The National Institute for Health and Care Excellence (NICE) claims that although cost-effectiveness is highly valued in its health technology appraisal process, it sates that there are other factors considered relevant. However, no explicit weight is assigned to these other factors. Do they matter?
A paper by Dakin et al. (2015) tries to answer this question by looking at a treatment’s cost-effectiveness as well as other factors such as:
- RCT Sample size: The number of patients in the randomized controlled trial
- Number of single-arm studies: Presence of single arm studies
- STA: Whether NICE approval was made with single technology appraisal.
- Unmet need: Whether the treatment is the only potential treatment for the decision
- Children: Whether the treatment was indicated for children <18 years old
- Advocacy groups: Whether any patient advocacy groups submitted letters in conjunction with the appraisal
- Disease severity: Mean disability-adjusted life years lost to the disease of interest
- Year: Linear time trend to see if NICE decision are changing
- Pharmaceutical: Whether the technology was a drug
- Orphan drug: Whether or not the technology was granted orphan status by the European Medicines Agency (EMEA)
- Systematic reviews: Whether systematic reviews have been performed for the evidence
The authors used data from 763 decisions from 229 appraisals and found:
As expected, the ICER had a significant effect on NICE decisions, with every £1000 increase in the ICER reducing the odds of NICE recommending the technology by 6.9% (95% confidence interval (CI): 4.3, 9.4%; p<0.001)…However, clinical evidence, having no alternative treatments, paediatric population, patient group submission, disease severity and date had no significant effect on NICE decisions…Nonetheless, omitting any variable from the model other than disease severity slightly reduced prediction accuracy, suggesting that these variables may help explain some NICE decisions.
There were some differences in approval likelihood. NICE was more likely to make a positive recommendation for musculoskeletal disease (5.7x) and cancer (3.1x), but 31% lower for treatments for respiratory disease (71% lower).
Our analyses demonstrate that cost-effectiveness is the principal determinant of most NICE decisions and that the probability of rejection increases significantly with increasing ICER. The finding was robust to extensive sensitivity analyses varying modelling approaches…We estimate that, in practice, the ICER at which the probability switches from more-likely-to accept to more-likely-to-reject is between £39 000 and £44 000: well above the stated £20 000–£30 000 range.
- Dakin, H, 2015), The Influence of Cost-Effectiveness and Other Factors on Nice Decisions. Health Econ., 24, 1256–1271. doi: 10.1002/hec.3086. , , , , and (