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

Biogen•Cambridge, MA

10d

About The Position

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.

Requirements

Advanced degree (MD, PhD, PharmD, or equivalent) with + 7 years of pharmaceutical, medical affairs, or clinical development experience, including substantial experience specific to evidence generation (registries, real-world data, or related methodologies); global medical experience preferred.
Demonstrated, hands-on experience designing, launching, and governing disease registries or natural history studies, including protocol and case report form design, data governance, site/network engagement, and long-term observational or post-marketing surveillance programs.
Proven track record analyzing registry-derived real-world data (longitudinal outcomes, safety signals, biomarker trends, effectiveness data) and translating findings into peer-reviewed publications, congress presentations, and medical strategy.
Experience serving on or engaging with registry steering committees, scientific advisory boards, or academic/consortium partnerships that govern shared disease registries.
Strong scientific knowledge of rare neuromuscular disease, SMA, ALS, genetics, or biomarkers, with the ability to lead through influence across a complex global matrix of regional teams, cross-functional partners, and registry governance stakeholders.
Strong communication and project leadership skills, with working knowledge of global medical affairs compliance, Good Clinical Practice, and registry data quality/governance requirements.

Nice To Haves

Deep therapeutic expertise in SMA, ALS, rare neuromuscular disease, neurodegeneration, genetics, or precision neurology.
Direct experience with SMA and ALS disease registries or natural history networks,
Experience with global evidence roadmaps, lifecycle management, product launches, registries, health outcomes research, comparative effectiveness, and patient-reported outcomes.
Familiarity with NfL or other biomarkers, intrathecal delivery platforms, genetic testing, newborn screening, treat-to-target frameworks, and rare disease patient identification strategies.
Experience partnering with registry steering committees, global medical experts, advocacy organizations, academic collaborators, and cross-regional medical teams.
Strong publication planning, scientific communication, and technical writing skills, including manuscripts, abstracts, congress presentations, advisory board materials, and internal scientific training.
Ability to create structure in ambiguity, anticipate external scientific shifts, and convert registry evidence gaps into bold, measurable, patient-centered medical strategies.

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.

Benefits

Medical, Dental, Vision, & Life insurances
Fitness & Wellness programs including a fitness reimbursement
Short- and Long-Term Disability insurance
A minimum of 15 days of paid vacation and an additional end-of-year shutdown time off (Dec 26-Dec 31)
Up to 12 company paid holidays + 3 paid days off for Personal Significance
80 hours of sick time per calendar year
Paid Maternity and Parental Leave benefit
401(k) program participation with company matched contributions
Employee stock purchase plan
Tuition reimbursement of up to $10,000 per calendar year
Employee Resource Groups participation