Quality Recruiting. Established Experts.

Senior Medical Director, Rare Genetic Diseases

Reference Number: 2795
Location: MA

Our client is a growing biopharmaceutical company.  They have asked us to assist them in their search for a Senior Medical Director, Rare Genetic Diseases.

The Senior Medical Director, Rare Genetic Diseases will assist with the advancement of our client’s research activities targeting Inborn Errors of Metabolism (IEMs), including several programs currently in the drug development process.

The incumbent will be responsible for the development and execution of clinical research and development programs for the company.  This individual, in conjunction with the Senior Director, Clinical Research will lead the strategic direction of one or more of the company’s clinical programs and be a key driver in drug development.  This position will develop and implement clinical strategies, serve as a liaison between company and clinical investigators and establish credible relationships with opinion leaders, medical directors, and key regulatory officials.

Major tasks and responsibilities will include:

  • Responsible for study design, protocol development, execution, and supervision of clinical studies, especially POC/Phase I studies with a strong focus in the rare or orphan drug areas.
  • Ensures that all clinical studies operate to the highest ethical and safety standards and in compliance with company, GCP and regulatory requirements.
  • Provides key input for strategic development to discovery teams as to the clinical feasibility of targets and small molecules, as well as being involved with the design, conduct, medical management, data interpretation and reporting of clinical trials and developing regulatory strategies.
  • Acts as a thought leader in rare/orphan diseases to drive interpretation and data display.
  • Direct involvement in trial design of Phase I/II research trials targeting rare or orphan diseases.
  • Directly supervises and monitors trial conduct as well as works closely with external medical monitor physicians to assure consistency of conduct across trials.
  • Works closely with all functions of the organization and external partners to manage trial logistics.
  • Identifies opportunities for external collaboration to optimize translational and clinical strategies.
  • Follows important developments and relevant trends in the scientific literature and develops/maintains contacts with external experts to support understanding of the candidate drug effects and to gain strategic insights into the further development and placement of study and the overall program.
  • Develops effective relationships with external providers to ensure successful medical oversight of outsourced studies.
  • Provides clinical assessments during disease/target evaluation, prioritization and selection, identifying novel therapeutics opportunities as well as critical study design and execution challenges.
  • Develops evidence for functional relevance of targets in human disease.
  • Supports qualification of pharmacodynamic/disease markers for assessment of efficacy.
  • Works closely with the project management representative(s) to track trial status.
  • Works closely with study physicians, as well as other project team members/functional areas to ensure regulatory compliance.
  • Writes, as well as provides editorial comments for clinical study reports and manuscripts.
  • Responsible for the scientific quality of all clinical work.
  • Ensures consistent practices with the highest ethical standards in compliance with internal SOPs, local regulations and laws.

We seek candidates with the following qualifications:

  • M.D. or equivalent degree (ideally in Genetics).
  • Board Certification, Board Eligibility or specialist accreditation in rare/orphan diseases; specific experience with genetic metabolic disorders is a plus.
  • A minimum of 5+ years of clinical and managerial experience within rare/orphan disease areas in pharmaceutical trial design and conduct (POC, Phase I/ Phase II) within the pharmaceutical industry, CRO or academia is required.  Experience with trials through NDA strongly preferred.
  • At least 3 years of early clinical research expertise and experience, including experience in designing, monitoring, executing and interpreting clinical trials, including understanding of biostatistics and safety reporting.
  • At least 3 years of translational research expertise and experience in rare/orphan disease development.
  • At least 3 years of experience in drug development, including team leadership and strategic influencing, either in academia working with industry or within industry.
  • Technical (medical and scientific) experience evaluating targets/agents for in-licensing or internal development.
  • Ability to be highly discriminating of potential targets and methodologies.
  • Experience supervising physicians and/or scientific staff in a management or team leader capacity is highly desirable.
  • Experience interacting with U.S. regulatory authorities required.
  • 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.
  • Up to 20% annual travel (domestic and international) is required for this position.


Do you have the skills and experience we seek and want to advance your career with one of the world’s leading companies? If so, please email your resume as a Word attachment to us, reference 2795. No calls or faxes, please. Include your daytime phone number and we will contact you confidentially or reply to your email.