Senior Clinical Research Director, Rare Disease

Position Overview
The Senior Clinical Research Director (sCRD), Rare Disease is a senior scientific and clinical leader responsible for shaping and executing clinical development strategy for assigned rare disease programs, driving them from early development through registration with a patient-centric focus.

Key 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 strategic clinical lead and medical spokesperson within governance/leadership forums
- Contribute to biomarker identification and provide due diligence support for business development opportunities

Scientific Leadership
- Maintain deep expertise in internal medicine, metabolic diseases, nephrology, and rare disease biology
- Integrate preclinical data, clinical pharmacology, and competitive intelligence to inform strategy
- Champion adaptive, natural history, and real-world-evidence trial designs for rare disease contexts

Regulatory & External Engagement
- Lead 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
- Drive publication of clinical data and lead advisory board meetings; maintain KOL and patient advocacy relationships

Required Qualifications
- MD required (MD/PhD strongly preferred); medical degree from LCME-accredited or equivalent institution
- 10+ years in drug development or clinical development (pharma/biotech)
- 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 cross-functional global leadership without direct authority; strong negotiation/decision-making
- Exceptional written/verbal English; ability to present to scientific, regulatory, executive, and patient audiences

Preferred Qualifications
- Board certification in metabolic medicine, medical genetics, nephrology, or cardiology
- Experience with lysosomal storage disorders, enzyme replacement therapy, or gene therapy
- Prior medical spokesperson experience in FDA/EMA meetings
- Familiarity with real-world evidence (RWE) in rare disease regulatory submissions
- Peer-reviewed publication record
- Experience with business development and scientific due diligence