Gene-Therapy Access and Cost

Why cost is part of the conversation

The U.S. Centers for Medicare & Medicaid Services (CMS) states that cell and gene therapies can transform the lives of people with rare and severe diseases but that access is difficult because they can cost millions of dollars. [1] CMS also says the high cost makes it difficult for state Medicaid agencies to pay for them. [1] MedlinePlus Genetics lists, among ethical questions about gene therapy, whether high costs will make it available only to the wealthy. [2]

What independent reviews say about value

Two examples concern gene therapies for sickle cell disease. They are modeling studies, not measurements of real-world outcomes.

• ICER. The Institute for Clinical and Economic Review (ICER), an independent group, reported in its 2023 assessment that despite uncertainty about longer-term outcomes, its appraisal committee voted that the evidence was adequate to show a net health benefit over standard of care. It estimated that the two therapies would meet common cost-effectiveness thresholds if priced between $1.35 million and $2.05 million. [3]
• Published model. A two-model analysis in the Annals of Internal Medicine, funded by the National Heart, Lung, and Blood Institute, assumed a $2 million price and estimated cost-effectiveness ratios of $193,000 and $427,000 per quality-adjusted life-year (health-care perspective). Results were sensitive to the cost of chemotherapy before gene therapy, caregiver quality of life and long-term survival, and the short-term effect on pain crises was extrapolated from one study. [4]

These numbers describe analyses at stated or assumed prices. They are not current list prices, and this page does not give prices because none were read from a regulator or manufacturer page.

Access barriers beyond price

CMS describes patients receiving gene therapy for sickle cell disease as needing an extended inpatient hospital stay and potentially multiple additional visits to a gene therapy center. [1] The preparation involves myeloablative chemotherapy, which CMS says typically renders patients infertile, so its model requires participating manufacturers to pay for a defined scope of fertility preservation services. [1] Other barriers named in the sources:

• Travel and lodging. CMS names support for travel expenses and lodging related to fertility preservation services. [1]
• Insurance rules. The CMS model includes standardized prior authorization criteria for eligible patients. [1]
• Evidence gaps. Cost-effectiveness depends on durability, which is still being followed. See off-target effects, durability and long-term monitoring.

A US example of a payment model

CMS describes the Cell and Gene Therapy (CGT) Access Model as a voluntary, multi-year model in which CMS negotiates outcomes-based agreements with manufacturers on behalf of state Medicaid agencies, with pricing discounts and outcomes-based rebates. [1] Its initial focus is sickle cell disease. [1] The page lists 34 state participants and 2 manufacturer participants, and participation is voluntary for both. [1] Eligibility requires, among other things, a documented diagnosis and Medicaid as the primary payer in a participating state. [1] Whether it improves access is being tested, and the model page frames it as a test. [1]

This is a United States program. Rules in other countries were not read for this page.

Cost and access are not evidence of effectiveness

A high price does not show a treatment works, and availability through a clinic does not show approval. The FDA warns that unapproved regenerative medicine products are broadly marketed, that people charged for such products outside a clinical trial are likely being offered a product illegally, and that listing on ClinicalTrials.gov does not mean a product is legally marketed. [5] See unapproved clinics and medical tourism for cell therapies.

Questions people ask their care team

• Is the product approved for my exact condition and age?
• What does the insurer cover, and what are the prior authorization steps?
• What costs fall outside the product itself (hospital stay, travel, fertility preservation)?
• Is a registered clinical trial or an expanded access route an option? See expanded access.

Related: approved gene and RNA therapies and rare-disease gene therapy research.