Vice President, Global RWE and HEOR Strategy

Responsibilities

• Provide strategic input to disease prioritization, target product profiles, clinical trial protocols, clinical development plans, and regulatory submissions; lead development of clinical sections for Investigator’s Brochures, briefing books, safety updates, IND/NDA materials, and responses to Health Authority questions.
• Develop, validate, and integrate patient-centered outcomes (PROs) into protocols and analyses.
• Interpret clinical trial results (including PROs and healthcare resource use) and partner with Clinical Development/Biostatistics on Statistical Analysis Plans (SAPs) and post-hoc analyses.
• Lead a fit-for-purpose RWE strategy (EHR, claims, registries, hybrid/open datasets) including natural history/registry design and governance to inform endpoints, recruitment, and post-authorization effectiveness/safety assessments.
• Develop and operationalize external/synthetic control arm approaches (e.g., matched registry cohorts, Bayesian borrowing) for single-arm or small-population studies typical in cell & gene therapy.
• Provide operational oversight of Medical Affairs evidence generation, including vendor/CRO governance, milestone/deliverable tracking, data-quality plans, risk management, and on-time execution across Medical Affairs-led studies; contribute to long-term capability building (digital health, real-world studies).
• Elicit and integrate perspectives from patients, KOLs, policy leaders, and internal stakeholders (market access, marketing, patient advocacy) to ensure plans meet the needs of patients, HCPs, and payers.
• Support Market Access on internal and external payer/policy initiatives (pricing and reimbursement, unmet medical need, regional market dynamics, evidence gaps) across the portfolio.
• Support business decisions around ex-US strategy including, but not limited to, scoping ex-US opportunities, prioritizing country engagement, early scientific advice / parallel consultations with EMA and HTA bodies (e.g., HTACG/JSC), providing advice to development programs on endpoint selection to support product and reimbursement approvals ex-US, etc.
• Anticipate payer evidence needs and budget-impact critiques (including preparation for ICER Reviews).
• Build and maintain economic models (cost-effectiveness, budget impact) tailored to one-time CGTs, addressing uncertainty/durability and caregiver burden.
• Collaborate with Market Access to design innovative payment models and outcomes-based agreements with pragmatic outcomes and tracking.
• Lead the AMCP dossier and pre-approval information exchange (PIE) strategy (6-12 months pre-launch).
• Work collaboratively with internal teams, consultants, and CROs to advance development of the portfolio.

Qualifications

• Master’s Degree or higher in Health Economics and Outcomes Research or a relevant discipline.
• Formal training in Epidemiology/Health Services Research required.
• 20+ years’ experience in observational research study management AND data analytics, either within industry or with an observational research consulting firm.
• Proven track record in drug development, with rare disease experience and an existing network of hematologists preferred.
• Demonstrated ability to lead in a matrixed, cross-functional environment, combining strategic agility with strong business acumen.
• Independent, proactive operator with strong ownership.
• Cross functional collaboration with Clinical, Regulatory, Commercial, Medical Operations, and Publications while maintaining medical/scientific integrity.
• Excellent communication, collaboration, and problem-solving skills.