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The Potential Impact of CAR T-Cell Treatment Delays on Society
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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.
Next, we estimated productivity gains from tisagenlecleucel. Because the value of QALYs gained to patients includes the value of the labor and leisure they afford, some of the health value from tisagenlecleucel is attributable to productivity gains. Nationally representative data on employment and wages by age and sex from the Bureau of Labor Statistics and the US Census Bureau were used to calculate productivity gains.21,22

The manufacturer profits were calculated next. We considered a range of production costs from $100,000 to $300,000 to reflect uncertainty and likely changes in the production process over time. The midpoint value, $200,000, was taken as the base-case value for sensitivity analysis. Given uncertainty about the future price of tisagenlecleucel with loss of exclusivity and competitor entry, we simplistically assumed a 30% price reduction in 2030 based on estimates from the literature.27 Finally, total social value was calculated by summing the patient value and manufacturer profit.

We calculated social value lost from treatment delays for the first pALL cohort. We examined the first cohort rather than all 20 cohorts given uncertainty around the extent of treatment delays in the future. We assumed that patients would take the SOC treatment while waiting for tisagenlecleucel and would initiate tisagenlecleucel treatment if they survived long enough to receive it. Survival of patients treated with clofarabine monotherapy was obtained from clinical trial data.28 For the first cohort, life-years, QALYs, and productivity were calculated conditional on patients receiving treatment after 1, 2, and 6 months of delay. Incremental value lost was calculated as the difference between the value obtained by patients who were treated immediately (0-month delay) and those who experienced delays.

The steps above were repeated to measure the value of axicabtagene ciloleucel for the treatment of 20 incident cohorts of patients with DLBCL, using a clinical trial for salvage chemotherapy13 as the SOC comparator in the treatment delays analysis. Table 110,18-26 contains the parameters used in the calculations for patients with DLBCL. All health and monetary values were discounted at a rate of 3.0% and costs were inflated to 2017 US dollars. Additional detail is available in the eAppendix (available at

We ran sensitivity analyses to test how consumer surplus, manufacturer profit, and social value changed by varying key model inputs. In 1-way sensitivity analyses, we adjusted the number of patients eligible for treatment, economic value of a QALY, price of CAR T, production costs, and future reduction in CAR T price individually from minimum to maximum values. We also varied life-year and QALY gains concurrently by ±50% and potential patient income by ±20%. In multiway sensitivity analyses, we conducted 1000 Monte Carlo simulations to vary each of the above parameters concurrently by selecting values of each parameter from its distribution, which measured the sensitivity of social value, manufacturer profit, and consumer surplus to the model assumptions. Each parameter was assumed to follow a beta distribution.



In the population with pALL (n = 20 × 400 = 8000), considering production costs of $100,000, $200,000, and $300,000 and a price of $475,000, we found that the total social values of tisagenlecleucel at each production cost were $6.5 billion, $5.8 billion, and $5.2 billion, respectively (Figure 1). The value accruing to patients was $4.4 billion regardless of production costs, representing 68.9%, 76.1%, and 85.0% of total social value, respectively. This translates to 48,485 life-years, 44,010 QALYs (worth $6.6 billion), and $352.0 million in productivity (worth 5.3% of QALY gains). The remaining 15.0% to 31.1% of total social value accrued to manufacturers.

Assuming no treatment delays, patients with pALL in the first cohort gained 2872 total QALYs. The value of those QALY gains totaled $430.8 million, of which $23.0 million (5.3%) was attributable to added patient productivity from employment gains. Accounting for the cost of acquiring CAR T, the total patient value was $271.2 million and the total social value was $381.2 million. This translates to 7.2 QALYs (worth $1.1 million), $57,423 in added productivity, and a social value of $952,991 per patient.

However, with 1, 2, or 6 months of treatment delay (assuming $200,000 production costs), the first pALL cohort lost 9.8%, 36.2%, and 67.3% of social value, respectively, relative to no treatment delays. Contributing to this were losses of 311, 1146, and 2128 total life-years; 282, 1040, and 1932 total QALYs; and $2.3 million, $8.3 million, and $15.4 million in total productivity, respectively. Each patient lost 0.8, 2.9, and 5.3 life-years; 0.7, 2.6, and 4.8 QALYs; $5638, $20,796, and $38,622 in productivity (Figure 213,28); and $93,560, $345,133, and $640,967 in social value, respectively. The loss of social value stems primarily from a high mortality rate in patients receiving SOC while awaiting treatment with tisagenlecleucel.28

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