We are seeking a talented, independent, and motivated clinician with interest in clinical research to help design, implement, and drive global clinical development for the company’s product candidate which has the potential to drastically improve the lives of patients with ultra-rare, life-threatening, and devastating genetic disorders. This role is a unique and exciting opportunity to join a company at a key inflexion point in its history. The selected candidate will be at the forefront of changing the standard of care and improving the treatment paradigm for patients with significant unmet need.
Essential Duties and Responsibilities:
- Aid in developing the clinical strategy and integrated development plan for our program and future programs in rare diseases, lead the execution of clinical trials, and work cross-functionally to implement, manage, interpret and communicate the results of clinical trials
- Be responsible for the clinical content of all clinical and regulatory documents, including protocols, INDs, CTAs, investigator brochures, CRF’s, and annual IND reports
- Active lead in the development of Study Protocols, Investigators Brochures, Clinical Development Plans and other key study documents in conjunction with other line functions
- Lead and supervise clinical trials, collaborating closely with the Clinical Operations team, and will be accountable for all deliverables of clinical trials under his/her direct responsibility
- Responsible for analysis of clinical data, including safety monitoring in collaboration with pharmacovigilance
- Develop and maintain relationships with key opinion leaders and PIs.
- Organize and present at relevant clinical advisory boards, DMC’s and medical/scientific meetings
- Help ensure Clinical Study Team compliance with FDA, EMEA, ICH and GCP guidelines and internal SOPs
- Maintain a high level of clinical and scientific expertise in the disease area(s) by reviewing the literature, attending medical/scientific meetings, and through personal interchanges with consultants, thought leaders, investigators and internal stakeholders
- Support medical affairs to help identify and effectively interact with thought leaders to engage in scientific and medical exchange as well as understand unmet medical needs and data gaps
- Travel, as needed, nationally and internationally
- MD, DO, or equivalent with completed residency in pediatrics or internal medicine and experience in rare diseases
- Fellowship training in cardiology, neurology, or endocrinology is a plus.
- 5-10 years clinical research experience in an academic or industry setting, with experience in analysis of research data; working knowledge of biostatistics and pharmacokinetics; working knowledge of GCP, scientific and clinical research methods and clinical study design, regulatory requirements for clinical studies
- Pharmaceutical/Biotech industry experience, including time managing clinical trials and/or clinical development program(s)
- Excellent oral and written communication skills
- Ability to work collaboratively in a fast-paced, team-based matrix environment and to function independently as appropriate