Solving the Cell & Gene Therapy Access Puzzle in the U.S.

Published January 10, 2025

Cell and gene therapies (CGTs) have ushered in a new era of medicine, offering patients transformative, even potentially curative solutions, with a single treatment. Despite the clinical promise, barriers to patient access remain given administration complexity, gaps in patient outcome evidence and high costs. As cell and gene therapies become available in new indications beyond oncology and rare pediatric diseases, developers of innovative therapies need to develop strategies targeting patients, providers and payers to overcome these challenges and promote access to cell and gene therapies in the U.S.

As we kick-off 2025, our team has summarized a few key issues relating to U.S. access from the patient, provider and payer perspectives, and outlined high-level trends for developers of innovative cell and gene therapy products to consider.

Treatments Are Mostly at Academic Centers, but Patients Are Not

As a result of complexities associated with administration, handling, monitoring and training requirements, cell and gene therapies today are primarily offered by a small number of academic medical centers. A major issue for patients therefore is geographical distance from treatment. It’s estimated that nearly 50% of patients live more than 60 minutes away from a designated treatment center. Beyond the cost of travel, accommodations and time off work, the patient also must be away from their home and family during treatment and build trust with a new care team. It is not surprising that patients hesitate in committing to a cell and gene therapy, especially when other options are available (e.g., Hemlibra for hemophilia).

A potential solution to this dilemma is to bring treatment closer to patients by shifting its administration out of academic centers and into community sites, creating a better-distributed network with fewer geographic gaps. Bringing non-specialized centers into the fold starts with developing cell and gene therapy technology to make administration and follow-up less complex, and generating evidence in support of the safety of the new treatment/process to alleviate any clinical concerns providers might have. Beyond technology improvements, manufacturers will also need to work with regulators to streamline onboarding/accreditation requirements, and work with payers to ensure these sites are integrated into their networks to address any financial concerns small providers might have (e.g., insufficient reimbursement).

Providers Need to Grow Capacity to Address Patient Volume

In addition to geographical challenges, the administrative burden placed on providers by payers can further limit or delay patient access. For gene therapies in particular, payers often require extensive documentation, including genetic testing, prior therapy records, lab work, fitness status, etc. These pre-authorization (PA) requirements, while intended to ensure that only eligible patients are treated, can be cumbersome for healthcare providers to manage.

Aligned with the goal of bringing therapies closer to where patients are, developers should invest into efforts that can lower barriers for non-academic sites to administer cell and gene therapies. As development progresses, manufacturers should engage institutions early on to understand needs and expectations and tailor a distribution provider support strategy accordingly. Provider support should be comprehensive in addressing infrastructural, training and administrative barriers to access, flattening the learning curve for speedy adoption. For treatments that will have to be administered in a specialized center, engagement can also focus on removing any barriers to referral of patients to appropriate facilities and raising awareness among specialists in new spaces who will be prescribing cell and gene therapies.

Gaps in Coverage

Today, U.S. payer organizations generally provide robust coverage for cell and gene therapies, albeit with significant requirements for submission of documentation, but there is a concern growing as more of this class of agents come to market. In a recent sign of this concern, Independence BCBS (PA) announced a policy starting in 2025, which will delay coverage for any products approved through FDA’s Accelerated Approval pathway by 18 months, as the manufacturer develops further evidence. It is unclear whether this policy will expand beyond a single organization, but if it does, cell and gene therapies would surely be one of the primary classes that will face restrictions.

Further adding to coverage gaps, employers have been reported to exclude cell and gene therapies from their benefit plans, which can leave a substantial portion of patients with no access. Medicaid policies are also shown to be more likely to have coverage restrictions compared to commercial insurance, with 68.4% of Medicaid policies imposing some form of restriction compared to 53.5% for commercial policies.

Thankfully, there are also positive developments that aim to balance these emerging gaps in coverage. For employers worried about the cost of cell and gene therapies, some third party plans provide coverage for these expensive therapies at set PMPM rates (e.g., Embarc, CVS) by creating broad risk-sharing pools. To manage high costs, payers and pharmaceutical manufacturers are also increasingly engaging in agreements designed to mitigate risk and make cell and gene therapies more accessible. Complexity and fragmentation of the U.S. system makes these agreements more challenging, but involvement of third parties facilitates operationalization. The recent steps taken by the CMS to establish the Cell and Gene Therapy Access Model will leverage similar outcome-based agreements to close gaps for Medicaid patients.

As our overview might imply, the manufacturers of cell and gene therapies will need to navigate an ever-expanding universe of payers and related stakeholders to manage potential gaps in coverage. Ahead of engaging with employers, traditional commercial and governmental payer organizations and other involved third parties, it is critical for manufacturers to clearly articulate what value their products bring for each stakeholder type and develop potential agreement frameworks that reinforce value proposition.

Conclusion: Moving Toward Greater Access and Coverage Consistency

The landscape of cell and gene therapy coverage remains fragmented and uncertain. Inconsistent policies across states, employers and insurers continue to create challenges for patients, healthcare providers and manufacturers alike. To improve access, it is essential that policymakers, insurers and pharmaceutical manufacturers work together to create more transparent and consistent coverage mechanisms with broad networks and expanded patient support to lessen the burden for patients.

To summarize, during development of innovative cell and gene therapies, manufacturers should:

• Articulate evidence-supported value propositions tailored for each stakeholder type including payers, physicians and provider organization decision-makers to communicate the benefits and differentiation of the product
• Take steps to expand administration network beyond specialized centers by improving safety/administration process and developing evidence to ease provider clinical concerns
• Identify potential community/non-academic providers early on and engage to understand and resolve barriers at every step from onboarding and capacity development to final reimbursement
• Design a distribution strategy and engage partners (e.g., specialty pharmacies/specialty distributors) that can meet the needs of a broader network 
• Evaluate innovative payment models and development agreement frameworks that can alleviate payer concerns of affordability and value for money

Ultimately, the future of cell and gene therapies lies in addressing the financial, logistical and policy-related barriers that hinder patient access. By creating a more consistent, transparent, and accessible framework for cell and gene therapy coverage, we can ensure that the promise of these groundbreaking therapies becomes a reality for a wider group of patients.

Trinity Life Sciences’ experts, under the umbrella of our Cell and Gene Therapy CoE, support cell and gene therapy developers in developing strategies to achieve their goals and optimize product potential, tailored to their target disease spaces, geographies and products. If you would like to discuss any topics outlined here in further detail, you can reach us at info@trinitylifesciences.com.

References:

https://avalere.com/insights/socioeconomic-factors-may-impact-patient-access-to-cell-therapies#:~:text=Patients%20requiring%20treatment%20with%20innovative,%2Dup%2C%20and%20care%20coordination

https://www.sciencedirect.com/science/article/pii/S2590229623000151

https://www.genengnews.com/topics/bioprocessing/2025-cell-gene-therapy-reimbursement-outlook/

https://global.lockton.com/us/en/news-insights/gene-therapy-or-what-it-means-for-employers

https://www.cms.gov/priorities/innovation/innovation-models/cgt

Authors

Helen Amata, Alexis Bryan, Kate Balicki and Ismail Ismailoglu