Incremental benefits of novel pharmaceuticals in the United Kingdom 67 4♣ provide full disclosure. The desire to maintain in confidence the incremental cost of their treatment, which would implicitly be made evident if both cost/benefit ratios and QALY values were simultaneously disclosed, may be the driving force behind redactions. In the Supplementary Material, we provide examples where we could retrieve estimates due to ineffective redaction. We also list the number of redacted estimates by disease area. The rates of redaction in oncology (37.2%) and hematology (44.9%), compared with other disease areas (such as cardiology, vascular medicine, endocrinology) where none of the values were redacted, may either represent the unwillingness to disclose high drug prices in these indications,27 or the unwillingness to disclose low benefits, the latter of which may make average QALY figures appear larger than they are for these disease areas. For withdrawn or terminated appraisals, no detailed information is available to the public on cost or QALYs. Although speculative, it is unlikely these appraisals discussed drugs that were cheaper and more effective than the current standard of care. Third, QALY estimates of individual products are sensitive to the choice of relevant comparator. Our results, however, show that the choice of comparator does not significantly affect the overall estimated QALY gain in our dataset. Alternatively, one may not be interested in the overall population, but only in specific (sub)populations reported in the appraisal documentation. This may give more specific estimates for individual patients but impedes the comparison of drugs across diseases. Fourth, estimates of median incremental QALY for each drug are associated with varying degrees of uncertainty. Although we have extracted the ‘preferred’ estimate from the evidence review group, the variance of these estimates is not routinely reported. Furthermore, distinct preferences in modelling choices, may result in substantial differences in benefit estimates. Our findings provide insight into the relative benefits of new pharmaceuticals across therapeutic areas. Additional health gains may be hindered by the difficulty of developing novel drugs for specific diseases, perhaps because major improvements have already been generated prior to our review period,28,29 or because scientific breakthroughs have not yet occurred. QALYs are a useful tool for comparison, but the measure omits important health-related variables, such as the extent to which a patient remains unable to live out a ‘normal’ life expectancy or achieve complete health. Other factors, such as lack of fundamental understanding of disease pathologies,30,31 or the abundance or absence of sufficient research funding may also limit health gains.32 Our figures evaluate the net present health-related benefits of drugs that are considered cost-effective by NICE over the past decade. In combination with indices measuring health needs, such as the Global Burden of Disease,33 as well as cost-effectiveness/cost-saving data of novel drugs that might produce similar QALYs as already available therapies, our findings can help provide context for the allocation of research funding and thereby shape health policy.
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