Company Overview:
Beam Therapeutics is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.
Position Overview:
Beam is seeking a dynamic, visionary, and strategic Senior Director, Value, Evidence, & Development Strategy to spearhead the global HEOR strategy for our sickle cell disease program (risto-cel) and other pipeline programs as they emerge . In this high-impact role, the Senior Director, Value, Evidence, & Development Strategy will develop and execute evidence-generation plans, manage stakeholder relationships, and shape external communication strategies. You will partner closely with Clinical Development, Medical Communications and Scientific Publications, Value and Evidence Strategy, Commercial, R&D, and additional cross-functional teams. If you thrive in a collaborative, mission-driven environment and are eager to shape the future of sickle cell disease care, we want you to bring your expertise and passion to Beam.
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.
- At least 10+ 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.