CASE STUDIES

Cost-Effectiveness Assessment for an Orphan Drug in a Rare Autoimmune Disease

Client: A Leading Pharmaceutical Company

Challenge:
A pharmaceutical company sought to adapt a cost-effectiveness model for an orphan drug targeting a rare autoimmune disease. The goal was to determine the incremental cost-effectiveness ratio (ICER), estimate the budget impact, and assist the company in negotiating pricing and a capped contract for utilization within NHS hospitals.

Solution Provided by Clevidence:
Clevidence was engaged to conduct a comprehensive assessment that included literature review, price analysis, and the organization of an expert panel:

  1. Cost-Effectiveness Model Adaptation:
    Our team adapted a cost-effectiveness model to reflect the drug’s value more accurately, which involved:

    – Evaluating clinical outcomes and healthcare resource utilization associated with both the orphan drug and alternative therapies.

    – Determining the ICER based on cost and Quality-Adjusted Life Year (QALY) comparisons with alternative therapies.

  2. Expert Panel Organization:
    We organized a panel of key opinion leaders (KOLs) to provide insights into local clinical practices for managing the rare autoimmune disease. This panel informed us about:

    – Health resources used for monitoring and managing the disease, critical for the cost-effectiveness and budget impact analyses.

    – Practical implications of adopting the orphan drug in clinical settings to ensure our models aligned with real-world practices.

  3. Budget Impact Analysis:
    We conducted a budget impact analysis to estimate the financial implications of the drug’s adoption within NHS hospitals. This included:

    – Analyzing epidemiological data to determine the estimated number of potential patients and reviewing current pricing lists for relevant health resources.

    – Projecting expenditure and savings for the NHS based on different adoption scenarios.

Results:
The adapted cost-effectiveness model and budget impact analysis provided the pharmaceutical company with essential data, including:

  • An ICER demonstrating the drug’s value relative to alternatives.
  • Detailed budget impact projections to inform pricing strategies.

Conclusion:
Through thorough analysis and expert engagement, Clevidence enabled the pharmaceutical company to present a compelling case for the orphan drug’s use within NHS hospitals. This evaluation clarified the drug’s economic viability and facilitated successful pricing negotiations, ensuring that patients with the rare autoimmune disease could access this vital treatment.

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At Clevidence, we are proud to showcase the results of our comprehensive and strategic health solutions. Our case studies highlight the tangible impact of our health technology assessments, market access strategies, post-marketing research, and real-world evidence generation. Discover how we have empowered clients to make informed decisions and achieve outstanding results.