Real World Data in Health Technology Assessment of Complex Health Technologies.

Milou A Hogervorst,Wim G Goettsch,Rick A Vreman,Johan Pontén,Aukje K Mantel-Teeuwisse

doi:10.3389/fphar.2022.837302

Abstract

The available evidence on relative effectiveness and risks of new health technologies is often limited at the time of health technology assessment (HTA). Additionally, a wide variety in real-world data (RWD) policies exist among HTA organizations. This study assessed which challenges, related to the increasingly complex nature of new health technologies, make the acceptance of RWD most likely. A questionnaire was disseminated among 33 EUnetHTA member HTA organizations. The questions focused on accepted data sources, circumstances that allowed for RWD acceptance and barriers to acceptance. The questionnaire was validated and tested for reliability by an expert panel, and pilot-tested before dissemination via LimeSurvey. Twenty-two HTA organizations completed the questionnaire (67%). All reported accepting randomized clinical trials. The most accepted RWD source were patient registries (19/22, 86%), the least accepted were editorials and expert opinions (8/22, 36%). With orphan treatments or companion diagnostics, organizations tended to be most likely to accept RWD sources, 4.3–3.2 on a 5-point Likert scale, respectively. Additional circumstances were reported to accept RWD (e.g., a high disease burden). The two most important barriers to accepting RWD were lacking necessary RWD sources and existing policy structures. European HTA organizations seem positive toward the (wider) use of RWD in HTA of complex therapies. Expanding the use of patient registries could be potentially useful, as a large share of the organizations already accepts this source. However, many barriers still exist to the widespread use of RWD. Our results can be used to prioritize circumstances in which RWD might be accepted.

Highlights

Sufficient amounts and quality of data on a treatment’s effects, safety and costs is of crucial importance in order to minimize uncertainty in decision-making on reimbursement (Vreman et al, 2020a; Hogervorst et al, 2021)
The available evidence for these evaluations can be limited (Hogervorst et al, 2021). Complex treatment strategies such as health technologies with concomitant genetic testing, advanced therapy medicinal products (ATMPs) or the assessment of sequences of treatments with disease-modifying capacities may be especially prone to these limitations
The HTx project is a Horizon 2020 project supported by the European Union lasting for 5 years from January 2019, with the aim to create a framework for the Generation health technology assessment (HTA) to support patient-centered, societally oriented, real-time decision-making on access to and reimbursement for health technologies throughout Europe (HTx, 2020)

Summary

Introduction

Sufficient amounts and quality of data on a treatment’s effects, safety and costs is of crucial importance in order to minimize uncertainty in decision-making on reimbursement (Vreman et al, 2020a; Hogervorst et al, 2021). Patients’ access to new health technologies may be requested more early in the authorization and reimbursement process because of high unmet medical needs, which has led to the development of expedited approval processes (Kesselheim et al, 2015; Leyens and Brand, 2016). This trend results in decreasing amounts and quality of data available at the time of the HTA process. Interim data or effectiveness data only based on surrogate endpoints is all that is available upon approval of a newly developed intervention (Larson et al, 2017; Pruce et al, 2017; Blagden et al, 2020; Ribeiro et al, 2020)

Methods

Results

Discussion

Conclusion