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The American Journal of Managed Care August 2019
Late Diagnosis of Hepatitis C Virus Infection, 2014-2016: Continuing Missed Intervention Opportunities
Anne C. Moorman, MPH; Jian Xing, PhD; Loralee B. Rupp, MSE; Stuart C. Gordon, MD; Mei Lu, PhD; Philip R. Spradling, MD; Joseph A. Boscarino, PhD; Mark A. Schmidt, PhD; Yihe G. Daida, PhD; and Eyasu H. Teshale, MD; for the CHeCS Investigators
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The Potential Impact of CAR T-Cell Treatment Delays on Society
Julia Thornton Snider, PhD; Michelle Brauer, BS; Rebecca Kee, BA; Katharine Batt, MD, MSc; Pinar Karaca-Mandic, PhD; Jie Zhang, PhD; and Dana P. Goldman, PhD
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Erin L. Duffy, PhD, MPH
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The Potential Impact of CAR T-Cell Treatment Delays on Society

Julia Thornton Snider, PhD; Michelle Brauer, BS; Rebecca Kee, BA; Katharine Batt, MD, MSc; Pinar Karaca-Mandic, PhD; Jie Zhang, PhD; and Dana P. Goldman, PhD
Treatment delays limit the social value generated by chimeric antigen receptor (CAR) T-cell therapy for the treatment of pediatric acute lymphoblastic leukemia and diffuse large B-cell lymphoma.
ABSTRACT

Objectives: To date, breakthrough chimeric antigen receptor (CAR) T-cell therapies, such as tisagenlecleucel, indicated for pediatric acute lymphoblastic leukemia (pALL) and diffuse large B-cell lymphoma (DLBCL), and axicabtagene ciloleucel, indicated for DLBCL, although clinically effective, have been limited by treatment delays. Our study measured the social value of CAR T-cell therapy (CAR T) for relapsed or refractory pALL and DLBCL in the United States and quantified social value lost due to treatment delays.

Study Design: We used an economic framework for therapy valuation, measuring social value as the sum of consumer surplus and manufacturer profit. Consumer surplus is the difference between the value of health gains from a therapy and its incremental cost, while accounting for indirect costs and benefits to patients.

Methods: For 20 incident cohorts of pALL (n = 20 × 400 = 8000) and DLBCL (n = 20 × 5902 = 118,040), we quantified patient value, calculated as the value of additional quality-adjusted life-years gained with CAR T, minus the incremental cost of CAR T compared with standard of care (SOC). We calculated manufacturer profits using a range of production costs given uncertainties in the production process. Patient value and manufacturer profits were summed to obtain total social value. We measured social value lost from treatment delays, assuming that patients received the SOC while awaiting CAR T-cell treatment.

Results: Depending on production costs, as much as $6.5 billion and $34.8 billion in social value was generated for patients with pALL and DLBCL, respectively. However, with 1, 2, or 6 months of treatment delay (assuming $200,000 production costs), the pALL population lost 9.8%, 36.2%, and 67.3% of social value, respectively, whereas the DLBCL population lost 4.2%, 11.5%, and 46.0%, relative to no delay.

Conclusions: The social value of CAR T is significantly limited by treatment delays. Efficient payment mechanisms, adequate capital, and payment policy reform are urgently needed to increase patient access and maximize the value of CAR T.

Am J Manag Care. 2019;25(8):379-386
Takeaway Points
  • Chimeric antigen receptor (CAR) T-cell therapies can provide significant benefit to patients with relapsed/refractory pediatric acute lymphoblastic leukemia (pALL) and diffuse large B-cell lymphoma (DLBCL) and to US society, generating up to $6.5 billion and $34.8 billion of social value for patients with pALL and DLBCL, respectively.
  • However, with 1, 2, or 6 months of treatment delay, patients with pALL lost 9.8%, 36.2%, and 67.3% of social value, respectively; patients with DLBCL lost 4.2%, 11.5%, and 46.0% of social value, respectively.
  • The magnitude of CAR T-cell therapy’s value depends on timely patient access. Efficient payment mechanisms, adequate physical and human capital, and payment policy reform could help reduce treatment delays.
Although there have been major advances in treatments for hematologic cancers such as pediatric acute lymphoblastic leukemia (pALL) and diffuse large B-cell lymphoma (DLBCL),1-3 efficacious treatments have historically remained limited for the population with relapsed or refractory disease.3,4 However, chimeric antigen receptor (CAR) T-cell therapies, such as tisagenlecleucel and axicabtagene ciloleucel, offer a possible cure for these patients.5-9 A recent review by the Institute for Clinical and Economic Review (ICER)10 concluded that tisagenlecleucel for pALL and axicabtagene ciloleucel for DLBCL are cost-effective treatments with incremental costs per quality-adjusted life-year (QALY) of $45,971 and $136,078, respectively.

Despite the recent approval of breakthrough therapies using CAR T cells in the United States, patients have faced barriers to treatment, including manufacturing challenges and a lack of formal coverage policies for CAR T-cell therapy (CAR T) in an inpatient setting,11,12 with delays as long as 90 days.11 Given the aggressive nature of relapsed/refractory disease, patients eligible for CAR T may have to settle for less efficacious third- or fourth-line therapies10,13 or even die while waiting for CAR T reimbursement approval.11

Cost-effectiveness analyses, like the ICER report, are useful for informing how resources may be allocated to treatments with the greatest QALY gains; however, stakeholders must consider the trade-off between treatment access today and incentivizing future treatment innovation. Social value analyses can complement cost-effectiveness analyses by shedding light on the access/innovation trade-off. Both types of analyses can inform coverage decisions, but they provide insight into different trade-offs that decision makers must weigh.

In this study, we measured the social value of treating pALL and DLBCL with CAR T in the United States and the social value lost from treatment delays as reported in the media.11,12,14-16 Social value analyses are used to quantify a therapy’s economic value from a societal perspective17 and determine the share of that value accruing to the manufacturer and patients. Expanded patient access and greater health benefits increase social value, whereas a greater requirement of society’s resources to produce the therapy (ie, higher production costs) reduces it. The higher the share of social value accruing to the manufacturer, the stronger the incentives for innovation. However, when treatment is delayed, social value is lost for both patients and manufacturers: Patients lose access to health gains from the treatment, and manufacturer profit is reduced.

METHODS

An economic framework for therapy valuation was used. Specifically, we measured social value as the sum of consumer surplus and manufacturer profit.17 In the health context, consumer surplus measures the difference between the value of the health gains from a therapy and its incremental cost to the patient. It also accounts for indirect costs and benefits to patients. We calculated the economic benefit of tisagenlecleucel for pALL relative to standard of care (SOC), clofarabine monotherapy, and of axicabtagene ciloleucel for DLBCL relative to salvage chemotherapy.10 In each case, social value was estimated for 20 incident cohorts in the United States over a lifetime horizon. In each year, a new incident cohort entered the model and the existing prevalent cohorts aged an additional year. Each cohort’s survival followed that of the average patient for each treatment. We explain our calculations using tisagenlecleucel as an example; calculations for axicabtagene ciloleucel were similar, unless otherwise noted.

We obtained clinical and cost parameters from the literature and ICER’s assessment of CAR T (Table 1 [part A and part B]10,18-26).10 ICER reported that 400 incident cases of relapsed or refractory pALL occur annually and estimated that the average patient with pALL treated with CAR T would gain 7.9 discounted life-years (12.1 undiscounted) and 7.2 discounted QALYs (10.9 undiscounted) over SOC. Costs to the patient of tisagenlecleucel and SOC were obtained from the literature.20 Other treatment-associated costs were obtained from ICER’s report and can be found in Table 1.10,18-26

We measured patient value, also known as consumer surplus, which is the difference between how much a consumer is willing to pay for a good or service and its price. We first estimated the health value that patients obtained, calculated as the value of QALYs gained with tisagenlecleucel compared with SOC, valuing each QALY gained at $150,000 (a midrange value from the literature).18 To obtain patient value, the incremental cost of tisagenlecleucel relative to SOC was subtracted from the health value.


 
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