Incremental benefits of novel pharmaceuticals in the United Kingdom 65 4♣ When selecting the next-best drug as a comparator instead of the best available comparator, the median added value slightly increases (0.31, IQR: 0.09 – 0.73), suggesting our results are robust under these different choices of comparators. DISCUSSION Novel pharmaceuticals that became publicly available to patients in the NHS over the past eleven years and that were favorably evaluated by NICE contributed the net present equivalent of between three to four months of life in perfect health relative to the best alternative therapy. The added benefit varied greatly, including eight drugs that were inferior in some cases to its alreadyavailable counterpart, and two that provided the equivalent of over five years in perfect health. To our knowledge, this analysis is the first to compare the therapeutic value of drugs across diverse disease areas using QALYs extracted from independent cost-effectiveness analyses conducted through a standardized framework. The largest benefits were observed in areas such as hematology or oncology, where drugs were shown to improve quality or duration of life by 0.70 and 0.46 QALY. Patients have least profited from pharmaceutical innovations in endocrinology and ophthalmology, where novel pharmaceuticals were associated with a median incremental benefit of 0.02 to 0.09 QALYs. The nature of each treatment (curative, palliative, symptomatic, preventive) may impact the incremental QALY. For example, adult patients that have undergone total hip or knee replacements may be treated with apixaban (TA245) to prevent venous thromboembolism. When used for this indication, apixaban provides an incremental benefit of 0.0016 QALY over the standard of care (low-molecular-weight heparin), equivalent to an additional fourteen hours of life in perfect health. The very low benefit reflected estimates that one venous thromboembolism event would be prevented for every 110–250 patients treated prophylactically for ten days following surgery.19–21 Although apixaban may prevent serious outcomes (death) in some patients, outcome heterogeneity led to the extremely low average incremental QALY. QALY evaluations are necessarily based on the data available at the time of drug approval, which are in turn increasingly based on earlier-phase trials, but later-generated evidence often fails to confirm promising early results.22 Furthermore, most (59%) drugs are now approved on the basis of surrogate endpoints,23 such as progression free survival, which for purposes of QALY calculations are assumed to correlate with clinical outcomes such as increased survival. However, studies have shown that this correlation is often poor or fair, particularly in oncology.24,25 Additionally, data on infrequent or longer-term harms cannot be known with certainty or incorporated in the appraisals, as these data only become apparent when the drug is available for broader use.
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