How do HTA agencies perceive conditional approval of medicines? Evidence from England, Scotland, France and Canada

There is a growing disconnect between regulatory agencies that are promoting expedited approval to medicines based on early phase clinical evidence and health technology assessment (HTA) agencies that require robust clinical evidence to inform coverage decisions. This paper provides an assessment of the evidence gap between regulatory and HTA agencies on medicines receiving conditional marketing authorisation (CMA) and examines how HTA agencies in France, England, Scotland, and Canada interpret and appraise evidence for these medicines. A mixed methods research design was used to identify the types and frequency of parameters raised in the context of HTA decision-making for all conditional approvals in Europe and Canada between 2010 and 2017. Significant heterogeneity was found across the HTA agencies in England, Scotland, France, and Canada in the assessment of medicines receiving CMA, with the highest likelihood of rejection present in Quebec (50%) and Scotland (25%). Rejected medicines were more likely to have unresolved uncertainties related to the magnitude of clinical benefit, study design, and issues in economic modelling. More systematic use of joint early dialogue and conditional reimbursement pathways would help clarify evidence requirements and avoid delays in patient access to innovative medicines

Alternative medicine · Authorization · Economics · Evidence-based policy · Geography · Health care · Health technology · Marketing authorization · Political science · Public economics · Reimbursement · Health Systems, Economic Evaluations, Quality of Life · Healthcare cost, quality, practices · Medicine · Pharmaceutical Economics and Policy · Public Administration

• Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond

  Open Access•Aris Angelis, Panos Kanavos•Social Science & Medicine•2017

  Add to list