{"id":14674,"date":"2021-04-05T00:00:00","date_gmt":"2021-04-05T00:00:00","guid":{"rendered":"https:\/\/www.nextias.com\/current_affairs\/uncategorized\/05-04-2021\/national-policy-for-rare-diseases-2021\/"},"modified":"2021-04-05T00:00:00","modified_gmt":"2021-04-05T00:00:00","slug":"national-policy-for-rare-diseases-2021","status":"publish","type":"post","link":"https:\/\/www.nextias.com\/ca\/current-affairs\/05-04-2021\/national-policy-for-rare-diseases-2021","title":{"rendered":"National Policy for Rare Diseases, 2021"},"content":{"rendered":"<p><strong>In News<\/strong><\/p>\n<p>Recently, the Government of India has announced the <strong>National Policy for Rare Diseases, 2021<\/strong>.<\/p>\n<p><strong>Rare Diseases<\/strong><\/p>\n<ul>\n<li>Rare diseases (also called \u201c<strong>Orphan<\/strong>\u201d<strong> diseases<\/strong>) are broadly defined as diseases that <strong>infrequently occur in a population<\/strong> and<strong> three markers<\/strong> are used (the total number of people with the disease, its prevalence, and the availability\/non-availability of treatment options).<\/li>\n<li>The <strong>World Health Organisation <\/strong>(WHO) defines a rare disease as having a<strong> frequency of less than 6.5-10 per 10,000 <\/strong>people.\n<ul>\n<li>As per an estimate, there are <strong>7,000 known rare diseases with an estimated 300 million patients <\/strong>in the world.<\/li>\n<\/ul>\n<\/li>\n<li><strong>Challenges in Fighting Rare Diseases<\/strong>\n<ul>\n<li>These diseases have <strong>differing definitions <\/strong>in various countries and <strong>present fundamentally different challenges<\/strong> from those of more common diseases.\n<ul>\n<li>According to the<strong> Organization for Rare Diseases India<\/strong>, rare diseases include inherited cancers, autoimmune disorders, congenital malformations, Hirschsprung\u2019s disease, Gaucher disease, cystic fibrosis, muscular dystrophies and Lysosomal Storage Disorders (LSDs)<\/li>\n<\/ul>\n<\/li>\n<li>Less than <strong>5% have therapies available to treat <\/strong>them and about <strong>95% have no approved treatment<\/strong> and <strong>less than 1 in 10 patients<\/strong> receive <strong>disease-specific treatment<\/strong>.<\/li>\n<li>Where drugs are available, they are <strong>prohibitively expensive<\/strong>, placing immense <strong>strain <\/strong>on resources and also the government has not been able to provide these for free.<\/li>\n<li>This is most apparent during the <strong>clinical development stage<\/strong> when<strong> rarity significantly complicates the task<\/strong>.\n<ul>\n<li>Problems include the small number of patients, the logistics involved in reaching widely dispersed patients, the lack of validated biomarkers and surrogate end-points, and limited clinical expertise and expert centres.<\/li>\n<\/ul>\n<\/li>\n<li>In India, there is a <strong>lack of epidemiological data on the prevalence<\/strong> here and hence has only <strong>classified certain diseases<\/strong> as \u2018rare\u2019.\n<ul>\n<li>There are an <strong>estimated 70 million patients<\/strong> in India.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<\/li>\n<li><strong>Steps Taken<\/strong>\n<ul>\n<li>The<strong> International Rare Diseases Research Consortium<\/strong> (IRDiRC) was launched in <strong>2011 <\/strong>to foster international collaboration in rare diseases research.<\/li>\n<li>However, despite these positive developments, the burden of rare diseases continues to persist.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p><strong>National Policy for Rare Diseases, 2021<\/strong><\/p>\n<ul>\n<li>It <strong>offers financial support<\/strong> for one-time treatment of up to Rs. 20 lakh, introduces a <strong>crowdfunding mechanism<\/strong>, creates a<strong> registry of rare diseases<\/strong> and <strong>provides for early detection<\/strong>.<\/li>\n<li>It was <strong>first prepared <\/strong>by the Centre in <strong>2017 <\/strong>but was put on hold due to questions on its formation, criterias, cost sharing, beneficiaries, etc.<\/li>\n<li>An<strong> expert group <\/strong>was constituted in <strong>2018 <\/strong>to review these questions, which submitted its <strong>report in January 2021<\/strong> and after a further round of consultation, the policy has been made public.<\/li>\n<li>It categories \u2018rare disease\u2019 into <strong>three groups<\/strong>.\n<ul>\n<li><strong>Group 1: <\/strong>Disorders amenable to one-time curative treatment.\n<ul>\n<li>Eligible for a one-time treatment cost of up to Rs. 20 lakh provided the beneficiaries conformed to definition of the <strong>Pradhan Mantri Jan Arogya Yojana<\/strong> and were treated in government tertiary care hospital, under the umbrella scheme of <strong>Rashtriya Arogya Nidhi<\/strong> (RAN).\n<ul>\n<li>RAN provides for<strong> financial assistance<\/strong> to patients, <strong>living below poverty line<\/strong> (BPL) and who are suffering from major life threatening diseases, to receive medical treatment at any of the <strong>super specialty Government hospitals\/institutes<\/strong>.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<\/li>\n<li><strong>Group 2:<\/strong> Diseases requiring long term\/lifelong treatment having relatively lower cost of treatment and benefit has been documented in literature and annual or more frequent surveillance is required.\n<ul>\n<li>States could \u201cconsider\u201d supporting patients of such rare diseases that could be managed with special diets or hormonal supplements.<\/li>\n<\/ul>\n<\/li>\n<li><strong>Group 3:<\/strong> Diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy.<\/li>\n<\/ul>\n<\/li>\n<li>The government would<strong> notify selected Centres of Excellence<\/strong> at premier government hospitals for comprehensive management of rare diseases.\n<ul>\n<li>The Centres of Excellence would be <strong>provided a one-time grant to a maximum of Rs. 5 crore each <\/strong>for infrastructure development for screening, tests, treatment.<\/li>\n<\/ul>\n<\/li>\n<li><strong>Significance<\/strong>\n<ul>\n<li>It will increase focus on<strong> indigenous research and local production of medicines<\/strong>.<\/li>\n<li>Under <strong>crowdfunding<\/strong>, <strong>corporates and individuals<\/strong> will be encouraged to extend financial support through a robust IT platform.<\/li>\n<li>The national hospital-based registry will <strong>ensure adequate data and comprehensive definitions<\/strong> of such diseases.<\/li>\n<li><strong>Early screening and detection<\/strong> will help the prevention of rare diseases.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p><strong>Concerns Related to the Policy<\/strong><\/p>\n<ul>\n<li>It offers <strong>no support to patients <\/strong>under the<strong> earlier National Policy for Treatment of Rare Diseases 2017<\/strong>.<\/li>\n<li>Patients with<strong> Group 3 <\/strong>rare diseases are left to fend for themselves due to<strong> lack of proper selection criteria<\/strong>.<\/li>\n<li>In the <strong>absence of a sustainable funding support<\/strong> for Group 3 patients, the lives of all patients, mostly children, are now at risk and at the mercy of crowdfunding.<\/li>\n<li>Even<strong> Group 1 is only for a few <\/strong>and <strong>Group 2 has been openly left for the State government<\/strong>.<\/li>\n<li>The policy <strong>fails to capture that these diseases last a lifetime<\/strong> and also does not realise that <strong>people might not be able to even make it to the prescribed tertiary hospitals<\/strong> for treatment.<\/li>\n<li>Diseases such as LSD for which definitive treatment is available, but costs are prohibitive, have been categorised as Group 3. However, <strong>no funding has been allocated<\/strong> for the <strong>immediate and lifelong treatment needs<\/strong>, for therapies already approved by the <strong>Drugs Controller General of India<\/strong> (DGCI).<\/li>\n<\/ul>\n<p><strong>Suggestions<\/strong><\/p>\n<ul>\n<li>There is a need to <strong>balance competing priorities of public health<\/strong> in \u201cresource-constrained\u201d settings.<\/li>\n<li>The Ministry of Health and Family Welfare should come up with an<strong> immediate seed-funding of Rs. 80 crore to Rs. 100 crore<\/strong> so that the life-saving therapy of all patients can be provided, thereby reducing further loss of life.<\/li>\n<li>It should also <strong>design and execute a 100-day roll-out plan<\/strong> after the national policy is notified <strong>to prioritise treatment <\/strong>of all eligible rare disease patients and <strong>prioritise and encourage States with a matching grant<\/strong>.<\/li>\n<li>If the Centre is able to extend the <strong>cost-sharing agreements <\/strong>that it has worked out with <strong>Kerala, Tamil Nadu and Karnataka<\/strong>, with other States too, its share of the annual costs will be halved.<\/li>\n<\/ul>\n<p>Source: <a href=\"https:\/\/www.thehindu.com\/news\/national\/disquiet-over-govts-new-policy-for-rare-diseases\/article34225606.ece\" target=\"_blank\" rel=\"noopener\">TH<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>In News Recently, the Government of India has announced the National Policy for Rare Diseases, 2021. Rare Diseases Rare diseases (also called \u201cOrphan\u201d diseases) are broadly defined as diseases that infrequently occur in a population and three markers are used (the total number of people with the disease, its prevalence, and the availability\/non-availability of treatment [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":14675,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[21],"tags":[31,30,47,39,55],"class_list":["post-14674","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-current-affairs","tag-government-policies-interventions","tag-gs-2","tag-health","tag-issues-arising-out-of-their-design-implementation","tag-welfare-schemes-for-vulnerable-sections-of-population-their-performance"],"acf":[],"jetpack_featured_media_url":"https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/ca\/uploads\/2023\/07\/3754785current-affairs.jpg","_links":{"self":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/14674","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\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/comments?post=14674"}],"version-history":[{"count":0,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/14674\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/media\/14675"}],"wp:attachment":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/media?parent=14674"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/categories?post=14674"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/tags?post=14674"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}