gms | German Medical Science

Background/research question: Rigorous evaluations of new health technologies are needed in health technology assessments (HTAs) to ensure safe, high-quality, and efficient healthcare. However, use of non-randomized studies for assessment and adaptive approaches to reimbursement have been recently proposed to accelerate availability of healthcare innovations [1]. Therefore, this scoping review was conducted to explore different methodological guidance to assess clinical effectiveness in HTAs in high income countries (HICs).

Methods: We searched websites of HTA organizations in HICs that are members of EUNetHTA and / or INAHTA. Methodological guidance documents that were published between January 2011 and October 2020 were considered. We extracted information on aims and focus, processing time, guidance for preparation, study types considered, certainty of evidence, determination of clinical benefits, and adaptive approaches to reimbursement using a standardized data extraction sheet. Thereafter, we used content analysis [2] method to describe similarities and differences between guidance documents for preparation of HTAs.

Results: 22 HTA documents from 19 HICs were identified. Besides presentation of methods for preparation of HTAs, frequently mentioned goals defined in guidance documents were to increase quality, transparency, and standardization of HTA processes. HTA guidance was generally based on high scientific standards for assessing clinical effectiveness. Almost all guidance documents mentioned systematic reviews (SRs), randomized controlled trials (RCTs) and cohort studies as representing the type and quality of evidence for an assessment of health effect that is required for an HTA. Furthermore, many documents reported that use of observational studies depends on certain prerequisites. For instances, prospective cohort studies are considered when limited or no evidence from RCTs is available and when investigating long-term effects and adverse effects, and in some cases data from registries and case studies are acceptable.

Conclusion: This scoping review provides an overview of current HTA guidance of different organizations, and it holds useful information that should be reflected in documents describing HTAs processes to ensure harmonization of methodological guidance for preparation of HTAs and assessment of clinical effectiveness. One limitation is that not all potentially relevant documents might have been included because the field is advancing dynamically.

Competing interests: The authors declare to have no conflicts of interest regarding this research.