Gene Therapies: Negotiating the Priceless-Insights from the Lyfegen 2024 Drug Contracting Trends Report
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Insights & Articles
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Newly approved gene therapies for hemophilia A and B, beta thalassemia and sickle cell disease come with multimillion dollar price tags that could bankrupt financially strapped state Medicaid programs. In many instances, the majority of patients affected by these diseases rely on Medicaid for healthcare coverage.
Last month, the federal government unveiled a novel “access model” - the Cell and Gene Therapy Access Model- designed to mitigate the costs state Medicaid programs incur when they pay for these potentially curative treatments and allow for patient access. The announcement by the Centers for Medicare and Medicaid Services marks a significant step towards addressing the access issue.
Similar to what is currently happening with a select group of prescription drugs under the Inflation Reduction Act, CMS plans to negotiate prices of these gene therapies with drug makers at the national level for all state Medicaid that decide to participate in the model. By facilitating access to potentially life-changing treatments and supporting outcomes-based agreements with manufacturers, the model has the potential to improve health outcomes and alleviate financial burdens on state Medicaid agencies.
At first, CMS intends to negotiate pricing and rebates with the drug manufacturers Vertex and bluebird bio over the next few months on behalf of state Medicaid agencies that voluntarily choose to join the program.
Related Post: Sickle cell disease gene therapies are here, but how is society going to pay for them?
Bluebird bio says it has already established an outcomes-based agreement for its SCD therapy, Lyfgenia, with commercial insurers. The product is listed at $3.1 million. The company did not divulge further details. Vertex, which priced its SCD therapy, Casgevy, at $2.2 million, has declined to discuss its plans.
The fact sheet that CMS provides contains few details as to what kinds of pricing arrangements would be put in place for products such as Lyfgenia and Casgevy. Prior to deciding to participate in the model and allow CMS to negotiate prices on their behalf, Medicaid agency directors will want to know specifically what kinds of outcomes-based agreements CMS can turn to.
The Lyfegen Model & Agreements Library offers CMS and state Medicaid agencies access to one central resource with more than 4,000 public pricing agreements and 20 innovative pricing models, including outcomes-based agreements. This invaluable resource has all the market research in one place for stakeholders to gather intelligence on novel ways to establish innovative payment models that are uniquely designed to suit the needs of Medicaid payers, CMS, and drug makers alike. To further enhance the practicality of these models, our value-based contracting platform mitigates the high costs and administrative challenges involved in managing these contracts. It equips CMS, the States, and Pharma with a streamlined, cost-effective means to implement outcome-based agreements efficiently.
Medicaid directors will also seek information on methods of evidence gathering, outcomes measurement, the length of time needed to test durability of gene therapies and how this information will be provided to CMS, presumably via a patient registry. Lyfegen offers solutions to identify the right drug pricing agreements in which evidence generation and the use of patient registries are key.
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With price tags in the millions, gene therapies are redefining medicine—and reshaping how we negotiate access to it. For both payers and pharmaceutical companies, these breakthrough treatments present a shared challenge: how do you fund what feels priceless?
From Zolgensma to Hemgenix, gene therapies promise one-time cures for rare and life-threatening diseases. But the financial model behind them can’t follow the traditional playbook. These treatments call for a smarter, more collaborative approach to pricing—and that’s exactly what’s taking root.
Why Payers and Pharma Need a New Playbook
Unlike conventional drugs, gene therapies frontload their cost while delivering benefits over time. That disconnect forces a fundamental rethink of how pricing, reimbursement, and risk-sharing are handled.
According to the Lyfegen 2024 Drug Contracting Trends Report, health systems worldwide are moving toward innovative agreements: outcome guarantees, installment plans, and subscription-based models. These aren’t just experiments—they’re becoming essential tools to balance patient access with financial responsibility.
For payers, it’s about managing risk while maintaining equity. For pharma, it’s about demonstrating value in a way that aligns with clinical reality. Either way, the direction is clear: shared risk, shared benefit.
Global Shifts That Are Shaping the Market
The trends are global and accelerating. In the United States, payers like Blue Cross Blue Shield and Medicaid are embracing outcome-based models for sickle cell gene therapies like Casgevy and Lyfgenia. Brazil’s Ministry of Health uses installment payments for Zolgensma, spreading risk over five years while tying reimbursement to real-world outcomes.
In Europe, countries like Spain and Italy combine restricted coverage with annual reassessments, ensuring that high-cost therapies are only reimbursed if they continue to deliver results.
The message? Pricing innovation is no longer a nice-to-have—it’s the only way forward.
How Lyfegen Bridges the Gap
At Lyfegen, we help payers and pharma move beyond the negotiation table—and into action.
• Our Agreements Library, the world’s largest digital repository of value-based contracts, helps you understand what others are doing and where the benchmarks lie.
• Our pricing simulation engine lets both sides explore scenarios before committing—making deals smarter from day one.
• And our automated platform handles everything from contract setup to rebate tracking, saving time, reducing risk, and driving transparency.
A Smarter Way to Fund the Future of Medicine
Gene therapies will continue to challenge the limits of what we think healthcare can afford. But with the right models and tools, both payers and pharma can find common ground—ensuring that innovation reaches the patients who need it most.
Curious about what’s next in drug contracting?
Download the 2024 Drug Contracting Trends Report for exclusive insights, real-world examples, and global benchmarks.
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The UK government is taking a bold step toward modernizing public services by cutting red tape, integrating AI into operations, and bringing NHS England back under direct ministerial control. This reform signals a shift toward efficiency, innovation, and better patient care—one where AI-driven solutions like Lyfegen can play a pivotal role.
NHS England was originally established in 2012 as an arm’s-length organization to insulate the health service from political interference. Over time, however, bureaucracy accumulated, slowing decision-making and increasing costs. With Starmer’s decision to fold NHS England’s functions back into the Department of Health and Social Care (DHSC), the system is poised for a fresh start. This restructuring aims to eliminate redundant roles, reduce administrative waste, and reallocate resources to frontline care—ushering in a new era of efficient and accountable healthcare management.
A key takeaway from Starmer’s announcement is his strong push for automation. The government is aiming to cut administrative costs by 25%, ensuring that resources are directed where they matter most: patient care.
Some of the expected changes include:
By integrating NHS England’s functions into the DHSC, the government is positioned to strengthen and streamline negotiations with pharmaceutical companies. This shift could lead to:
One of the most promising aspects of this reform is the government’s commitment to leveraging AI to transform operations. For an AI-powered platform like Lyfegen, this presents a significant opportunity to deliver real-world benefits in healthcare management. Here’s how Lyfegen can help:
While AI promises to revolutionize healthcare efficiency, successful implementation will require overcoming hurdles such as:
Transforming the NHS is no small task. Beyond balancing innovation and cost, the government must manage vast amounts of healthcare data and navigate the complexities of implementing change at scale. However, Starmer’s announcement represents more than just another cycle of NHS reforms—it’s a meaningful step toward a future where efficiency and technology-driven innovation deliver real, lasting benefits to patients.
At Lyfegen, we’re ready to support this transformation by delivering AI-powered solutions that drive real savings and faster patient access. Let’s build a smarter, more efficient NHS together.
Want to see how our AI-powered solutions can support smarter drug pricing and better healthcare access? Let’s schedule a demo today.
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