Acknowledging the Challenges in Gene Therapy: Perspectives on ICER's White Paper

Today marks the release of the Institute for Clinical and Economic Review (ICER) and the NEW Drug Development ParadIGmS (NEWDIGS) at Tufts Medical Center’s white paper addressing the expanding gene therapy market.1 It is projected that by 2030, there will be more than 60 cell and gene therapies and indications approved in the United States,2 approximately 10-20 per year.3 This paper outlines a handful of complexities facing healthcare payers as new gene therapies enter the market, a challenge that is as intricate as it is vital. We appreciate ICER/NEWDIGS’ engagement in this conversation and their discussion of the potential advantages and key barriers related to gene therapy payment. With this NPC publication, we note ongoing concerns and recommendations on framing, scope, and proposed payment methods that were not addressed through the authors’ white paper.

Gene Therapy Value, Access, and Innovation Requires Multi-Stakeholder Solutions

The white paper acknowledges that not all payment methods will suit all gene therapies or all payers. As the authors put it, “None of these tools are ‘silver bullets’ that can singlehandedly solve all the barriers and tensions inherent in the current healthcare insurance and payment landscape.”1 This is a critical understanding as we advance into an era of personalized medicine, where one-size-fits-all solutions fall short.4 5

Moreover, the complexity of one-time treatments that promise long-term, potentially curative outcomes introduces significant challenges within the U.S. health insurance model. Particularly, 'beneficiary churn'—where members frequently switch insurers—risks creating misaligned incentives if churn is not bidirectional.5 Insurers, especially small payers and self-insured employers, may face the upfront costs of these transformative treatments without reaping the long-term financial benefits, underscoring the need for innovative approaches to health insurance and payment systems that accommodate the unique nature of gene therapies.

We commend the authors’ support for early dialogue between manufacturers and payers. Proactive communication is a cornerstone of sustainable innovation, especially for innovative payment methods. It can ensure that as new therapies come to market, they do so with a framework for access and reimbursement that aligns with their clinical impact. We are also happy to see ICER/NEWDIGS’ recommendations for policy reforms that advance value-based purchasing arrangements, as this is one promising avenue for promoting patient access to innovative therapies.6

A Focus on Payer Challenges and Solutions Is Only Part of the Story

However, any discussion of gene therapy payment methods must go hand in hand with discussions of value. While the authors describe several broad challenges associated with payment for gene therapies, it is nearly devoid of any meaningful lessons learned from the 12 gene therapies that have been approved by the FDA in the last eight years.7 8 A more comprehensive review of the current landscape, highlighting what has worked in the past, what has failed, and how patients have been affected, would have deepened the value of the white paper.  

NPC has previously shared concerns related to the framing and scope of ICER/NEWDIGS’ analysis that were not addressed in this paper, including:   

• Narrowly focusing on the payer perspective. The white paper exclusively describes payment and reimbursement challenges from the payer perspective and neglects to commensurately explore challenges facing other stakeholders, including patients, provider groups, and manufacturers. 

• Focusing on “fair pricing” vs. “fair value.” The white paper's lack of discussion on "value assessment" points to a concerning focus on cost containment rather than evaluating and delivering value to patients and the healthcare system. The paper’s emphasis on establishing a "fair price" simplifies the nuanced challenge of assessing the value of innovative therapies at launch, which should consider diverse patient segments and payer populations. 

• Relying on several assumptions and hypothetical assertions that aren’t borne out by empirical evidence or real-world market conditions. The white paper relies on unsupported assertions, such as the exaggerated economic surplus for gene therapy producers compared to traditional treatments and a premature suggestion that gene therapies will not encounter competition, painting a scenario that seems to demand precipitous changes in value assessment. This narrow view fails to account for recent market trends, including the slow uptake of gene therapies for conditions like hemophilia9 and the notable risks for manufacturers.10  

• Excluding an exploration of real-world applications and market conditions. By not acknowledging the lessons from several years of gene therapy availability in the U.S. market, the white paper misses the chance to leverage established practices for access and reimbursement.  

• Relying on unsupported methods such as ICER’s “shared savings.” The white paper continues to advance ICER’s shared savings methods despite public pushback and lack of supporting evidence.11 12 13  Reliance on unsupported methods weakens the authors’ proposed strategies and their applicability to the unique challenges and potentials of gene therapies. 

Moving Toward a Meaningful Discourse on Value That Extends Beyond Mere Pricing 

For gene therapies, NPC believes that a meaningful discourse on value—extending beyond mere pricing—is paramount. Gene therapies have the potential to deliver groundbreaking improvements in patient health outcomes. Yet, realizing this potential is contingent upon recognizing the treatments' value and dismantling the structural and financial barriers that curtail meaningful patient access.14 This aligns with our guiding practices that emphasize value assessment as a nuanced tool—not a rule—for setting drug prices, ensuring they reflect the full spectrum of benefits provided to patients, caregivers, healthcare systems, and society.15

Ultimately, the white paper identifies critical challenges related to paying for gene therapies and advocates for innovative pricing and payment solutions to address concerns about financial sustainability. NPC has often said that we are living in a golden age of biopharmaceutical innovation and need as much innovation in how we pay for medicines as we see in new medicines. However, any policy discussion about payment must equally consider the value of these treatment options and ensure that these therapeutic advances reach all individuals who can benefit. Moving forward, it is imperative that we collectively strive for a valuation framework that is equitable, comprehensive, and sensitive to the innovative nature of gene therapies, thereby guaranteeing that their full potential is realized and made available to those in need.  

Authors: Tyler D. Wagner, Lisabeth Buelt, Jon D. Campbell, Kimberly Westrich. 

The authors thank Robert J. Nordyke for his insights and contributions to the writing of this article.  

• 1 a b Phares S, Trusheim M, Emond SK, Pearson SD. Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform. 2024. April 23, 2024.
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• 3Gottlieb S. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. FDA. Updated January 19, 2019. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics
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• 6Ciarametaro M, Graff JS. RE: Medicaid Program; Establishing Minimum Standards in Medicaid State Drug Utilization Review (DUR) and Supporting Value-Based Purchasing (VBP) for Drugs Covered in Medicaid, Revising Medicaid Drug Rebate and Third-Party Liability (TPL) Requirements. In: Centers for Medicare & Medicaid Services, editor. Washington, D.C.2020.
• 7NEWDIGS. Approved cell and gene therapy (CGT) products. Tufts Medicine Tufts Medical Center. Accessed April 5, 2024. https://newdigs.tuftsmedicalcenter.org/payingforcures/defining-disruption/cell-and-gene-therapy-products-and-pipeline/approved-cell-and-gene-therapy-products/
• 8The ICER/NEWDIGS white paper states that 17 gene therapies have been approved by the FDA in less than seven years, but the NEWDIGS website lists 12 FDA-approved gene therapies since 2017. 
• 9Beasley D. Uptake of new hemophilia gene therapies slow as field assesses options. Reuters. Updated December 15, 2023. https://www.reuters.com/business/healthcare-pharmaceuticals/uptake-new-hemophilia-gene-therapies-slow-field-assesses-options-2023-12-15/ 
• 10Shah-Neville W. Cell and gene therapy manufacturing: trends, advancements and challenges. Labiotech. Updated July 27, 2023. https://www.labiotech.eu/in-depth/cell-and-gene-therapy-manufacturing/#:~:text=Scalability%3A%20a%20key%20challenge,both%20time%2Dconsuming%20and%20expensive. 
• 11Westrich K. Why is ICER Charging Ahead with It’s Flawed “Shared Savings” Approach? LinkedIn. Updated February 1, 2024. https://www.linkedin.com/pulse/why-icer-charging-ahead-its-flawed-shared-savings-kimberly-westrich-rkbre/ 
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• 13Cencora. Valuing potential cures: An examination of ICER’s approach to assessing high-impact single and short-term therapies. 2023. October 2023. https://www.amerisourcebergen.com/-/media/assets/amerisourcebergen/phrma-sst-issue-brief-xf.pdf
• 14Abrahams E, Wells CJ. Personalized medicine: We’re not there yet. STAT News. April 5, 2024. https://www.statnews.com/2024/04/05/personalized-medicine-not-here-yet/ 
• 15National Pharmaceutical Council. Guiding Practices for Patient-Centered Value Assessment. 2024. https://www.npcnow.org/sites/default/files/2024-01/2024%20Guiding%20Practices%20for%20Patient-Centered%20Value%20Assessment%20January.pdf