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2. European Medicines Agency (EMA), Guideline on registry-based studies, 2021.
3. FDA Guidance for Industry and Food and Drug Administration Staff: Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices, 2017.
4. FDA Guidance for Industry: Rare diseases: common issues in drug development. https://www.fda.gov/media/120091/download. February 2019 (Revision 1).
5. FDA Guidance for Industry: Rare diseases: natural history studies for drug development. https://www.fda.gov/media/122425/download. March 2019.