Accelerated drug approvals compromise safety and efficacy

Our previous blog showed how missing or misrepresented data from clinical or preclinical trials can harm patients by allowing medicines to be approved with insufficient evidence of safety and/or efficacy. It is very disturbing how often pharmaceutical companies fail to provide requisite data to the drug regulators (FDA in America, MHRA in UK, EMA in Europe) and how often regulators fail to demand that data or take any action when companies fail to comply. More disturbing still is that schemes which were originally intended to help patients access life-saving treatments more quickly are being misused as an easier, faster and cheaper route to approval because they require less evidence of risks and benefits. These accelerated access schemes include the FDA’s Fast Track process, the Early Access to Medicines Scheme in the UK, and the EU’s Compassionate Use and Early Access Adaptive Licensing program. They all allow the use of truncated testing regimes, where the normal requirements for evidence of safety are relaxed to varying degrees, making the much-criticised bar for approval even lower.
Early access to potentially life-saving medicines for seriously ill patients may sound compassionate in theory. However, we know that more than 90% of medicines fail in human trials due to lack of safety or benefit, even after the full standard package of preclinical studies. To expose the most vulnerable patients to potential new drugs whose likelihood of success is clearly lower than 10% seems far from compassionate in reality. Human-relevant testing approaches could play an invaluable role here, to improve the safety and efficacy profiles of these experimental medicines, which may not have completed any human clinical trials. Adverse effects which are too subtle or rare to be detected before exposure to large numbers of patients following drug approval can be detected by human-relevant testing, e.g. using organ-on-chip or patient-on-chip devices. This would reduce the risks – not only to the vulnerable patients for whom these schemes were designed – but to all of us, since many treatments are now gaining approval via such reduced-evidence pathways.
Related: FDA allows drugs without proven clinical benefit to languish for years on accelerated pathwayApprovals of drugs with uncertain benefit–risk profiles in EuropeFulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway – The Need for ReformsThe FDA has devalued the gold standard on R&D. And that threatens everyone in drug development; Should regulatory authorities approve drugs based on surrogate endpoints?The next step in immorality: charging to create and cure disease

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