Balancing Value with Affordability: Cell Immunotherapy for Cancer Treatment in the U.S.

Abstract

Despite the Centers for Medicare and Medicaid Services’ recent approval to increase payments for inpatient‐delivered chimeric antigen receptor T‐cell therapy (CAR‐T) for adult lymphoma, reimbursement remains far below the costs of the product and overall treatment of the therapy. We surveyed 92 CAR‐T‐certified centers in the U.S. to assess the perceived financial viability and related challenges for treating adult patients with lymphoma. Of 92 certified CAR‐T centers in the U.S., 20 (22%) directors or chief medical officers responded. More than three quarters of facilities reported treating patients in an inpatient setting, and 60% reported that the majority of their patients were covered under commercial/private insurance. The financial viability rating across centers (median: 62; interquartile range: 48–69; scale 1–100) signals that economic sustainability of institutional programs for adult lymphoma is a concern. These dynamics may limit access to CAR‐T for Medicare beneficiaries and lead to greater outpatient use of the therapy, which may limit access for medically complex patients.

Short abstract

CAR‐T represents one of the first of many anticipated ultra‐high‐priced treatments for rare or previously incurable conditions, making its successful integration into clinical practice and reimbursement models a useful example for gene and cell therapy adoption. This article focuses on the perceived financial viability and related challenges for CAR‐T among officials at CAR‐T certified centers across the U.S. for adult patients with lymphoma.

Introduction

Chimeric antigen receptor T‐cell therapy (CAR‐T) has been an area of exceptional promise for patients with treatment‐resistant cancers and the physicians treating them. However, treatment is complex, carrying substantial side effects for patients and financial challenges for hospital systems delivering them. Reimbursement has been slow, and in some cases, the amount is well below the cost of delivering CAR‐T therapy 1, 2, 3. For example, total inpatient reimbursement for CAR‐T therapy set by the Centers for Medicare and Medicaid Services (CMS) was initially only $186,500 for adult patients with lymphoma, far below the costs of the CAR‐T product itself ($373,000). Even with recent increases in inpatient reimbursement to $242,450 per case, reimbursement remains well below costs 4. With inpatient treatment‐related costs reaching $1 million or more in some cases 2, 3, access to CAR‐T for Medicare beneficiaries is at risk. These dynamics may also lead to greater outpatient use of the therapy (paid via fee‐for‐service medical billing under Medicare part B), which may limit access for some medically complex patients. There are fewer clinical data for outpatient use, however, than inpatient administration for currently approved therapies.

CAR‐T represents one of the first of many anticipated ultra‐high‐priced treatments for rare or previously incurable conditions, making its successful integration into clinical practice and reimbursement models a useful example for gene and cell therapy adoption. We sought to quantify the perceived financial viability and related challenges for CAR‐T among officials at CAR‐T‐certified centers across the U.S. for adult patients with lymphoma.

Materials and Methods

Between April and May 2019, we e‐mailed an 18‐item survey to cancer center directors or chief medical officers (CMOs) of 92 CAR‐T‐certified centers in the U.S. Survey domains included geographic region, whether facilities currently deliver axicabtagene ciloleucel (Yescarta; Kite Pharma, Santa Monica, CA) or tisagenlecleucel (Kymriah; Novartis, Basel, Switzerland) for adult patients with lymphoma, location of treatment (inpatient/outpatient), estimated number of patients treated and their insurance status, perceived financial viability (on a scale from 0 [lowest] to 100 [highest]) of the CAR‐T program, and the extent to which institutions leveraged markups to maximize reimbursement for CAR‐T.

Results

Of 92 certified CAR‐T centers in the U.S., 20 (22%) directors or CMOs responded, 70% (14/20) of whom were located in the southern or western U.S. Across the centers, respondents reported more than 500 patients treated for axicabtagene ciloleucel and 45 for tisagenlecleucel. All facilities reported delivering axicabtagene ciloleucel, whereas 79% (15/19) reported delivering tisagenlecleucel. More than three quarters of facilities reported treating patients in an inpatient setting, and 60% reported that the majority of their patients were covered under commercial/private insurance (Table 1).

Table 1.

Survey results of chimeric antigen receptor T‐cell therapy–certified centers in the U.S. (n = 20)

Characteristic	n (%)
Geographic region
Northeast	4 (20)
Midwest	2 (10)
South	8 (40)
West	6 (30)
Delivers axicabtagene ciloleucel	20 (100)
Inpatient only	17 (85)
Delivers tisagenlecleucel (n = 19; missing = 1)	15 (79)
Inpatient only (n = 14; missing = 1)	10 (71)
Estimated insurance breakdown for treated patients
Majority commercial/private	12 (60)
Institutional leverage of markups to maximize reimbursement (n = 18; missing = 2)	6 (33)
If facility reported <100 for financial viability, factors that need to change (n = 15; missing = 4)a
Reimbursement	10 (67)
Cost of product	5 (33)

^aOne respondent rated their institution's financial viability at 100.

The median respondents’ rating of the financial viability of their institution's CAR‐T program for adult lymphoma was 62 (interquartile range:48–69; Fig. 1). Among 15 respondents who rated their CAR‐T program financial viability below 100, nearly three quarters responded that reimbursement needed to change, whereas just over 30% responded that the high cost of the product needed to change. There was also evidence of centers leveraging institutional markups to increase aggregate charges, which would result in additional outlier payments from Medicare or stop‐loss payments from insurers. Approximately 33% of respondents in our sample acknowledged using such markups to maximize reimbursement.

Figure 1.

Respondent financial viability rating of chimeric antigen receptor T‐cell therapy programs for adult patients with lymphoma. Financial viability scale: 0 = lowest; 100 = highest. Bubble size represents the number of respondents reporting each viability score range (i.e., larger bubble size represents a higher number of respondents indicating a particular financial viability score).

Discussion

The financial viability rating across certified CAR‐T centers signals that economic sustainability of institutional programs for adult lymphoma is a concern, with Medicare reimbursement rates and manufacturer prices for CAR‐T the primary drivers of viability concerns. Manufacturer price reductions may be necessary to ensure continued access to treatment. It is too soon to tell what impact reimbursement levels will have on Medicare beneficiary access to CAR‐T, particularly for those who are not healthy enough to receive outpatient treatment. Notably, most patients treated to date among surveyed sites were privately insured, despite the national median age at diagnosis of 66 5. With centers now finding ways to offset losses such as through outpatient use of the therapy and institutional markups, CMS should closely monitor access for Medicare beneficiaries, as well as spending per beneficiary.

Limitations of this assessment include self‐reported data, participation bias, and the relatively low response rates (although rates are comparable to other physician‐administered surveys, particularly among web‐based and specialty physician surveys 6, 7, 8). A higher proportion of respondents were located in the southern and western parts of the U.S., and we did not collect information on 340B status and whether sites were Prospective Payment System exempt (standalone cancer centers). Although responses may not fully generalize to all CAR‐T treatment sites, the study results provide a snapshot of practices currently offering CAR‐T to adult patients. Despite the potential value of cellular and gene therapies like CAR‐T, affordability presents new challenges for health systems and payers. CAR‐T represents 2 of 17 U.S. Food and Drug Administration–approved cellular and gene therapy products 9. Whether therapies will be available at a price that both the patient and health sector can afford is unclear.

Conclusion

Access to costly therapies like CAR‐T often faults the most vulnerable populations. Although centers are quickly finding ways to redeem financial losses, payers, manufacturers, and health systems have an obligation to work together on more sustainable financing arrangements to enable patients to realize the full benefits of these promising new therapies.

Disclosures

Joshua T. Cohen: AbbVie, Axovant, Novartis, Partnership for Health Analytic Research, Pharmerit, Precision Health Economics, Sage Therapeutics, Sarepta Therapeutics (C/A); Peter J. Neumann: Abbvie, Amgen, Avexis, Bayer, Congressional Budget Office, Vertex, Veritech, Janssen, Merck, Novartis, Novo Nordisk, Precision Health Economics (C/A), The CEA Registry Sponsors by various pharmaceutical and medical device companies, Amgen, Lundbeck, Bill and Melinda Gates Foundation, NPC, Alzheimer's Association (RF). The other authors indicated no financial relationships.

(C/A) Consulting/advisory relationship; (RF) Research funding; (E) Employment; (ET) Expert testimony; (H) Honoraria received; (OI) Ownership interests; (IP) Intellectual property rights/inventor/patent holder; (SAB) Scientific advisory board