Medical Lead Hemophilia A, Sr. Director Clinical Development
Employment Type: Full-Time
Industry: Executive Management
Lead Hemophilia A, Senior Director Clinical Development
uniQure is dedicated to bringing innovative therapies or unmet medical needs to patients. In doing so each function in uniQure is directly or indirectly responsible for the lives of patients. This responsibility is part of our day-to-day working lives by (i) living up to high quality standards and (ii) by recognizing the sense of urgency in areas of high medical need.
Medical Lead Hemophilia A, Senior Director Clinical Development will serve a key role of providing expert medical input to the company's ongoing portfolio of gene therapy-based rare diseases programs with a focus and ownership over the hemophilia A program. This will include the design, implementation, execution, medical monitoring and reporting of requisite clinical studies at all phases of pipeline development.
Medical Lead Hemophilia A, Senior Director Clinical Development, will report to the Vice President, Clinical Development Hemophilia, and collaborate closely with the CMO, to identify current and future pipeline needs for uniQure moving forward.
A summary of the responsibilities includes the following:
- Serve as primary Medical leader in the matrixed Core Project Teams (with Project Management, Clinical Operations, Medical Affairs, Regulatory Affairs, Database Management & Programming, Biostatistics, Commercial, and Pre-Clinical)
- Design, development and implementation of IND for hemophilia A program
- Proactively identify program risks, and create and implement mitigation strategies
- Serve as primary liaison with key opinion leaders, and potential clinical sites
- Handle day-to-day clinical, medical, scientific questions from various internal and external sources
- Collaborate cross-functionally in the preparation of the IND, CDP, clinical protocols, investigator brochures and target product profiles
- Participate in the ongoing interpretation and analysis of pre-clinical and clinical trial results including pharmacokinetics, pharmacodynamics, laboratory, imaging, and antibody data, review and approve clinical trial coding; safety monitoring in collaboration with pharmacovigilance expert
- Participate in and lead investigator meetings
- Participate as medical lead in various scientific advice meetings and other interactions with the EMA and FDA, or other Regulatory bodies
- Continual review and analysis of the pertinent medical (disease-specific) literature
- Preparation and/or delivery of abstracts, posters, and slide presentation at external Conference Symposia, and manuscripts for peer-reviewed journals
- A key role will be to collaboratively interface with the company's Discovery Research department to bring a medical disease perspective and analysis of unmet need to new gene therapy product candidates (either in-licensed or internally developed).
- Focused medical review of emerging pre-clinical product candidates at Stage Gate #1, with design of an early high-level clinical development plan (CDP)
- Collaborate with the commercial part of the organization as needed for perspective on disease incidence and prevalence of orphan indications and to ensure development programs are aligned with commercial expectations
- With Business Development, participate as medical expert in Company due-diligence exercises on potential in-licensing of pipeline gene therapy programs
- Other duties as assigned, including support as necessary with deliverables for hemophilia B program
The successful candidate we hire will be a sophisticated leader who has the right degree of drive and independence with a very strong track record in drug development. S/he will have the following mix of personal and professional characteristics:
- Medical education (MD) and clinical development industry experience required
- Strong scientific, medical and regulatory expertise, knowledge and experience in hemophilia, gene therapy, or biologics required
- Experience leading or collaborating on IND preparations and/or approvals preferred
- Although not a prerequisite, special consideration in title, grade, and responsibility will be given to candidates with the following backgrounds: (1) MD physicians with PhDs or with strong scientific postdoctoral research experience; (2) Specialty experience or training in hematology
- Excellent working knowledge of applicable global, regional, local clinical research regulatory requirements; i.e. Good Clinical Practice (GCP) and International Conference of Harmonization (ICH) guidelines, SOPs.
- Proven ability to meet strict deadlines; manage competing priorities and changing demands
- Sound organization and time management skills.
- Good attention to detail and accuracy whilst maintaining high quality GCP standards.
- Ability to follow instructions/guidelines, work independently and proactively on own initiative.
- Ability to be flexible and receptive to changing process demands
- Willingness and aptitude to learn new skills, and support activities across service lines.
- Ability to establish and maintain effective working relationships with coworkers, managers and clients - many with diverse cultural backgrounds
- Skill required to create buy-in from functional groups and departments
- Outstanding communication skills; comfortable representing the company and vision to internal and investor communities.
- Confident, independent and decisive; a self-starter, proactive and results-oriented with high performance standards. Passionate, persistent and tenacious. Strong ability to prioritize and operate with a sense of urgency.
- \"Hands-on\", \"roll-up-the-sleeves\" leader who understands the need for involvement at all levels of activity within a small company environment. Extremely bright with a highly strategic nature but also balanced with a biotech mindset and an attitude that gets things done.
- Understanding of and willingness to meet applicable regulatory, quality and compliance standards. Trustworthy with highest integrity, committed to ethics and scientific standards.
- Willingness to travel (domestic/international, approximately 20% time) to meet investigators, attend conferences, and participate in advisory boards.
Associated topics: design, engineering, manufacture, product, product manager, production, program manager, quality
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