Responsibilities:
- Provide strategic input to disease prioritization, target product profiles, clinical trial protocols, clinical development plans, and regulatory submissions; lead clinical sections for Investigator’s Brochures, briefing books, safety updates, IND/NDA materials, and Health Authority responses.
- Develop, validate, and integrate patient-centered outcomes (PROs) into protocols and analyses.
- Interpret clinical trial results (including PROs and healthcare resource use); partner with Clinical Development/Biostatistics on SAPs and post-hoc analyses.
- Lead fit-for-purpose RWE strategy (EHR, claims, registries, hybrid/open datasets), including natural history/registry design and governance.
- Develop and operationalize external/synthetic control arm approaches (e.g., matched registry cohorts, Bayesian borrowing) for single-arm/small-population studies.
- Provide operational oversight of Medical Affairs evidence generation (vendor/CRO governance, milestone tracking, data-quality plans, risk management, on-time execution) and build long-term capability (digital health, real-world studies).
- Elicit and integrate perspectives from patients, KOLs, policy leaders, and internal stakeholders (market access, marketing, patient advocacy).
- Support Market Access on payer/policy initiatives (pricing/reimbursement, unmet need, regional dynamics, evidence gaps) and ex-US strategy.
- Anticipate payer evidence needs and budget-impact critiques (including ICER Reviews); build cost-effectiveness and budget-impact models for one-time CGTs.
- Collaborate on innovative payment models and outcomes-based agreements.
- Lead AMCP dossier and pre-approval information exchange (PIE) strategy (6–12 months pre-launch).
Qualifications:
- Master’s degree or higher in Health Economics and Outcomes Research (or relevant discipline).
- Formal training in Epidemiology/Health Services Research (required).
- 20+ years’ experience in observational research study management and data analytics.
- Proven drug development track record; rare disease experience; existing hematologists network (preferred).
- Matrix leadership, strategic agility, strong business acumen; independent, proactive ownership.
- Cross-functional collaboration with Clinical, Regulatory, Commercial, Medical Operations, and Publications while maintaining scientific integrity.
- Excellent communication, collaboration, and problem-solving.
- Onsite: 1–3 days per week.