Description
*BioMarin has a preferred candidate identified, however, all others are encouraged to apply*
Who We Are
BioMarin is a leading rare disease biotechnology company focused on genetically defined conditions.
Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options. More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease, expanding our ability to reach more people living with rare genetic conditions.
Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world-class manufacturing capabilities. At the heart of BioMarin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.
About Worldwide Research and Development
From research and discovery to post-market clinical development, our R&D engine 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.
The US Medical Director, Skeletal Conditions (SC) is a critical strategic and execution focused role on diseases and products within the Skeletal Business Unit. The role reports into the Head of US Medical, SC and represents US Medical Affairs on core cross-functional teams, key governance meetings and review committees and will be responsible for communicating updates to the US Medical team.
The role partners with the Global Medical Leads (GMLs) to develop the medical plans for therapeutic products for patients with skeletal related disorders and ensures the implementation and alignment of medical activities across the US. In addition, the US Medical Director will work collaboratively with Global Medical Affairs functions to execute on data generation and drive US skeletal conditions publication planning and support medical communications.
The US Medical Director will ensure that medical activities delivered across the region are aligned to the overall Global Strategy and objectives for the Skeletal Business Unit. The US Medical Director will partner with US Medical Affairs, US Marketing and Patient Advocacy to ensure scientifically accurate content development, lead and support advice-seeking activities, and assist with training development and delivery as needed.
The US Medical Director will be responsible for establishing and maintaining strong working relationships and partnerships with external Global and National KOLs, US Skeletal Dysplasia Centers and Patient Advocacy Organizations as appropriate.
Key Responsibilities
Disease Area Strategy & Execution
- Develop and implement the US Medical Plan and lead US medical activities in coordination with Medical Affairs, Commercial and Regulatory colleagues
- Ensure that the medical activities are aligned to the Global Medical Strategy for the disease area and product(s) and adapted to fit US regional needs
- Accountable to deliver clinical and scientific input from the US region into the development of plans at Global Medical planning teams and other cross-functional teams as required
- Ensure adherence to US regulatory and clinical practice guidelines in supporting development and marketing of BioMarin compounds for SC
- Act as the US product and disease area expert and primary contact point for all compound or disease related issues both within Medical Affairs and for all other BioMarin departments
Medical Engagement
- Develop and strengthen key US medical expert relationships to expand potential collaborative opportunities aligned with Global and US Medical strategy
- Contribute to the development of the Medical Engagement strategy with the Medical Engagement Lead ensuring inclusion of key stakeholders, development of tools to support execution, and mechanisms are in place to measure impact
- Develop and lead US scientific advice seeking events, coordinating with insights from the field, and delivering outputs through appropriate channels and support Global scientific advice seeking events as appropriate
- Collaborate with Scientific Communications on relevant publications related to skeletal related disease states and management
- In partnership with Global Integrated Evidence, develop and lead US SC collaborative research proposals, propose and support US RWE and HEOR analyses, and US focused sub-analyses from ongoing and completed clinical research studies that support the Medical strategy
- Develop and lead US scientific educational programs and events for healthcare providers in partnership with Medical and cross functional teams
- Review US Medical Information requests, standard response documents and FAQs
- Contribute US perspective to scientific messaging discussions
Launch and Pre-Launch Excellence
- Act as a key member on the Medical SMART team to provide medical recommendations and collaboration with the Clinical Development Team, Integrated Evidence Team, Asset Team and Integrated Brand Team
- Develop US launch plans in collaboration with Medical Affairs and Commercial colleagues and execute on the US specific tactics in support of global product launches
Filing / Regulatory
- Assist in preparation and review of relevant product BLAs or sBLAs and other regulatory documents including protocols, protocol amendments and briefing materials
Post Marketing Commitments
- Lead for development/design of US post-marketing commitments, registries and studies falling under Medical Affairs scope in coordination with Global Study Operations
- Active member of Study Engagement Teams (SET) related to ongoing PKU post-marketing studies
- Collaboration with pharmacovigilance to evaluate safety issues associated with products and promotional activities for BioMarin products in accordance with respected regulatory requirements
- Post Marketing Cross Functional Leadership
- Represent Medical Affairs on relevant US Commercial teams including Brand Team
- Represent Medical Affairs in the Promotional Review Board (PRB) and Medical Material Review Committee (MMRC) to ensure maximum effectiveness of materials while adhering to the company's ethical standards and relevant Codes of Practice for the region
- Provide medical oversight for Commercial symposiums and advisory boards
External Research Projects
- In partnership with Global Integrated Evidence, develop and lead US SC collaborative research proposals, propose and support US RWE and HEOR analyses, and US focused sub-analyses from ongoing and completed clinical research studies that support the Medical strategy
- Presentation of US external research project proposals to SMART and External Research Committee for endorsement
- Assist and collaborate with internal and external stakeholders in critically evaluating external research protocols, study results for statistical and final reports; Ongoing reviews with study investigators, resolving problems arising in the course of clinical investigations
Training
- Responsible for clinical and scientific training, including disease and product related training events, for US Medical and cross-functional stakeholders in collaboration with Medical and Commercial Learning and Development
Desired Skills
The US Medical Director is expected to exhibit mastery-level understanding of multiple competencies, including communication, scientific knowledge, medical diagnostics and management, as well advances skills in strategic thinking, problem solving, business acumen and drug development, regulatory and study design.
The Medical Director must be able to:
- Be recognized as a highly capable facilitator of information sharing across all levels of the organization with ability to explain complex, challenging topics across various audiences
- Actively foster and maintain product strategy working relationships with key influential stakeholders inside and outside of BioMarin
- Be the US subject matter expert, demonstrating deep medical, technical and scientific knowledge for relevant BioMarin therapeutic areas
- Demonstrate understanding of cross-functional inter-dependencies across the drug development life cycle
- Ability to support study design and protocol development for pre and post marketing studies
- Demonstrate strong leadership skills
- Work independently and collaboratively within diverse cross functional teams
- Show strong organizational skills with the ability to prioritize and manage multiple time-sensitive projects simultaneously
- Demonstrate business acumen and analytical thinking skills
People Management
- The US Medical Director may have the opportunity to manage direct reports
Education and Experience
- MD, PhD, or PharmD, or other advanced life science degree required
- 5 or more years of relevant experience in clinical science, medical affairs, and/or drug development required
- Previous experience as an Associate Medical Director/Medical Director within Medical Affairs strongly preferred
- Skeletal conditions knowledge/clinical experience ideal
- Prior experience in orphan drugs, rare diseases and genetic disorders strongly preferred
- Experience working in cross functional teams at the brand or disease area levels in country, regional or global organizations preferred
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.
Equal Opportunity Employer/Veterans/Disabled
An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.
Ireland Safety Responsibility
Support the organisation’s commitment to environmental protection, injury prevention, and continual improvement of EHS management systems. Actively contribute to a safe and environmentally responsible workplace by identifying hazards, reporting incidents and participating in EHS initiatives and training.
