Comparison of post-authorisation measures from regulatory authorities with additional evidence requirements from the HTA body in Germany – are additional data requirements by the Federal Joint Committee justified?

Abstract

ObjectivesThe aim of this study was to compare post-authorisation measures (PAMs) from the European Medicines Agency (EMA) with data requests in fixed-termed conditional appraisals of early benefit assessments from the German Federal Joint Committee (G-BA).MethodsMedicinal products with completed benefit assessments during an assessment period of 3.5 years were considered. PAMs extracted from European Public Assessment Reports (EPARs) were compared with data requests issued by the G-BA in the context of conditional appraisals.ResultsTwenty conditional appraisals (19 products) and 34 EPARs containing PAMs (33 products) were identified. Data categories (efficacy, safety, etc.), data types (type of study required to address the request) and clarity of requests were determined. Conditional appraisals disproportionately focused on oncology products (13/19 products with conditional appraisals vs. 14/33 products with PAMs). No clear rationale for the G-BA issuing conditional appraisals could be identified in public sources. Both EMA and G-BA requested mainly efficacy and safety data (44/54 and 23/35 categories requested, respectively); however, 28/35 G-BA data requirements went beyond requests made by the EMA. Almost half of the G-BA requests (9/20), but no PAMs, were unclear, and no methodological guidance for fulfilling the data requirements was provided by the G-BA.ConclusionsBetter alignment between data requests from regulatory authorities and health technology assessment bodies is strongly recommended.

Building Synergy between Regulatory and HTA Agencies beyond Processes and Procedures—Can We Effectively Align the Evidentiary Requirements? A Survey of Stakeholder Perceptions

“…Our survey respondents suggested that the requirements need to be aligned not only at the initial approval stage but also during postauthorization to best fulfill the follow-up evidentiary requirements of regulatory and HTA agencies. A recent study by Ruof et al [24] assessed the postauthorization data request from EMA and the German HTA body Der Gemeinsame Bundesausschuss (G-BA), and found that G-BA made additional requests with less clear instructions compared with those made by the EMA.…”

How can a joint European health technology assessment provide an ‘additional benefit’ over the current standard of national assessments?

“…Within their report on the implementation of the EMA-EUnetHTA work plan 2017–2021 EMA and EUnetHTA provide in-depth insights regarding their discussion of controversial methodological challenges [ 15 ]. Continuation of those discussions and clarification of evidence requirements within the regulatory and HTA framework including parallel early advice, early access schemes, as well as regulatory and HTAs is urgently required to ‘ensuring a strong EU voice globally’ in line with chapter 5 of the European Commission’s pharmaceutical strategy for Europe [ 13 , 16 , 17 ]. The management of uncertainty after successful EMA approval leading to additional national data requirements as a toll needed for covering the gaps and discrepancies between the regulatory and HTA data needs is another key challenge affecting any future oncology HTA within Europe [ 18 ].…”

“…Within their report on the implementation of the EMA-EUnetHTA work plan 2017–2021 EMA and EUnetHTA provide in-depth insights regarding their discussion of controversial methodological challenges [ 15 ]. Continuation of those discussions and clarification of evidence requirements within the regulatory and HTA framework including parallel early advice, early access schemes, as well as regulatory and HTAs is urgently required to ‘ensuring a strong EU voice globally’ in line with chapter 5 of the European Commission’s pharmaceutical strategy for Europe [ 13 , 16 , 17 ].…”

AMNOG Arbitral Awards: Analysis of the Decisions of the Arbitration Board Ten Years after AMNOG Implementation

“…The two-step process begins with an early benefit assessment initiated at the launch of the pharmaceutical. The objective of the early benefit assessment is to assess whether a pharmaceutical has an additional benefit in comparison to the appropriate comparator therapy (ACT) 5 , 6 , 7 .…”