Associate Director, Health Economics & Outcomes Research (HEOR)

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) Associate Director supports 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 a specific therapeutic area of interest. This role contributes to shaping patient-relevant outcomes requirements within the clinical programs, helping ensure alignment with Regulatory, HTA and access needs. In partnership with internal cross-functional and external stakeholders, the Associate Director supports the design and execution of patient-relevant outcomes research initiatives.

The Associate Director builds effective working relationships with cross-functional partners across Value & Access, Commercial, Medical Affairs, Clinical Development, Regulatory Affairs and Patient Advocacy, while supporting engagement with external stakeholders including HTA agencies, payers and patient organizations. This role requires solid expertise in patient-relevant outcomes research and a strong understanding of market access, value and HTA frameworks. The position reports to the Senior Director, HEOR within the Global HEOR team which sits within the Pricing Access Value & Evidence (PAVE) organization.

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.
• Contribute to organizational understanding of payer and HTA requirements related to patient-relevant outcomes and health utility measurement.
• Support the strategic assessment of 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.
• Support patient-relevant endpoint planning and assist with 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.
• Support 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 support presentation and publication of research in peer-reviewed journals.
• Support the assessment of business development opportunities against payer and HTA requirements to identify risks, evidence gaps, and access considerations.

Education & Experience:

• 7+ years of Pharma Industry experience with 4+ years of experience in HEOR roles in pharmaceutical companies or consulting, and at least 2 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.
• Strong understanding of market access, value and global HTA frameworks.
• Experience supporting the development and execution of patient-relevant outcomes strategies to meet payer and HTA requirements.
• Prior experience contributing to 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.
• Ability to collaborate and build alignment across cross-functional stakeholders, including Value & Access, Commercial, Medical Affairs, Clinical Development & Regulatory Affairs.
• Ability to manage multiple initiatives and effectively prioritize in a dynamic, fast-paced environment.
• Developing expertise within the patient-reported outcomes field, with a growing professional network.
• Experience supporting engagement with external stakeholders, including HTA agencies, payers, health economists, clinical experts, and patient advocacy organizations.
• Rare disease experience 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.