June 2018 Edition Vol.11, Issue 6

CAR T-Cell Therapies: Early Insights into Access and Affordability

By Christina Bennett, MS

Chimeric antigen receptor (CAR) T-cell therapies have ignited enthusiasm with the FDA approvals of Kymriah [tisagenlecleucel; Novartis] and Yescarta [axicabtagene ciloleucel; Kite/Gilead], but with this burgeoning enthusiasm comes questions on how patients may access these drugs and how much payers cover for these expensive, one-time therapies. While early trends into access and affordability have emerged, it is still unclear long-term how payers and providers will employ CAR T-cell therapies and other exceedingly expensive therapies in the cancer care armamentarium.

Patients Line Up—To Wait

Kymriah is indicated for patients up to age 25 who have relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) and, as of May 1, is also indicated for adult patients with relapsed or refractory B-ALL.1,2 Yescarta is approved to treat adult patients with relapsed or refractory large B-cell lymphoma.3

“We do see that there has been a limited number of patients treated [with CAR Ts],” said Jeff Liepman, Executive Vice President at Promidian, during a recent OBR webinar: “Challenging Dynamics in Immuno-oncology.”4 About 60 sites are certified and active in using CAR T-cell therapies, but the adoption has been “protracted.” Some of the reasons for this, Liepman said, include geography constraints for patients, reimbursement considerations, and new codes and processes for payers. As a result, there are long wait lists, ranging from 50 to 100 patients at certain cancer treatment sites.

Access and Attitudes Emerge

Currently, CAR T-cell therapy administration consists of a combination of outpatient and inpatient procedures and drugs, but the CAR T-cell infusion itself is done in the inpatient setting for most patients.5

“I can’t claim to have an extensive knowledge of what’s happening across the country,” said Caron Jacobson, MD, Medical Director of the Immune Effector Cell Therapy program at Dana-Farber/Brigham and Women’s Cancer Center, “but I’m not aware of any center that’s giving outpatient CAR T-cell yet for commercial products.”

At Dana-Farber, adult patients are admitted into the hospital the night before the CAR T-cell infusion, and once infusion is completed, patients are required to stay for a minimum of seven days to monitor for side effects. Patients can stay longer if they have ongoing toxicities.

Children at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center also receive CAR T-cell infusion inpatient, but are hospitalized for an average of 30 days.6

Commercial and federal payers are approving use of CAR T-cell therapies, but only on a single user agreement basis, which requires payers to review each patient case before granting coverage.

“I’ve been impressed with the payers because they understand the importance of time in this instance and they’re working diligently, in my opinion,” said Dr. Jacobson. For patients who are eligible to receive a CAR T-cell therapy, time is essential, as these patients are very sick and may die while waiting for the drug to be manufactured or if there’s a long wait list. “There’s not a patient yet that we have identified that we have been unable to treat,” she noted.

Because Kymriah and Yescarta are available only at authorized treatment centers, some patients may have to travel out-of-state or a long distance to access care, which can result in delays.

Amy Emmert, MScPH, Vice President, Hematopoietic Stem Cell Transplantation and Cell Therapies at Dana-Farber Cancer Center, said there’s been “a little bit” of slowness to pick up coverage among patients who come to Dana-Farber from out-of-state Medicaid programs. On the other hand, commercial payers have been “quite supportive.”

Ms. Emmert explained that once an out-of-state Medicaid program or commercial insurer gets through a single user agreement, “they’re much quicker to move forward with patients the second time around.”

Patient-related Costs

Patient costs and hospital reimbursement rates have slowly begun to surface, and Ms. Emmert provided insights into cost and reimbursement.

“We don’t know where these prices are going to go long-term once there are more products on the market, but for right now, Medicare is covering these charges from hospitals and patients are not experiencing large out-of-pocket,” said Ms. Emmert. However, she said, “It’s not as black and white as you might think in terms of what patients will be required to cover; it depends on what they’re buying into.”

Patients receive additional procedures, like leukapheresis, and drugs, such as chemotherapy before CAR T-cell infusion or tocilizumab to manage cytokine release syndrome. The cost of these procedures and drugs can range from $30,000 to $36,000 for the average patient, according to a recent study, bringing the mean total cost of treatment with Kymriah or Yescarta to $432,000 or $402,000, respectively.7 However, as reported in Medscape, these are conservative estimates and additional costs are much higher, in the realm of $400,000 to $450,000, which effectively doubles the total cost of therapy to about $1 million.8

Also, expenses outside of the hospital setting, such as travel, time off from work, and housing might pose a financial burden to patients.

Following the CAR T-cell therapy hospitalization at Dana-Farber, patients stay within a two-hour radius of the medical center. For some patients, that may require up to three weeks in temporary housing after discharge. Resources are available to help patients cover these costs.

Novartis has a patient support system where they cover hotel and travel, as well as some out-of-pocket expenses associated with copays. “It seems to be a very generous program,” said Ms. Emmert.

Gilead has a different approach. According to Ms. Emmert, Gilead attempts to help find resources for patients and each hospital also identifies resources. For example, “Dana-Farber has a subsidized housing program for patients who need to stay locally, or free depending on income,” she said.

Hospital Reimbursement

The Centers for Medicare and Medicaid Services (CMS) has initiated statements on how it will reimburse hospitals for CAR T-cell administration.

Effective April 1, 2018, a CMS spokesperson told OBR, the agency is capping Medicare beneficiaries’ copayments for Kymriah and Yescarta in the outpatient setting at $1,340. CMS also set the hospital reimbursement rate at about $400,000 for Yescarta and $500,000 for Kymriah in the outpatient setting; however, CAR T-cell therapies are typically administered in the inpatient setting, so those rates may not apply.

The 2019 Medicare Inpatient Prospective Payment System proposed rule, which takes effect on October 1, 2018, proposes assigning ICD-10-PCS procedure codes XW033C3 and XW043C3 for CAR T-cell therapies to an existing code (MS-DRG 016), but they are also considering the idea of creating a new code.9

Entities like the American Society for Blood and Marrow Transplantation (ASBMT) have been advocating for CAR T-cell reimbursement updates.5 Stephanie Farnia, Director of Health Policy and Strategic Relations of ASBMT, said CMS has responded to their requests to create more specificity around which MS-DRG will be paid when a CAR T-cell product is delivered in the inpatient setting, but that there are concerns around the MS-DRG 016 code, in particular, that ASBMT is analyzing to better understand it.

She said, “The clarity that CMS provided regarding the ICD-10 codes is helpful and welcome – providers now know that they can use the procedure code created last year at Kite’s request for both CAR T-cell products.”

CMS has also put plans in motion to provide additional reimbursement guidance over the next year by opening a National Coverage Analysis for CAR T-cell therapies. The National Coverage Analysis aims to develop a consistent national coverage policy for Medicare beneficiaries by May of 2019; a proposed decision is expected by February 16, 2019. To accomplish this analysis, the evidence on CAR T-cell therapies will be reviewed on August 22, 2018, during a Medicare Evidence Development and Coverage Advisory Committee meeting.10

Spotlight Shines on New Payment Model

Novartis announced last year that they would collaborate with CMS to provide Kymriah via an outcomes-based payment arrangement.11

The outcomes-based agreement for Kymriah stipulates that payment is only required for patients who respond to Kymriah by the end of the first month and that “other value-based approaches related to future indications for Kymriah and CAR T-cell therapies are under discussion,” according to the press release.11

Adams Dudley, MD, MBA, Director of the Center for Healthcare Value at the University of California, San Francisco, thought that Novartis’ outcomes-based agreement may not be as good as it sounds.

“We do lots of things that you don’t know whether they’re going to work, and you don’t want to necessarily set up context where if it doesn’t work, there isn’t payment and have that become a standard thing,” he said. To him, “it feels like a ploy to be able to say we only get paid if we save lives, that’s why we charge so much money.”

Throughout the healthcare system, reimbursement is undoubtedly shifting from fee-for-service to value-based payments, but how value-based payments are setup can vary. Payments can be based on the population or individual patient level.

“I don’t see a strong rationale for moving it to the individual,” said Dr. Dudley. He thinks payment based on a specific patient’s outcome “introduces an enormous bonus for claiming that the patient has had a benefit, whether they have or not.” For example, he explained, a physician with a relationship with a drug company would know the company only gets paid if the physician says the drug works for a specific patient—which means the physician has an incentive to argue the therapy worked. In contrast, if payment is based on evidence for population-level effectiveness, such as traditional studies, there’s not the same incentive to claim the therapy worked.

For Gilead, a spokesperson told OBR that although they don’t have any outcomes-based agreements, there has been varying degrees of interest and ability to execute these types of arrangements with payers.

Sustainability Called into Question

According to an Institute for Clinical and Economic Review (ICER) March report, it was determined that both Kymriah and Yescarta were cost-effective at their wholesale acquisition costs of $475,000 and $373,000, respectively; however, only 38% of the estimated 5,900 eligible patients could be treated at Yescarta’s current price in a given year before crossing the budget threshold.12

As a result, ICER issued an Affordability and Access Alert for Yescarta noting that “the added health care costs associated with a new treatment may be difficult for the system to absorb over the short term without displacing other needed services or contributing to unsustainable growth in health care insurance costs.”12

Dr. Dudley commented on ICER’s economic assessment of Yescarta, saying, “It is just a difficult calculation we have to make as a society about whether this [therapy] that appears promising is priced at a level that makes it implementable.” He felt prices are set at what the market will bear and that prices for these innovative therapies are climbing at an unsustainable rate. “Even if we could pay for everyone for this therapy, we couldn’t pay that amount of money for everyone who has cancer,” he said.

As a result, he thought, hospitals won’t want to be put in the position of having to decide who gets treatment or not. “[It’s] not at all a traditional doctor or hospital role to say, ‘No, society has run out of money for you; therefore, even though I know what you need, and I’ve got it, I can’t give it to you,’” he said. “That’s not a role that doctors or nurses have ever had to play before and if that’s how this plays out, there will be a very big backlash from the profession.”

Editor’s Note: For an in-depth discussion of the issues shaping the access, adoption and reimbursement of the new immuno-oncology agents for patients, providers and payers, listen to a replay of OBR’s recent webinar, “Challenging Dynamics in Immuno-Oncology,” at http://obroncology.com/webinar/challenging-dynamics-in-immuno-oncology-access-reimbursement-and-provider-perspectives/.

 References

  1. FDA.gov. FDA approval brings first gene therapy to the United States. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.html. Accessed April 25, 2018.
  2. FDA.gov. FDA approves tisagenlecleucel for adults with relapsed or refractory large B-cell lymphoma. https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm606540.html. Accessed June 14, 2018.
  3. FDA.gov. FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm581216.html. Accessed April 25, 2018.
  4. OBR webinar. Challenging dynamics in immuno-oncology: Access, reimbursement, and provider perspectives. April 25, 2018. http://obroncology.com/webinar/challenging-dynamics-in-immuno-oncology-access-reimbursement-and-provider-perspectives
  5. ASBMT. Request for new MS-DRGs for CAR-T therapy for FY 2019. https://higherlogicdownload.s3.amazonaws.com/ASBMT/43a1f41f-55cb-4c97-9e78-c03e867db505/UploadedImages/ASBMT_Request__FY2019_New_CAR-T_MS-DRGs_.pdf. Accessed April 25, 2018.
  6. Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. Kymriah™ CAR T-cell therapy. http://www.danafarberbostonchildrens.org/uploadedfiles/content/page_content/innovative_approaches/gene_therapy/kymriah-car-t-cell-therapy-faq.pdf. Accessed April 25, 2018.
  7. Hernandez I, Prasad V, and Gellad W. Total costs of chimeric antigen receptor T-cell immunotherapy. JAMA Oncol. April 26, 2018 [Epub ahead of print].
  8. Mulcahy N. What’s the total cost of one CAR T-cell treatment? Medscape. https://www.medscape.com/viewarticle/895735. Accessed April 27, 2018.
  9. Centers for Medicare & Medicaid Services. Fiscal Year (FY) 2019 Medicare hospital inpatient prospective payment system (IPPS) and long term acute care hospital (LTCH) prospective payment system proposed rule, and request for information. https://s3.amazonaws.com/public-inspection.federalregister.gov/2018-08705.pdf. Accessed April 25, 2018.
  10. Novartis. Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah™ (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice. https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019. Accessed April 25, 2018.
  11. Centers for Medicare and Medicaid Services. National Coverage Analysis (NCA) tracking sheet for chimeric antigen receptor (CAR) T-cell therapy for cancers (CAG-00451N). https://www.cms.gov/medicare-coverage-database/details/nca-tracking-sheet.aspx?NCAId=291. Accessed June 14, 2018.
  12. Institute for Clinical and Economic Review. A look at CAR-T therapies. March 2018. https://icer-review.org/wp-content/uploads/2018/03/ICER_CAR-T_RAAG_032318.pdf. Accessed April 25, 2018.

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