The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

Abstract

Italy has a well-established prominent system of national registries to support managed entry agreements (MEAs), monitoring innovative medicinal products (MPs) with clinical as well as economic uncertainties to ensure appropriate use and best value for money. The technological architecture of the registries is funded by pharmaceutical companies, but fully governed by the national medicines agency (AIFA). A desktop analysis was undertaken of data over a 15-year timeframe of all AIFA indication-based registries and associated EMA information. The characteristics of registries were evaluated, comparing orphan MPs vs. all MPs exploring cancer and non-cancer indications. OMP (orphan medicinal product) registries’ type vs. AIFA innovation status and EMA approval was reviewed. Of the 283 registries, 182 are appropriateness registries (35.2% relate to OMPs, with an almost equal split of cancer vs. non-cancer for OMPs and MPs), 35 include financial-based agreements [20% OMPs (2 non-cancer, 5 cancer)], and 60 registries are payment by result agreements [23.3% OMPs (4 non-cancer, 10 cancer)]. Most OMPs (53/88) came through the normal regulatory route. With the strengthening of the system for evaluation of innovation, fewer outcomes-based registries have been instigated. AIFA has overcome many of the challenges experienced with MEA through developing an integrated national web-based data collection system: the challenge that remains for AIFA is to move from using the system for individual patient decisions about treatment to reviewing the wealth of data it now holds to optimize healthcare.

Impact of the COVID‐19 pandemic on prescription of sacubitril/valsartan in Italy

“…The specifics of AIFA monitoring registries, including their position within the Italian regulatory framework, have been discussed previously. 7 , 8 The inclusion in a monitoring registry is mandatory to obtain the reimbursement from the Italian National Healthcare System (NHS) for the prescription of sacubitril/valsartan to treat heart failure in Italy. Therefore, data presented in this work represent a census of patients treated with sacubitril/valsartan during the observational period.…”

“…The analysis was conducted using data from the Italian Medicines Agency (AIFA) monitoring registries. The specifics of AIFA monitoring registries, including their position within the Italian regulatory framework, have been discussed previously 7,8 . The inclusion in a monitoring registry is mandatory to obtain the reimbursement from the Italian National Healthcare System (NHS) for the prescription of sacubitril/valsartan to treat heart failure in Italy.…”

A Proposal for Value-Based Managed Entry Agreements in an Environment of Technological Change and Economic Challenge for Publicly Funded Healthcare Systems

“…AIFA registries are an important operational part of the PR negotiation between AIFA and the pharmaceutical industry that constitutes a key element of the contract between the two parties. The MEAs implemented in the AIFA registries are also part of the PR contracts and therefore have the same validity as the registries ( 11 ). The peculiarity of these instruments is that they are not planned by the industry but are decided during the PR procedure by the Italian payer.…”

“…Italy has a distinct system of national registries to support MEAs that has been in place for fifteen years, verifying the appropriateness of innovative hospital MPs with consideration of clinical as well as economic uncertainties to ensure best value for money ( 11 ). The Italian regulation recognizes registries as an integral part of the NHS information system, while the additional policies that were introduced for the purposes of attributing specific responsibilities for PR re-negotiation of medicinal products ( 14 – 17 ).…”

Postlaunch evidence generation practices among health technology assessment bodies in Europe

“…Approaches for generating quality real-world evidence (RWE) with different purposes (coverage with evidence development, managed entry agreement, clinical evaluation after launch or licensure, etc.) have been launched by many countries, including several European countries such as France (4), Italy (5;6), Spain (7;8), UK (9;10), Germany, Sweden, and The Netherlands (11). Other initiatives such as the HTAi Global Policy Forum (GPF) meeting in November 2018 aimed to discuss the future availability and use of RWE in the context of HTA processes to inform decision making (12).…”