Clinical Scientist, Rare Disease

We are seeking a highly motivated and experienced Rare Disease Clinical Scientist to join our team. This role is integral to our efforts in developing innovative therapies for patients with rare and orphan diseases. The successful candidate will collaborate with multidisciplinary teams, including clinical operations, regulatory affairs, medical writing, and biostatistics, to drive clinical development programs from early-stage trials through to regulatory submissions.

This is an exciting opportunity to contribute to pioneering research in rare disease treatment and make a significant impact on the lives of patients with unmet medical needs.

Key Responsibilities:

• Clinical Strategy & Trial Design :
• Contribute to the design, execution, and analysis of clinical trials focused on rare diseases.
• Develop clinical protocols, amendments, and study-related documents in collaboration with clinical investigators, regulatory experts, and cross-functional teams.
• Provide scientific leadership for study designs and endpoints specific to rare disease populations.
• Data Interpretation & Reporting :
• Analyze and interpret clinical trial data, ensuring scientific rigor and clinical relevance.
• Author and contribute to clinical study reports, investigator brochures, publications, and regulatory submissions (IND/CTA/BLA/NDA).
• Work closely with biostatisticians to ensure appropriate data analysis methodologies are applied for rare disease studies.
• Cross-functional Collaboration :
• Serve as the scientific point of contact for internal stakeholders (clinical operations, regulatory, safety, pharmacovigilance) and external stakeholders (key opinion leaders, principal investigators, patients’ advocacy groups).
• Participate in strategic discussions regarding product development, providing clinical expertise on rare disease populations.
• Collaborate with the regulatory team on clinical development strategy to ensure alignment with global regulatory requirements for rare disease products.
• Patient Engagement & Advocacy :
• Engage with patient advocacy groups and contribute to patient-centric development plans that address the unique needs of rare disease patients.
• Ensure that the patient experience is integrated into trial design and execution.
• Medical Monitoring & Safety :
• Provide medical/scientific oversight during clinical trials, working closely with the safety team to monitor for adverse events and ensure patient safety.
• Participate in data safety monitoring board (DSMB) reviews and other safety oversight activities.
• Regulatory Interaction :
• Support the preparation of regulatory submissions (e.g., orphan drug designation applications, Fast Track, or Breakthrough Therapy designations).
• Participate in meetings with regulatory agencies, presenting clinical data and addressing agency inquiries.
• Innovation & Continuous Learning :
• Stay current on scientific literature and competitive landscape related to rare disease research and treatments.
• Identify new technologies, biomarkers, and methodologies to enhance the clinical development of rare disease therapies.

Qualifications:

• Education :
• PhD, PharmD, or MD in a related scientific field (e.g., genetics, molecular biology, immunology, rare diseases) required.
• Experience :
• Minimum 3-5 years of experience in clinical research, with specific experience in rare disease drug development preferred.
• Proven track record of success in designing and executing clinical trials, ideally for rare or orphan diseases.
• Experience with regulatory submissions for rare disease drugs is highly desirable.
• Technical Skills :
• Strong understanding of clinical trial methodology, biostatistics, and regulatory requirements.
• Expertise in interpreting clinical data and managing clinical projects from inception to completion.
• Familiarity with FDA and EMA guidance for rare disease therapies, including orphan drug designations and accelerated approval pathways.
• Soft Skills :
• Exceptional communication and presentation skills, with the ability to effectively collaborate across multidisciplinary teams.
• Strong leadership and project management capabilities.
• Highly organized with the ability to manage multiple priorities and timelines.
• Ability to work effectively in a fast-paced, dynamic environment and adapt to rapidly changing needs in the rare disease field

Preferred Qualifications:

• Experience working directly with patient advocacy groups and rare disease communities.
• Knowledge of the genetics and pathophysiology of rare diseases.
• Experience with gene therapy, biologics, or precision medicine is a plus.
• Demonstrated involvement in successful regulatory interactions for orphan drug designations, Breakthrough Therapy designations, or similar pathways.