Senior Director, Health Economics & Outcomes Research (HEOR) in Vauxhall

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.

The Health Economics & Outcomes Research (HEOR) Senior Director leads asset-specific payer evidence strategies, including patient-relevant outcomes strategy, to generate differentiated evidence, strengthen the payer value proposition, and optimize access for BioMarin assets across all therapeutic areas of interest. The role is responsible for shaping patient-relevant outcomes requirements within the clinical programs, ensuring alignment with Regulatory, HTA and access needs. In partnership with internal cross-functional and external stakeholders, the Senior Director leads the design and execution of patient-relevant outcomes research initiatives.

The Sr. Director cultivates strong partnerships with cross-functional partners across Value & Access, Commercial, Medical Affairs, Clinical Development, Regulatory Affairs and Patient Advocacy, while engaging with external stakeholders including HTA agencies, payers and patient organizations. This role requires deep expertise in patient-relevant outcomes research and a strong understanding of market access, value and HTA frameworks.

Responsibilities:

• Lead the development, execution and communication of payer evidence strategies aligned with brand and Value & Access priorities for early-stage pipeline assets.
• Collaborate closely with Value & Access, Commercial, Medical Affairs, Clinical Development & Regulatory Affairs & Patient Advocacy organizations.
• Drive organizational understanding of payer and HTA requirements related to patient-relevant outcomes and health utility measurement.
• Drive the strategic assessment for patient-relevant outcomes requirements for HTA and access to support differentiation and value demonstration.
• Ensure payer evidence requirements are incorporated into clinical development plans to enable optimal patient access.
• Lead the development of cross-functionally aligned, asset-specific patient-relevant outcomes strategies informed by disease conceptual models.
• Lead patient-relevant endpoint planning and provide oversight of implementation within clinical trials.
• Lead early Joint Clinical Assessment (JCA) strategy development, including assessment of access implications and trade-offs across markets.
• Assess payer evidence gaps and inform future integrated evidence planning for early-stage pipeline assets.
• Manage and partner with external partners in the execution of asset-level patient-relevant outcomes strategies, including PRO development and validation, health state valuation studies (time trade-off or standard gamble), and patient preference research, as appropriate.
• Lead scientific interactions with HTA agencies, particularly scientific advice (e.g., EU HTA JSC, NICE, CADTH), including preparation of briefing materials and evidence packages related to PRO endpoints, MCIDs, etc.
• Support scientific interactions with regulatory agencies and patient advocacy organizations.
• In partnership with the Integrated Evidence Teams (IET), lead the exploration, analysis and interpretation of patient-relevant outcomes data from clinical trials to generate insights, and strengthen value propositions for HTA, payer and other stakeholders.
• Shape strategic scientific communication plans, and lead presentation and publication of research in peer-reviewed journals.
• Assess business development opportunities against payer and HTA requirements to identify risks, evidence gaps, and access considerations.

Education & Experience:

• 12+ years of Pharma Industry experience with 7+ years of experience in HEOR roles in pharmaceutical companies or consulting, and at least 5 years of direct experience with patient-relevant outcomes research.
• Advanced degree in health economics, outcomes research, epidemiology, public health, health services research, pharmacy, or a related scientific discipline.
• Deep understanding of market access, value and global HTA frameworks.
• Demonstrated experience developing and executing patient-relevant outcomes strategies to meet payer and HTA requirements.
• Prior experience in the evaluation, development, and validation of PRO measures to support regulatory, payer, and HTA decision making.
• Experience supporting HTA submissions, value dossiers, and patient-relevant briefing books.
• Proven ability to influence and drive alignment across senior cross-functional stakeholders, including Value & Access, Commercial, Medical Affairs, Clinical Development & Regulatory Affairs.
• Ability to lead multiple complex initiatives and effectively prioritise in a dynamic, fast-paced environment.
• Recognised leader within the patient-reported outcomes field, with a strong professional network and external visibility.
• Experience engaging with external stakeholders, including HTA agencies, payers, health economists, clinical experts, and patient advocacy organizations.
• Rare disease experience preferred.
• Prior people leadership experience, including talent development preferred.

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, colour, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.

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.