The Orphan Drugs Access in Italy: The Role of the EARLY Access Tools in the Last FIVE YEARS
Author(s)
Prada M, Mariano EE, Molchanova E, Rossi L
Intexo Società Benefit, Rome, RM, Italy
INTRODUCTION: In Italy, several provisions govern the access of drugs approved by EMA before the NHS reimbursement. It is the so-called early access. OBJECTIVES: This study aims to analyse the impact of early access tools (Law 648, Law 326 and compassionate use programmes (CUPs)) on access to orphan drugs (ODs). METHODS: The drugs’ panel was built with all the ODs approved by EMA between 2015 and 2020. Info about Law 648, 326 and CUPs are available on AIFA’s website, OSMED Report and Italian OJ. RESULTS: Between 2015 and 2020 EMA’s CHMP recommended 64 ODs. 41/64 ODs(64%) are marketed. Of these, 22/41(54%) are classified in H class, 9/41(22%) in A, 7/41(17%) in C and 3/41(7%) in C(nn). 20/41(49%) ODs currently marketed had an early access: 10/41(24%) through CUPs, 6/41 (15%) through Law 326, 4/41(10%) through Law 648 and 3/41(7%) ODs launched in C(nn) class before their reimbursement. 13/41 ODs marketed (32%) with EA were also recognized as innovative by AIFA. Of the 23 ODs not marketed, 7(30%) are available through EAPs (4/23 (17%) CUP; 3/23(13%) 326). Of the 16 ODs not marketed nor available via EAP, 5 are under AIFA negotiation, 2 are in A class, 1 is in H class, 1 is in C class and 5 are in C(nn) class. The median negotiation time was 287 days for the ODs with early access vs 327 for ODs without . The access to the AIFA Fund 5% (Law 326) was requested for 20 patients (expenditure of about 7.4 MIO €) in 2016 and for 60 patients (expenditure of about 12.9 MIO €) in 2017. CONCLUSIONS: Our analysis highlighted a positive impact of various early access tools on the availability of ODs.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PRO71
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Pricing Policy & Schemes
Disease
Rare and Orphan Diseases