In the early stages of development, a lack of comprehensive data can be a major hurdle in the rare disease sector. We help you to:

• Synthesise information on the natural history of the disease, unmet needs, and patient burden to identify missing data.
• Create early economic models to explore pricing thresholds and assess the potential viability of pipeline candidates.
• Integrate the patient voice from the outset to ensure that outcomes and quality-of-life measures are relevant and meaningful to patients and their families.
• Conduct literature reviews to support the justification of surrogate endpoints and evaluate the clinical validity of outcome measures in data-limited, rare disease populations.

Developing a compelling value case for a rare disease product requires creative and methodologically sound approaches. We support you to:

• Design and execute targeted studies to generate real-world evidence and other data needed to complement clinical trial results.
• Leverage diverse data sources, including patient-reported outcomes, caregiver perspectives and disease registries, to build a comprehensive picture of the product’s impact.
• Address key uncertainties by helping you develop a defensible evidence base that can withstand HTA scrutiny.
• Develop a clear and defensible value story that highlights the unique benefits of your innovation, even when traditional comparative data are limited.

In this complex landscape, clear and effective communication is essential to ensure your value proposition is understood by stakeholders. We help you to:

• Develop economic models and HTA submissions to address specific challenges facing the rare disease sector, such as limited long-term data, small patient populations, and high uncertainty.
• Tailor your value proposition and communications to meet the specific requirements of different HTA bodies, payers and patient groups.
• Create compelling materials that are easy to interpret and clearly explain the clinical, humanistic, and economic benefits of your product.
• Prepare for payer and decision-maker meetings by anticipating their expectations and equipping you to address potential challenges proactively.

Demonstrating value is an ongoing process, and post-market development is important to comprehensively understand a rare disease product’s performance in real-world settings. We assist you to:

• Strengthen your value proposition post-launch with new data that reflects the lived experiences of patients in the real world.
• Address new evidentiary challenges that emerge in the real world.
• Prepare for evolving healthcare expectations and adapt to changes in policy or methodology.