REF: Expedited Pathways & Designations

Accelerated Approval | FDA. (2023, February 24). Retrieved from Food and Drug Administration: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval

Breakthrough Therapy | FDA. (2018, April 01). Retrieved from Food and Drug Administration: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy

Designating an Orphan Product: Drugs and Biological Products | FDA. (2022, 08 07). Retrieved from Food and Drug Administration: https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Guidance for Industry (fda.gov). (2019, February). Retrieved from Food and Drug Administration: https://www.fda.gov/media/120267/download

Expedited Programs for Serious Conditions – Drugs and Biologics. (2020, 06 25). Retrieved from Food and Drug Administration: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics

Fast Track | FDA. (2018, April 01). Retrieved from Food and Drug Administration: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track

FDA Designations For Rare Disease Products, Part 3: Rare Pediatric Disease Designation | Premier Consulting. (2021, 03 25). Retrieved from Premier Consulting: https://premierconsulting.com/resources/blog/fda-designations-for-rare-disease-products-part-3-rare-pediatric-disease-designation/#:~:text=A%20rare%20pediatric%20disease%20is,infants%2C%20children%2C%20and%20adolescents

Orphan Drug Designation Request Form | FDA. (2021, July 09). Retrieved from Food and Drug Administration: https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-designation-request-form

Priority Review | FDA. (2018, April 01). Retrieved from Food and Drug Administration: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review

Rare Diseases at FDA. (2022, 12 13). Retrieved from Food and Drug Administration: https://www.fda.gov/patients/rare-diseases-fda

Rare Pediatric Disease (RPD) Designation and Voucher Programs | FDA. (2023, 06 02). Retrieved from Food and Drug Administration: https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs

Rare Pediatric Disease Priority Review Vouchers (fda.gov). (2019, 07). Retrieved from Food and Drug Administration.

Regenerative Medicine Advanced Therapy Designation | FDA. (2023, April 19). Retrieved from Food and Drug Administration: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience - PMC (nih.gov). (2023, 03). Retrieved from PubMed: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10031144/