Ensuring Value: Our Health Economics & Market Access (HEOR & MA) Strategy

Translating Innovation into Access in the Rare Disease Space

In the world of rare diseases, innovation alone is not enough. To truly make an impact, life-changing therapies must also be accessible, affordable, and sustainable. At Aegerion Pharmaceuticals UK, our Health Economics and Market Access (HEOR & MA) strategy ensures that the value of our therapies is clearly demonstrated—and that patients across the UK can access them without unnecessary delay or burden.

We operate at the intersection of science, policy, and economics, transforming advanced medicines into meaningful solutions for patients and health systems alike.

Why Access Requires Strategy in Rare Disease

Rare diseases often involve:

• High unmet medical need

• Small, dispersed patient populations

• Specialist care requirements

• Significant long-term burden on healthcare systems

Therapies developed for these conditions can be high-cost but high-impact. That’s why it’s essential to demonstrate their value in real-world terms—clinical, economic, and societal.

Aegerion’s Approach to Health Economics & Market Access

Our HEOR & MA function is focused on removing barriers to access while aligning with UK health system priorities and budget constraints.

1. Evidence-Driven Value Demonstration

We build robust, localised value dossiers that clearly communicate:

• Clinical effectiveness and safety

• Improvement in patient quality of life

• Cost-effectiveness vs. current standard of care

• Impact on healthcare resource utilization

These models are tailored to UK-specific payer frameworks and supported by both clinical trial data and real-world evidence (RWE).

2. Strategic HTA and Payer Engagement

Aegerion engages early and constructively with:

• NICE, SMC, and All Wales Medicines Strategy Group (AWMSG)

• Integrated Care Boards (ICBs) and NHS procurement teams

• Private healthcare and insurance groups

Our goal is to align on shared outcomes and create sustainable access models through:

• Health Technology Assessment (HTA) submissions

• Budget impact and affordability modeling

• Risk-sharing and outcome-based agreements where applicable

3. Real-World Evidence Generation

We recognize the importance of post-launch data, especially in rare conditions. We invest in:

• Patient registries and long-term follow-up studies

• Observational and burden-of-illness research

• Collaborations with clinical centers and NHS digital data platforms

This data not only supports value reassessment but also helps improve care pathways and long-term outcomes.

4. Early Access and Innovative Funding Solutions

For therapies addressing urgent unmet needs, we support:

• Early Access to Medicines Schemes (EAMS)

• Compassionate use and named patient supply

• Collaboration with NHS England for specialised commissioning

We adapt to evolving NHS structures and funding mechanisms to reduce time to access.

Aligning with National and NHS Priorities

Our HEOR & Market Access strategy is built to support:

• The UK Rare Diseases Framework

• NHS priorities around health equity, patient outcomes, and innovation adoption

• ABPI-compliant, transparent pricing strategies

We believe in fair pricing and responsible access solutions that reflect both the value of innovation and the realities of public healthcare delivery.

Collaborating for Impact

We work cross-functionally with:

• Medical Affairs to integrate real-world insights

• Regulatory Affairs to ensure data readiness

• Patient advocacy groups to incorporate lived experiences

• Policy makers and HTA experts to refine value arguments

This collaboration ensures a patient-centric, evidence-based approach to every pricing and access conversation.

From Value to Access—With Patients at the Center

At Aegerion Pharmaceuticals UK, we understand that true value is realized only when a therapy is available to the people who need it most.

Through transparent dialogue, rigorous data, and adaptive access models, our HEOR & Market Access team ensures that innovation leads to impact, equity, and sustainable care.