PHP226 - Introducing the ENCePP Working Group (WG) on Health Technology Assessment (HTA): Enhancing the Generation of Additional Evidence for HTA Processes

Abstract

The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) is a project led by the European Medicines Agency aimed at further strengthening the post-authorisation monitoring of medicinal products in Europe by facilitating the conduct of multi-centre, independent post-authorisation studies focusing on safety and benefit/risk assessment. ENCePP has recently established a WG on HTA to develop methodological guidance to supplement the efficacy and safety aspects of medicines known at the time of marketing authorisation (MA) and to bridge the needs of HTA and post-marketing benefit-risk assessments. Decision makers are often faced with the challenge that long-term, real-world data on safety and effectiveness is lacking at the time of MA. This creates uncertainty around the medicines' risk-benefit profile while manufacturers, health care providers, and patient groups exert pres sure by demanding early decisions and rapid access. Decision makers may unduly delay potential benefits to patients by waiting for stronger evidence, or may endorse medicines that later turn out to have a less robust benefit-risk ratio, to be ineffective cost-ineffective, or even harmful. Hence, many countries have developed mechanisms that allow temporary access to promising medicines while concurrently requesting the generation of additional evidence to reduce uncertainty. Their objective is an optimal trade-off between different stakeholder needs, flexibility, responsiveness, and rigor as well as the flexibility to revise decisions on access when new evidence becomes available. The ENCePP WG on HTA has the potential to become a capacity building tool for regulatory and HTA agencies to develop research structures aimed at complementing the evidence generation for MA and market access. Post-authorisation studies developed under the auspices of ENCePP could provide new safety and clinical effectiveness information of marketed medicines. ENCePP expertise, research experience and health care databases may also contribute to the coordination, methodological guidance and data sourcing for the enhancement of HTA processes.