In 2020, COVID-19 had only a minimal effect on drug approvals, which were considerably higher in comparison with 2019. The US Food and Drug Administration (FDA) approved more than 50 new medicines and therapeutic biologics in 2020, more than a third of which were oncology/hematology treatments. Similarly, the European Medicines Agency’s Committee for Medicinal Products for Human Use issued positive opinions on over 50 new medicines, therapeutic biologics, and vaccines in 2020.
Newly approved gene and cell therapies expanded the use of technology to new disease states; for example, Gilead’s new chimeric antigen receptor T-cell therapy (Tecartus™ [brexucabtagene autoleucel]) was the first of its kind approved for treatment of mantle cell lymphoma. Other innovative medicines included Immunomedics’ sacituzumab govitecan-hziy for metastatic triple-negative breast cancer and AZ/Daiichi Sankyo’s fam‐trastuzumab deruxtecan-nxki for human epidermal growth factor receptor 2-positive breast cancer. Both of these agents have the potential to positively impact the lives of women with breast cancer and to see significant growth in 2021. In the US, the FDA approved 4 tyrosine kinase inhibitors and 2 RET inhibitors, as well as several new oral therapies for various tumor types. Interestingly, progress with new biologic agents has slowed, with only 8 new products licensed in 2020 (compared with 22 in 2019, and 21 in 2018).
These new approvals in solid tumors and hematologic malignancies provide insight into clinical practice treatment trends. Several of the products recommended for approval in the US and EU were orphan medicines, indicating a continued shift to more specialty drugs and targeted medicines. For example, Incyte’s newly approved pemigatinib is the first FDA-approved treatment for advanced or metastatic fibroblast growth factor receptor 2 fusion bile duct cancer. However, 2021 remains unpredictable in terms of expectations for new approvals in the fields of oncology and hematology. It has been speculated that the anticipated increase in submissions for COVID-19 therapies and vaccine approvals in 2021 may impact overall drug approvals. Additionally, many cancer trials were suspended during the pandemic, which may lead to delays in study completions for new therapies.
Very close cooperation and collaboration among all stakeholders within the healthcare system is needed to allow more focused and targeted/cellular therapies to be created, tested in clinical trials, and ultimately administered to patients upon approval. The trend toward more specialty drugs is likely to persist, as researchers continue to identify specific therapeutic targets for solid tumors and hematologic malignancies. While these targeted therapies typically offer increased efficacy, they also require additional tests to determine whether a specific patient can benefit from them. The past year has taught us that stakeholders at all levels must be flexible, and at times creative, to ensure that patients are able to get the care and treatments they need and to have access to pivotal clinical trials.
The increase in specialty drug approvals indicates a continued shift toward increased use of therapies with specific targets. As a result, many new and ongoing clinical trials include niche target populations. When appropriate, community oncologists should consider ordering genetic testing upon diagnosis to help inform treatment decisions and provide support for prior authorizations, if required.