Medical Director - Spinal Muscular Atrophy

Medasource; United States; Remote; USD 330,000 - 387,000; Full time

Job summary

An innovative firm seeks a Medical Director for Spinal Muscular Atrophy, focusing on evidence generation strategies to improve patient outcomes. This role involves collaboration with cross-functional teams and oversight of observational studies. The ideal candidate will have deep expertise in rare neurological diseases and a proven track record in clinical research. Join a forward-thinking organization dedicated to advancing medical science and enhancing patient care in a remote capacity. If you are passionate about making a difference in the field, this opportunity is perfect for you.

Benefits

Medical Insurance

Vision Insurance

Qualifications

• Advanced degree in life sciences required.
• 5+ years in pharmaceutical industry or academia.

Responsibilities

• Support global medical strategic imperatives and communication frameworks.
• Lead evidence generation projects aligned with global strategy.

Skills

Clinical Research

Neurology Expertise

Evidence Generation

Project Management

Communication Skills

Education

MD or equivalent

PhD in Life Sciences

Job description

Medical Director - Spinal Muscular Atrophy

Medical Director - Spinal Muscular Atrophy

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Medical Director – Spinal Muscular Atrophy (SMA)

Contract through December 2025 to start

Remote (EST)

About This Role

As a key member of the Global Medical SMA team within the worldwide medical organization, the Associate Medical Director, reporting to the Global Medical Lead, will be responsible for supporting the development and implementation of global evidence generation strategies in Spinal Muscular Atrophy (SMA). This role involves oversight of Real-World Data (RWD), Observational Research (OR), and Collaborative Research activities, ensuring alignment with the Integrated Evidence Generation Plan (IEGP) and global medical priorities.

The ideal candidate has deep expertise in SMA, a strong background in evidence generation methodologies, and a passion for improving patient outcomes in rare neurological diseases.

What You’ll Do

• In collaboration with the Global Medical SMA team, support the development of global medical strategic imperatives and a communication framework in partnership with SMA scientific collaborators.
• Lead, co-lead, or support high-impact evidence generation projects aligned with the IEGP, serving as a key global medical contact for cross-functional teams (e.g., Clinical Development, Commercial, Market Access, PDC, Regional Medical).
• Lead or co-lead planning, design, and execution of observational studies and clinical trials (e.g., defining target populations, developing protocols, calculating sample sizes).
• Assess and evaluate the strengths and limitations of external Real-World Data sources to advance the SMA evidence generation strategy.
• Drive and implement evidence generation plans, conducting gap analyses and aligning efforts with medical and strategic needs.
• Collaborate with the Therapeutic Area Lead and cross-functional stakeholders to ensure key insights and data needs from affiliates are identified and integrated into the evidence strategy.
• Partner with regional and affiliate medical teams to gather insights that inform clinical trial planning and data generation efforts globally.
• Perform additional responsibilities as assigned; the scope of the role may evolve based on business needs.

Who You Are

You are a scientific or clinical professional with in-depth knowledge of rare neurological diseases and a track record of success in evidence generation. You are collaborative, detail-oriented, and driven by a commitment to advancing medical science and patient care. You understand the evolving SMA landscape and are adept at translating insights into action.

Required Skills & Qualifications

• Advanced degree (MD, PhD, PharmD, MPH, or equivalent) in the life sciences is required.
• Clinical or scientific expertise in neurology, rare diseases, and clinical research, with a strong preference for experience in Spinal Muscular Atrophy (SMA).
• Experience supporting or leading medical activities for an SMA product launch is highly desirable.
• 5+ years of experience in the pharmaceutical industry, consulting, or academia, with a proven track record of conducting and publishing RWE studies.
• Deep understanding of Real-World Evidence (RWE), observational research, and collaborative study models.
• Strong interpersonal and project management skills, with the ability to operate effectively in a matrixed global organization.
• Excellent written and verbal communication skills, with the ability to present complex data clearly and persuasively.
• Demonstrated experience working with regional/affiliate medical teams to ensure alignment between global strategy and local execution.
• Must be willing and able to travel, as required for meetings, congresses, or field engagement.

Seniority level

• Seniority level

  Director

Employment type

• Employment type

  Full-time

Job function

• Job function

  Research

• Industries

  Pharmaceutical Manufacturing

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Medical insurance

Vision insurance

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