Ultra-High-Cost Gene Therapies Set to Influence Major Pharmacy Shifts

The advent of “ultra-high-cost” treatments like gene and cell therapies will create exciting new therapies for patients, but will also likely create economic, equity, and workforce issues for health system pharmacies, a new report warns. The 2025 American Society of Health-System Pharmacists (ASHP)/ASHP Pharmacy Forecast and its foundation urges health systems to carefully prepare for the emerging new era.1

To date, the FDA has approved 21 gene and cell therapies for cancer, blood disorders, and retinal disease, among others. That number is expected to climb significantly in future years. While such therapies can lead to dramatic improvements for patients, their price tags are also dramatic—the most expensive gene therapies cost more than $3.5 million, the report notes.

Novel, ultra-high-cost gene and cell therapies are set to have a major influence on pharmaceutical systems | image credit: Vink Fan - stock.adobe.come

“The groundbreaking therapies and technologies that are the most challenging to our healthcare system, like gene therapies and generative artificial intelligence, also provide promising, curative opportunities,” said ASHP CEO Paul W. Abramowitz, PharmD, ScD, in a press release.2

The report highlights 3 particular challenges associated with gene and cell therapies: finding ways to pay for them, ensuring patients can access them, and preparing health system pharmacies to handle and manage the treatments.1

Right now, only 0.8% of total pharmaceutical spending in the US is spent on gene and cell therapies, but the report notes that the percentage is likely to increase. A 2023 analysis taking into account current drug company pipelines and the prevalence of the diseases targeted by new therapies suggested that spending on gene therapy could rise to $25.3 billion in 2026 before leveling off to $21.0 billion by 2034.3 About half of that spending will be non-Medicare spending.

The ASHP report notes that payment for gene therapies is risky for insurers, since the benefits of the often-one-time treatments—including avoided healthcare spending—could be realized by another insurer if the patient switches insurance providers. Moreover, the category of gene therapy is so new that the long-term, real-world impacts of particular therapies remain unclear.1

One possible solution is outcomes-based payment arrangements,

“For example, starting in 2025, state Medicaid programs can participate in the Cell and Gene Therapy Access Model through which the Centers for Medicare and Medicaid Services will develop outcomes-based agreements with manufacturers of gene and cell therapies for sickle cell disease, a disease that disproportionately affects Medicaid enrollees,” the report notes.

However, only 11% of the respondents to ASHP’s forecast panelist survey said such programs were “very likely” to happen.

Subscription-based models, stop-loss insurance to help smaller insurers, and a national reinsurance program may also help the problem, the report said.

Even if the therapies can be paid for, not all health systems have the equipment and expertise to handle them, the report notes.

When survey respondents were asked to forecast whether they would be prepared for a world in which pharmacies were in charge of managing cell and gene therapies for health systems, fully half of respondents said they would consider their health systems “very unprepared” (18%) or “somewhat unprepared” (32%).

Health systems may be able to partner with manufacturers to expand access, but the report cautions that a number of details, such as who will receive compensation, and how compensation will be handled if outcomes are not achieved, should be sorted out in advance.

In terms of staffing, the forecast panelists said they expect Medicare—or perhaps manufacturers themselves—will require a pharmacist’s involvement in the management of gene and cell therapies.

“Pharmacists are poised to play a key role, working alongside other healthcare professionals, to ensure new advances are integrated smoothly and accessibly into health systems for the benefit of all patients,” said Joseph T. DiPiro, PharmD, the report’s editor, in the press release.2

Playing that role, the report states, will require health systems to build appropriate infrastructure, facilities, and policies.

References:

1. DiPiro JT, Tichy E, Shea S, et al. ASHP Foundation Pharmacy Forecast 2025: Strategic Planning Guidance for Pharmacy Departments in Hospitals and Health Systems. Am J Health Syst Pharm.2024;, zxae280. doi.org:10.1093/ajhp/zxae280
2. Multi-Million Drug Therapy Innovations Bring Challenges to Healthcare. ASHP. News Release. December 9, 2024. Accessed December 9, 2024. https://www.prnewswire.com/news-releases/multi-million-drug-therapy-innovations-bring-challenges-to-healthcare-302326264.html
3. Wong CH, Li D, Wang N, Gruber J, Lo AW, Conti RM. The estimated annual financial impact of gene therapy in the United States. Gene Ther. 2023;30(10-11):761-773. doi:10.1038/s41434-023-00419-9