Real-World Evidence for Health Technology Assessment of Pharmaceuticals: Opportunities and Challenges

Abstract

When conducting health technology assessments (HTA) to reach decisions on the reimbursement of health interventions, HTA agencies often resort to scientific evidence of the highest degree available; thus conventionally randomized controlled clinical trials (RCTs). Several limitations pertaining to RCTs (e.g. highly selected patient populations and controlled follow-up of patients) make them less suitable for answering all questions of relevance to HTA agencies, which aim to assess the value of drugs for the entire patient population under conditions of routine clinical practice. A potential alternative data source for scientific evidence which may complement RCTs in this regard is so-called real-world data (RWD). At the moment, little is known with regards to RWD use in HTA of pharmaceutical drugs and subsequent decision making. This thesis aims to address this gap in knowledge. Several aspects are explored throughout this thesis, namely: the definition of RWD and real-world evidence (RWE), policies for RWD use in HTA, the use of RWD in actual practice, practical and cultural obstacles associated with RWD use and the generation of RWD through novel sources. Real-World Data (and RWE) bears promise for HTA and decision making on the reimbursement of drugs. However, a number of challenges complicate the potential implementation of RWE in HTA and subsequently for decision making on resource allocation in healthcare systems. The challenges pertain to a number of issues, including: unresolved differences amongst stakeholders on the definition of RWE, dissimilarities in policies for RWE use amongst HTA agencies, a fragmented reality of RWE use in standard HTA and MEAs, inaccessibility to RWE and the need for alternate governance structures. On the other hand, numerous initiatives are ongoing to address the challenges cited above. Many of these will provide critical insights that may help improve the methodology behind RWE collection and analysis, create awareness for alternative approaches in RWE generation throughout the product lifecycle and stimulate joint action amongst all relevant stakeholders for developing appropriate governance structures for RWE repositories. Alongside policy and research recommendations made in this thesis, the outcomes of such initiatives may lead to progressively optimal use of RWE for HTA and decision making. It is our aspiration that in doing so, HTA agencies and decision makers will inch ever closer to realizing the full potential healthcare systems can bear for all citizens.