Insights & Articles

Rare Disease Therapies and the Case for Outcomes-Based Agreements

Treatments for rare diseases, such as spinal muscular atrophy or CAR-T therapies like tisagenlecleucel, hold transformative potential for patients. Yet, they often come with significant challenges—uncertainties around long-term efficacy, high costs, and the need for tailored patient selection. Outcomes-Based Agreements (OBAs) offer a structured way to address these challenges, aligning financial risk with therapeutic outcomes. However, their implementation requires careful consideration and planning.

The Promise and Practicalities of OBAs

1. What Makes OBAs Valuable?

OBAs shift the focus from upfront costs to real-world outcomes, creating a more sustainable framework for funding innovative therapies. They enable:

Risk Sharing: Payers and manufacturers align costs with actual therapeutic results.

Patient-Centric Focus: Treatments are tied to measurable improvements, emphasizing value rather than volume.

Increased Access: By mitigating cost risks, OBAs can support the introduction of high-cost therapies in resource-constrained settings.

2. Implementation Challenges

Despite their promise, OBAs are not without hurdles:

Administrative Complexity: Managing OBA agreements involves data sharing, contract monitoring, and performance assessments—all requiring robust systems.

Data Availability and Quality: Real-world evidence is critical, but gaps in data collection, reporting, and standardization can limit success.

Stakeholder Collaboration: Successful OBAs require alignment between payers, manufacturers, and healthcare providers. Misaligned priorities or unclear accountability can derail agreements.

How Lyfegen Supports OBA Implementation

Learning from Global Examples

Lyfegen’s Agreements Library—featuring 6,700 public agreements and 20 pricing models from 33 countries—offers invaluable insights into how OBAs have been implemented worldwide. By analyzing these examples, stakeholders can identify models that best suit their unique challenges, reducing the trial-and-error phase of implementation.

Streamlined Scenario Analysis

The Lyfegen Drug Contracting Simulator enables stakeholders to simulate OBA scenarios using real-world data. From adherence-based contracts to outcome guarantees, the Simulator helps users:

• Assess feasibility through scenario modeling.

• Forecast financial implications with real-world inputs.

• Compare multiple pricing models to find the most suitable solution.

Simplifying Administration

Managing the administrative burden of OBAs is crucial. Lyfegen’s tools offer:

• Centralized contract management for version control and compliance tracking.

• Automated data processing to ensure performance metrics are accurately reported.

• Detailed dashboards and trend reports to facilitate collaborative decision-making.

Key Considerations for OBA Success

1. Feasibility Studies Are Essential

Not every therapy or market is suited for OBAs. Conducting thorough feasibility assessments helps determine the viability of such agreements.

2. Data Plans Need Clarity

Reliable outcomes-based contracts depend on well-defined metrics and data collection processes. Establishing these frameworks early is crucial.

3. Commitment from All Stakeholders

OBAs thrive on collaboration. Shared goals, transparent communication, and clear accountability among all parties can ensure smoother execution.

Conclusion

Outcomes-Based Agreements represent an important step forward in addressing the challenges of high-cost, high-impact therapies for rare diseases. With the right tools, insights, and preparation, healthcare stakeholders can unlock the potential of OBAs to improve access, manage costs, and focus on patient outcomes.

Discover how Lyfegen can simplify your journey to outcomes-based contracting. Schedule a demo today to explore our solutions in action.

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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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A Bright New Chapter in UK Healthcare: How AI-Driven Reform Will Transform Drug Pricing and Access

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.

A New Dawn for NHS England

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.

What’s Changing in the UK’s Healthcare System?

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:

  • Investing in AI & Digital Tools – Automating processes to enhance efficiency and decision-making.
  • Hiring More Digital Experts – Training 2,000 new tech apprentices by 2030, with 10% of civil servants working in tech roles.
  • Reducing Administrative Waste – Freeing up time and funds by using AI to handle repetitive tasks, allowing healthcare professionals to focus on patient outcomes.

The Impact on Drug Prices and Healthcare Access

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:

  • Lower Drug Prices – Reduced bureaucracy means more direct resources for securing better pricing.
  • Stronger Negotiating Power – Direct ministerial oversight can drive sustained cost reductions rather than short-term fixes.
  • Faster Access to Medicines – Savings from automation and efficiency gains can be reinvested into reducing wait times and improving treatment availability.
  • Enhanced Value-Based Care – AI tools will optimize drug pricing strategies, ensuring maximum rebates and cost efficiency.
  • Short-Term Disruptions, Long-Term Gains – The transition may temporarily affect drug supply and pricing, but AI-driven analytics will help stabilize and reduce costs in the long run.

The AI Revolution: Powering Efficiency and Innovation

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:

  • Optimized Negotiations – With NHS England now under ministerial control, data-driven pricing will be crucial. Lyfegen’s Agreements Library can benchmark UK drug prices against global agreements, ensuring smarter, fairer negotiations.
  • Automated Contracting – Our AI-powered platform streamlines drug contract creation and management, reducing paperwork and making negotiations faster and more efficient.
  • Real-Time Pricing Simulations – Before finalizing agreements, Lyfegen runs real-time simulations to test different pricing scenarios, identifying the most financially and operationally beneficial outcomes.
  • Capturing Hidden Savings – By automating drug rebate management, Lyfegen detects missed savings, ensuring that every possible dollar is recovered and reinvested into patient care.

Challenges to Watch

While AI promises to revolutionize healthcare efficiency, successful implementation will require overcoming hurdles such as:

  • Data Integration – Ensuring AI systems can seamlessly access and analyze NHS data.
  • Change Management – Encouraging widespread adoption of digital tools among healthcare professionals.
  • Regulatory Compliance – Navigating evolving policies around AI-driven decision-making in healthcare.

A Bright Future for Lyfegen and the Healthcare Sector

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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