Formulary Submissions: Value Claims, Protocols and Outcomes Based Contracting in Rare Disease

Area of Science:

• Health Economics
• Pharmacoeconomics
• Health Technology Assessment

Background:

• Outcomes-based payment contracting is emerging, particularly for rare diseases.
• Rare diseases present unique challenges: limited evidence, high costs, and small patient populations.
• Current health technology assessment (HTA) models, like those from the Institute for Clinical and Economic Review (ICER), pose barriers to innovative therapies.

Purpose of the Study:

• To propose a new analytical framework for HTA in rare diseases.
• To advocate for a shift from assumption-driven cost-effectiveness simulations to evidence-based value claims.
• To facilitate effective outcomes-based contracting between payers and manufacturers.

Main Methods:

• Critique of current HTA methodologies, specifically those relying on modeled cost-effectiveness.
• Proposal of a new framework focused on single attribute value claims.
• Development of a new formulary submission package integrating value claims and assessment protocols.

Main Results:

• The proposed framework can overcome the limitations of current ICER-like modeling.
• A new approach can support evidence-based value claims for rare disease therapies.
• This facilitates the integration of value assessment with contracting protocols.

Conclusions:

• A new HTA framework focusing on single attribute value claims is essential for rare diseases.
• This approach can replace problematic modeling and enable outcome-based contracting.
• Outcome-based contracting should become the standard for future payer negotiations.