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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
Access to Chiropractic Care and the Cost of Spine Conditions Among Older Adults
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Tools to Improve Referrals From Primary Care to Specialty Care
Varsha G. Vimalananda, MD, MPH; Mark Meterko, PhD; Molly E. Waring, PhD; Shirley Qian, MS; Amanda Solch, MSW; Jolie B. Wormwood, PhD; and B. Graeme Fincke, MD
Influence of Out-of-Network Payment Standards on Insurer–Provider Bargaining: California’s Experience
Erin L. Duffy, PhD, MPH
Cost of Dementia in Medicare Managed Care: A Systematic Literature Review
Paul Fishman, PhD; Norma B. Coe, PhD; Lindsay White, PhD; Paul K. Crane, MD, MPH; Sungchul Park, PhD; Bailey Ingraham, MS; and Eric B. Larson, MD, MPH

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.

In the population with DLBCL (n = 20 × 5902 = 118,040), given production costs of $100,000, $200,000, and $300,000 and a price of $373,000, the total social values of axicabtagene ciloleucel were $34.8 billion, $25.8 billion, and $16.7 billion, respectively (Figure 3). The value accruing to patients was $13.5 billion regardless of production costs, which represents 38.7%, 52.2%, and 80.5% of total social value, respectively. This translates to gains of 372,617 life-years, 306,595 QALYs (worth $46.0 billion), and $12.5 billion in productivity (worth 27.3% of QALY gains). The remaining 19.5% to 61.3% of total social value accrued to manufacturers.

The first cohort of patients with DLBCL gained 20,008 total QALYs, assuming no treatment delays. The value of those QALY gains totaled $3.0 billion, of which $818.9 million (27.3%) was attributable to added patient productivity from employment gains. Accounting for the cost of acquiring CAR T, the total patient value was $659.5 million, and the total social value was $1.68 billion. This translates to 3.39 QALYs (worth $508,500), $138,742 in added productivity, and a social value of $284,743 per patient.

However, with 1, 2, or 6 months of treatment delay (assuming $200,000 production costs), the first DLBCL cohort lost 4.2%, 11.5%, and 46.0% of social value, respectively, relative to no treatment delays. Contributing to this were losses of 1021, 2796, and 11,185 total life-years; 840, 2301, and 9204 total QALYs; and $34.4 million, $94.2 million, and $376.7 million in total productivity, respectively. Each patient lost 0.2, 0.5, and 1.9 life-years; 0.1, 0.4, and 1.6 QALYs; and $5827, $15,955, and $63,821 in productivity (Figure 213,28), resulting in losses of $11,959, $32,745, and $130,982 in social value, respectively.

Sensitivity Analyses

In the pALL analysis, results of 1-way sensitivity analyses showed that social value was most sensitive to the discount rate, value of a QALY, and survival gains (Table 2). When key parameter assumptions were varied simultaneously to test the sensitivity of the model to those inputs, the social value and patient value results were most sensitive to the discount rate, value of a QALY, and survival gains (eAppendix Figures 1 and 2), and the manufacturer profits were most sensitive to the production costs, discount rate, and number of patients eligible for tisagenlecleucel (eAppendix Figure 3).

In the DLBCL analysis, results of 1-way sensitivity analyses indicated that social value was most sensitive to the survival gains, value of a QALY, and production costs of axicabtagene ciloleucel (Table 2). These findings are similar in the multiway sensitivity analysis of social value (eAppendix Figure 4). Meanwhile, multiway sensitivity analyses indicated that patient value was most sensitive to survival gains, value of a QALY, and discount rate, whereas manufacturer profits were most sensitive to production costs, number of patients eligible for axicabtagene ciloleucel, and discount rate (eAppendix Figures 5 and 6).

Because the total social value of CAR T is determined by the survival gains and the production costs, it is expected that social value is most sensitive to the aforementioned parameters. Meanwhile, the price of CAR T, future reduction in its price, and patient income had no effect on total social value because the former 2 parameters affect only the patients’ and manufacturers’ shares of social value, whereas the latter affects only the amount of patient value attributable to productivity.


CAR T has provided the hope of a cure to patients who otherwise have limited treatment options and poor prognoses.29 Patients receiving CAR T are expected to experience meaningful improvements in life expectancy and QALYs, enabling them to contribute to overall productivity and generate social value. In both pALL and DLBCL, patients lost a substantial share of social value with treatment delays.

Various reasons have been reported for the treatment delays.11,14 One-time curative treatments such as tisagenlecleucel and axicabtagene ciloleucel present a challenge to existing payment systems because their costs accrue up front, whereas benefits accrue over a lifetime, in contrast with other cancer therapies that are administered over an extended time period. To address this challenge, novel financing mechanisms, such as an outcomes-based approach to reimbursement for tisagenlecleucel, are currently being discussed.16,30 One reimbursement approach under consideration would allow participating payers to pay for tisagenlecleucel only when patients respond within 1 month of treatment,31 allowing payers and manufacturers to share the financial risk. Additionally, aspects of the US healthcare system present challenges to outcomes-based contracts for curative therapies like CAR T. Because the average American changes health insurers every few years,32 the payers that pay the up-front costs of treatment with CAR T may not be the same ones that cover the cured individual years down the line. Thus, the payer may benefit from only a fraction of the savings, which reduces the incentive to invest in curative therapies.33 Creative solutions have been proposed to combat this “free-rider” problem.33,34 Although an outcomes-based contract developed for CAR T may help reduce payers’ risk of paying for nonresponse, the issue of up-front costs disincentivizing innovation remains.

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