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In this Viewpoint, the authors discuss the FDA's strategies for increasing access to new medicines for patients with serious or life-threatening illnesses. The article describes the currently available access mechanisms, and highlights the FDA's planned regulation revisions for expanding access.

The Bloomberg New Economy International Cancer Coalition brings together academia, industry, government, patient advocacy and policy think tanks to leverage technology and collaboration to improve patient access to clinical trials and to harmonize regulations aiming to accelerate cancer cures and prevention worldwide in the post-pandemic era.

In 2016, FDA Oncology approved five new molecular entities and 17 efficacy supplements, including six accelerated approvals, 17 priority reviews, and 11 approvals of breakthrough-designated therapies. The FDA also approved five companion diagnostics, including a liquid biopsy test. One new anti-PD-L1 antibody was approved, along with six supplementary approvals of anti-PD-1/PD-L1 antibodies.

In 2023, the US FDA approved several new cancer drugs and biologic agents, including seven small-molecule inhibitors, four bispecific T cell engagers, two anti-PD-1 antibodies and one cell therapy product. Regulatory focus areas included analyses of biomarker-positive subgroups that drive efficacy, equipoise in randomized controlled trials and a new authority to require confirmatory trials be underway before accelerated approval.

In 2020, despite challenges related to the COVID-19 pandemic, the US FDA approved 30 new drugs and biologic agents, 45 supplemental drug and biologic applications and 1 biosimilar application in oncology.

In 2021, policy areas of focus for FDA Oncology included the Accelerated Approval programme, expanding eligibility criteria, dose optimization and patient-reported outcomes. The FDA continued to be active with approvals of both new drugs and supplementary applications, including three new chimeric antigen receptor T cell products, two antibody–drug conjugates and several new targeted agents.

In 2019, the FDA Oncology Center of Excellence (OCE) approved 11 new drugs and biologic agents, 30 supplemental drug and biologic applications, and four biosimilar applications in oncology. These included two landmark approvals involving collaboration among international regulators as part of OCE Project Orbis, as well as the approval of three novel antibody–drug conjugates.

In 2022, the FDA approved numerous new drug and biologic agents, including targeted small molecules, immunotherapeutics, a gene therapy and a radiopharmaceutical. Several drug development challenges were also addressed, and key focus areas for the FDA Oncology Center of Excellence included ongoing monitoring of the Accelerated Approval programme and drug dose optimization.

In 2018, the FDA approved 19 new drug and biologic applications, 38 supplemental drug and biologic applications and 4 biosimilar applications in oncology. These advances in anticancer therapy included a landmark approval of the first histology-agnostic, biomarker-defined new molecular entity and approvals based on real-time data review and novel end points, such as minimal residual disease rate and metastasis-free survival.

Information Exchange and Data Transformation (INFORMED), a multidisciplinary initiative anchored in the FDA Oncology Center of Excellence, is a decentralized science and technology incubator designed to harness the power of big data and advanced analytics to improve disease outcomes.

In 2017, FDA Oncology approved 17 new drug and biologic applications, 32 supplemental drug and biologic applications, and two biosimilar applications in oncology. These actions included landmark approvals of two chimeric antigen receptor T cell therapies and the first site-agnostic, biomarker-defined approval. Three next-generation sequencing 'oncopanels' designed to detect hundreds of somatic genetic aberrations were also approved.

Trials evaluating novel therapies in the neoadjuvant setting must have clearly defined study elements and appropriately selected end points to ensure the applicability of the trial and enable interpretation of the study results. In this Perspectives, the authors describe the findings of a public workshop jointly sponsored by the US Food and Drug Administration and the Bladder Cancer Advocacy Network, which discussed key elements and end points when designing trials of neoadjuvant therapy for muscle-invasive bladder cancer.

Cancer therapy has changed substantially since the beginning of the century, with advances including kinase inhibitors and immunotherapies resulting in substantial improvements in treatment outcomes. This Review summarizes trends in the approval of oncology therapeutic products by the FDA since 2000 and discusses the implications for the future of anticancer drug development.

Here, the authors discuss issues relating to the co-development of targeted cancer therapies and companion diagnostics that were not covered in depth in the draft guidance released by the US Food and Drug Administration in 2011. They propose potential strategies that will be useful to mitigate challenges and to help guide the future co-development of drugs and diagnostics.