Introduction Cancer treatment has advanced rapidly in recent decades. Regulatory approval of cancer therapies relies on pivotal clinical trials, with phase III, randomised controlled trials (RCTs) regarded as the gold standard. However, there is growing concern about the increasing use of single-arm trials in cancer approvals. This study provides an overview of cancer drug approvals in Switzerland over the past 20 years. We assessed changes in approval patterns, characteristics of pivotal trials and the use of biomarkers in approvals. Research design and methods We collected data on Swiss cancer drug approvals from 2001 to 2020, and their respective pivotal trials from Swissmedic’s internal database and performed descriptive analyses. Results Over 20 years, Swissmedic approved 362 cancer-related indications, 40.6% as new active substances. Solid tumours accounted for 63.3% of approvals, with lung cancer achieving top ranking with 22.3%. Approvals rose from 6 in 2001 to 34 in 2020, with biomarker-associated indications increasing from 16.7% in 2001 to 55.9% in 2020. Indications were mainly approved based on RCTs (71.3%). However, the proportion of single-arm trials supporting approvals for solid tumours increased from 14.3% in 2005 to 34.6% in 2020, with most approvals covering rare cancer entities and biomarker-associated indications. Conclusions The notable rise in cancer drug approvals and biomarker-associated indications has expanded treatment options. While RCTs remained the primary basis for approvals, the growing reliance on single-arm trials, specifically for biomarker-associated approvals, introduces uncertainty for regulatory agencies in assessing benefit-risk profiles. This highlights the importance of postapproval evidence generation to confirm long-term safety and efficacy.
Rahimzadeh et al. (Tue,) studied this question.
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