Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case

• DOI: 10.1186/s40545-023-00570-z
• Publication Date: 2023-05-17T13:02:34Z
• Authors: Alice Tarantola; Monica Hildegard ...; Patrizio Armeni; Francesco Costa; Francesco Malandrini; Claudio Jommi
• Keywords: Reimbursement

Early access programs (EAPs) generally refer to patient medicines/indications before marketing authorization, possibly extended price and reimbursement approval. These include compassionate use, which is usually covered by pharmaceutical companies, EAPs reimbursed third-party payers. This paper aims at comparing in four European countries (France, Italy, Spain, UK) providing empirical evidence on Italy. The comparative analysis was conducted through a literature review (including scientific grey literature), complemented 30-min semi-structured interviews with local experts. Italian employed data available the National Medicines Agency website. Although are very different across countries, they exhibit some common features: (i) eligibility criteria absence of valid therapeutic alternatives presumed favourable risk-benefit profile; (ii) payers do not allocate pre-determined budget these programs; (iii) total spending unknown. French seem be most structured, financed social insurance, covering pre-marketing, post-marketing pre-reimbursement phases for collection. Italy's approach has been varied, several payers, including cohort-based 648 List (for both early off-label use), nominal-based 5% Fund, Compassionate Use. Most applications from Antineoplastic immunomodulating drug class (ATC L). Some 62% indications either under clinical development or have never approved (pure use). For those subsequently approved, coincide EAPs. Only Fund provides economic impact (€ 81.2 million 2021; average cost per € 61.5K). Diverse possible source inequalities medicines Europe. A harmonization programs, though difficult achieve, could modelled provide key advantages, least effort collect real-world parallel trials clear separation between use programs.