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- Patient-Focused Drug Development Guidance: Key Highlights From FDA’s Second Public Meeting
- Patient-Focused Drug Development Guidance: Key Highlights From FDA’s Second Public Meeting
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- White Paper: Adapting an Existing Instrument for a Rare Disease: A Valuable Resource within Your Reach
- White Paper: Can Early Collaboration between Multiple European Stakeholders Increase Access for Patients Living with Rare Diseases?
- White Paper: Clinical Outcome Assessment Selection for Rare Disease Trial Programs
- White Paper: Evidence Requirements for Orphan Drugs from a Payer Perspective: How Can Early Scientific Advice Help?
- White Paper: FDA Updates Draft Guidance on Rare Diseases: Some Key Takeaways You Need to Know
- White Paper: How Social Media Can Be Used to Understand What Matters to People with Rare Diseases
- White Paper: More Evidence Needed for Orphan Drugs in Germany: A Hurdle for Access and an Opportunity for Real-World Evidence
- White Paper: Natural History Studies in Rare Diseases and Genetic Biomarkers
- White Paper: Patient Engagement in Clinical Trial Protocol Design and Recruitment Strategies: What Does It Mean for Orphan Drug Manufacturers?
- White Paper: Policy Trends Update: CADTH Launches Parallel Scientific Advice Programs with NICE and Health Canada
- White Paper: Rare Disease Treatments Aren’t So Rare Any More
- White Paper: Registries in Rare Disease Research: Approaches to Optimize Success