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From Science Fiction to Reality: Blockbuster Drug Approvals Offer Innovative Approaches to Life-Threatening Diseases

November 9, 2017

Could this be the beginning of the end of cancer?iStock-868432664_asian_woman_fighting_disease_duo.jpg

Chimeric antigen receptor T-cell (CAR-T cell) therapy sounds like language out of a science fiction novel, but it’s making a major impact in the cancer community as a breakthrough treatment. The therapy involves engineering a patient's T cells to express a receptor that targets the protein on the surface of a cancer cell. In other words, the cancer patient’s own cells are reprogrammed to attack and kill the cancer.

The U.S. Food and Drug Administration (FDA) approved KYMRIAH™ (tisagenlecleucel), the first drug utilizing gene therapy, at the end of August 2017 and YESCARTA™ (axicabtagene ciloleucel) shortly thereafter in October. These two CAR-T therapies have the potential to revolutionize medicine, leading to new treatments and possibly cures.

Kymriah, a product of Novartis Pharmaceuticals Corp, was approved to treat certain pediatric and young adult patients up to 25 years of age with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). ALL is a cancer of the bone marrow and blood in which the body makes abnormal lymphocytes. It is the most common childhood cancer in the United States. Despite overall improvements in outcomes of pediatric/young adult B-cell ALL, effective treatment options for patients that have relapsed or are refractory to treatment are limited.

Manufactured by Kite Pharma, Yescarta treats adults with certain types of large B-cell non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant. NHL is a cancer that begins in the lymph organs, which is part of the body’s system that fights infection and disease. Patients with aggressive lymphoma who do not respond to their previous treatments have a very poor prognosis, typically an 8% chance of achieving a complete response to current therapies.[1]

According to the FDA, in clinical trials, the overall remission rate for ALL patients within three months of Kymriah treatment was 83%.[2] For Yescarta, remission rate for NHL patients after treatment was more than half, at 51%.[3] Novartis announced last week that it is seeking approval for a second indication for Kymriah, which will also treat NHL.[4]

Although innovative and clinical results are promising, both drugs carry a boxed warning for cytokine release syndrome (CRS), which can be life-threatening. Because of this, both require a risk evaluation and mitigation strategy (REMS) to ensure safe use. The FDA approved Actemra (tocilizumab) to treat CAR T-cell-induced CRS.

Potential impact for Plan Sponsors:

These blockbuster drugs can be catastrophic to a plan’s budget. The one-time treatment of Kymriah launched with an average wholesale price (AWP) of $570,000 per dosage, not inclusive of in-patient critical medical care typically required. Although less expensive, a one-time treatment of Yescarta has a list price of $373,000.[5] Due to the nature of the drug’s administration, most claims will occur within the medical benefit rather than traditional pharmacy benefit claims processing. However, medications commonly needed post-infusion to treat side effects, such as Actemra (tocilizumab) and IV immunoglobulin, may be seen in the pharmacy benefit.

Icon_Pins_ targetred.pngEnvisionRx has taken proactive steps to ensure all pieces of this complex regimen—from the primary infusion to the side effect therapies—have a disciplined approach to positive patient experience care. Ask your account team how these therapies may affect your plan and discuss ways to mitigate risk.


Human blood provides yet another life-saving role in new treatment

On June 22, 2017 the FDA announced the approval of HAEGARDA® (C1 esterase inhibitor, human), the first self-administered option to prevent potentially life-threatening episodes of hereditary angioedema (HAE) in adolescents and adults. A rare genetic disease, HAE is caused by a deficient or dysfunctional C1-INH protein found in the blood that helps control inflammation. Patients suffer from regular, painful and unpredictable episodes of swelling in the body that can be life-threatening, as in the case in laryngeal attacks, which can block the airway and be fatal.

According to the FDA, Haegarda is a human plasma-derived, purified, pasteurized, freeze-dried concentrate prepared from large pools of human plasma from U.S. donors.[6] The drug works by replacing the faulty protein through a self-administered, plasma-derived concentrate of C1-INH injected twice weekly subcutaneously (just under the skin). Clinical trials show that Haegarda reduced HAE attacks by 95% and decreased the use of rescue medication by more than 99%. According to the manufacturer, CSL Behring, subcutaneous administration of Haegarda builds and maintains steady-state functional C1-INH levels within three to four doses.[7]

Along with showing effectiveness, safety was evaluated and the most common adverse reactions (>4%) reported were injection site reactions, hypersensitivity, nasopharyngitis, and dizziness. It is approved for routine prophylaxis to prevent HAE attacks, but is not indicated for treatment of acute HAE attacks.[8]

Potential impact for Plan Sponsors:

With this approval of Haegarda as the first self-administered option for prevention of acute swelling attacks, there is the potential for a significant increase in utilizers and a resulting increase in client spend. Haegarda is available to be dosed from designated vial sizes that will create an issue of potential product waste resulting in soaring costs if not managed properly, with yearly cost estimates per patient ranging from $197,400 to $592,200.

Icon_Pins_ targetred.pngEnvisionRx uses a multitude of management strategies to protect clients from inappropriate or over usage. To assess which patients may switch to Haegarda, watch your medical utilization for this class of medications (review diagnosis codes ICD-10:D84.1 or J0597; J0596; J0598). Ask your account team to see if you currently have members with HAE that may be affected by this new medication.


Balancing clinical effectiveness and economic impact with the member experience

The Envision Clinical Steering Committee brings together leaders across the enterprise to monitor the drug landscape and prepare stakeholders for changes in the market, with the goal of balancing the impact from a clinical, economic and member experience perspective. This involves clinical reviews of best practices for evaluation of safety and efficacy, analysis of cost impact and ways to mitigate higher costs, and collaborating with specialty vendor partners to develop a patient-centered care approach.


[1] Crump M, Neelapu SS, Farooq U, et al. (2016). Outcomes in refractory aggressive diffuse large B-cell lymphoma (DLBCL): results from the international SCHOLAR-1 study. Presented at: the 2016 American Society of Clinical Oncology Annual Meeting; Chicago, Illinois; June 3-7, 2016. Abstract 7516
[2] U.S. Food & Drug Administration (2017). FDA approval brings first gene therapy to the United States. August 30, 2017. Retrieved from:
[3] U.S. Food & Drug Administration (2017). FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma. October 18, 2017. Retrieved from:
[4] Toich, L. (2017). Novartis seeks new indication for gene therapy Kymriah. American Journal of Pharmacy Benefits. November 1, 2017. Retrieved from:
[5] Ramsey, L. (2017). A cancer treatment that’s part of a ‘big new field of medicine’ just got approved. Business Insider. October 18, 2017. Retrieved from:
[6] U.S. Food and Drug Administration (2017). FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease. June 22, 2017. Retrieved from:
[7] CSL Behring (2017). FDA approves HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]), first and only subcutaneous preventive treatment for Hereditary Angioedema. June 23, 2017. Retrieved from:
[8] U.S. Food and Drug Administration (2017). FDA approves first subcutaneous C1 Esterase Inhibitor.