[Risk sharing agreements: with orphan drugs?]

Area of Science:

• Pharmacoeconomics
• Health Technology Assessment
• Orphan Drug Policy

Background:

• Share Risk Agreements (RSA) are an emerging mechanism for introducing orphan drugs (OD) into clinical practice.
• Available information and rigorous evaluations on the outcomes of RSAs are currently limited.

Discussion:

• RSAs offer theoretical advantages for OD, including value-based pricing, enhanced pharmacovigilance, and improved understanding of comparative effectiveness.
• Key challenges include designing, implementing, and monitoring these agreements, which often hinder their success.
• Two primary RSA types exist: financially based and performance-outcome based.

Key Insights:

• Despite scant experience, orphan drugs appear suitable for introduction via RSAs.
• Few RSAs have demonstrably succeeded to date, highlighting implementation difficulties.
• RSAs can potentially reduce uncertainty for patients, industry, payers, and clinicians.

Outlook:

• Successful RSA management requires harmonized assessments, overcoming implementation hurdles, and strong political/institutional support.
• Technical expertise exists to manage RSAs effectively.
• RSAs hold potential for optimizing orphan drug access and value if challenges are addressed.