Sr. Director/Director, Medical Affairs, Achondroplasia

Location:  remote, United States Category: Medical Affairs


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’s Research & Development group is responsible for everything from research and discovery to post-market clinical development. Research & Development involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with rare diseases. Come join our team and make a meaningful impact on patients’ lives.


This position is responsible for providing regional medical leadership to achondroplasia in North America. This individual will be a key member of the team charged with creating a product-based franchise around Vosoritide. This individual has a regional scope of responsibility in the Medical Affairs function and is expected to excel at aligning with colleagues across matrix/functions proactively.

This individual will interface externally with healthcare professionals, clinical investigators, regulatory agencies amongst others as well as with internal BioMarin departments providing medical insights and supporting the product development strategy. Within the latter ones, this person’s responsibility will also include providing input and collaborating on other cross-functional activities and initiatives, from Commercial (Brand Management, Market Access, Sales) Patient Advocacy, Regulatory, Clinical Sciences and other departments, aligning with colleagues across matrix/functions proactively.

The person holding this position will be the scientific reference at the regional level, representing the department and the company at key internal and external activities and events. 



  • Responsible for direct management of projects and matrix collaboration within and outside of the department.
  • Responsible for the development of a Regional Medical Affairs Plan for the respective product.
  • Responsible for direct interaction with investigators, KOLs and the research community to support company initiatives.
  • Responsible for representing BioMarin at scientific, clinical and business development meetings.
  • Promote effective communication between internal and external groups regarding Medical Affairs initiatives.
  • Act as a key member of different internal committees ensuring NORAM input is incorporated into the global strategy.
  • Interact in collaborations with strategic partners
  • Responsible for management and medical input in support of key medical messaging such as core claims document, product information, etc. S/he will contribute to promotional activities for BioMarin products being done in accordance within the respective regulatory and compliance requirements.
  • Responsible for providing medical input into development, execution and interpretation of clinical protocols.  This includes review of draft independent research protocols, reports and manuscripts. May need to assist in critically evaluating study results in statistical and final reports and ongoing reviews with study investigators, resolving problems arising in the course of clinical investigations and preparing reports to the management and medical community.
  • Direct medical writing activities associated with Medical Affairs to ensure accurate execution of product dossiers, product related slide decks, standard response letters, FAQs as well as corporate abstracts, posters, oral presentation and manuscripts when appropriate.
  • Serve as medical and technical resource for clinical issues raised by internal and external collaborators, investigators, consultants and contract resources
  • Ensure compliance with policies, SOPs, Code of Business Conduct and Corporate Social Responsibility, as well as relevant legislation and regulations


  • Ability to work proactively and effectively, with exceptional creative problem solving skills.
  • Able to thrive in an ambiguous and demanding environment, with high capacity for effective relationship building and teamwork.
  • Flexibility and adaptability. Sensitivity to a multicultural environment.
  • Excellent project management skills, with a drive for results coupled with the highest medical and ethical standards. High scientific curiosity.
  • Excellent written and oral communication skills. Able to convince, inspire and motivate people with a variety of backgrounds.
  • Commercially astute



Advanced Degree (M.D. or equivalent / Ph.D., or both) in Medicine and/or a medical sciences field. Board certification desirable, but not required. Experience in paediatrics highly desirable, either in a pharmaceutical or clinical research or a clinical environment.

Very strong Medical Affairs experience is a must.


  • Strong industry experience, especially in Medical Affairs (≥ 7 years). Previous experience in orphan diseases is desirable but not required.
  • Knowledgeable and current in GCP guidelines and compliance rules in US and Canada. Clinical trial and publication experience is desirable.
  • Launch experience is desirable.
  • Demonstrate excellent medical affairs skills
  • Working knowledge in the therapy area is desirable but not a must.
  • Willingness to travel (also international)




Will be expected to interact on a daily basis with various members of regional commercial team, NORAM team and Developmental Sciences team, Market Access team, Market Planning team, Clinical Sciences team. S/he will be required to interact closely with Global Medical Affairs and colleagues internationally.


Will be required to interact with, inter alia, KOLs, payer bodies, patient associations, in collaboration with other departments.

Note:  This description is not intended to be all-inclusive or a limitation of the duties of the position.  It is intended to describe the general nature of the job that may include other duties as assumed or assigned.

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.