BIOMARIN

Who We Are BioMarin is a global biotechnology company that relentlessly pursues bold science to translate genetic discoveries into new medicines that advance the future of human health. Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Using our unparalleled expertise in genetics and molecular biology, we develop medicines for patients with significant unmet medical need. We enlist the best of the best - people with the right technical expertise and a relentless drive to solve real problems - and create an environment that empowers our teams to pursue bold, innovative science. With this distinctive approach to drug discovery, we've produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options. SUMMARY DESCRIPTION The Health Economics & Outcomes Research Associate Directorwill be responsible for the access evidence planning and execution for a program within BioMarin's Enzyme Therapies business. The individual will lead the global compound access evidence strategic plan and tactical execution in alignment with the compound value & access strategy. They will: Plan, develop, and disseminate HEOR evidence to demonstrate the value of BioMarin's assets. Actively influence and shape evidence generation delivered by cross-functional partners (i.e. Clinical Sciences, Medical Affairs) to ensure that they meet the needs of Market Access. Be an acknowledged subject matter expert supporting country and regional market access leads with evidence communication to external stakeholders including payers and health technology assessment (HTA) agencies. The role is part of the Global HEOR team which sits within Global Value Access & Strategic Pricing. It will include a high degree of cross-functional collaboration with medical affairs, regulatory affairs, clinical sciences, data sciences, patient affairs and with regional and local market access teams. The role will also involve liaising with external stakeholders including payers, HTA agencies, clinicians and patient groups. RESPONSIBILITIES Market access evidence strategy Lead the global compound access evidence plan in support of the access strategy. Provide the market access voice in global cross-functional evidence forums including the Value & Access Team and the Integrated Evidence Team. Become a subject matter expert in value demonstration methods and communication. Market access evidence generation Lead HEOR evidence generation, synthesis and dissemination. Project manage vendors to deliver high quality deliverables on time and on budget. Review and influence clinical and medical activities including trial design, endpoints and analytical approaches to ensure they meet payer needs. Lead secondary analyses of clinical data to address market access needs. Country access and reimbursement support Interpret and communicate evidence to internal stakeholders including country and regional market access teams and cross-functional partners. Support country access teams with adaptation of materials and review/sign-off of reimbursement submissions. Represent BioMarin at external meetings with payers and HTA agencies as required. EXPERIENCE 7+ years of HEOR experience obtained within the pharmaceutical industry. Demonstrated ability to deliver a range of HEOR evidence projects including cost-effectiveness models, budget impact models, systematic literature reviews and real-world data analysis. History of working cross functionally, influencing, and gaining alignment across Market Access, Commercial, Medical, Clinical and Regulatory departments. Comfortable interacting with external decision makers, health economists, clinical experts and patient organizations. Sound knowledge and experience of different payer architypes and appreciation for the broader market access environment - global or multi-country experience preferred. Excellent verbal and written communication, collaboration, influence, strategic, analytical and problem-solving skills are required. Working knowledge of common statistical packages and reference management software preferred. EDUCATION Postgraduate qualifications (Masters or PhD) in public health, health economics, epidemiology or statistics 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.

Who We Are BioMarin is a global biotechnology company that relentlessly pursues bold science to translate genetic discoveries into new medicines that advance the future of human health. Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Using our unparalleled expertise in genetics and molecular biology, we develop medicines for patients with significant unmet medical need. We enlist the best of the best - people with the right technical expertise and a relentless drive to solve real problems - and create an environment that empowers our teams to pursue bold, innovative science. With this distinctive approach to drug discovery, we've produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options. About Technical Operations BioMarin's Technical Operations group is responsible for creating our drugs for use in clinical trials and for scaling production of those drugs for the commercial market. These engineers, technicians, scientists and support staff build and maintain BioMarin's cutting-edge manufacturing processes and sites, provide quality assurance and quality control to ensure we meet regulatory standards, and procure the needed goods and services to support manufacturing and coordinating the worldwide movement of our drugs to patients. SUMMARY DESCRIPTION The Director of Launch Excellence is a new role in the Product Supply Chain Management organization. Director, Launch Excellence will ensure enterprise governance and frameworks for new product launches and artwork and labelling are implemented with quality and timeliness to elevate BioMarin's launch discipline. Reporting to the Executive Director, Product Supply Chain Management, the Director, Launch Excellence will partner closely with Product Teams and cross-functional launch teams to develop launch disciplines and establish consistent tools, processes, and KPI dashboards, ensuring launch excellence against specific business deliverables. The ideal candidate will have strong leadership and team development skills, a deep understanding of Product Launch (New Product Introduction), Project Management and Artwork & Labelling with demonstrated ability to drive continuous improvement initiatives. Note that individual responsibilities may include all or part of responsibilities defined below depending on individual experience and abilities and may change to best support on going business needs. RESPONSIBILITIES Mange team to drive launch processes, disciplines, and toolkits including blueprints, frameworks, archetypes, playbooks, governance models that are standardized and utilizes across all products and launch teams. Create communities of practice across for upcoming launches to be able to gather, aggregate and share best practices and cross-functional perspectives across teams, ensuring that teams are building on launch and artwork and labelling learnings across the organization Manage a consistent and robust product launch process and governance as a 'control tower' structure to provide oversight and to ensure consistency of tracking and incorporating disciplines across launches Work with internal launch stakeholders to ensure launch process management and successful completion of launch readiness tasks (standardized approach across business units - including Blueprint / Playbook, Governance, and Dashboards /KPIs. Work with cross-functional team to understand the impact of changing regulations, product requirements and new market entry regarding artwork and labelling. Coordinate Launch Readiness Review Assessments across launch brands to ensure strategic and tactical plans are reviewed and aligned with CtF (commit to file) and CtL (commit to launch) dates with BU and TOPs leadership Establish reporting dashboards to manage reporting of key metrics and KPIs including leading and lagging indicators to ensure launch success, providing mechanism for teams to share critical updates. Leading overall business planning working with the Product Teams, Commercial / Country leaders, Planning & Logistics as well as cross-functionally to ensure that strategic planning & financial-planning deliverables are created at a high-level and in a timely manner Designs and establishes new processes, as required, for new modalities and emerging technologies that warrant deviation / modification of standard processes. Create and manage change control packages in product life cycle management system, ensure documentation is accurate and appropriate. Team Leadership: Lead, mentor, and develop a matrix team of TOPS professionals, fostering a culture of excellence and continuous improvement. Compliance: Ensure compliance with all relevant TOPS and Regulatory policies and processes. Stakeholder Collaboration: Collaborate and consistently communicate with key stakeholders, including BU leadership, TOPS product teams and leadership. Technology Utilization: Leverage appropriate technology and systems to streamline processes and improve data accuracy to support Product Launch Dashboards. Change Leadership:As a member of the Product Supply Chain Team(s) support and drive the change management requirements through communication internally and externally, developing the vision for Launch Excellence and broader PSCM and GSC organization and empowering team members to make effective decisions. SCOPE This role is responsible for supporting the on-time Launch and availability of products and artwork and labelling in market in compliance with applicable regulations. Decisions made in this role are of critical nature in mitigating risks for the business while ensuring BioMarin's commercial products are delivered to patients in a timely and compliant manner. The candidate should demonstrate the following attributes: A firm commitment to BioMarin's cultural beliefs and values Consistent track record of exceeding objectives within area of influence Ability to multitask effectively within a highly dynamic environment Ability to achieve results through others without direct reporting lines Strong problem-solving skills with a global focus both internally and externally Possess a high level of accountability and demonstrate an ability to be a change agent in a highly regulated industry Flexibility and ability to deal with changing priorities without losing sight of the overall business, product and market needs Strong interpersonal, communication and influencing skills working with all levels and within an environment of constant change Proactive and energetic outlook with an unwillingness to accept the status quo Interaction and collaboration with team members, peers and senior management Ability to work independently with strong time management skills Ability to operate globally, across different time-zones, work with global and regional functions. EDUCATION Bachelor's degree in Life Science, Engineering, Supply Chain Management, Business Administration, or a related field. Preferred: MBA in supply chain management, finance or business operations EXPERIENCE Minimum 10 years of experience in Supply Chain Product Launch or related discipline. Experience developing and operationalizing processes, systems and plans for New Product Launches inclusive of Artwork and Labelling. Strong leadership and management skills, excellent communication and interpersonal skills. Lean-DMAIC expertise, preferably Green Belt Certified or higher Project management certification, PMP or equivalent (preferred). Strong regulatory and GMP acumen Detail-oriented, analytical thinker, problem-solver, and able to work in a fast-paced environment. Proven ability to lead change initiatives and drive improvements within and across functions which enable compliance and mitigate risk Ability to effectively work autonomously and independently Proficiency in Microsoft applications and understanding of Office Timeline, MS Project / Project Online, MS Teams, MS PowerBI, SAP, or like technologies CONTACTS TOPS and PSCT product teams, GSC Planning, Site SC and Operations, Regulatory (CMC, Labeling & International / EU), Commercial Ops., Master Data team, Logistics, Artwork / Packaging Operations, Quality SUPERVISOR RESPONSIBILITY Manage a direct team of 3 - 5 professionals responsible for Product Launches and Artwork & Labelling across BioMarin portfolio of products. 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.

Who We Are BioMarin is a global biotechnology company that relentlessly pursues bold science to translate genetic discoveries into new medicines that advance the future of human health. Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Using our unparalleled expertise in genetics and molecular biology, we develop medicines for patients with significant unmet medical need. We enlist the best of the best - people with the right technical expertise and a relentless drive to solve real problems - and create an environment that empowers our teams to pursue bold, innovative science. With this distinctive approach to drug discovery, we've produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options. About Worldwide Research and Development From research and discovery to post-market clinical development, our WWRD 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 genetic diseases. The Associate Director inGlobal Study Operations(GSO) is expected to have a multifaceted role including program, project, and team management. The expectation is that they have proven ability at the clinical program lead role and can be relied upon to lead and execute complex programs. In a wider context the Associate Director is an excellent communicator, who can efficiently synthesize information from a variety of sources proactively identifyinginterdependencies andleveraging a solutions-focused mindset to develop options to work through setbacks. The ability to maintain a strong leadership presence, display sound decision-making and presentation skills, and is highly adept at representing the program both internally and externally as essential components of their success. The Associate Director will collaborate with cross-functional teams to ensure the successful execution of clinical programs, fostering a collaborative environment that drives innovation and excellence. The Associate Director is an accomplished manager with proficient people management and cross-functional management experience within a matrix organization. From a technical standpoint the ideal candidate has experience with early to late-stage clinical trial delivery, clinical trial recruitment and retention practices, clinical trial diversity practices, a strong interest in process improvement projects and cross functional initiative leadership, skills in metrics and dashboard creation, drive compliance and quality and use innovative technology to reduce burden for sites, vendors, and study teams alike. Within the role the candidate is expected to have advanced knowledge and experience in the following competencies: Communication and Collaboration Leadership Influence and Organizational Awareness Decision Making, Strategic Thinking, and Problem Solving Agility and Proactivity With advanced technical competencies in the following areas: Drug Development and Study Design Product & Therapeutic Area Knowledge Vendor Management and Oversight Data analytics/interpretation Key responsibilities include but are not limited to: Clinical Development strategy and planning: Contribute to the clinical development strategy for a program or programs providing input on the design and operationalization of the Clinical Development Plan (CDP) and supporting studies. Provide operational expertise and guidance on all relevant program and study deliverables. Ensure development and implementation of recruitment and retention strategies as applicable to support enrolment according to projections. Overall program operational accountability: Provide program-level oversight, leadership, and direction, ensuring that Global Study Operations team members and the cross-functional Study Execution Team achieve assigned deliverables within timelines, budget, and in accordance with BioMarin's quality standards. Ensure consistent study execution practices to drive successful outcomes and deliverables across all studies in a program. Ensure effective communication across all key program stakeholders. The primary point of escalation for program and study executional issues or risks related to deliverables, vendors, quality, timelines, and budget when staff or study team members are not able to resolve independently; take ownership of appropriately escalated issues and drive to resolution. Provides strategic and tactical input, guidance, and reports on program progress, risks, issues, metrics as needed to executive leadership across WWRD and BioMarin as appropriate Departmental contributions: Drive and implement critical cross functional initiatives across the portfolio of programs and/or Global Study Operations to improve efficiency and effectiveness of clinical program development Champion change management and operational projects, including developing communications and supporting staff through change Contribute to and support the establishment and evolution of the use of innovative technology and systems management across the portfolio and department deliverables. Contribute to the development of a cohesive, balanced, and high-functioning team focused on driving continuous improvement for the organization. Mentor and manage staff in alignment with a matrix team environment Education BA/BS degree (advanced degree preferred) with a focus on life sciences, nursing, or related technical or scientific discipline Experience 10+ years clinical trial or drug development experience in biotechnology, pharmaceuticals, medical device organizations with BA/BS; 8+ years with MA/MS;6+ years with PhD 3 or more years of team management experience 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.

Who We Are For more than two decades, going our own way has led to countless breakthroughs, bettering the lives of those suffering from rare genetic disease. In 1997 we were founded to make a big difference in small patient populations. Now we seek to make an even greater impact by applying the same science-driven, patient-forward approach that propelled our last 25 years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Through our unparalleled expertise in genetics and molecular biology, we will continue to develop targeted therapies that address the root cause of the conditions we seek to treat. Applying our knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The end goal has always been better lives and now we can reach more. And the more people we reach, the more our impact can grow. We transform lives through genetic discovery. Our Culture Our desire to make a positive impact on our patients extends to our employees and BioMarin is committed to fostering an inclusive environment where every person feels seen, valued, and heard - so employees can thrive in all areas of their lives, in and outside of work. We seek to provide an open, flexible, and friendly work environment to empower people and to provide them with the ability to develop their long-term careers. Ultimately, we want to be an organization where people enjoy coming to work and take pride in our efforts to help patients. The Head of Early Programs is a leadership position responsible for the portfolio of programs from initiation of investigational new drug (IND) to completed proof of concept (POC) studies. This position is responsible for the clinical oversight of all early clinical studies, and the articulation of the strategic goals of early development. BioMarin plans to support 2 new INDs and 2 completed POCs each year based on a development track record of success. BioMarin is a genetic medicines company with a broad therapeutic portfolio and multiple treatment modalities including viral and non-viral gene therapy, oligonucleotides for gene targeting, biologics, and small molecules. This position reports to the newly created senior position of Head of Experimental Medicine and Early Clinical Development and will be a critical function in the continued growth and diversification of the clinical development pipeline of BioMarin. This role will have key strategic interfaces internally with Late-Stage Development, Translational Sciences, and the Product Portfolio Team amongst others. Externally, this position is expected to be a dynamic catalyst for support of the pipeline and help build strong relationships with world class key opinion leaders. Success requires sound understanding of the evaluation of the scientific programs, translational results, development of key mechanistic endpoints, and leveraging early studies to understand therapeutic margins and estimates of effect size. This role is expected to contribute to portfolio growth and strategy in the context of market assessment and competitive analysis. This position will have oversight and responsibility for all Early Clinical Development (ECD) functions: Manage a motivated group of physician-scientists engaged in the programs. within the ECD portfolio, with an expectation to scale and build the team. Author strategic content of clinical development plans and protocol development for ECD. Advancement of programs to support corporate growth goals. Cross-functional collaboration with Regulatory to author strategy for first in human studies. Develop key internal and external stakeholder collaborations. Representation of ECD at key research and development governance forums. Lead teams responsible for designing and executing early clinical programs. Contributions in cross-functional setting Identify therapeutic targets, disease mapping and pipeline strategy. Synthesize and communicate results/outcomes of early clinical studies. Assimilate advancement of early studies into the assessment of risk and investment in programs. Lead external advisory process for new development programs and develop high level KOL support for key programs. Regular interactions with senior research, senior leaders in business development, research and commercial to align development goals with functional and corporate initiatives. The Head of Early Programs will be a member of leadership teams in Translational Science, Clinical Development, and participate in governance and strategic forums. This role is expected to maintain highest scientific and ethical integrity, manage publication and communication plans. The development of disease mapping, patient centered outcomes, and integrated evidence plans for a diverse portfolio in a cross-functional is considered a key deliverable. The Head of Early Programs is at the nexus of translational and clinical research, leveraging detailed scientific, genetic, translational, and phenotype information into a development plan that supports data-rich advancement decisions for the early portfolio. This is a leadership and management role of significant impact to the growth of the development organization. Commensurate with the intended impact of this role, candidate must be a thoughtful and mentoring manager. In addition, they must also have excellent technical understanding of science and development. The candidate must be facile communicating technical and corporate initiatives to key external thought leaders and partners. 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.

We're looking for a candidate to fill this position in an exciting company. Undertake critical appraisal of available scientific and medical information, identify gaps and drive activities to address deficiencies Review and approve product materials, including product educational materials, information resources, study documentation, regulatory dossiers, internal briefing material, medical information response documents, slide decks, etc. Enable scientific messaging and gap analysis regarding product or disease area Contribute to the development of, and review and approve the Program Publication Plans Medical degree preferred 5 or more years of relevant regional level Medical Affairs experience; global experience preferred