Clinical Research Director, Rare Disease

Benefits

Qualifications

• •5+ years in drug development in pharmaceutical/biotech industry with experience in clinical development
• •Demonstrated experience with orphan drug frameworks and small patient population trial design
• •Expertise in clinical pharmacology, biomarker strategy, benefit-risk assessment, and innovative trial designs
• •Proven ability to lead cross-functional global teams without direct authority; strong negotiation and decision-making skills
• •Exceptional written and verbal English; ability to present to scientific, regulatory, executive, and patient audiences
• •2 more items(s)

Responsibilities

• •Lead design and authorship of study synopses, CDPs, and clinical sections of IDPs
• •Oversee end-to-end trial execution through cross-functional units, ensuring GCP and regulatory compliance
• •Define and manage timelines, budgets, and risk mitigation strategies with Clinical Operations, Project Management, and Procurement
• •Serve as clinical lead and medical spokesperson within the Global Project Team and Protocol Review Committee
• •Contribute to biomarker identification and provide due diligence support for business development opportunities
• •Maintain deep expertise in internal medicine, metabolic diseases, and rare disease biology
• •Integrate preclinical data, clinical pharmacology, and competitive intelligence to inform strategy
• •Champion innovative trial designs - adaptive, natural history, and real-world evidence - appropriate for rare disease contexts
• •Influence product value proposition through evidence-based input on unmet needs and development approaches
• •Represent programs at key regulatory interactions (FDA, EMA, PMDA) as primary medical spokesperson
• •Develop engagement strategies for pre-IND, End-of-Phase meetings, and Advisory Committee preparations
• •Support label development, registration submissions, and post-approval modifications
• •Ensure all activities comply with FDA, EMA, CHMP, ICH guidance and Sanofi policies
• •Drive timely publication of clinical data in peer-reviewed journals and at scientific congresses
• •Lead advisory board meetings and maintain strategic relationships with KOLs, patient advocacy organizations, and rare disease consortia
• •Align cross-functional stakeholders around a unified development vision; present CDPs to governance and senior leadership
• •Partner with Medical Affairs, Biostatistics, Translational Medicine, Regulatory, Market Access, and Commercial functions
• •Mentor junior clinical scientists and contribute to talent development within the Rare Disease TA
• •15 more items(s)