Global cooperation and early access—clinical outcomes matter

In modern cancer drug development, surrogate measures are commonly used as clinical trial endpoints instead of more definitive clinical outcomes such as overall survival, with the goal of expediting the development and approval of new drugs. The prevalence of surrogate measures is driven by improved understanding of the basis of cancer and a desire to reduce costs and time, resulting in studies that require smaller sample sizes and shorter follow-up. Regulatory bodies around the world, such as the US Food and Drug Administration (FDA), also accept trials using surrogate measures to expedite patient access to new treatments. But many intermediate endpoints have not been shown to be adequate surrogates for definitive outcomes, adverse effects that can affect overall survival are under-recognised at market entry, and there is a limited understanding of clinical benefit. 1 Tannock IF Amir E Booth CM et al. Relevance of randomised controlled trials in oncology. Lancet Oncol. 2016; 17: e560-e567 Summary Full Text Full Text PDF PubMed Scopus (69) Google Scholar Thus, rigorous testing after approval and close follow-up are needed, but might not be provided. Overall survival benefits of cancer drugs initially approved by the US Food and Drug Administration on the basis of immature survival data: a retrospective analysisFewer than a third of indications approved with immature survival data showed a statistically significant overall survival benefit after approval. Notable inconsistencies in timing and availability of information after approval across different sources emphasise the need for better reporting standards. Full-Text PDF Open AccessEffect of Project Orbis participation by the Swiss regulator on submission gaps, review times, and drug approval decisions between 2020 and 2022: a comparative analysisSubmission gap and review time for oncology applications at Swissmedic were significantly reduced by participation in Project Orbis, and approval consensus decisions were increased between agencies. These findings suggests that participating in Project Orbis could lead to faster patient access to drugs. Full-Text PDF