Senior Clinical Research Director, Rare

Role Summary

Senior Clinical Research Director (Senior CRD) within the RARE Therapeutic Area, acting as the expert clinical lead for one or several indications in the development of an R&D program. The role requires medical leadership, strong organizational skills, and a strategically and operationally focused mindset. The Senior CRD collaborates with internal and external stakeholders, demonstrates high emotional intelligence, self-motivation, and the ability to manage multiple operational tasks.

Responsibilities

• Provide medical expertise in the study team to conduct clinical studies from early phases to lifecycle management programs.
• Collaborate with other medical and clinical science experts and stakeholders to deliver development strategy and support regulatory interactions.
• Develop study-level regulatory documentation (Abbreviated Protocol, Protocol, Amended Protocol) and lead study start-up activities (e.g., pressure test, competitive intelligence, patient engagement, cluster feasibility, risk assessment).
• Provide medical input and support for activities related to clinical studies (answers to IRBs/HA questions, protocol medical training, centralized data review, medical information for the study team, and investigators).
• Develop the clinical portion of briefing documents and the Common Technical Document for regulatory submissions (FDA, EMA, PMDA, etc.) and respond to health authority questions.
• Serve as the medical reference in the Clinical Study team, mentoring other team members and training on medical/scientific strategy by indication.
• Lead the clinical development plan strategies, including clinical development plans and sections of integrated development plans (IDP).
• Contribute to defining product value proposition (TVP), Target Product Profile (TPP), and market access strategy; guide research strategy focus by clinical needs.
• Prepare internal governance documents and contribute to medical aspects of TPP, TVP, and GPT documents for one or more indications; collaborate with project leadership to resolve issues and share lessons.
• Coordinate with external partners, regulators, scientific experts, and internal stakeholders; provide operational expertise to project sub-teams as needed.
• Review and evaluate relevant medical literature and status of competitive products.
• Lead, support, and oversee execution of clinical development and studies activities, including collaboration with all function stakeholders, ensuring timelines, budgets, and risk management in compliance with GCP and SOPs.
• Develop/renew study protocols, Core Study Informed Consent Form (CSICF), and Study Committee Charters; collaborate on feasibility results and competitive intelligence.
• Provide clinical input to study documents such as study plans and risk management/monitoring plans; participate in investigator meetings and trainings; respond to medical questions from authorities and sites.
• Ensure data quality and availability of trial master file documents; oversee study committees (IDMC, steering committee, adjudication) with operational support; co-develop the SAP with biostatistics; develop key results and the clinical study report.
• Represent the project in regulatory interactions, lead clinical sections of CTD, briefing documents, and support registration/labelling; contribute to clinical sections of Investigator’s Brochure, CTA/IND/DSUR/DRMP/RMP and BLA/CTD.
• Ensure clinical data meets regulatory standards; collaborate with Patient Safety to detect safety signals; participate in Advisory Committee preparation.
• Participate in authorship of manuscripts and abstracts; establish collaborations with knowledge experts or advisory boards; serve as clinical advisor to research teams for potential in-licensing candidates.
• Lead or contribute to ongoing scientific data evaluation and authorship activities.

Qualifications

• At least 4 years of experience in clinical development in the pharmaceutical industry or CRO, or medical experience in clinical development within a healthcare institution.
• Language: English fluent (spoken and written).

Education

• Medical Doctor (MD) preferred: GP or specialist; training in pediatrics or pediatric neurology is a plus.

Skills

• Very good communication skills (verbal and written) for cross-functional interactions and regulatory agency engagement.
• Excellent problem-solving ability; ability to challenge decisions with a risk-management approach.
• Quality-focused with data-driven and risk-based practices; ability to handle multiple tasks and prioritize effectively.
• Negotiation skills to secure operational resources; mentoring and resource evaluation capabilities.
• Ability to work within a matrix organization; international/intercultural working skills; openness to applying new digital solutions.
• Understanding of pharmaceutical product development and lifecycle management; strong scientific and medical/clinical expertise; proficiency in clinical development methodologies; familiarity with Health Authority interactions.

Additional Requirements

• Location: Cambridge, MA or Morristown, NJ.
• Travel expectations may be required as part of role.