Orphan Drug Act & Drug Pricing: A Modern Analysis

Each year, February is recognized as Rare Disease Month, highlighting the experiences and breakthroughs shaping progress in this space—chosen for its rare distinction as a 28-day month. Hello, my name is Maggie Shaw, lead editor with The American Journal of Managed Care^®and welcome to the latest episode of Managed Care Cast.

In this episode, Kristi Martin, a longtime Washington, DC–based policy strategist with deep experience implementing major legislation like the Affordable Care Act and the Inflation Reduction Act, explores how the Orphan Drug Act, passed in 1983 as a bipartisan, patient-driven effort, transformed rare disease treatment by aligning scientific advances, government investment, and market incentives.

She also explains how today’s policy landscape has fundamentally changed, creating new cost and equity challenges as the Orphan Drug Act intersects with modern drug pricing policy, including the shielding of high-spending orphan oncology drugs from negotiation. While acknowledging the law’s life-saving impact, Martin argues that reform may now be necessary—rethinking incentives for blockbuster drugs, refining orphan drug eligibility, and shifting toward more targeted, evidence-based negotiation—while emphasizing the federal government’s continued role in sustaining innovation through agencies such as the NIH, FDA, and CMS.

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