- Project ORBIS
- FDA Oncology Center of Excellence (OCE) framework for concurrent submission and review of oncology products among international partner agencies. Review types: Type A (concurrent submission, all partners), Type B (submission within ~30 days of FDA / partners), Type C (submission after FDA, review can begin later).
- Accelerated vs traditional approval
- Accelerated approval is based on a surrogate/intermediate endpoint reasonably likely to predict clinical benefit, with a required confirmatory trial. Traditional (regular) approval rests on direct evidence of clinical benefit. Always state which one a precedent used.
- iPSP / PSP
- (initial) Pediatric Study Plan — the FDA mechanism, required under PREA, in which a sponsor lays out planned pediatric studies (or a deferral/waiver request) early in development.
- PIP / PDCO
- Paediatric Investigation Plan — the EMA counterpart to the iPSP, agreed with the Paediatric Committee (PDCO) governing pediatric studies for an EU application.
- PREA
- Pediatric Research Equity Act — US law requiring pediatric assessment of certain applications (with deferrals/waivers); the statutory basis for the iPSP.
- RACE for Children Act
- Research to Accelerate Cures and Equity Act — amended PREA so that molecularly targeted oncology drugs directed at a relevant pediatric molecular target must be studied in children, even where the adult indication doesn’t apply.
- PMR / PMC
- Post-Marketing Requirement (legally required, e.g. an accelerated-approval confirmatory trial) vs Post-Marketing Commitment (agreed but not statutorily required).
- MIDD
- Model-Informed Drug Development — use of modeling & simulation (PK/PD, exposure-response, extrapolation) to inform dose, design, and pediatric extrapolation decisions.
- RAPNO vs RANO
- Response Assessment in Pediatric Neuro-Oncology vs Response Assessment in Neuro-Oncology — the pediatric-specific CNS response criteria vs the adult framework; name which criterion a trial used.
- Conditional approval (EMA)
- EU marketing authorisation granted on less complete data for an unmet need, subject to specific obligations and annual renewal — the EMA analogue to (not identical with) US accelerated approval.
- Tissue-agnostic
- An approval or indication defined by a molecular alteration (e.g. a BRAF class or fusion) across tumor types, rather than by tumor site of origin.
- Basket trial
- A trial enrolling multiple tumor types sharing a common molecular alteration into parallel cohorts under one protocol — the design that often supports tissue-agnostic claims.
- HITL
- Human-in-the-loop — required regulatory and legal sign-off before anything leaves as a claim; this app is decision support, not regulatory or legal advice.
- IBC orchestrator
- The platform layer that routes each question to the right specialist agent and integrates their views.
- Plixorafenib
- Fore Biotherapeutics’ investigational next-generation BRAF (“paradox-breaker”) inhibitor — the program this regulatory support is oriented around, including its pediatric BRAF-CNS / orphan setting.