NICE accountability for reasonableness: a qualitative study of its appraisal of treatments for...

The UK National Institute for Health and Clinical Excellence (NICE) is widely regarded a role model for the implementation of Health Technology Assessments including cost-effectiveness evaluation. The aim of the present study was to explore the real-life robustness of the NICE technology appraisal process when addressing complex clinical problems, using the Accountability for Reasonableness (A4R) framework proposed by Daniels and Sabin as a reference.

A qualitative case study of NICE Technology Appraisal No. 98, 'Treatments for Attention-Deficit/Hyperactivity Disorder (ADHD)', analyzing each step of the appraisal process.

Scoping was narrower than that for corresponding clinical guidelines. Economic evaluation for assessment was primarily based on six short-term studies, was unable to differentiate compounds on grounds of effectiveness, and cost-effectiveness modeling suggested a clear recommendation driven by acquisition costs. After appraisal, all treatment options assessed were recommended within their licensed indications. With estimated costs per quality-adjusted life years (QALYs) compared to no treatment generally falling below 7000 pounds, NICE guidance specified that choice of drug should be primarily based on clinical considerations, followed by cost.

The appraisal process adhered to predefined timelines, which were sensibly adapted by NICE to changes in the environment. A4R criteria most pertinent to the case study were 'publicity' and 'relevance'. The 'publicity' condition was greatly fulfilled, except for commercial-in-confidence data and economic model. 'Relevance' requires appraisals to reflect concerns for fairness and to be evidence-based; in that respect, principles and realization of the assessment deserve further scrutiny. Questions also remain regarding the 'appeal' and 'enforcement' conditions under A4R.