Medical/Senior Medical Director, Rare Genetic Diseases
Middlesex County, MA
Medical Director/Senior Medical Director Rare Genetic Diseases (RGD)
Agios (agios.com) is a biopharmaceutical company committed to applying our scientific leadership in cellular metabolism to transform the lives of patients with cancer and rare genetic diseases. We are growing rapidly with an active research and discovery pipeline across both therapeutic areas. Agios has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development.
We are currently seeking an innovative Clinical Development leader to help drive our rare disease development program. Reporting into our Vice President for Rare Genetic Diseases (RGD), this person will advance Agios research activities and pipeline targeting Inborn Errors of Metabolism (IEMs).
The Medical Director/Senior Medical Director will be responsible for the development and execution of clinical research and development programs for Agios Pharmaceuticals including the leadership of the pivotal trials, NDA and filing activities. This role will give strategic medical and tactical input to the RGD discovery teams from early discovery to development candidates to IND submission. This position will serve as a key liaison between company and clinical investigators and establish credible relationships with opinion leaders, and regulatory officials.
Specific Strategic and Tactical Responsibilities:
- Direct involvement in trial design of Phase I/II/III research trials targeting rare or orphan diseases.
- Directly supervise and monitor trial conduct as well as work closely with external medical monitor physicians to assure consistency of conduct across trials. Work closely with all functions of the organization and external partners to manage trial logistics.
- Provide medical input to global regulatory plans and regulatory meetings in partnership with regulatory affairs.
- Follow important developments and relevant trends in the scientific literature and develop/maintain contracts with external experts to support understanding of the candidate drug effects and to gain strategic insights to the further development and placement of study and overall program.
- Develop effective relationships with external providers to ensure successful medical oversight of outsourced studies.
- Provide clinical assessments during disease/target evaluation, prioritization and selection, identifying novel therapeutics opportunities as well as critical study design and execution challenges.
- Develop evidence for functional relevance of targets in human disease.
- Support qualifications of pharmacodynamic/disease markers of assessment of efficacy.
- Work closely with the project management representative(s) to track trial status.
- Work closely with study physicians, as well as other project team members/functional areas to ensure regulatory compliance.
- Review written materials and provide editorial comments for clinical study reports and manuscripts.
- Responsible for the scientific quality of all clinical work.
- Ensure consistent practices with the highest ethical standards in compliance with internal SOPs, local regulations and laws.
- M.D. or equivalent degree (desirable areas include Genetics or Hematology).
- Board Certification, Board Eligibility or specialist accreditation in internal medicine or pediatrics; specific experience with genetic metabolic disorders desirable.
- A minimum of 3+ years of clinical and managerial experience within pharmaceutical trial design and conduct (POC, Phase I/Phase II) within the pharmaceutical industry CRO or Academia is required. Experience with trials though NDA strongly preferred.
- Translational research expertise and experience in rare/Orphan disease development desirable.
- Excellent interpersonal and public speaking skills are required for this high visibility position.
- Demonstrated ability to work in a matrix environment with cross-functional teams.
- Demonstrated leadership experience.
- Approximately 10% annual travel (domestic and international) is required for this position.