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    Four therapeutic areas to watch in 2018



    Oncology is a fertile therapeutic category with 15 drugs approved in 2017 by press time. Aimee Tharaldson, a senior clinical pharmacist at Express Scripts, says breakthrough cancer therapies—several of which are immunotherapies such as programmed cell death protein (PD-1) inhibitors and chimeric antigen receptor T-cell (CAR T) therapy—are going to continue to reach the market quickly based on early data. 

    She also says more oral drugs will gain approval; 40% of those in the pipeline are administered orally.

    Because personalized medicine is making inroads into oncology, many cancer drugs will be accompanied by pharmacogenetic tests to identify appropriate candidates for therapy.

    Sandy Smith, vice president, U.S. oncology research, McKesson Specialty Health, says 2017 brought better understanding of immunotherapy, and how drugs are clinically benefiting patients based on individual characteristics and biomarkers and why certain drugs succeed in one person and not another. While immunotherapy is primarily targeting oncology, she anticipates that use will expand to other disease states.  

    In November, the FDA introduced a comprehensive policy framework for the development and oversight of regenerative medicine products that could help spur the development of treatments involving human cells and tissues, including gene therapy.

    One of the challenges, Smith says, is that often immunotherapy doesn’t immediately reduce the size of a tumor but could initially indicate pseudo progression. “Without sufficient education, patients won’t want to use immunotherapy,” Smith says. On the other hand, she is optimistic about immunotherapy’s effect on long-term survival, patient toleration, and quality of life. 

    Smith says these therapies could be quite expensive, especially when used in combination with other medications. But she says costs could become sustainable when genetic modifications improve and when these drugs are used only for select patients. 

    In August 2017, the FDA approved the first gene therapy—Kymriah (tisagenlecleucel), a CAR T therapy from Novartis for the treatment of certain pediatric and young adult patients with a form of acute lymphoblastic leukemia who have relapsed at least twice or no longer respond to standard treatments. 

    Although one multicenter trial indicates an overall remission rate of 83% within three months of treatment, the price tag for a one-time infusion could be out of reach at $475,000. A Risk Evaluation and Mitigation Strategy (REMS) is attached to the drug.

    A second CAR T therapy, Yescarta (axicabtagene ciloleucel) from Kite/Gilead, followed on the heels of Kymriah, with approval in October 2017. It is indicated for adult patients with relapsed or refractory forms of non-Hodgkin lymphoma with a one-time infusion cost of $373,000. It also is accompanied by REMS, as well as a boxed warning.

    Tharaldson anticipates that a third CAR T drug might be approved in the second half of 2018, as a second-line treatment for a form of non-Hodgkin lymphoma. Kymriah is expected to gain this expanded indication possibly in the second half of 2018.

    Sanofi and Regeneron’s Cemiplimab (REGN2810), a breakthrough therapy in development for the treatment of adults with locally advanced, unresectable or metastatic cutaneous squamous cell carcinoma (CSCC), is expected to be the next PD-1 inhibitor to be approved, also in the second half of 2018. While other PD-1 inhibitors are on the market, Cemiplimab is the only one that targets CSCC.


    Next: Autoimmune


    Mari Edlin
    Mari Edlin is a frequent contributor to Managed Healthcare Executive. She is based in Sonoma, California.


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