Regulatory Pathway Explorer

FDA designations that modify timeline or review intensity — not the evidence standard. May be held concurrently; requested at different development stages.

Chemistry, Manufacturing & Controls development is a continuous regulatory obligation — not a discrete stage. Pre-approval inspection (PAI) of manufacturing sites occurs during NDA review. Post-approval changes require notification or approval under 21 CFR 314.70.

FDA programs that modify pathway incentives or timeline — without changing evidence standards:

• →Breakthrough Device Designation — expedited development & enhanced FDA interaction
• →Humanitarian Device Exemption (HDE) — for rare conditions (<8,000 patients/year)
• →De Novo (pathway + designation hybrid) — creates new device classification & controls
• →Priority Review — limited applicability compared to drugs; affects interaction intensity

Continuous process governed by Design Controls (21 CFR 820.30), running in parallel across all pathway stages:

• →User needs → design inputs → outputs → verification → validation
• →Risk management per ISO 14971 integrated throughout all stages
• →Usability engineering and human factors validation (FDA guidance)

Programs that modify development timeline and FDA interaction intensity — not the evidence standard for licensure:

• →Breakthrough Therapy Designation — intensive FDA guidance from early development
• →Fast Track — rolling BLA review & increased FDA meeting access
• →Accelerated Approval — approval on a surrogate endpoint; PMR required to verify clinical benefit
• →Priority Review — 6-month PDUFA goal vs. 10-month standard
• →RMAT — Regenerative Medicine Advanced Therapy; biologics-specific, critical for cell & gene therapies

The continuous, dominant workstream across the entire biologics lifecycle:

• →Cell line development → upstream/downstream processing
• →Analytical characterization — identity, purity, potency
• →Process validation & comparability
• →Facility readiness & GMP compliance
• →"The process is the product." Changes require comparability data and regulatory approval.