ASHP Midyear: High Costs of Gene Therapies Present Major Challenges to Implementation

Although gene therapies have incredible potential for health outcomes and could have long-term savings, a report presented at the American Society of Health-System Pharmacists (ASHP) 2024 Midyear Clinical Meeting demonstrate that their substantial up-front costs are a major hurdle.¹

Gene therapies work by altering part of the patient’s genome by replacing, deleting, or inserting genetic material. With this approach, these therapies could treat or even cure diseases that previously had few, if any, options. However, researchers have consistently found that the high costs of these drugs present challenges.² Currently, the FDA has approved 21 gene and cell therapies for conditions such as cancer, blood disorders, leukodystrophies, muscular dystrophies, and retinal disease. Many more are in development.³

Hand holding a strand of DNA | Image credit: © neirfy | stock.adobe.com

In research findings published in Gene Therapy, investigators developed an original simulation model that identified the 109 late-stage gene therapy clinical trials that were underway prior to January 2020, estimated the prevalence and incidence of their corresponding diseases, applied a model of the increase in quality-adjusted life years for each therapy, and simulated the launch prices and anticipated spending of all available gene therapies annually. The findings showed that annual spending on gene therapies will be approximately $20.4 billion, with conservative assumptions.²

The investigators also noted high costs on the individual patient level. For example, voretigene neparvovec (Luxturna; Spark Therapeutics) is indicated to treat Leber congenital amaurosis, a rare inherited form of vision loss. At launch, it was priced at $425,000 per eye. Similarly, onasemnogene abeparvovec-xioi (Zolgensma; Novartis) is indicated for children younger than 2 years with spinal muscular dystrophy and was priced at $2.1 million per patient.²

These therapies also require specialized management for safe, effective, and equitable access, according to the ASHP Pharmacy Forecast. Pharmacists can play a vital role here, with the report demonstrating that 75% of survey participants expect pharmacists to oversee these therapies and 58% expect Medicare will require pharmacist involvement.³

“We are witnessing major innovations in health care, and strong collaboration is essential,” said Joseph T. DiPiro, PharmD, editor of the Forecast, in a news release. “Pharmacists are poised to play a key role, working alongside other health care professionals, to ensure new advances are integrated smoothly and accessibly into health systems for the benefit of all patients.”¹

The report presented noted that current spending on gene and cell therapies in the United States is just 0.8% of total pharmaceutical spending, but that is expected to grow. Approximately half of the spending on gene therapies is expected to occur in commercial insurance and Medicaid because some treatments are specifically for younger patients.³

Alternative and sustainable payment models may be beneficial. The report notes that gene and cell therapies are already being paid for using outcomes-based payment models, although only 11% of the pharmacy leaders surveyed believed that outcomes-based payments were very likely. Other potential models could include subscription-based models and stop-loss insurance for smaller health plans, in which employers can pay an additional per-member fee to ensure access.³

Finally, the report included some strategic recommendations for pharmacy leaders to prepare and access these treatments. Recommendations include the following³:

• Determine whether the health system has financial resources, service lines, and clinical expertise to provide these therapies.
• Establish relationships with state Medicaid programs to determine whether their state plans to participate in the Cell and Gene Therapy Access Model, and how their state plans to provide access to these medications.
• Partner with manufacturers to become a preferred site of administration. Health systems with investigational drug programs can also proactively work with manufacturers to ensure ongoing access.
• Pharmacists should work to ensure that they are involved to work with organizational revenue cycle and contracting teams to determine the potential full costs of an episode of care with cell and gene therapies.
• Pharmacy leaders should work with C-suite administrators to prepare for financial variances and lagging reimbursements.
• Pharmacy leaders across health systems should develop training programs for pharmacists and technicians to ensure they have the knowledge and skills to manage and administer these therapies.

The high cost of gene therapies presents a significant hurdle for both patients and health care systems. Although these treatments offer unprecedented potential for curing diseases, the costs raise concerns about equitable access and long-term sustainability. Innovative payment models such as outcomes-based contracts and subscription models may be crucial to ensure these life-changing therapies reach patients who need them. Additionally, pharmacists will play a vital role in managing these complex treatments, ensuring their safe and effective use while advocating for patient access and affordability.

“The groundbreaking therapies and technologies that are the most challenging to our health care system, like gene therapies and generative artificial intelligence, also provide promising, curative opportunities,” said ASHP CEO Paul W. Abramowitz, PharmD, ScD (Hon), FASHP, in the news release. “This year’s Pharmacy Forecast focuses on the future and rethinking how we deliver care, moving beyond traditional models to concentrate on health outcomes that align with patients’ individual goals.”¹

REFERENCES

1. Multi-million drug therapy innovations bring challenges to healthcare. News release. American Society of Health-System Pharmacists. December 9, 2024. Accessed December 11, 2024. https://news.ashp.org/News/ashp-news/2024/12/09/multi-million-drug-therapy-innovations-bring-challenges-to-healthcare

2. 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 Therapy. 2023;30:761-773. doi:10.1038/s41434-023-00419-9

3. DiPiro JT, Hoffman JM, Tichy E, et al. ASHP and ASHP Foundation Pharmacy Forecast 2025: Strategic planning guidance for pharmacy departments in hospitals and health systems. Am J Health-Syst Pharm. 2024;zxae280. doi:10.1093/ajhp/zxae280