n<\/ul>\n\n\n\n<p><strong>Source: <\/strong><a href=\"https:\/\/indianexpress.com\/article\/explained\/explained-health\/issues-in-the-treatment-of-rare-diseases-and-what-the-govt-can-do-9618942\/\" target=\"_blank\" rel=\"noopener\"><strong>IE<\/strong><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>The Delhi High Court issued directions aimed at improving the availability of orphan drugs, which are medications used to treat rare diseases.<\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[21],"tags":[],"class_list":["post-30880","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\/30880","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\/4"}],"replies":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/comments?post=30880"}],"version-history":[{"count":1,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/30880\/revisions"}],"predecessor-version":[{"id":30881,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/30880\/revisions\/30881"}],"wp:attachment":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/media?parent=30880"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/categories?post=30880"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/tags?post=30880"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}