Clinical Research Director, Rare Disease

Job Title: Clinical Research Director, Rare Disease

Location: Cambridge, MA, Morristoen, NJ

About the Job

Join the engine of Sanofi’s mission — where deep immunoscience meets bold, AI-powered research. In R&D, you’ll drive breakthroughs that could turn the impossible into possible for millions.

Position Overview

The Clinical Research Director (CRD), 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.

The CRD serves as a recognized scientific authority within the Rare Disease TA, championing clinical excellence, challenging conventional approaches, and advancing Sanofi's mission to deliver transformative therapies to underserved patients.

Key Responsibilities

Clinical Development Strategy & Execution

• 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

• Scientific Leadership

• 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

• Regulatory Strategy

• 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

• External Engagement & Dissemination

• 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

• Cross-Functional Collaboration

• 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

Required Qualifications

• Education: MD required; MD/PhD strongly preferred; medical degree from LCME-accredited or equivalent institution

• Experience: 5+ years in drug development in pharmaceutical/biotech industry with experience in clinical development

• Rare Disease: Demonstrated experience with orphan drug frameworks and small patient population trial design

• Technical Skills: Expertise in clinical pharmacology, biomarker strategy, benefit-risk assessment, and innovative trial designs

• Leadership: Proven ability to lead cross-functional global teams without direct authority; strong negotiation and decision-making skills

• Communication: Exceptional written and verbal English; ability to present to scientific, regulatory, executive, and patient audiences

Preferred Qualifications

• Board certification in metabolic medicine, medical genetics, nephrology, or cardiology

• Direct experience with lysosomal storage disorders, enzyme replacement therapy, or gene therapy

• Prior experience as medical spokesperson in FDA/EMA meetings

• Familiarity with real-world evidence (RWE) in rare disease regulatory submissions

• Peer-reviewed publication record in the relevant therapeutic area

• Experience with business development and scientific due diligence

Why Choose Us?

• Bring the miracles o