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Clinical Pathways GPS

CMS Policies Concerning CAR T-Cell Therapy

Authored by

Matthew Pakizegee, PharmD, MS; Richard G Stefanacci, DO, MGH, MBA, AGSF, CMD—Column Editor

Affiliation

EVERSANA™, Berkeley Heights, NJ

Disclosures

Dr Stefanacci is the chief medical director for the managed markets agency of EVERSANA™. Dr Pakizegee is the clinical content lead for Government Policy Systems at the managed markets agency of EVERSANA™. 

Citation

J Clin Pathways. 2019;5(4):32-33. doi:10.25270/jcp.2019.05.00076

Chimeric antigen receptor (CAR) T-cell therapy is a new form of cancer therapy that uses a patient’s own immune system to fight the disease. This type of cell therapy uses an individualized process to develop a treatment that is currently predominantly provided in the inpatient setting. The two approved CAR T-cell therapies on the market are tisagenlecleucel (Kymriah; Novartis) indicated for acute lymphoblastic leukemia and diffuse large B-cell lymphoma (DLBCL)1 and axicabtagene ciloleucel (Yescarta; Kite) indicated for DLBCL.2 Medicare members make up most DLBCL patients who receive these therapies based on the indications for which they are approved. Unfortunately, even with new Centers for Medicare & Medicaid Services (CMS) rules and proposed solutions, inpatient CAR T-cell therapy use has insufficient reimbursement.


Tisagenlecleucel and axicabtagene ciloleucel are the only CAR-T therapies on the US market so far, and both are approved for DLBCL.1,2 The administration and reimbursement of both therapies are affected my CMS policies, since at least half of DLBCL patients are covered by Medicare.3 Experts believe these cell therapies have the potential for sustained efficacy. However, long-term, real-world data is not currently available for these therapies, as they were only approved in 2017 based on phase 2 data. This creates a challenge for clinical pathways and payers since direct comparisons with standard of care is not possible given these current data gaps.4

CAR T-Cell Therapy Proposed National Coverage Determination

National coverage determinations (NCDs) are used by CMS to ensure consistent coverage for the therapies across the United States and to prevent access issues in certain regions based on inappropriate local Medicare Administrative Contractors.

CMS recently proposed Coverage with Evidence Development (CED) for hospital-administered CAR T-cell therapy in patients with relapsed or refractory cancer as a proposed NCD.5 The NCD would require Medicare to cover CAR-T therapy nationwide when it is offered in a CMS-approved registry or clinical trial in which patients are monitored for at least 2 years post-treatment. This CED is meant to ensure that Medicare patients receiving these novel treatments are followed to gather data on the ongoing efficacy and safety. This data, including patient-reported outcome (PRO) data, will be used to determine coverage of these two products for patients in the future. The CED may create new access barriers for patients receiving the two approved therapies. In addition, the requirements for a hospital to participate in the CED would result in additional administrative costs in tracking and maintaining data. Many hospitals may choose not to participate in the CED based on disincentives for reimbursement in the inpatient setting, resulting in challenges for patients trying to find providers in participating hospitals. All of these CED requirements need to be built into current clinical pathways as well as navigation options, such as site of care, to ensure appropriate patient access.

Both CAR T-cell therapy manufacturers have partnered with the Center for International Blood and Marrow Transplant Research (CIBMTR) to form registries.6,7 It is not clear whether this same registry will be used to track PRO and quality-of-life data. Many stakeholders have assumed CMS will adopt using CIBMTR, since any alternative would increase cancer centers’ burden of reporting patient data, but CIBMTR may need to make changes to reporting requirements to be consistent with CMS rules.8

Before the NCD proposal was presented, local Medicare Administrative Contractors had discretion over whether to pay for this new therapy, since there is no national policy. Medicare NCDs have not been used very often for cancer immunotherapy. The last time it was used for an immunotherapy was for sipuleucel-T (Provenge; Dendreon) in 2011.9 NCDs for cell therapies provide Medicare administrators and commercial payers that follow their lead an approach that takes into account the uncertainty associated with the lack of long-term efficacy data.

Inpatient Reimbursement Hurdles

Former Food and Drug Administration Commissioner Scott Gottlieb, MD, recently raised concerns that hospitals are being underpaid for CAR T-cell therapies when they are administered in the inpatient setting and how reimbursement issues may lead to reductions in future pharmaceutical industry investment in CAR T-cell therapy and other innovative treatments.10,11

Current CMS policies disadvantage inpatient administration of CAR T-cell products when it is not deemed clinically appropriate or safe, based on the proportion of patients who experience cytokine release syndrome. Outpatient administration is reimbursed by CMS based on average sales price, but most cancer centers that utilize this reimbursement option are engaged in clinical trials. CMS announced payment rates for outpatient administration of the two therapies at $395,380 for Yescarta and $500,839 for Kymriah, well above their list prices.12 Unfortunately for health systems administering these products, most payments for Medicare patients will be in the inpatient setting because of the accompanying clinical needs. 

In 2018, CMS categorized CAR T-cell therapy with an inpatient diagnosis-related group (DRG) 016 – Autologous Bone Marrow Transplant with CC/MCC or T-cell Immunotherapy. This DRG code reimburses hospitals $39,951 for infusing a CAR T-cell therapy along with an add-on outlier payment under the Inpatient Prospective Payment System.8 Additionally, hospitals get reimbursed new technology add-on payment (NTAP) for these therapies up to $186,500.13 In April 2019, CMS announced increase in the NTAP to $242,450 that will take effect October 2019.14 When all these payments are added up, the hospital still loses about $100,000 for each Medicare patient who receives CAR T-cell therapy based on the list prices of either product in the DLBCL indication at $373,000. Additionally, patients may face side effects like cytokine release syndrome and neurotoxicity that may result in costly intensive care unit stays without additional reimbursement for the hospital.15

In general, if medical treatment is provided in an outpatient setting for a Medicare patient and the patient requires inpatient care within 72 hours, all payments prior to that 72-hour window become part of the inpatient stay, according to CMS. When applied to a CAR T-cell therapy, Medicare patient therapy administered in an outpatient setting, monitoring and side effects management would make the administered cell therapy a part of an inpatient payment based on Medicare’s 3-day payment window rule.16

Implications for Clinical Pathways

Clinical pathways have a role in determining the best administration setting and process for these treatments. Based on the side-effect profile of CAR T-cell therapy products on the market, many cancer centers are not quite familiar enough with the new therapy to give it on an outpatient basis. 

For commercial payers, there are few barriers to inpatient reimbursement. Separate pathways need to be developed for commercial and Medicare patients based on reimbursement. The recent NCD proposal from CMS is specifically for relapsed or refractory cancer patients. CAR T-cell therapies will likely be approved for first-line treatment of hematologic malignancies in the future and may depend on timely updates to the NCD to prevent delays in coverage. 

A concern is whether CED data will be shared with payers and clinical pathway organizations for coverage determinations, as they are with CMS. It is not clear whether the data can be shared by CMS, manufacturers, or by health systems collecting the data. This information could be valuable in shaping future pathways and recommendations to providers treating hematologic malignancies. Another issue is whether patients will be allowed to opt out of data collection based on privacy concerns and whether coverage will be at risk if they choose not to participate in data collection. 

Conclusion

Clinical pathways that include CAR T-cell therapy as an option must integrate appropriate guidance on its use and settings of care. While these issues are extremely complex, guidance is important to facilitate ideal clinical and financial outcomes for all stakeholders.

References

1. Kymriah [package insert]. East Hanover, NJ: Novartis Pharmaceuticals Corporation; 2017. 

2. Yescarta [package insert]. Santa Monica, CA: Kite Pharma Inc; 2017.

3. Farnia S. Money Matters: CAR-T. Presented at: 2018 BMT Tandem Meetings; February 21-25, 2018; Salt Lake City, UT.  https://higherlogicdownload.s3.amazonaws.com/ASBMT/43a1f41f-55cb-4c97-9e78-c03e867db505/UploadedImages/Tandem_CAR_T_2018_Final.pdf. Accessed April 24, 2019.

4. American Society for Blood and Marrow Transplantation. ASBMT CMS IPPS FY 2019
Proposed Rule Comment Letter. June 22, 2018. https://higherlogicdownload.s3.
amazonaws.com/ASBMT/43a1f41f-55cb-4c97-9e78-c03e867db505/UploadedImages/ASBMT_CMS_Comment_Letter_IPPS_FY2019_6_2018_FINAL.pdf
.
Accessed April 24, 2019.

5. Centers for Medicare & Medicaid Services. Proposed decision memo for chimeric
antigen receptor (CAR) T-cell therapy for cancers (CAG-00451N). https://www.cms.gov/
medicare-coverage-database/details/nca-proposed-decision-memo.aspx?NCAId
=291
. Published February 15, 2019. Accessed April 24, 2019.

6. CIBMTR announces collaboration with Kite to track long-term outcomes data for YESCARTA [press release]. Milwaukee, WI: Center for International Blood and Marrow Transplant Research; May 17, 2018. https://www.eurekalert.org/pub_releases/2018
-05/btm-cac051618.php
. Accessed April 24, 2019.

7. CIBMTR to track long-term outcomes data for Kymriah [press release]. Minneapolis, MN: Be The Match BioTherapies; September 14, 2018. https://bethematchbiotherapies.com/
newsroom/cibmtr-to-track-long-term-outcomes-data-for-kymriah/
. Accessed April 24, 2019.

8. Shah J, Miller E. CMS super session: CAR-T & cellular therapy covrage, coding, reimbursement & policy updates. Presented at: 2019 Transplantation and Cellular Therapy Meetings; February 20-24, 2019; Houston, TX. https://higherlogicdownload.s3.amazonaws.com/ASBMT/43a1f41f-55cb-4c97-9e78-c03e867db505/UploadedImages/2019_Admin_Slides/Paper13316_1.pdf. Accessed April 24, 2019.

9. Centers for Medicare & Medicaid Services. Decision memo for autologous cellular immunotherapy treatment of metastatic prostate cancer (CAG-00422N). June 30, 2011.  https://www.cms.gov/medicare-coverage-database/details/nca-decision-memo.aspx?NCAId=247. Accessed April 24, 2019.

10. Dunn A. FDA’s Gottlieb ‘extremely worried’ on CAR-T reimbursement. BioPharmaDive website. https://www.biopharmadive.com/news/fdas-gottlieb-extremely-worried-on-car-t-reimbursement/540512/. Published October 24, 2018. Accessed April 24, 2019.

11. A Conversation With Scott Gottlieb, U.S. Food and Drug Administration [video]. Santa Monica, CA: Milken Institute; 2018. http://www.milkeninstitute.org/videos/view/a-conversation-
with-scott-gottlieb-commissioner-u-s-food-and-drug-administration
. Accessed April 24, 2019.

12. Gallegos A. Medicare sets outpatient CAR T-cell therapy rates. MDedge. April 27, 2018. https://www.mdedge.com/hematology-oncology/article/164403/business-medicine/medicare-sets-outpatient-car-t-cell-therapy. Accessed April 24, 2019.

13. Inserro A. CMS approves extra payments for CAR T, increases other payments in final rule. ajmc.com website. https://www.ajmc.com/newsroom/cms-approves-extra-payments-for-car-t-increases-other-payments-in-final-rule. Published August 3, 2018. Accessed April 24, 2019.

14. Beasley D. Medicare offers to partially raise payment for cancer CAR-Ts. Reuters. April 23, 2019. https://www.reuters.com/article/us-health-medicare-cancer/medicare-offers-to-partially-raise-payment-for-cancer-car-ts-idUSKCN1RZ2JC. Accessed April 30, 2019.

15. Tat T, Li H, Constantinescu CS, et al. Genetically enhanced T lymphocytes and the intensive care unit. Oncotarget. 2018;9(23):16557-16572. doi:10.18632/oncotarget.24637

16. Centers for Medicare & Medicaid Services. Three day payment window. Cms.gov website. https://www.cms.gov/Medicare/Medicare-Fee-for-Service-Payment/AcuteInpatientPPS/Three_Day_Payment_Window.html. Updated May 6, 2014. Accessed April 24, 2019.

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