yoinka

Global Medical Director, Rare Disease ( SMA/ALS), Evidence Generation Lead

Biogen

Cambridge, MAStaffH-1B sponsor company
Sign in to applyVerified 1h ago
Location
Cambridge, MA
Work model
On-Site
Level
Staff
H-1B history
30 approvals (FY2023)
Posted
5h ago

About this role

About the Role

The Global Medical Director, Rare Disease Evidence Generation – ALS and SMA is a key member of the Global Medical Rare Disease team, responsible for leading all evidence generation efforts — including registry-based studies, real-world data initiatives, and other evidence sources — across Biogen's amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) portfolios. This role will design, oversee, and translate evidence to inform medical practice, support lifecycle strategy, strengthen scientific leadership, and improve meaningful outcomes for people living with these rare neuromuscular diseases. By leveraging your deep knowledge of rare diseases, and evidence generation, you will directly contribute to shaping the future of personalized outcomes research and medical advancements. This is a unique opportunity to make a meaningful impact in a highly matrixed and dynamic environment.

Key Responsibilities

Lead the Global Integrated Evidence Generation strategy/ plan development for Rare portfolio (SMA and ALS) by leveraging registries, real-world data, and other evidence sources in alignment with Global Medical Plan priorities and regional medical needs. Design, prioritize, and oversee evidence generation initiatives — including registry-based studies leveraging established disease registries and natural history networks, real-world data analyses, and other complementary evidence sources — to generate long-term safety, effectiveness, and outcomes data. Develop and maintain an integrated evidence generation roadmap across rare portfolio, incorporating registries, real-world data, neurofilament light chain (NfL) and other biomarkers, motor function outcomes, and long-term safety surveillance. Develop a communication plan Collaborate closely with Clinical Development, Biostatistics, Epidemiology, Health Economics and Outcomes Research, Value and Access, Regulatory, Patient Advocacy, Regional Medical, and Commercial partners to ensure registry evidence plans are scientifically robust, strategically aligned, and globally actionable. Lead or contribute to registry protocol development, statistical analysis plans, study reports, publication strategy, data interpretation, and scientific communication plans for SMA and ALS registry programs. Strengthen Biogen's scientific leadership in rare neuromuscular disease by advancing registry-derived evidence on treatment optimization, sequencing, durability, biomarker use, novel endpoints, patient identification, access, and long-term outcomes. Synthesize global medical insights, registry data, external evidence, competitive intelligence, and regional feedback to refine evidence priorities and inform medical strategy. Serve as a credible scientific expert and medical representative for SMA/ALS portfolio registry-based evidence generation in internal governance forums, advisory boards, congresses, scientific meetings, and external expert engagements. Partner with publications, medical communications, and regional medical teams to translate registry evidence into medically accurate, balanced, and compliant materials, including manuscripts, abstracts, posters, training resources, scientific narratives, and congress content. Build and nurture relationships with key external stakeholders, including registry steering committees, global medical experts, healthcare institutions, registry partners, and advocacy organizations across the SMA and ALS landscape. Support governance and operating rhythms for cross-functional registry evidence review, prioritization, decision-making, budget tracking, and delivery against measurable evidence-generation milestones. Ensure all registry-based evidence generation activities are conducted in accordance with Biogen's compliance standards, Good Clinical Practice, relevant regulations, and applicable internal policies.

Who You Are

You are a scientifically rigorous, globally minded medical leader who can connect evidence strategy to

Global Medical Director, Rare Disease ( SMA/ALS), Evidence Generation Lead at Biogen, Cambridge, MA | Yoinka