Making Rare Disease Treatments Economically Sustainable
Access to rare disease treatments depends on proving their clinical value and economic impact. Our Health Economics & Outcomes Research (HEOR) and Market Access teams work directly with national reimbursement agencies (e.g., NICE in the UK) to develop compelling value arguments for our therapies.
Health Economics & Market Access
We provide:
Health technology assessment (HTA) dossiers and cost-effectiveness models
Engagement strategies for payers, commissioners, and pricing authorities
Real-world evidence generation to support long-term clinical and financial value
Collaboration with patient advocacy groups to support policy change
We advocate for sustainable pricing and reimbursement models that ensure patients receive care — and health systems receive value.
Health Economics & Market Access
Demonstrating Value. Enabling Access. Supporting Sustainability.
At Aegerion Pharmaceuticals UK, our Health Economics and Market Access (HEOR & MA) team plays a pivotal role in ensuring that life-changing therapies for rare diseases reach the patients who need them—equitably, affordably, and sustainably.
We work across the healthcare ecosystem to deliver compelling, data-driven value propositions for our therapies—balancing clinical innovation with the economic realities of modern healthcare systems.
Our Approach: Value Beyond the Molecule
Orphan drugs often serve small, underserved patient populations, but come with significant innovation, clinical benefits, and transformative outcomes. Our HEOR & Market Access strategy focuses on translating that value into actionable, reimbursable solutions.
1. Evidence-Based Value Demonstration
We build robust, locally relevant value dossiers that reflect:
Clinical outcomes and quality-of-life gains
Burden of illness and unmet medical need
Cost-effectiveness and budget impact modelling
Real-world evidence (RWE) and post-market data to support long-term value
2. Payer & HTA Engagement
We maintain open and proactive relationships with:
NHS England and devolved UK nations
Health Technology Assessment (HTA) bodies such as NICE and the Scottish Medicines Consortium (SMC)
Private insurers and integrated care systems (ICS)
Our team works early in the product lifecycle to align with assessment criteria and submit fit-for-purpose evidence packages that address clinical benefit, affordability, and societal impact.
3. Innovative Access Solutions
Given the complexity of rare diseases and orphan drug pricing, we work with healthcare systems to implement:
Value-based agreements
Managed access programs
Risk-sharing or outcome-based reimbursement models
Early access or named patient supply mechanisms, where appropriate
4. Local Market Adaptation
While we operate as part of a global organization, our UK HEOR & MA team brings deep understanding of local policy frameworks, ensuring:
Tailored pricing strategies aligned with UK health priorities
Compliance with ABPI guidelines and NHS frameworks
Strategic alignment with national rare disease plans and access equity goals
Collaborating Across Stakeholders
We work closely with internal teams and external partners, including:
Medical Affairs for real-world data generation
Regulatory Affairs to align clinical outcomes with HTA needs
Patient advocacy groups to reflect lived experiences in economic models
Healthcare professionals and KOLs to validate burden and treatment pathways
By bridging science, policy, and patient perspectives, we create a unified narrative that supports both access and long-term adoption.
Delivering Sustainable Access to Rare Disease Innovation
At Aegerion Pharmaceuticals UK, our HEOR & Market Access efforts are driven by a simple goal: to ensure that no patient is denied access to effective treatment due to economic barriers.
By investing in health outcomes research, engaging with payers early, and adapting to the needs of local health systems, we deliver rare disease solutions that are as sustainable as they are transformative.
To learn more about our access strategy or discuss potential collaborations, please contact us.
Public Sector & Government Partnerships
Collaborating with Health Authorities to Serve Public Needs
Aegerion Pharmaceuticals UK is an experienced and trusted government partner, supplying vital orphan drugs through public tenders, framework agreements, and named-patient supply programs. Our work with NHS Trusts and other health bodies reflects our commitment to public health priorities and access equity.
