{"id":23150,"date":"2020-08-12T12:20:04","date_gmt":"2020-08-12T12:20:04","guid":{"rendered":"https:\/\/technopolis-group.com\/?p=23150"},"modified":"2020-09-04T08:59:53","modified_gmt":"2020-09-04T08:59:53","slug":"20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises","status":"publish","type":"post","link":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/","title":{"rendered":"20 years on: did the EU Orphan Regulation deliver on its promises?"},"content":{"rendered":"\n

In 2000, European Member States adopted the \u2018EU Orphan Regulation\u2019. Its purpose was to stimulate the development of new medicines for treatment of rare diseases, better known as \u2018orphan medicines\u2019. There are an estimated 30 million people living with one of around 7,000 known rare diseases in the EU alone, but because each of these diseases affects no more than 1 in 2,000 people, for a long time pharmaceutical companies did not see a sufficient business case for investing in research and development of treatments.<\/p>\n\n\n\n

When the EU\nintroduced the Regulation, it followed the examples of the US, which had introduced\nthe US Orphan Drug Act already back in 1983, and Japan. Like its American\ncounterpart, the EU Orphan Regulation offers developers of orphan medicines a\nfinancial incentive in the form of so-called \u2018market exclusivity\u2019. This\nexclusivity gives a marketing authorisation holder of a designated orphan\nmedicine the sole right to market the medicine in the EU for a period of 10\nyears, provided there are no better treatment alternatives for the designated\nindication. The hope was that by giving companies the ability to protect their\nrevenue streams for longer, they would be incentivised to invest in R&D in\nareas that would otherwise be too unattractive.<\/p>\n\n\n\n

Twenty years on, the significant increase in orphan medicines on the market would suggest that the regulation has succeeded in what it set out to do. On 11 August 2020, the European Commission published its own evaluation<\/a> of the Orphan and Paediatric Regulations. This evaluation draws heavily on the simultaneously published evaluation of the EU Orphan Regulation<\/a> prepared by Technopolis Group and Ecorys. Our evaluation finds that, by the end of 2017, 142 new orphan medicines had been authorised in the EU for 107 unique conditions, including some very rare ones. Additionally, these medicines have become available in more EU markets and did so faster than before.<\/p>\n\n\n\n

Pulling the\nright strings<\/strong><\/h3>\n\n\n\n

So does this mean\nthe regulation should be considered an unfettered success? Unfortunately, in\nthe real world few things are never that simple. For one, for the vast majority\nof rare diseases there are not only no treatments on the market, but also no\npromising products in the pipelines. One area where the impact of the\nregulation has been particularly disappointing is in delivering new  treatments for rare diseases that primarily\naffect children. This had been noted previously in the evaluation of the EU Paediatric Regulation<\/a>, also led by Technopolis Group. Meanwhile, orphan\ndesignations are increasingly being granted for conditions where there are\nalready other treatments available. Clearly, the regulation is not sufficiently\nmanaging to steer industry toward the areas of greatest unmet need. Of course\nthe blame for this cannot be placed solely at the feet of the regulation, as\nproduct development can be done only where basic reseach has already provided\ninsights into mechanisms of disease. Nonetheless, the attention of the industry\nappears strongly correlated with areas of highest profits, most notably the\nlucrative field of anti-cancer medicines.<\/p>\n\n\n\n

The high\nprice of innovation<\/strong><\/h3>\n\n\n\n

Anyone who has\npaid attention to the debates concerning the role of Big Pharma will be at\nleast somewhat familiar with another major issue associated with the\nregulation: that of affordability and availability of orphan medicines. The\nregulation grants a period of market exclusivity to the marketing authorisation\nholder but does not ask for any guarantees on availability and affordability of\nthose medicines in return. In recent years, there have been numerous examples\nof orphan medicines that have been brought to market with eye-watering price\ntags. These prices cause many to question whether a regulation that is meant to\nincentivise development in areas where there is no commercial potential, is not\ngiving unnecessary hand-outs to Big Pharma. Our analysis finds that indeed the\nrisk of \u2018overcompensation\u2019 exists, although it also suggests that on average\nthere is a reasonable balance between the costs of development and the rewards\noffered by the regulation. Nonetheless, the balance may be lost in cases where\nthere has been little investment in R&D, such as with repurposed products,\nor where there is an annual turnover of over \u20ac100 million. Moreover, the\navailability of orphan medicines is highly uneven across the EU and this inequality\nof access may be set to increase even further. Unfortunately, the regulation\noffers no tools to address this issue.<\/p>\n\n\n\n

Keeping up\nwith science<\/strong><\/h3>\n\n\n\n

The evaluation furthermore\nidentifies a number of potential challenges to the regulation associated with\nadvances in medical science, such as personalised medicine, new clinical trial\ndesigns and real world evidence. Whilst these advances are set to offer\nimportant benefits to patients, by improving the effectiveness and safety of\nmedicines, and getting these to patients more quickly, the current regulatory\nframework is ill-equipped to deal with them.<\/p>\n\n\n\n

Where do we\ngo from here?<\/strong><\/h3>\n\n\n\n

The evaluation\nmakes clear that, for all of its positive impacts, the regulation has also had\nsome consequences that at the time of introduction policy makers did not\nforesee. Meanwhile, the field of medicine and the dynamics in the market for\norphan medicines have evolved. It is thus timely to reconsider what measures\nshould remain, which ones should be added, which removed and how the overall\narchitecture of the pharmaceutical regulatory framework can be improved. <\/p>\n\n\n\n

As the EC embarks\non the process of the European Pharmaceutical Review, policy makers face the difficult\ntask of deciding how the regulation can be amended to improve its effectiveness\nand minimise the unintended consequences. In doing so, they should be careful\nnot to let the perfect become the enemy of the good. <\/p>\n","protected":false},"excerpt":{"rendered":"

In 2000, European Member States adopted the \u2018EU Orphan Regulation\u2019. […]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[4],"tags":[],"solution":[137],"sector":[73,74],"class_list":["post-23150","post","type-post","status-publish","format-standard","hentry","category-non-classifiee","solution-evaluation-fr","sector-health-life-sciences-fr","sector-higher-education-fr"],"acf":[],"yoast_head":"\n20 years on: did the EU Orphan Regulation deliver on its promises? - Technopolis Group<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"20 years on: did the EU Orphan Regulation deliver on its promises? - Technopolis Group\" \/>\n<meta property=\"og:description\" content=\"In 2000, European Member States adopted the \u2018EU Orphan Regulation\u2019. […]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/\" \/>\n<meta property=\"og:site_name\" content=\"Technopolis Group\" \/>\n<meta property=\"article:published_time\" content=\"2020-08-12T12:20:04+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2020-09-04T08:59:53+00:00\" \/>\n<meta name=\"author\" content=\"cedric\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"\u00c9crit par\" \/>\n\t<meta name=\"twitter:data1\" content=\"cedric\" \/>\n\t<meta name=\"twitter:label2\" content=\"Dur\u00e9e de lecture estim\u00e9e\" \/>\n\t<meta name=\"twitter:data2\" content=\"5 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/\"},\"author\":{\"name\":\"cedric\",\"@id\":\"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/eeed59af0b9e4a5e3a50d148502a3256\"},\"headline\":\"20 years on: did the EU Orphan Regulation deliver on its promises?\",\"datePublished\":\"2020-08-12T12:20:04+00:00\",\"dateModified\":\"2020-09-04T08:59:53+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/\"},\"wordCount\":926,\"commentCount\":0,\"articleSection\":[\"Non classifi\u00e9(e)\"],\"inLanguage\":\"fr-FR\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/\",\"url\":\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/\",\"name\":\"20 years on: did the EU Orphan Regulation deliver on its promises? - Technopolis Group\",\"isPartOf\":{\"@id\":\"https:\/\/technopolis-group.com\/nl\/#website\"},\"datePublished\":\"2020-08-12T12:20:04+00:00\",\"dateModified\":\"2020-09-04T08:59:53+00:00\",\"author\":{\"@id\":\"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/eeed59af0b9e4a5e3a50d148502a3256\"},\"breadcrumb\":{\"@id\":\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#breadcrumb\"},\"inLanguage\":\"fr-FR\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\/\/technopolis-group.com\/fr\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"20 years on: did the EU Orphan Regulation deliver on its promises?\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\/\/technopolis-group.com\/nl\/#website\",\"url\":\"https:\/\/technopolis-group.com\/nl\/\",\"name\":\"Technopolis Group\",\"description\":\"Technopolis-group\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\/\/technopolis-group.com\/nl\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"fr-FR\"},{\"@type\":\"Person\",\"@id\":\"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/eeed59af0b9e4a5e3a50d148502a3256\",\"name\":\"cedric\",\"image\":{\"@type\":\"ImageObject\",\"inLanguage\":\"fr-FR\",\"@id\":\"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/image\/\",\"url\":\"https:\/\/secure.gravatar.com\/avatar\/497d412f48cec5d772f00904ec9eeece7ccdc8c4c13e9c38824de5ce441fab90?s=96&d=mm&r=g\",\"contentUrl\":\"https:\/\/secure.gravatar.com\/avatar\/497d412f48cec5d772f00904ec9eeece7ccdc8c4c13e9c38824de5ce441fab90?s=96&d=mm&r=g\",\"caption\":\"cedric\"},\"url\":\"https:\/\/technopolis-group.com\/fr\/author\/cedric\/\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"20 years on: did the EU Orphan Regulation deliver on its promises? - Technopolis Group","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/","og_locale":"fr_FR","og_type":"article","og_title":"20 years on: did the EU Orphan Regulation deliver on its promises? - Technopolis Group","og_description":"In 2000, European Member States adopted the \u2018EU Orphan Regulation\u2019. […]","og_url":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/","og_site_name":"Technopolis Group","article_published_time":"2020-08-12T12:20:04+00:00","article_modified_time":"2020-09-04T08:59:53+00:00","author":"cedric","twitter_card":"summary_large_image","twitter_misc":{"\u00c9crit par":"cedric","Dur\u00e9e de lecture estim\u00e9e":"5 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#article","isPartOf":{"@id":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/"},"author":{"name":"cedric","@id":"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/eeed59af0b9e4a5e3a50d148502a3256"},"headline":"20 years on: did the EU Orphan Regulation deliver on its promises?","datePublished":"2020-08-12T12:20:04+00:00","dateModified":"2020-09-04T08:59:53+00:00","mainEntityOfPage":{"@id":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/"},"wordCount":926,"commentCount":0,"articleSection":["Non classifi\u00e9(e)"],"inLanguage":"fr-FR","potentialAction":[{"@type":"CommentAction","name":"Comment","target":["https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#respond"]}]},{"@type":"WebPage","@id":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/","url":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/","name":"20 years on: did the EU Orphan Regulation deliver on its promises? - Technopolis Group","isPartOf":{"@id":"https:\/\/technopolis-group.com\/nl\/#website"},"datePublished":"2020-08-12T12:20:04+00:00","dateModified":"2020-09-04T08:59:53+00:00","author":{"@id":"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/eeed59af0b9e4a5e3a50d148502a3256"},"breadcrumb":{"@id":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#breadcrumb"},"inLanguage":"fr-FR","potentialAction":[{"@type":"ReadAction","target":["https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/technopolis-group.com\/fr\/20-years-on-did-the-eu-orphan-regulation-deliver-on-its-promises\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/technopolis-group.com\/fr\/"},{"@type":"ListItem","position":2,"name":"20 years on: did the EU Orphan Regulation deliver on its promises?"}]},{"@type":"WebSite","@id":"https:\/\/technopolis-group.com\/nl\/#website","url":"https:\/\/technopolis-group.com\/nl\/","name":"Technopolis Group","description":"Technopolis-group","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/technopolis-group.com\/nl\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"fr-FR"},{"@type":"Person","@id":"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/eeed59af0b9e4a5e3a50d148502a3256","name":"cedric","image":{"@type":"ImageObject","inLanguage":"fr-FR","@id":"https:\/\/technopolis-group.com\/nl\/#\/schema\/person\/image\/","url":"https:\/\/secure.gravatar.com\/avatar\/497d412f48cec5d772f00904ec9eeece7ccdc8c4c13e9c38824de5ce441fab90?s=96&d=mm&r=g","contentUrl":"https:\/\/secure.gravatar.com\/avatar\/497d412f48cec5d772f00904ec9eeece7ccdc8c4c13e9c38824de5ce441fab90?s=96&d=mm&r=g","caption":"cedric"},"url":"https:\/\/technopolis-group.com\/fr\/author\/cedric\/"}]}},"publishpress_future_action":{"enabled":false,"date":"2026-04-11 09:40:35","action":"change-status","newStatus":"draft","terms":[],"taxonomy":"category","extraData":[]},"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/posts\/23150","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/comments?post=23150"}],"version-history":[{"count":0,"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/posts\/23150\/revisions"}],"wp:attachment":[{"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/media?parent=23150"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/categories?post=23150"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/tags?post=23150"},{"taxonomy":"solution","embeddable":true,"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/solution?post=23150"},{"taxonomy":"sector","embeddable":true,"href":"https:\/\/technopolis-group.com\/fr\/wp-json\/wp\/v2\/sector?post=23150"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}