Mirum focuses on finding and developing drugs for difficult to treat liver diseases. Our lead program is focused on two severe liver diseases in children, Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). Both conditions can result in severe itching, liver damage and a need for liver transplant. Based on a wealth of data from clinical trials spanning multiple years and involving more than one hundred patients, we believe our approach can address the underlying cause of symptoms and liver damage. Our goal is to give families their lives back.
The Medical Director will join the Clinical Development group and support deliverables for our clinical trials and programs associated with the clinical development of molecules in the stages of clinical development. The optimal candidate will have the desire to proactively participate within a cross-functional team of internal clinical, regulatory, scientific, operational and commercial colleagues and have the ability to interact with external experts, investigators and government agencies. This role is instrumental in supporting the growth of Mirum's pipeline in rare liver diseases. The successful candidate will have hands-on responsibility designing and executing clinical trials, in addition to developing and implementing clinical strategies. In addition, the candidate will ensure data quality and delivery of outputs including but not limited to, publications and clinical study reports. The successful candidate will participate in health authority interaction activities to support NDA submissions, and will serve as a medical monitor, including evaluation of the safety, pharmacology and efficacy in ongoing and completed studies in close collaboration with the clinical operations team. As the medical lead of a program, the successful candidate is expected to work closely and in collaboration with the regulatory, clinical operations, biometrics and medical writing teams.
- Leadership and management of all aspects of complex, global development programs, including clinical trials focused on multiple indications, from phase 1 through post-commercialization.
- Primary point of contact for medical inquiries from sites, CRO and site monitors; answer or triage questions as appropriate integration of clinical/regulatory/manufacturing strategy into global product development strategy.
- Primary liaison for internal Clinical Operations for site selection, site start-up activities, study management and closeout activities, etc.
- Support the creation of and/or review of clinical slides for internal and external meetings (i.e. Investigator Meetings, SIVs, Advisory Boards, Steering Committee Meetings, etc.)
- Assist in authoring key clinical study documents, including Protocols, Clinical Study Reports, Investigator Brochures, Regulatory Authority briefing books, Development Safety Update Reports etc.
- Conduct literature searches, draft background sections, and ensure that standardized disease/molecule specific language in incorporated (i.e. eligibility and dose modification criteria).
- Evaluate medical rational in new indications, discuss proof-of-concept and registration trial designs with external experts.
- MD, with a minimum of 2 years+ in Clinical Development.
- Experience in Liver or rare disease preferred.
- Knowledge and understanding of regulatory requirements for product approval and compliance in US and Europe.
- Demonstrated accountability for delivery on complex development plans.
- Experience working on cross-functional team.
- Experience with basic statistical principles and techniques of data analysis, interpretation and clinical relevance.
- Experience presenting data to regulatory agencies and advisory committees.
Knowledge, Skills and Abilities:
- Ability to understand and communicate risks, as well as develop and execute contingency plans.
- Ability to think creatively and develop and execute in a fast-paced environment.
- Detail oriented with exceptional organizational skills.
- Effective communicator with strong verbal and written skills.
- Ability to identify issues, analyze situations and provide effective solutions.
- Solid understanding of the clinical development process.
- Knowledge of applicable regulatory.