Updates - Rare Coalition

Thought Leadership

Project GUARDIAN: Pioneering Genomic Newborn Screening for Rare Genetic Diseases

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How QALY Discriminates Against People Living with Rare Diseases

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Congress Can Unite to Help Kids Living with Rare Diseases

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Celebrating 40 Years of the Orphan Drug Act: Renewing a Commitment to Advancing Innovation for Rare Disease Patients

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The State of the Biotech Industry

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Policy Must Keep Pace with Technology if we are to Reap the Benefits of AI Drug Discovery for Rare Diseases

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The Case for Rare Diseases as a Public Health Priority

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“One-Size-Fits-All” HTA would be a Major Setback for Rare Disease Patients

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The Unique Clinical and Economic Challenges of Rare Disease Diagnosis

Cutting the Orphan Drug Tax Credit is Not Drug Pricing Reform

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Rightsizing the U.S. Newborn Screening System

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The Perils of One-Size-Fits-All Public Policy

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Accelerated Approval Offers a Promising Roadmap for Rare Neurological Diseases