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Principal Clinical Director - Clinical Sciences – Hemophilia/Hematology

Location United States, Remote

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 Principal Clinical Director [PCD] is responsible for leading important initiatives and partnering with key functions to further the clinical development strategy, both within and beyond Global Clinical Development. She/He is a key member of the Clinical Sciences team and charged with supporting the hematology/gene therapy franchise on an accelerated timeline. This individual has a global scope of responsibility as the Clinical Sciences Lead for valoctocogene roxaparvovec (BMN 270), working closely with the Clinical Development Team (CDT) Lead, and provide leadership across matrix/functions pro-actively.

As the Clinical Sciences Lead, the PCD will provide crucial input on the direction, design and planning, execution and interpretation of clinical trials, as well as oversight of individual Medical Monitors for 1 or more clinical studies.

Responsibilities include providing feedback on the Clinical Development Plan with the CDT and Core team, subject matter expertise in the development of concept sheets and protocol designs beyond Phase 1, preferably Phase 0-4, review of CSRs, ICFs, SAPs, Safety Reviews, including handling of AEs, SAEs, SUSARs, and substantial amendments, as needed.  In addition, the individual may be responsible for selecting, leading and managing Clinical Advisory Boards, DRBs, and DMCs.

The PCD has to provide input to Investigators' Brochures, support Regulatory Affairs in interactions with regulatory agencies worldwide and responses to queries related to PIs, as well as help conceptualize the best pathway towards product approval.

Other critical supportive roles include support of Clinical Operations in site selections and recruitment adherence to GCP and all aspects of study conduct; Medical Affairs in collaborative projects/studies, development of medical education tools, and training; Business Development and Marketing in product launches across geographies.  There will be opportunities to represent Bio Marin at speaking engagements, advisory boards, symposia, and/or Congresses, as well as internal strategy discussions.

In addition to providing critical contributions for drug development, the incumbent will cultivate a close working relationship with Global Medical Affairs in order to help guide medical and strategic research efforts to better meet unmet medical needs. This also includes providing critical clinical and scientific input into the resolution of issues, when requested, arising in safe and effective utilization of these complex biological products.


  • This position reports directly to the Global Clinical Development Hematology Franchise Lead, BioMarin Pharmaceutical Inc.
  • The candidate is responsible for interpreting and evaluating results of research and making recommendations.
  • She/He is responsible to see that the design and conduct of studies under her/his management are carried out with the highest medical and ethical standards with national government regulations including the U.S. FDA.
  • Maintenance of expert scientific and medical knowledge is achieved, among other means, by attendance and participation at medical meetings and seminars and review of pertinent current medical and scientific literature.


  • This position requires a Medical Degree (MD).
  • Candidate should have considerable experience within drug development beyond Phase 1/2; therapeutic area expertise in Hematology/Hemophilia required.
  • Strong academic/teaching background preferred.
  • Candidate must have demonstrated originality in problem solving as applied to drug development and have strong interpersonal skills, leadership capabilities and strong organizational skills.
  • She/He must have demonstrated success in leading both internal and cross-functional project teams and can establish credibility across a range of relevant scientific and clinical 'issues, and be able to generate and communicate a comprehensive body of clinical data related to assigned molecule/drug.
  • The candidate should have both strong presentation and speaking skills.

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We treat rare diseases, and that gives us rare opportunities. Chris Brodeur, Director, Corporate Compliance
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