{
  "abstract": "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.",
  "authors": [
    {
      "affiliations": [
        "University of Zurich, Faculty of Medicine, Zurich, Switzerland"
      ],
      "name": "Parisa Rahimzadeh"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Qiyu Li"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Leonie Rudofsky"
    },
    {
      "affiliations": [
        "Division Regulatory Operations and Development, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Simon Dalla Torre di Sanguinetto"
    },
    {
      "affiliations": [
        "Department of Medical Oncology and Hematology, University Hospital Zurich, Zurich, Switzerland"
      ],
      "name": "Nicola Miglino"
    },
    {
      "affiliations": [
        "Department of Medical Oncology and Hematology, University Hospital Zurich, Zurich, Switzerland"
      ],
      "name": "Eva Camarillo-Retamosa"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Jeanette Bachir"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Nicole Bodmer"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Arunas Gircys"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Mario Iovino"
    },
    {
      "affiliations": [
        "University of Zurich, Faculty of Medicine, Zurich, Switzerland"
      ],
      "name": "Elija Brunschwiler"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Stephanie Juritz"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Kathrin Espinoza"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Bettina Maier"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Claudia Ruch"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Elisa Zaninotto"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Matea Zosso-Pavic"
    },
    {
      "affiliations": [
        "Human Medicines Expert Committee, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland",
        "Department of Oncology, Hôpitaux Universitaires de Genève, Service d’oncologie, Geneva, Switzerland"
      ],
      "name": "Anita Wolfer"
    },
    {
      "affiliations": [
        "Division Clinical Assessment, Authorization Sector, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Ulrich-Peter Rohr"
    },
    {
      "affiliations": [
        "University of Zurich, Faculty of Medicine, Zurich, Switzerland",
        "Department of Medical Oncology and Hematology, University Hospital Zurich, Zurich, Switzerland",
        "Human Medicines Expert Committee, Swissmedic Swiss Agency for Therapeutic Products, Bern, Switzerland"
      ],
      "name": "Andreas Wicki"
    }
  ],
  "title": "Trends in pivotal clinical trial design and biomarker use: a retrospective analysis of oncology drug approval in Switzerland from 2001 to 2020",
  "uid": "649985ea-e6b0-501e-9a1d-2b49e8836585"
}