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Gene and Cell Therapies Hold Potential—but How Can Payers Manage Their Costs?


Presenters at the Academy of Managed Care Pharmacy (AMCP) 2024 annual meeting discussed the current promise and future potential of gene and cell therapies, as well as payer management strategies for these costly treatments.

Gene and cell therapies have been popular topics over the years at the Academy of Managed Care Pharmacy (AMCP) annual meeting. At this year’s event, presenters took the stage on April 17 to discuss the current state and future direction of gene and cell therapy management, as well as contextualize these treatments beyond the realm of value-based contracting (VBC).

Close Up Cellular View | image credit: pinkeyes - stock.adobe.com

Close Up Cellular View | image credit: pinkeyes - stock.adobe.com

Gene and cell therapies are frequently used and described together; however, these interventions are not the same. Yugian Liu, senior director of Specialty Clinical Solutions, Prime Therapeutics and Magellan Rx, began by describing the differences between the 2 treatments.

Gene therapy aims to alter human genetic composition to effectively change defective genes, produce the correct proteins, and ultimately have a curative impact on patients. Cell therapy refers to the manipulation of cells that are then infused back into the body with the capacity to target and diminish other cellular malignancies. Additionally, cell therapy can involve replenishing the human body with healthy cells to promote better functioning.

“The ultimate take home message,” Liu said, “is that potentially these are one-time dose treatments that provide a curative solution for our patients.”

To date, there are 13 approved gene therapy products on the market. When the first treatment, voretigene neparvovec-rzyl (Luxturna), was introduced for biallelic RPE65 mutation-associated retinal dystrophy, Liu noted how shocking its price tag—$850,000 per injection—was to the managed care world.1 Fast-forward to the recent approval of atidarsagene autotemcel (Lenmeldy; Orchard Therapeutics) this past March, which is indicated for the treatment of metachromatic leukodystrophy and costs $4.25 million. “We have great medicine coming to the market. However, the prices of these treatments are skyrocketing. And that's the message for why managed care should and needs to care about this topic,” Liu said.

As medical technologies have advanced, they have enabled the consistent development of these therapies. At present, Liu added, the pipeline for gene therapy currently has 70 agents in phase 2 development, 22 in phase 3, and 5 with scheduled FDA review dates this year1—and these numbers can change monthly.

“When you look at it, none of these [scheduled] gene therapies are for the mass population. They're all for ultra-rare diseases. But combined, if you think about how many of these are on the market and how many are to come, it absolutely will impact every health plan.”

Liu continued by noting the 10 approved cell therapies on the market, which are largely indicated in oncology,2 although cell therapies in general are on pace to expand into other areas.1 Gene therapies, however, are meant to target specific pieces of a gene. This aspect makes expanding to other areas difficult, unless that part of the gene is indicated in various clinical expressions (such as β-thalassemia sickle cell, Liu added).

Evidence-based medicine is invaluable for guiding decisions in patient care, which is why managed care professionals should care about the data surrounding gene and cell therapy, Liu said. Their durability—how long the effects of these products can be sustained—is of utmost importance, he added.

Liu then presented current data in the field demonstrating the durability of various therapies. She pointed to a study on onasemnogene abeparvovec (Zolgensma; Novartis), which is a gene therapy indicated in the treatment of spinal muscular atrophy (SMA). In the 7-year follow-up study that includes 10 patients, 3 notably achieved new milestones of standing with assistance3—“something that’s completely unthinkable historically for this population.” Additionally, all these patients were free from permanent ventilation, were able to eat orally, and maintained their milestone achievements. Six of these patients also had risdiplam or nusinersen as an add-on therapy.

Data for axicabtagene ciloleucel (Yescarta; Kite Pharmaceuticals), a cell therapy indicated in the treatment of large B-cell lymphoma, were also presented. In a 5-year analysis, 31% of patients were exhibiting an ongoing response to treatment at year 5, and duration of complete response was a median of 62.2 months. Overall survival (OS) rates in treated patients reached 42.6%, with over 64% achieving complete remission.4

Liu noted data from additional gene and cell therapy studies that demonstrate the efficacy, durability, and great potential of these treatments across conditions such as hemophilia, bladder cancer, and various forms of lymphoma.

As Liu concluded her presentation, she stressed the need to keep patient impact in mind among considerations of costs and available data.

“These are life-changing therapies,” Liu said. “What we need help with is understanding who we can give treatments to to maximize their clinical outcome. How can we sustain a healthcare model with these costs of therapies continuously to increase fast and furiously at the end of the day? What does that all mean for our patients?”

Andy Killpack, PharmD, director of specialty clinical solutions at Prime Therapeutics, followed these questions by discussing payer management and operation strategies to finance gene and cell therapies. His talk largely explored the pros and cons of various approaches in this area.

Of the various potential strategies, he noted prior authorization as one of the most common methods to ensure the right patients receive the correct treatments, which can influence intended outcomes. However, a study from 2021 revealed that only 67% of health plans were utilizing prior authorization in their management of gene and cell therapeutics.5 While the process of prior authorization is improving, Killpack said, many providers cite its added administrative burden as a challenge to delivering care.

Outcomes-based agreements are another method of payer management and operation. Essentially, this approach aligns payments by or to manufacturers with pre-determined, post-treatment outcomes in mind. Should those outcomes fail to be reached, then forms of reimbursement or discounts on future payments are given to payers.6 While this type of agreement can protect payers from the financial risks of covering a therapy with limited long-term data, Killpack pointed to the challenge of negotiating power for payers when it comes to outcome measurements or definitions of treatment success, as well as the challenge of streamlining and financing ways to gather outcomes data in this approach.6, 7

Outcomes-based product solutions are another potential avenue. A major benefit with this solution is that the health plan will not need to harbor the contract. Instead, a vendor would hold it for them and keep a health plan’s legal team from needing to involve themselves in the agreements. Additionally, this route allows for the pooling of risks from other health plans to increase patient populations and let payers better predict the financial outlooks related to treatments.8

Patient tracking and portability generally present a big challenge because, as Killpack explained, “health plans will oftentimes see a member enroll, get treatment, and then leave their plan within the next year or two, therefore leaving the plan without the ability to follow up and execute on a value-based agreement because that patient is no longer with their health plan.” An outcomes-based product solution, he argued, may be able to aid in this area and keep track of patients moving between plans.

Killpack then elaborated on the use of reinsurance and stop-loss coverage. These solutions are used by small and large payers alike, he said, and can aid in covering part of high-cost claims.6 This can prevent payers from exceeding their budgets in cases where their members need high-cost therapies.7 Yet, there are multiple weaknesses to this strategy, including increasingly costly premiums and that it may not be a great long-term solution given the plethora of gene and cell therapies that are approaching the market that could drive premiums up further.9

High-cost drug amortization, which allows a payer to pay off drug costs to a manufacturer over the course of several years, constitutes another potential solution. This strategy may benefit a payer as they can save on costs while paying off high-cost, curative treatments,6 but if a member changes their health plan during this course, the payer could lose out on these gains before they are seen.8

With these considerations, Killpack emphasized that payers should always consider the additional costs and services that might arise during cell or gene therapies. This may include patients who need to make additional travel accommodations to reach nearby treatment centers, the compilation of lab fees, caregiver burden, and more.

As Killpack brought the talk to a close, he focused on a case study regarding an individual diagnosed with Leber congenital amaurosis, a retinal dystrophy the severely impacts one’s vision, at a very young age. She was told she would end up completely blind, but quickly after the administration of the approved gene therapy voretigene neparvovec-rzl (Luxturna), this individual saw improvements in their sight, which helped them stay committed to school, eventually graduate, and maintain their independence in adulthood.10

With stories like this and data in support of therapies like these, Killpack concluded, “we need to keep that in mind as we're looking at policies and creating criteria and looking at the costs and solutions to manage the high-cost therapies . . . that there's patients that will benefit. So what we need to do as managed care professionals, both from the payer side and manufacturer standpoint, is [ask] how can we come together to develop solutions models that make sense for both parties to make sure that there's access for these patients who are going to be benefiting from these drugs?”


1. Life-Cycle Insights. US Clinical Development [IPD Analytics]. 2024. hhtps://secure.ipdanalytics.com/User/Pharma/RxStrategy

2. CodeSource. US Clinical Development [IPD Analytics]. 2024. hhtps://secure.ipdanalytics.com/User/Pharma/RxStrategy

3. Mendell J, Wigderson M, Alecu I, Yang L, Mehl L, Connolly A. Long-term follow-up of onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1. J Neuromuscu Dis. 2023;33(1):S91. doi:10.1016/j.nmd.2023.07.105

4. Nastoupil LJ, Jain MD, Feng L, et al. Standard-of-care axicabtagene ciloleucel for relapsed or refractory large B-cell lymphoma: Results from the US lymphoma CAR T consortium. J Clin Oncol. 2020;38(27):3119-3128. doi:10.1200/JCO.19.02104

5. Myshko D. Study: Patients face access restrictions for cell and gene therapies. Managed Healthcare Executives. November 10, 2021. Accessed April 18, 2024. https://www.managedhealthcareexecutive.com/view/study-patients-face-access-restrictions-for-cell-and-gene-therapies

6. Slocomb T, Werner M, Valluri S, et al. New payment and financing models for curative regenerative medicines. Alliance for regenerative Medicine. July 2017. Accessed April 18, 2024. https://alliancerm.org/wp-content/uploads/2018/05/ARM_Curative_Regenerative_IV1707_LRS.pdf

7. Marsden G, Towse A, Pearson SD, et al. Gene therapy: Understanding the science, assessing the evidence, and paying for value. Institute for Clinic and Economic Review. March 2017. Accessed April 18, 2024. https://icer.org/wp-content/uploads/2020/11/ICER-Gene-Therapy-White-Paper-030317.pdf

8. Emerging market solutions for financing and reimbursement of durable cell and gene therapies. Financing and Reimbursement of Cures in the US. June 16, 2021. Accessed April 18, 2024. https://newdigs.tuftsmedicalcenter.org/wp-content/uploads/2022/06/FoCUS-WP-CGT-Market-Solutions.pdf

9. Kleinke JD, McGee N. Breaking the bank: Three financing models for addressing the drug innovation cost crisis. Am Health Drug Benefits. 2015;8(3):118-26

10. Salzman R, Cook F, Hunt T, et al. Addressing the value of gene therapy and enhancing patient access to transformative treatments. Mol Ther. 2018;26(12):2717-2726. doi:10.1016/j.ymthe.2018.10.017

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