The analysis also noted that any survival gains over existing treatment/placebo were often marginal.
An editorial notes that this study extends the findings of another study on this issue, which found that between 2008 and 2012 the US FDA approved most uses of cancer drugs without evidence of survival or improved quality of life (67%, 36/54). Among the 36 such approvals, only five (14%) uses were shown later to improve survival compared with existing treatments or placebo after a median of 4.4 years on the market. It attempts to characterise the current regulatory climate. Firstly, when drugs do offer survival advantages, the gains are often marginal. Secondly, the small benefits of cancer drugs typically occur in trials conducted in unrepresentative patient populations. Lastly, many of the surrogate outcomes used for drug approval are poorly correlated with survival. It suggests that “the default path to market for all cancer drugs should include rigorous testing against the best standard of care in randomised trials powered to rule in or rule out a clinically meaningful difference in patient centred outcomes in a representative population. The use of uncontrolled study designs or surrogate endpoints should be the exception not the rule. When surrogates are used, postmarketing studies with clinically meaningful and patient centred outcomes must be started, completed, and published. Patient level data should be shared. Health technology assessment programmes should reject modelled measurements of survival, which may unintentionally incentivise the industry not to conduct trials that evaluate survival directly and rely instead on modelling.”
A feature article in the BMJ notes methodological problems with trials that the European Medicines Agency has either failed to identify or overlooked, including the trials’ design, conduct, analysis, and reporting, which can lead to bias and further difficulties in identifying the true effectiveness. It warns that “unless there’s thorough scrutiny of this regulatory evidence after approval, governments may make poor decisions about how to prioritise health budgets.”
An article from the patient’s perspective expresses concern that the current research and development model has failed. It highlights ‘Just Treatment’, a patient led campaign with no ties to the pharmaceutical industry that is calling for a new system that rewards and promotes innovation, so that more effective and accessible cancer medicines are brought within reach. It points out an alternative model such as delinkage—whereby drug prices are decoupled from research and development costs—would reward drug companies for bringing new, effective drugs to market while ensuring these medicines remain an affordable public good.