Decision-making in the midst of uncertainty: appraising expensive medicines in England.

Michael Calnan

doi:10.1590/1413-812320212611.41872020

Abstract

Decisions need to be made about which services or technologies should be prioritized for provision in the NHS in England .The analysis focuses specifically on the National Institute for Health and Care Excellence (NICE), and on how they appraise expensive medicines. This analysis takes a sociological perspective on decision-making in relation to uncertainty and how uncertainties are managed, drawing on evidence from a scoping study and an ethnographic study. Uncertainties were central to these rationing decisions. Three types of layers of uncertainty -epistemic, procedural and interpersonal - were shown to be salient. Another form of uncertainty was associated with the complexity of the science and that included the level of technicality of the information provided. The analysis highlighted the salience of uncertainties associated with interpersonal relations and the relations between the committees and the drug industry, clinical and patient experts. A key element in these relationships was trust. Decision makers adopted a mixture of formal and informal, collective and individual strategies in making decisions and a need to exercise pragmatism within a more formal institutional framework. The paper concludes by considering more recent policy developments in relation to appraising expensive medicines.

Highlights

Background contextHealth care in England is predominantly provided by the National Health Service (NHS) which is a universal system free at the point of access funded by the government, primarily from public taxation
The National Health Service, like many other publicly funded health systems, has limited resources so decisions need to be made about which services or technologies should be prioritised or in a more negative sense rationed
The introduction of National Institute for Health and Care Excellence (NICE) created a ‘fourth hurdle’ in the regulation of pharmaceuticals in England NICE and cost effectiveness evaluation was added to the assessment of quality, efficacy and safety by other medicines regulatory bodies[7]

Summary

Background context

Health care in England is predominantly provided by the National Health Service (NHS) which is a universal system free at the point of access funded by the government, primarily from public taxation. The introduction of NICE created a ‘fourth hurdle’ in the regulation of pharmaceuticals in England NICE and cost effectiveness evaluation was added to the assessment of quality, efficacy and safety by other medicines regulatory bodies[7] It could be characterised as adopting a role as an explicit rationing agency to ensure consistent equitable patient access to drugs across the entire NHS and the efficient use of public finances by regulating NHS consumption of new and expensive drugs by cost effectiveness criteria. This was to be based on rigorous appraisals of scientific evidence and NICE was to manage uncertainty through a calculative and evidence-based approach. The focus here is on the social influences which are more implicit in the decision-making in relation to uncertainty and how they are managed using evidence primarily from a scoping study using documentary analysis and an ethnographic study[12,13,14]

Uncertainty and decision making about appraising medicines

Heightened uncertainty and recent policy developments