Strategic Clinical Development Flashcards
What characterises clinical development in biotech startups?
Single-asset focus, aim to partner/sell post-PoC, full outsourcing of clinical trial execution.
What are the two main teams in large pharma clinical development?
Clinical Development Strategy Teams and Clinical Trial Execution Teams.
What is the difference between boutique and large CROs?
Boutique CROs offer agility and fast turnaround; large CROs provide stable, scalable full-service capabilities.
What services does Strategic Development Consulting (SDC) cover?
Preclinical toxicology, clinical trial design, regulatory strategy, market access, and vendor selection.
List the phases in drug development.
Preclinical, Phase 1 (FIH), Phase 2 (PoC), Phase 3 (Pivotal), Phase 4 (Post-marketing).
Why are Pre-IND and EoP2 meetings important?
They facilitate early engagement with regulators, helping to avoid clinical holds and align Phase 3 plans.
What are three US FDA expedited approval pathways?
Fast Track, Breakthrough Therapy, and Accelerated Approval.
What is EMA’s equivalent to FDA Fast Track?
PRIME – Priority Medicines scheme to accelerate review for high-need drugs.
What power is typically used in Phase 2 vs Phase 3 trials?
Phase 2: 80% power; Phase 3: 95% power with p-value < 0.05.
What biomarker was used in the ALS gene therapy study?
Neurofilament Light (NfL), though its regulatory acceptance was uncertain.
What was the rationale for using Tofersen-treated patients in ALS trials?
To ease recruitment by targeting patients already familiar with treatment options.
What are the major challenges in recruiting NASH patients?
High biopsy-driven screen failure rates (70-80%) and low disease awareness.
What’s the difference between Accelerated and Traditional Approval in NASH?
Accelerated uses surrogate endpoints; traditional requires survival/clinical benefit data.
What are the benefits of precision medicine in clinical development?
Smaller, faster trials, higher approval probability, and optimised pricing/reimbursement.
Why is early FDA/EMA engagement essential?
To prevent trial delays, align endpoints, and ensure smoother regulatory paths.
