{"id":10796,"date":"2023-05-26T12:08:42","date_gmt":"2023-05-26T10:08:42","guid":{"rendered":"https:\/\/pharmavalue.it\/?p=10796"},"modified":"2023-05-26T12:08:42","modified_gmt":"2023-05-26T10:08:42","slug":"pharma-value-analysis-of-the-national-plan-for-rare-diseases-2023-2026-focus-drugs","status":"publish","type":"post","link":"https:\/\/pharmavalue.it\/en\/pharma-value-analysis-of-the-national-plan-for-rare-diseases-2023-2026-focus-drugs\/","title":{"rendered":"Pharma Value analysis of the National Rare Disease Plan 2023-2026 - focus drugs"},"content":{"rendered":"<p>It was <strong>ratified<\/strong> Monday, <strong>24 May 2023<\/strong>in the State-Regions Conference,<strong> the approval of the National Plan for Rare Diseases 2023-2026<\/strong>, realised after 7 years of work, with the aim of giving concrete answers to people with rare diseases, some 2 million in Italy, and their families.<\/p>\n<p><strong>Pharma Value analysed the chapter on 'Pharmacological Treatments'.<\/strong><\/p>\n<p>The concise text is structured in: Foreword; Objectives; Actions; Instruments; Indicators.<\/p>\n<p><strong>The Logical Framework methodology was used to verify the feasibility of the Plan<\/strong>This is a matrix tool, used in programmes promoted by the European Commission and other international bodies, which is useful for clearly and concisely describing the different elements into which a project idea or plan is articulated, both in the strategy development phase and in the monitoring and evaluation phases.<\/p>\n<p>The analysis was based on the cause-effect relationship between the different project hubs, to verify the sequentiality between general and specific objectives, expected results, indicators and activities.<\/p>\n<p>In general, <strong>we have noticed<\/strong>:<\/p>\n<ul>\n<li><strong>a discrepancy and\/or absence of actions and indicators for certain objectives,<\/strong><\/li>\n<li><strong>ineffective formulation of instruments<\/strong> which, in our opinion, if stated more clearly and consistently should be called 'outputs' or 'results'.<\/li>\n<\/ul>\n<p>In project management terminology, tools are the techniques used to support the realisation of activities, while results are the end point, or product, of a series of activities, aimed at achieving the formulated objectives.<\/p>\n<p>You can see how, even <strong>the use of appropriate terminology to represent the focal points of the National Plan for Rare Diseases can affect<\/strong>not only in understanding the text, but <strong>also in the operational phases of its implementation.<\/strong><\/p>\n<p>Following our analysis <strong>several doubts remain<\/strong>:<\/p>\n<ul>\n<li>Without indicators and actions describing how the objectives of the plan will be implemented, how can the plan be implemented by the regions and autonomous provinces?<\/li>\n<li>Will the territorial gap with different actions region by region continue to widen?<\/li>\n<li>Doesn't the absence of certain actions and indicators constrain planning and could it accentuate the heterogeneity of access to drugs for rare diseases?<\/li>\n<\/ul>\n<p>Our opinion in this regard is that <strong>The National Plan for Rare Diseases 2023-2026, as it is currently formulated, represents a missed opportunity for patients and patients with rare diseases who need the country to work as a system.<\/strong><\/p>\n<p>&nbsp;<\/p>\n<p>If you are interested in learning more, you can read the article on the <a href=\"https:\/\/pharmavalue.it\/en\/summary-of-the-single-text-on-rare-diseases\/\">summary of the Consolidated Text on Diseases<\/a> rare approved on 3 November 2021.<\/p>\n<div class=\"_df_book df-lite\" id=\"df_10800\"  _slug=\"analisi-pharma-value-piano-nazionale-malattie-rare-focus-farmaci\" data-title=\"analisi-pharma-value-piano-nazionale-malattie-rare-focus-farmaci\" wpoptions=\"true\" thumbtype=\"\" ><\/div><script class=\"df-shortcode-script\" nowprocket type=\"application\/javascript\">window.option_df_10800 = {\"outline\":[],\"backgroundColor\":\"eeeeee\",\"duration\":\"0\",\"autoEnableOutline\":\"false\",\"autoEnableThumbnail\":\"false\",\"overwritePDFOutline\":\"false\",\"enableDownload\":\"true\",\"direction\":\"1\",\"pageSize\":\"0\",\"pageMode\":\"1\",\"soundEnable\":\"false\",\"autoPlay\":\"false\",\"source\":\"https:\\\/\\\/pharmavalue.it\\\/wp-content\\\/uploads\\\/2023\\\/05\\\/Analisi-Pharma-Value-Piano-Nazionale-Malattie-Rare-focus-farmaci.pdf\",\"wpOptions\":\"true\"}; if(window.DFLIP && window.DFLIP.parseBooks){window.DFLIP.parseBooks();}<\/script>","protected":false},"excerpt":{"rendered":"<p>\u00c8 stata ratificata luned\u00ec, 24 maggio 2023, in Conferenza Stato-Regioni, l\u2019approvazione del Piano Nazionale Malattie Rare 2023-2026, realizzato dopo 7 anni di lavori, con il fine di dare risposte concrete alle persone con malattia rara, circa 2 milioni in Italia, e ai loro familiari. Pharma Value ha analizzato il capitolo relativo ai \u201cTrattamenti farmacologici\u201d. Il [&hellip;]<\/p>\n","protected":false},"author":314,"featured_media":10810,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_seopress_robots_primary_cat":"none","_seopress_titles_title":"","_seopress_titles_desc":"","_seopress_robots_index":"","footnotes":""},"categories":[9],"tags":[],"class_list":["post-10796","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-appunti"],"_links":{"self":[{"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/posts\/10796","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/users\/314"}],"replies":[{"embeddable":true,"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/comments?post=10796"}],"version-history":[{"count":0,"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/posts\/10796\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/media\/10810"}],"wp:attachment":[{"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/media?parent=10796"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/categories?post=10796"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/pharmavalue.it\/en\/wp-json\/wp\/v2\/tags?post=10796"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}