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Increasing Focus on Value Related to Cancer Drugs: A Payer Perspective

Breast cancer is a hot topic among payers. Approximately 12% of women will have invasive breast cancer in their lifetime.1 In 2017 alone, more than 250,000 new cases of invasive breast cancer are projected to be diagnosed in the United States, in addition to the estimated 63,410 cases of noninvasive breast cancer that will be diagnosed, making breast cancer the most common type of cancer.1,2

Responsible for approximately 40,000 deaths annually, breast cancer is the fourth leading cause of cancer-related death, after lung, colorectal, and pancreatic cancer.2 Not surprisingly, this necessitates dissecting the cost of therapy relative to its efficacy and safety. Based on the prevalence of invasive breast cancer, the amount of drug therapy required among a managed patient population is considerable, and significantly affects the top cancer trend therapies within the cancer budget for payers.

Outside of initial clinical trials, few tools are available to assess the real-world impact of breast cancer on patients. In a research letter published in February 2017 in the Journal of the American Medical Association, the authors compared the impact of several new cancer drugs on progression-free survival (PFS) with their impact on the patient’s quality of life and annual cost.3

Two breast cancer drugs were included in the analysis–bevacizumab, which lost its indication for breast cancer in 2011, and everolimus.3 The authors concluded that bevacizumab demonstrated no statistical difference in quality of life versus placebo, at a cost of $134,124 annually, and everolimus demonstrated mixed results for quality of life compared with placebo, at an annual cost of $144,840.3

One ongoing study with ribociclib includes the combination of ribociclib plus everolimus and exemestane in patients who received no more than 1 previous line of therapy.4 If approved, this combination will come with a substantial financial cost. However, the hope is that the combination’s efficacy and quality of life will offset its financial burden and adverse effects.

Palbociclib, first approved by the US Food and Drug Administration (FDA) in 2015, has little data to support its real-world cost-effectiveness compared with other treatments. Because palbociclib shares a similar mechanism of action with that of ribociclib, it is the best comparable treatment that is currently FDA approved. Palbociclib plus letrozole demonstrated a 10-month improvement in PFS compared with placebo plus letrozole in women who had not received previous systemic therapy.5 In women with disease progression after previous therapy, palbociclib plus fulvestrant increased PFS by more than 5 months compared with placebo plus fulvestrant.6 Both of these results were significant. The initial data for ribociclib plus letrozole look promising, because this combination has met its primary end point of PFS compared with placebo plus letrozole, despite not yet having mature end points.7

Similar to palbociclib, neutropenia is the most common and worrisome adverse event in patients who receive ribociclib. In addition to posing significant risks, and possibly requiring the discontinuation of regularly scheduled therapy, neutropenia also contributes to additional downstream expenses through the use of colony-stimulating agents and other healthcare resource utilization such as hospitalizations. Dose interruption, reduction, and the discontinuation of ribociclib are all strategies to reduce the incidence of neutropenia, which can be well-managed if it does occur.8

The initial wholesale acquisition price of palbociclib was $469 in February 2015.9 As of January 1, 2017, after an 11% increase in price, palbociclib costs $552 per capsule, amounting to more than $140,000 annually. The wholesale acquisition cost (WAC) of ribociclib for a 28-day cycle is $10,950 for the 600-mg once daily dose, $8760 for the 400-mg dose, and $4380 for the 200-mg dose. The flexible pricing structure for ribociclib allows physicians to easily adjust dosing, as necessary, according to the patient’s tolerability and without treatment interruption. Based on these prices, ribociclib will be the CDK4/CDK6 inhibitor with the lowest WAC at its launch.10

In addition to breast cancer, ribociclib is being studied in non–small-cell lung cancer, head and neck cancer, melanoma, high-grade glioma, and other cancers. All these cancer types are in need of a greater number of targeted therapies that produce substantially improved results. Because drugs such as ribociclib are approved in broad patient populations, this trend continues to highlight the unsustainable cost of oncolytic drugs as a whole, and the burden this cost places on individuals, employer groups, and government programs.

It is always exciting to have new drugs approved for the treatment of cancers for which there is no cure. With the high prevalence of breast cancer, new therapies may reach a large number of patients who are looking for new treatment options. Ribociclib may provide a new option to be added to the armamentarium of choices for physicians and patients in the treatment of breast cancer.

Payers will continue to evaluate drugs such as ribociclib based on clinical outcomes. Even without direct head-to-head studies, payers will continue to scrutinize available clinical and cost data, with the goal of differentiating drugs. In addition, payers will increasingly prefer therapies that have a better perceived overall value.

Author Disclosure Statement
Dr Mitchell reported no conflicts of interest.


  1. U.S. breast cancer statistics. Accessed February 3, 2017.
  2. National Cancer Institute. Common cancer types. Accessed February 3, 2017.
  3. Rupp T, Zuckerman D. Quality of life, overall survival, and costs of cancer drugs approved based on surrogate endpoints. JAMA Intern Med. 2017;177:276-277.
  4. Phase Ib trial of LEE011 with everolimus (RAD001) and exemestane in the treatment of hormone receptor positive HER2 negative advanced breast cancer. Accessed February 23, 2017.
  5. Finn RS, Martin M, Rugo HS, et al. Palbociclib and letrozole in advanced breast cancer. N Engl J Med. 2016;375:1925-1936.
  6. Turner NC, Ro J, André F, et al. Palbociclib in hormone-receptor-positive advanced breast cancer. N Engl J Med. 2015;373:209-219.
  7. European Society for Medical Oncology. ESMO 2016 press release: ribociclib improves progression-free survival in advanced breast cancer. October 8, 2016. Accessed December 15, 2016.
  8. Kisqali (ribociclib) tablets [prescribing information]. East Hanover, NJ: Novartis; March 2017.
  9. Medi-Span cost data. Accessed February 3, 2017.
  10. Pagliarulo N. Novartis locks up Kisqali approval, eyes Pfizer’s lead. March 13, 2017. Accessed June 8, 2017.
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