Chapter 4 60 of their drugs in comparison to the standard of care.15 An evidence review group—generally a group of university based researchers contracted by NICE—then appraises the evidence in ‘single technology appraisals’ and produces independent estimates of health benefits, measured in QALYs. Using data from NICE evidence review groups, we sought to better understand the incremental value of all new therapies assessed from 2010 to 2020. Although these data are used to inform public health decisions, we here present their implications from a patient’s perspective. Specifically, we sought to identify disease areas where the greatest gains from novel therapies have occurred, and the differing average amounts of gain per drug for individual patients in each disease area. MATERIALS AND METHODS We identified all single technology appraisals of novel pharmaceuticals that were submitted to NICE between January 1st, 2010 and December 31st, 2020.16 Data were extracted on May 1st, 2021. We excluded drug appraisals resulting in negative coverage decisions, appraisals for which no data were available because of termination, withdrawal, or reconsideration, and appraisals that addressed only cost-saving issues and lacked QALY data. Two authors (TBP and DGJC) independently extracted QALY estimates from each drug’s appraisal documents. Discordance was resolved by discussion with the last author (MMV). As per NICE guidance,17 QALYs are calculated over the remainder lifetime of patients, and future health benefits are discounted at a 3.5% annual rate. We extracted these ‘net present’ values. When appraisal documents included multiple comparators, we extracted the QALY value that corresponded to the best alternative therapy. As a sensitivity analysis, in the case of multiple comparators, we also computed the added value compared with the next-best alternative. We disregarded cost, as we focused on health gains for individual patients and not on health care systems. The evidence review group usually specified which of the modelled QALYs was its preferred estimate of health benefit (i.e., which modelling assumptions were deemed most appropriate to the review group). If the evidence review group did not clearly document their preference and this could not be determined after deliberation with the last author (MMV), we discarded the appraisal from our analysis. Although manufacturers frequently report the incremental cost-effectiveness ratio in cost (British pounds) per QALY, they are not required to disclose the individual components of this ratio. We therefore removed appraisals in which the manufacturer redacted all estimates of incremental QALYs (also see: Supplementary Material). A schematic overview of our appraisal selection and data extraction method is depicted in Figure 3.
RkJQdWJsaXNoZXIy MTk4NDMw