POSA218 Hospital Exemptions for Atmps: Friend or Foe for Pharma?

Abstract

Hospital exemption (HE), under EU Regulation (EC) No 1394/2007 Article 28(2), allows for production of unlicensed advanced therapy medicinal products (ATMPs) custom-made for individual patients for use in a hospital setting on a non-routine basis. For patients with unmet clinical needs, HEs grant access to unlicensed, potentially life-saving therapies. Although HE’s increase access to innovative treatments, open interpretation of legislation by national authorities could foster different motives and capacity for ATMP production, encroaching on the traditional pharma model. In conducting a targeted literature review, national differences in HE interpretation, motivation, and scale of use were investigated. Case studies examining past product market withdrawals, successful collaborations between academia and industry, and disruptive innovation were analysed to discern the risks and opportunities faced by industry. There are variations in how countries interpret HE regulations, with some imposing additional requirements for use. This results in inconsistencies across countries in the application of HE, especially in relation to large scale production. Additionally, HE use is increasingly encouraged through policy, with national payers viewing this as a means to drive innovation and reduce treatment costs. From an industry perspective, HEs could potentially be exploited by institutions to circumvent the centralised market authorisation process and produce therapies at lower costs, leading to unfair competition and the creation of a fragmented market. As such, industry is pushing for greater transparency and restrictions on ATMP production under HE. A paradigm shift is already occurring, regardless of objections from industry. With governments and healthcare authorities believing that tighter regulation may harm innovation and lead to decreased patient access, alternative solutions have become imperative. Accordingly, industry must find approaches to collaborate with academia and key centres to leverage rapid innovation, and get treatments to patients earlier whilst ensuring budget sustainability for healthcare systems.