Article type
Year
Abstract
Background: As regulators are turning to early and conditional licensing of new pharmaceuticals to mitigate market access delays; payers such as the Department of Health in the UK are simultaneously considering value-based pricing (VBP) as a new scheme to efficiently allocate scare funds to promising medicines. These on-going regulatory and pricing changes will have significant implications for the conduct of health technology assessment (HTA) in coming years.
Objectives: First, to identify key evidential and methodological issues associated with early drug evaluations, conditional licensing and VBP; second, to identify potential analytical and statistical solutions to address these.
Methods: A systematic literature review in Medline®, EMBASE, and the Cochrane Library; as well as a comprehensive search of relevant institutional and government websites was conducted. In addition, a horizon scan across different research areas was performed to identify methodological developments not currently used in the health sciences that could be applicable to the issues raised. Relevant documentation was reviewed and expert opinion considered for discussion.
Results: Earlier assessment of relative effectiveness necessitates the consideration of wider sources of evidence than purely RCTs, in addition to issues surrounding the analysis of small numbers/subgroups, heterogeneity, immature/incomplete datasets, and issues of bias. Additional limitations were considered for VBP, particularly in combination with the above, such as data constraints for (network) meta-analysis and uncertainty. A number of statistical approaches are currently used to address these issues such as stratified analysis according to study ‘quality’, bias modelling, model averaging, etc.
Conclusions: HTA practice needs to evolve to embrace the regulatory and pricing changes in the UK. Tools are available to tackle the evidential and methodological issues arising from new clinical data requirements; however, these have not necessarily been used jointly nor been evaluated in the context of HTA.
Objectives: First, to identify key evidential and methodological issues associated with early drug evaluations, conditional licensing and VBP; second, to identify potential analytical and statistical solutions to address these.
Methods: A systematic literature review in Medline®, EMBASE, and the Cochrane Library; as well as a comprehensive search of relevant institutional and government websites was conducted. In addition, a horizon scan across different research areas was performed to identify methodological developments not currently used in the health sciences that could be applicable to the issues raised. Relevant documentation was reviewed and expert opinion considered for discussion.
Results: Earlier assessment of relative effectiveness necessitates the consideration of wider sources of evidence than purely RCTs, in addition to issues surrounding the analysis of small numbers/subgroups, heterogeneity, immature/incomplete datasets, and issues of bias. Additional limitations were considered for VBP, particularly in combination with the above, such as data constraints for (network) meta-analysis and uncertainty. A number of statistical approaches are currently used to address these issues such as stratified analysis according to study ‘quality’, bias modelling, model averaging, etc.
Conclusions: HTA practice needs to evolve to embrace the regulatory and pricing changes in the UK. Tools are available to tackle the evidential and methodological issues arising from new clinical data requirements; however, these have not necessarily been used jointly nor been evaluated in the context of HTA.