Chapter 4 68 Eight drugs improved life by more than two incremental QALYs, which may justify their superlative epithets of ‘ground-breaking’ or ‘game-changing’.2 Half of the drugs in our study were likely to improve life by the equivalent of three to four months in perfect health, and 84.8% of novel drugs did not add more than one such year. Unfortunately, 25% of appraisals have covered drugs that contributed the equivalent of no more than one month in perfect health, and 23 (12.5%) drug-indication pairs were estimated to add several hours to just a week of perfect health. For example, eluxadoline for prevention of diarrhea and abdominal pain in patients with irritable bowel syndrome yielded a total QALY gain of 0.015—equivalent to 5.5 days in perfect health— compared with placebo. Given the uncertainty around cost-effectiveness estimates—models that require ample assumptions and extrapolations over lifetime horizons can hardly be expected to accurately forecast a week of health gained—drafting extensive cost-effectiveness reports in these situations is not likely to be a cost-effective use of time. Drugs that have little health benefit relative to the best alternative may still promote price competition and thereby free funds for other public health initiatives or treatments. To avoid wasting public resources in needless evaluations, guideline committees could determine a threshold of incremental benefit that is clinically relevant to each disease area.34 Drugs that do not pass this threshold based on early assessments of their value should be rejected without a full evaluation unless they are offered at lower cost. Patients and physicians can use the QALY data presented here to put the effectiveness of treatments in perspective. The frequently employed metric of ‘number needed to treat’ provides important information about the effectiveness of drugs on the principal disease-specific outcome. For example, the efficacy of eluxadoline could be described in terms of the number of patients that would need to be treated three months to avoid one episode of abdominal pain or diarrhea, in this case between eight and 33 patients over three months.35 Metrics such as this, however, do not account for adverse events. Using the incremental QALY estimate that integrates gains and losses into a single measure (for eluxadoline, 0.015), it is possible to calculate that 67 patients would need to be treated over their lifetime horizons to gain the equivalent of one year in perfect health. As such, the QALY provides an estimate of both duration and quality of life, which are arguably the two most important factors from the perspective of a patient. Conclusions Novel pharmaceuticals that received a positive coverage decision by NICE from 2010 to 2020 provided patients with an average of 0.27 additional QALYs over the best alternative therapy, the equivalent of three to four additional months of life in perfect health. One in four drugs does not improve quality and quantity of life by more than one month, and incremental benefit varies
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