Executive Medical Director, Clinical Development - Hemophilia
Location United States, RemoteApply
BioMarin is the world leader in delivering therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. These conditions are often inherited, difficult to diagnose, progressively debilitating and have few, if any, treatment options. BioMarin will continue to focus on advancing therapies that are the first or best of their kind.
BioMarin Clinical Science (CLS) is responsible for overseeing clinical programs across various phases, from proof-of concept to Phase 3 and BLA/NDA/MAA filing. The Clinical Science team provides leadership for clinical strategy and oversight to ensure excellence in clinical trial conduct, data analysis and interpretation, publication preparation, and safety monitoring.
The Executive Medical Director (S/E MD) has a key leadership role in BioMarin, accountable for the successful guidance and execution of the cross-functional investigational program team and deliverables.
Responsibilities include being accountable for ensuring a Clinical Development Plan (CDP) is developed and maintained; acting as a subject matter expert on program clinical strategy and plan for the business; and leading a cross functional Clinical Development Team.
This particular position is for the Clinical Development Team Lead for valoctocogene roxaparvovec (BMN 270), AAV gene therapy for hemophilia A, who will report to VP Clinical Science.
The Executive Medical Director will also mentor team members and establish a team environment that fosters the development of high-quality deliverables; identify, assess and mitigate risks; and drive decision making as it relates to the CDP.
Scientific planning and oversight
- Accountable for ensuring that global CDPs are established and incorporate the appropriate degree of scientific, statistical and regulatory rigor:
- Drive the Clinical Development Team (CDT) deliverables for the creation, review, approval and updating of CDPs
- Assess pros & cons of all potential clinical development paths in collaboration with members of the CDT
- Integrate strategic inputs from all critical functions represented on the CDT to generate “decision-quality” data from clinical studies under its purview; ensure that the CDT references and complies with the Target Product Profile (TPP) and can support product registrations worldwide.
- Accountable for the development of high quality and compliant Clinical Science outputs, such as protocols, IBs, concept sheets, CSRs, and briefing documents
- Anticipate potential challenges and risks to the CDP; lead the CDT to develop mitigation steps and escalate to appropriate forums and/or functional heads, as appropriate. (Note: this effort is meant to complement not replace individual clinical study risk mitigation strategies developed by Study Execution Teams (SETs))
- Review and provide comments on all company-sponsored program-related abstracts, manuscripts and publications of original research and press releases, as requested (This may be delegated to appropriate CDT members as needed)
Governance and Leadership
- Lead and hold the CDT members to be accountable for achieving assigned deliverables within timelines, within budget, and in accordance with BioMarin’s quality standards
- Act as the single point of accountability in driving critical strategic decision making at CDT and resolving any impasse through clear communications and decision-making methodology
- Lead, facilitate and manage CDT meetings, including approval of minutes
- Act as escalation point for issues and move toward resolution
- Align with functional area representatives on the CDT to ensure that appropriate resources are assigned to a given product
- Ensure alignment between CDT and the Core Team (and other CT partner teams), assuming primary responsibility for communication with the Core Team and/or CLS Core Team Representative for strategic aspects of the program.
- Assume overall accountability for clinical development deliverables within agreed upon timelines and budget
- Reconcile CDP deliverable with the TPP and lifecycle strategy
- Lead discussions/presentations to the XDRC on behalf of the CDT. (Note: The CDTL is expected to draw directly upon the expertise of all members of the GCD functions in his/her interactions with the XDRC (i.e. through direct participation), as needed.)
- Inform Core Team and/or CLS Core Team Representative on clinical study execution/progress and highlight any items that impact key aspects of planning and strategy
- Partner with the Core Team and/or CLS Core Team Representative to identify potential lifecycle opportunities
- Escalate issue(s) from CDT or CDT partner teams (along with proposed risk mitigation strategies) that could have an impact on product viability, (e.g., lack of efficacy, unacceptable patient safety), the team’s ability to meet deliverables or timelines, have cross-functional implications or could potentially impact BioMarin’s reputation
- Leverage the decision-making and endorsement functionalities of the Core Team as appropriate, particularly where issues have the potential to shift program direction or have cross-functional impact
- Represent the overall program clinical strategy internally across all levels of the company, and externally to investigators, advisors, KOLs, service providers, business partners and other collaborators. (This responsibility may be delegated to applicable CDT members in certain instances where appropriate and applicable)
- Accountable for acting as program subject matter expert at discussions with governance bodies, such as Cross-functional Drug Review Committee (XDRC) and Product Approval Committee (PAC)
- Accountable for review and distribution of study update reports to management and other key stakeholders (i.e. monthly study reports, presentation at team meetings)
People DevelopmentMentor Clinical Science and CDT team members to foster collaborative and effective working relationship, professional growth and the development of high-quality deliverables
The Executive Medical Director takes the lead in setting the direction for Clinical Science. In doing so, s/he models mastery of several essential CLS core and technical competencies, including communication and collaboration, problem solving, proactivity, regulations and compliance, and external stakeholder engagement. The / Executive Medical Director demonstrates that s/he is:
- Able to articulate alignment between functional initiatives and corporate strategy such that employees, at all levels, fully understand their roles in achieving success
- Recognized as an innovative problem solver through contributions to medical, commercial and corporate strategies
- Able to establish a culture of taking accountability for identifying issues and risks and seizing opportunities; creates open dialogue with all levels of organization to facilitate issue / opportunity identification and resolution
- Demonstrating robust knowledge of GCP guidelines and their impact on each stage of the study and can actively contribute to discussions on GCP compliance issues
- Capable of defending product value proposition with internal and external leaders using sound and extensive scientific and medical rationale
The Executive Medical Director will also demonstrate mastery of relevant clinical and therapeutic area knowledge to support clinical decision making, problem solving, safety surveillance, and as well as collaboration with external physician stakeholders.
BioMarin offers comprehensive career development programming, including guidance, targeted training opportunities, and mentorship programming. A career development pathway for Clinical Science has been defined and is available to employees of BioMarin.
- MD, MD/PhD (or equivalent)
- 6-8 or more years of relevant experience in clinical science/clinical development of genetic diseases, metabolic diseases, specialty care (e.g., hematology), rare diseases, and/or gene therapy desired
- 5 or more years of line management experience preferred
- The Executive Medical Director may manage several direct reports
- The employee will be required to travel 30-40% travel requirement and include weekends.
Internal: Clinical Operations, Pharmacovigilance, Regulatory Affairs, Patient Advocacy, Biometrics, Development Sciences Quality (DSQ), Global Medical Affairs, Legal, Corporate Ethics & Compliance, Corporate Communications, Pipeline Commercialization, Business OperationsExternal: Key Opinion Leaders, Clinical Trial Site MDs and Staff, vendors and suppliers
We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, sexual orientation, national origin, disability status, protected veteran status, or any other characteristic protected by law.