{"id":199,"date":"2026-07-07T12:43:17","date_gmt":"2026-07-07T10:43:17","guid":{"rendered":"https:\/\/www.pqra.gr\/market-access-for-rare-disease-therapies-navigating-uncertainty\/"},"modified":"2026-07-07T12:46:22","modified_gmt":"2026-07-07T10:46:22","slug":"market-access-for-rare-disease-therapies-navigating-uncertainty","status":"publish","type":"post","link":"https:\/\/www.pqra.gr\/el\/market-access-for-rare-disease-therapies-navigating-uncertainty\/","title":{"rendered":"Market Access for Rare Disease Therapies: Navigating Uncertainty"},"content":{"rendered":"<p style=\"font-size:19px;line-height:1.7;color:#33414d;\"><strong>Rare disease therapies can transform lives &mdash; but getting them reimbursed is one of the hardest challenges in market access. Small populations, limited data and high prices collide with payer budgets and cost-effectiveness thresholds.<\/strong><\/p>\n<p>In the EU, orphan designation is granted under Regulation (EC) No 141\/2000 for conditions affecting no more than 5 in 10,000 people, bringing incentives including ten years&#8217; market exclusivity. But designation is not reimbursement &mdash; access still depends on national HTA and pricing decisions.<\/p>\n<h2>The evidence Catch-22<\/h2>\n<p>With so few patients, large randomised trials are often impossible. Orphan dossiers frequently rest on <strong>single-arm studies, surrogate endpoints or small, immature datasets<\/strong>. The result is a familiar bind: rapid approval on limited evidence, followed by high clinical uncertainty when payers assess value.<\/p>\n<h2>Delays and inequity<\/h2>\n<p>The average time from EU marketing authorisation to actual patient access for orphan medicines is roughly 18 months, and it varies enormously between countries &mdash; leaving patients with the same condition facing very different access depending on where they live.<\/p>\n<h2>Bridging the gap<\/h2>\n<p>High per-patient prices, needed to recoup R&#038;D across tiny populations, sit uneasily with cost-effectiveness thresholds. The main tools to bridge the uncertainty are <strong>managed-entry and risk-sharing agreements<\/strong> and greater use of <strong>real-world evidence<\/strong> to confirm value after launch. From January 2028, orphan medicines also enter the scope of the EU Joint Clinical Assessment, which may harmonise the clinical evaluation &mdash; though pricing and reimbursement stay national.<\/p>\n<h2>The Greek picture<\/h2>\n<p>Greece gives orphan medicines some eased pricing rules &mdash; for example, they can be priced even where reference prices exist in only two Eurozone Member States, and are exempt from the usual requirement to be reimbursed in two-thirds of Member States. Reimbursement runs through EOPYY and the HTA Committee, with an annual horizon-scanning process in place since 2022. Clawback and rebate obligations still weigh heavily on high-cost products.<\/p>\n<h2>How PQRA helps<\/h2>\n<p>PQRA builds orphan-drug value dossiers, designs managed-entry proposals and manages the EOPYY reimbursement route &mdash; turning limited evidence into a defensible case for access.<\/p>\n<p><strong><a href=\"\/el\/contact\/\">Plan your rare-disease access strategy &rarr;<\/a><\/strong><\/p>","protected":false},"excerpt":{"rendered":"<p>Rare disease therapies can transform lives &mdash; but getting them reimbursed is one of the hardest challenges in market access. Small populations, limited data and high prices collide with payer budgets and cost-effectiveness thresholds. In the EU, orphan designation is granted under Regulation (EC) No 141\/2000 for conditions affecting no more than 5 in 10,000 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":210,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"saved_in_kubio":false,"_kadence_starter_templates_imported_post":false,"_swpsp_post_exclude":false,"footnotes":""},"categories":[1],"tags":[],"class_list":["post-199","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Market Access for Rare Disease Therapies: Navigating Uncertainty - PQRA<\/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:\/\/www.pqra.gr\/el\/market-access-for-rare-disease-therapies-navigating-uncertainty\/\" \/>\n<meta property=\"og:locale\" content=\"el_GR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Market Access for Rare Disease Therapies: Navigating Uncertainty - PQRA\" \/>\n<meta property=\"og:description\" content=\"Rare disease therapies can transform lives &mdash; but getting them reimbursed is one of the hardest challenges in market access. 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