{"id":972,"date":"2020-02-18T11:14:33","date_gmt":"2020-02-18T05:44:33","guid":{"rendered":"https:\/\/medicineplexus.com\/?p=972"},"modified":"2020-02-18T11:14:33","modified_gmt":"2020-02-18T05:44:33","slug":"orphan-drugs","status":"publish","type":"post","link":"https:\/\/medicineplexus.com\/orphan-drugs\/","title":{"rendered":"Orphan Drugs"},"content":{"rendered":"\n<p><strong>Orphan Drugs<\/strong><\/p>\n\n\n\n<p>The Orphan Drug Designation program of US-FDA provides orphan status<strong> to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases\/disorders.<\/strong><\/p>\n\n\n\n<p><strong>What is Rare<\/strong><\/p>\n\n\n\n<ul><li>The World Health Organization <strong>(W.H.O.)<\/strong> has suggested that a rare disease should be defined as one with frequency less than <strong>6.5 \u2013 10 per 10,000 people<\/strong><\/li><li>Considering the large population of India, <strong><em>ORDI<\/em> (<em>Organisation for Rare Diseases in India<\/em>)<\/strong> suggests a disease to be defined as rare if it affects less than <strong>1 in 5,000 people<\/strong><strong><\/strong><\/li><\/ul>\n\n\n\n<p>A rare disease occurs infrequently in a population, but there is no universal definition.<\/p>\n\n\n\n<p>3 elements to the definition as used in various countries are as follows:<\/p>\n\n\n\n<p>1. The total number of people having the disease<\/p>\n\n\n\n<p>2. Its prevalence<\/p>\n\n\n\n<p>3. Non-availability of treatment for the disorder<\/p>\n\n\n\n<ul><li><strong>Authorities in different countries or regions use different cut-off values:<\/strong><ul><li>&nbsp;WHO defines an incidence of &lt;0.65-1\/1000 as \u2018Rare\u2019<\/li><\/ul><ul><li>&nbsp;<strong>US defines &lt;2,00,000 (<\/strong>1\/1500) patients as \u2018Rare\u2019<\/li><\/ul><ul><li>&nbsp;EU defines &lt;5\/10,000 as \u2018Rare\u2019<\/li><\/ul><ul><li>&nbsp;Japan defines &lt;50,000 as \u2018Rare\u2019<\/li><\/ul><ul><li>&nbsp;Australia defines &lt;2000 as \u2018Rare<\/li><\/ul><\/li><\/ul>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"467\" height=\"265\" src=\"https:\/\/medicineplexus.com\/wp-content\/uploads\/2020\/02\/image-429.png?w=467\" alt=\"\" class=\"wp-image-989\" srcset=\"https:\/\/medicineplexus.com\/wp-content\/uploads\/2020\/02\/image-429.png 467w, https:\/\/medicineplexus.com\/wp-content\/uploads\/2020\/02\/image-429-300x170.png 300w\" sizes=\"(max-width: 467px) 100vw, 467px\" \/><\/figure>\n\n\n\n<p>As of 2014, there were 281 marketed orphan drugs and more than 400 orphan-designated drugs in clinical trials. More than 60% of orphan drugs were biologics. The U.S. dominated development of orphan drugs, with more than 300 in clinical trials, followed by Europe.<\/p>\n\n\n\n<p><strong><u>ORPHAN DRUG ACT <\/u>\u2013<\/strong><\/p>\n\n\n\n<ul><li>The Orphan Drug Act (ODA) of January <strong>1983, passed in the <\/strong><a href=\"http:\/\/en.wikipedia.org\/wiki\/United_States\"><strong>United States<\/strong><\/a>, is meant to encourage <a href=\"http:\/\/en.wikipedia.org\/wiki\/Pharmaceutical_company\"><strong>pharmaceutical companies<\/strong><\/a><strong> to develop drugs for diseases that have a small market<\/strong><\/li><li>The law is designed to facilitate the <strong>development and commercialization of drugs to treat rare diseases<\/strong><\/li><li>The <strong>salient features<\/strong> of the act are as follows:<ul><li><strong>Incentives<\/strong> to Pharma companies investing in Orphan Drugs R &amp; D<\/li><\/ul><\/li><li><strong>Tax Credits-<\/strong>50% of Clinical Investigations Expenses<\/li><li><strong>Protocol Assistance<\/strong> in Drug Development Process<\/li><li><strong>Grant Support<\/strong>-Medical Foods and Medical Services also<\/li><li>Open Protocols to increase drug availability for patients<ul><li><strong>Faster\/Priority clearance<\/strong> of Orphan Drugs Application through FDA<\/li><\/ul><ul><li><strong>Market exclusivity<\/strong><strong> for 7 years<\/strong> for Pharma companies to recover costs<\/li><\/ul><\/li><\/ul>\n\n\n\n<p><strong>Organisation for Rare Diseases India (ORDI)<\/strong><\/p>\n\n\n\n<ul><li>Launched on Feb. 18, <strong>2014<\/strong><\/li><li>Theoretically, 400 US FDA &amp; 80 European Medicines Agency (EMA) approved orphan drugs are available<\/li><li>Practically,&nbsp; most are either not accessible to most patients in India or are unaffordable<\/li><li>It is ORDI\u2019s <strong>mission to make these and new approved orphan drugs easily accessible and affordable to patients with rare diseases in India<\/strong><\/li><li>Currently, drug developers in India are receiving no formal incentives from the Government and hence are more focused on developing affordable drugs<\/li><\/ul>\n\n\n\n<p><strong>How to apply for Orphan Drug Designation<\/strong><\/p>\n\n\n\n<p>A <strong>sponsor shall submit two copies of a completed, dated and signed request<\/strong> for designation that contains the following:<\/p>\n\n\n\n<ol type=\"1\"><li>A statement that the sponsor requests <strong>orphan-drug designatio<\/strong>n for a rare disease or condition<\/li><li>The <strong>name and address of the sponsor<\/strong>; the name of the sponsor&#8217;s primary contact person<\/li><li>A <strong>description of the rare disease<\/strong> or condition for which the drug is being or will be investigated, the <strong>proposed use of the drug, and the reasons why such therapy is needed<\/strong>.<\/li><li>A description of the drug, to include the identity of the active moiety, <strong>all relevant data from in vitro laboratory studies, preclinical efficacy studies.<\/strong><\/li><li>Where the <strong>sponsor seeks approval of an already approved orphan-drug, stating an explanation as to why the proposed variation in his drug may be clinically superior to the first drug<\/strong>.<\/li><li>A <strong>summary of the regulatory status and marketing history of the drug in the United States and in foreign countries<\/strong><\/li><li>Documentation, with authoritative references<\/li><\/ol>\n\n\n\n<p><strong>After Designation Request Is Submitted\u2026<\/strong><\/p>\n\n\n\n<ul><li>Typical review cycle ~ 90 days (often less)<\/li><li>Will either receive:<\/li><\/ul>\n\n\n\n<p>-Designation Letter OR<\/p>\n\n\n\n<p>-Deficiency Letter<\/p>\n\n\n\n<ul><li>Once designated, sponsor is required to <strong>submit annual reports until drug is approved<\/strong><\/li><\/ul>\n\n\n\n<p><strong>Development of Orphan drugs is costlier because:<\/strong><\/p>\n\n\n\n<ul><li>The <strong>limitations <\/strong>of medical and scientific <strong>knowledge related to the pathogenesis<\/strong>&nbsp;<\/li><li>A <strong>limited market<\/strong>&nbsp;<\/li><li><strong>High research and manufacturing costs<\/strong> (50 to 80 per cent of rare diseases are from genetic origin)<\/li><li>&nbsp;Use of high-tech products, genetic therapies, cellular therapies, recombinant proteins&nbsp;<\/li><li>Impossibility of patenting certain ingredients<\/li><\/ul>\n\n\n\n<p><strong>Financial incentives available to orphan drug developers in India<\/strong><\/p>\n\n\n\n<ul><li>Currently, drug developers in India are receiving <strong>no formal incentives from the Government<\/strong> and hence they are more focused on o developing affordable drugs for more common diseases such as oral insulin, statins for preventing or slowing the progression of cardiovascular disease, vaccines and antibiotics for a number of preventable infectious diseases, etc.<\/li><li>Hence, <strong>patients with rare diseases in India have to rely on imported drugs from western countries<\/strong> which makes these treatments (even when available) unaffordable.<\/li><\/ul>\n\n\n\n<ul><li>Many infectious diseases qualify for incentives to develop orphan drugs in developed countries<\/li><\/ul>\n\n\n\n<p>&nbsp;&nbsp;&nbsp; e.g. Two of the top \ufb01ve infectious disease killers in the world, qualify as rare diseases in developed&nbsp; nations<\/p>\n\n\n\n<ul><li>&nbsp;Tuberculosis: Rifampicin and Rifapentine&nbsp;&nbsp;<\/li><li>Malaria: Halofantrine, Me\ufb02oquine, Quinine sulphate<\/li><\/ul>\n","protected":false},"excerpt":{"rendered":"<p>Orphan Drugs The Orphan Drug Designation program of US-FDA provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases\/disorders. What is Rare The World Health Organization (W.H.O.) has suggested that a rare disease should be defined as one with frequency[&#8230;]\n","protected":false},"author":3,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_seopress_robots_primary_cat":"","_seopress_titles_title":"","_seopress_titles_desc":"","_seopress_robots_index":"","footnotes":""},"categories":[3],"tags":[],"_links":{"self":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/posts\/972"}],"collection":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/comments?post=972"}],"version-history":[{"count":0,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/posts\/972\/revisions"}],"wp:attachment":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/media?parent=972"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/categories?post=972"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/tags?post=972"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}