Postlaunch evidence generation practices among health technology assessment bodies in Europe

Abstract

Objectives The term Postlaunch Evidence Generation (PLEG) refers to evidence generated after the launch or licensing of a health technology. The aim of this paper is to provide an overview of the implementation of these practices in the European Union in order to explore cross-border cooperation opportunities. Methods In December 2019, a survey composed of nine closed-ended questions with multiple choice answers about the PLEG practices in each country was sent to all twenty-five dedicated work package (WP5B) partners of the European Network of Health Technology Assessment (EUnetHTA) Joint Action 3. In addition to the survey, the national practices were discussed during a face-to-face meeting with WP5B partners. Results Twelve Health TechnologyAssessment (HTA) bodies completed the survey. Of these, eleven reported procedures in place for official requests for PLEGs in their remit. In the large majority of cases, the requests are made at the time of the assessment/appraisal. Several agencies participate in the definition of the scope of the PLEG or review of its protocol. Data collection and analysis mainly lie with companies for pharmaceuticals, whereas it is more the responsibility of the HTA bodies for medical devices. Only one agency owns the data and is able to exchange them without asking permission. Conclusions Most agencies recommend European collaboration on PLEG commence once the evidence gaps have been defined or during the production of the HTA report in the case of European joint assessment.

Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries

“…This may be because, for example, such platforms do not collect the appropriate diagnosis, treatment or outcome variables. The new regulation on European cooperation on HTA does not have any provision for collaboration on post-launch evidence generation (PLEG) ( Puñal-Riobóo et al, 2022 ). This would have enabled the development of common protocols and standards ( Iorio et al, 2018 ; COMET, 2022 ).…”

Can requests for real-world evidence by the French HTA body be planned? An exhaustive retrospective case–control study of medicinal products appraisals from 2016 to 2021

“…A protocol assistance procedure is proposed by HAS to further help health technology developers in conceptualizing the design of the PRS according to the request (11). Similar postlaunch data collection exists in other HTA jurisdictions (12). Germany, for example, amended the AMNOG law in 2019 to allow the Federal Joint Committee (G-BA) to request nonrandomized comparative studies under certain conditions where the clinical benefit cannot be quantified (13)(14)(15).…”

Financial Outcomes of Managed Entry Agreements for Pharmaceuticals in Italy

“…Health outcome-based MEAs generally take either of these 2 forms: (1) coverage with evidence development, in which reimbursement is contingent on the initiation of postlaunch evidence generation, and (2) performance-linked schemes, in which reimbursement of covered products is associated with measures of clinical outcomes in practice settings. 4,[6][7][8][9] Performance-linked reimbursement schemes aim to enhance the costeffectiveness of a new technology in clinical practice and may have a role in the financial sustainability of pharmaceutical spending. 5 Italy is one of the first countries in Europe to have adopted MEAs; the Agenzia Italiana del Farmaco (AIFA/Italian Medicines Agency) agreed on its first contract in July 2006.…”

“…According to price-volume agreements with ceiling, a maximum volume/budget that may be sold is established, and if the sales volume or budget is exceeded, the pharmaceutical manufacturer usually reduces the price of the medicine (ie, discount) or pays back the amount of sales exceeding the agreed levels (ie, rebate). Health outcome–based MEAs generally take either of these 2 forms: (1) coverage with evidence development, in which reimbursement is contingent on the initiation of postlaunch evidence generation, and (2) performance-linked schemes, in which reimbursement of covered products is associated with measures of clinical outcomes in practice settings . Performance-linked reimbursement schemes aim to enhance the cost-effectiveness of a new technology in clinical practice and may have a role in the financial sustainability of pharmaceutical spending …”