What Are the Emerging State-of-the-Art HTA Methods That Are Relevant to Harmonization of HTA Across Jurisdictions?
Author(s)
Laughlin W1, D'Ausilio A2, Bretton D1, Olid Gonzalez A3, Bending MW1
1Evidera Value and Access Consulting, London, UK, 2Evidera Ltd, London, UK, 3Evidera, London, UK
Presentation Documents
OBJECTIVES:
Whilst the remit of Health Technology Assessment (HTA) bodies has evolved, the development of innovative therapies has revealed new methodological challenges to address. There have been reviews on best-practice HTA methods; for example, the European Federation of Pharmaceutical Industries and Associations (EFPIA) reviewed state-of-the-art HTA methods in 2018/2019. However, considering recent developments, including the new EU HTA Regulation, there is a need to assess emerging methods to inform future harmonization.METHODS:
We defined two areas with potential needs to improve appraisals of innovative therapies: i) comparisons, including simulated control arms and indirect comparisons; ii) outcomes, including surrogate and long-term extrapolation of outcomes. For each, we conducted a pragmatic literature review on PubMed, HTA websites, HTAi and ISPOR conferences, and reviewed HTA methods.RESULTS:
The pragmatic search retrieved >400 hits, with 20 papers selected for review. HTA methods from NICE, HAS and IQWiG were also reviewed. For comparators, we identified emerging state-of-the-art methods such as simulated control arms, but noted variability on implementation. HTAs appear conservative in accepting newer methods in indirect comparisons, such as in non-randomised evidence and population adjustments. Clarity and consensus in appropriate outcome measures (for instance, with availability of core outcome sets) is needed. In their 2022 methods, NICE mentions such methods, but current use is limited. The varied implementation of state-of-the-art methods may present a barrier to their use in HTA harmonization. Given the need for compromise, there is risk that European HTA does not enable flexibility for acceptance of new types of evidence for innovative therapies, limiting their uptake.CONCLUSIONS:
Despite emerging state-of-the-art methods, their limited and variable uptake poses challenges to future assessments of innovative pharmaceuticals. Further research is needed on domains such as populations and study designs to fully capture the current environment.Conference/Value in Health Info
2022-11, ISPOR Europe 2022, Vienna, Austria
Value in Health, Volume 25, Issue 12S (December 2022)
Code
HTA269
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas