Flexibility in assessment of rare disease technologies via NICE’s single technology appraisal route: a thematic analysis
Summary
Aim: NICE’s highly specialized technology (HST) evaluations are highly restrictive in terms of entry criteria and as a consequence, the vast majority of rare disease medicines are assessed through NICE’s standard, single technology appraisal (STA) route. We explored whether NICE shows flexibility and pragmatism when evaluating treatments for rare diseases through its STA process. Materials & methods: We matched a sample of recent, randomly selected STAs for rare diseases to STAs for non-rare diseases and conducted a thematic analysis to identify patterns in NICE’s decision-making, with a specific focus on the application of NICE’s published methods and the handling of uncertainty. Results: Three themes emerged where some flexibility was shown: ‘handling of uncertainty and discretion’, ‘application of NICE methods’ and ‘commercial arrangements’. Rare disease technologies were generally subject to longer appraisal times than those for non-rare diseases. Conclusion: Although NICE shows a degree of flexibility and pragmatism toward uncertainties in the evidence base for rare disease medicines, this is often off-set by a lengthy appraisal process, which can lead to delays in patients receiving vital treatment.
Plain language summary
What is this article about?
In England and Wales, the National Institute for Health and Care Excellence (NICE) decides whether to recommend medicines for use by the National Health Service. It has a process for evaluating very rare disease medicines – the highly specialized technology route. However, the entry criteria for this process are very restrictive, so many rare disease medicines end up in NICE’s standard STA process, which is deemed inadequate to account for the complex issues associated with rare diseases. We wanted to find out whether NICE considers these issues systematically when evaluating rare disease medicines via STA.
What were the results?
We randomly selected six treatments for rare diseases and six treatments for non-rare diseases and compared information taken from documents published on NICE’s website. We found three main areas where NICE showed some flexibility for rare diseases: 1) taking into account a lack of clinical evidence; 2) lenient adherence to, and interpretation of, the NICE methods guide; 3) negotiating commercial arrangements that provide access to treatment while giving the manufacturer time to gather more information. However, appraisals generally took much longer for rare diseases than for non-rare diseases.
What do the results of the study mean?
The results show that NICE shows some flexibility when evaluating rare disease medicines. However, they also show that treatments for rare diseases can be disadvantaged by going through the STA process relative to non-rare diseases, resulting in the potential for substantially delayed access for patients.