Actionable Insights: Enabling Market Access As CGTs Reach More Patients
Cell and gene therapies (CGTs) are revolutionizing the treatment of complex diseases with the promise of potentially curative outcomes. To fully realize this potential, CGT manufacturers must proactively address key market access hurdles.
Success hinges on strategies such as enhancing payer understanding for informed coverage decisions, adapting pricing and reimbursement models, and evolving treatment delivery towards outpatient settings to improve accessibility and reduce costs. Ultimately, comprehensive education across all stakeholders is crucial for seamless integration of CGTs into standard clinical practice.
The following recommendations offer actionable insights to navigate these market access challenges within the U.S. healthcare landscape.
Increasing payer awareness
A significant challenge in ensuring market access for CGTs lies in increasing payer awareness and facilitating well-informed coverage decisions. This is especially crucial when innovative CGTs enter therapeutic areas already served by established treatments, requiring them to compete for attention and reimbursement.
Payers are tasked with navigating the complexities of CGT evaluation, balancing potentially substantial upfront costs with the prospect of long-term benefits and potential cures. Achieving this necessitates a comprehensive understanding of the unique clinical and economic value proposition of CGTs compared to existing options. Adding to this complexity, payers are indicating a trend toward more rigorous review for CGTs entering markets where similar therapies are already approved, potentially creating obstacles to coverage as they strive to discern therapeutic advantages.
Recommendation
Manufacturers are advised to prioritize early and consistent engagement with payers through appropriate pre-approval communication. This communication should focus on delivering compelling evidence demonstrating the clinical safety, efficacy, and economic value of their CGTs. Post-FDA approval, a proactive communication strategy should be deployed to specifically address potential hurdles to access, such as cost considerations, the long-term durability of the therapy, the intricacies of patient and product logistics, and challenges related to patient portability across state lines for treatment administration.
By actively enhancing payer understanding and providing robust data to inform their decisions, manufacturers can pave a more effective pathway towards widespread coverage and the successful incorporation of CGTs into routine clinical practice.
Innovative pricing and reimbursement models
With a growing number of CGTs now targeting larger patient populations, either due to disease prevalence or earlier positioning in the treatment pathway, payers are increasingly focused on the potential budgetary impact. As these therapies become applicable to a broader range of patients and earlier lines of treatment, the financial implications for payers' portfolios are becoming a significant concern, driving the urgent need for innovative pricing and reimbursement models.
The substantial upfront costs associated with CGTs necessitate the development of creative financial solutions that ensure both affordability and accessibility for patients while preserving the economic sustainability for manufacturers.
Recommendation
To facilitate broader access to CGTs in earlier treatment lines, manufacturers should clearly articulate the economic value proposition alongside clinical efficacy. Demonstrating how earlier intervention can lead to significant cost savings by reducing or preventing chronic comorbidities and treatment failures is essential. Simultaneously, manufacturers should actively partner with healthcare providers and payers to design integrated care pathways that optimize administration and monitoring, thereby mitigating logistical challenges.
These strategies will not only enhance the accessibility of CGTs but also maximize their impact on patient outcomes. Moreover, collaborating with payers on innovative pricing models, substantiated by comprehensive data on long-term benefits and cost-effectiveness, is key to achieving wider adoption and seamless integration into standard healthcare practice.
Transitioning treatment models
Transitioning CGT administration from inpatient to outpatient settings presents both challenges and opportunities for patients, healthcare providers, and payers, including:
From a patient perspective, the shift could negatively impact patient out-of-pocket costs because outpatient services often involve different cost-sharing arrangements than inpatient care. Payers may need to help address these disparities to prevent financial barriers to access.
From a provider perspective, this transition requires adaptations in infrastructure, training, and patient management protocols. The standard of Centers of Excellence will likely evolve from mostly academic institutions to include community and office settings. This leads to different logistical and administrative considerations, as well as reimbursement structures for the providers and facilities.
Payers may implement stricter utilization management protocols, such as prior authorization or step therapy, to control access and ensure appropriate use. These measures aim to manage costs while maintaining quality care. Additionally, payers will increasingly rely on real-world data to assess long-term outcomes and effectiveness. This data is vital for maintaining flexible and informed coverage decisions as therapies evolve.
This evolution in treatment models is driven by therapeutic innovation leading to strong safety profiles, the need to improve patient convenience, a push to reduce healthcare costs, and the common desire to expand patient access
Recommendation
Manufacturers must proactively anticipate the evolution of payer management strategies aimed at balancing cost, access, and quality outcomes. To this end, manufacturers should be forward-looking and actively support the necessary education, infrastructure, and logistical frameworks, particularly concerning the specialized storage and administration requirements of CGTs.
This includes strategic partnerships with specialized treatment centers and comprehensive training programs for healthcare professionals to effectively manage the unique aspects of these therapies. Furthermore, providing clarity on the reimbursement pathway is essential for manufacturers to offer appropriate support, recognizing that outpatient treatments often necessitate different payment structures compared to traditional inpatient care.
While this transition holds the promise of enhanced accessibility and affordability for a broader patient population, it also requires diligent navigation of regulatory, operational, and economic hurdles by all stakeholders to ensure successful implementation and equitable access.
References:
- As of 27 February 2025, Cencora has supported 27 CGTs. 33 gene therapies have been approved (including genetically modified cell therapies), as reported by the American Society of Cell and Gene Therapy (ASCGT). Gene, Cell, & RNA Therapy Landscape Report: Q4 2024 Quarterly Data Report. 1 January 2025. Accessed on 29 April 2025. Available online at: https://www.asgct.org/global/ documents/asgct-citeline-q4-2024-report.aspx
- IPG Health. “Million-dollar medicine: Insights on payer management of cell and gene therapies (CGTs).” 11 September 2024. Accessed 29 April 2025. Available online at: https://ipghealth.com/news/million-dollar-medicineinsights-on-payer-management-of-cell-and-genetherapies-cgts
- Allowable actions related to pre-approval information exchange (PIE) are set by a biopharmaceutical manufacturer’s compliance guidelines within the bounds set by H.R.2617 - Consolidated Appropriations Act, 2023 and the FDA guidance on PIE, “Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities,” June 2016. Available online at: https://www. fda.gov/media/133620/download.