Rare disease
A rigorous approach
to value generation
Rare disease
Rare diseases present unique challenges. Small patient populations, fragmented evidence, evolving standards of care, and complex patient journeys can make demonstrating value difficult
HEOR helps organisations navigate these challenges by combining clinical, biostatistical, HEOR, and market access expertise within a single multidisciplinary team.
We generate evidence where data are scarce, develop robust value frameworks, and translate scientific innovation into compelling value propositions that support patient access worldwide
One partner –
Multiple perspectives
Rare disease development requires clinical, scientific, and commercial considerations to be aligned from the outset
Our experts work as a coordinated team, providing strategic guidance, technical expertise, and operational support across the product lifecycle. By connecting stakeholders, aligning evidence generation activities, and maintaining a consistent value strategy, we help organisations navigate complexity with confidence
Building access
into development from the start
We work with development teams, clinicians, and patient communities to ensure trial design, endpoint selection, and evidence generation plans address the needs of regulators, HTA bodies, and payers
By integrating access considerations early, we help maximise the future value of every evidence-generation activity
Supporting
global access strategies
Healthcare systems differ, but the need for a clear and credible value story is universal
HEOR supports clients across global, regional, and local markets, helping them navigate the expectations of HTA agencies, payers, and reimbursement bodies worldwide. By connecting global strategy with local requirements, we help maximise the reach and impact of rare disease innovations
Modelling for
complex evidence environments
Our team develops transparent, HTA-ready models designed specifically for rare disease settings where traditional approaches may be insufficient
From evidence synthesis and comparative effectiveness analyses to innovative modelling techniques that address uncertainty, we create rigorous frameworks that support confident decision-making and withstand external scrutiny
Clinically grounded
evidence and insight
Limited published evidence is a reality in many rare diseases.
Through structured engagement with clinicians, patient advocates, and specialist centres, we build a deeper understanding of disease burden, unmet need, and real-world patient experiences
These insights strengthen economic models, value assessments, and reimbursement submissions, ensuring that value stories reflect both scientific evidence and clinical reality
Why HEOR
- Integrated clinical, biostatistical, HEOR, and market access expertise
- Early incorporation of payer and HTA perspectives
- Deep experience addressing evidence gaps and uncertainty
- Clinically grounded insights from expert and patient engagement
- Robust, HTA-ready modelling and value demonstration approaches
- Global delivery informed by local market understanding
One multidisciplinary partner
From first-in-human to reimbursement
Across every market that matters
rare disease services
De novo value framework development
Multidisciplinary strategic partnership
Early-stage HEOR and HTA integration
Regulatory and payer alignment
Clinically grounded evidence generation
Advanced modelling and analytics
Global market access strategy
Collaborative, challenge-led delivery