At BioMarin, our operating principles begin and end with patients. We are driven by the understanding that patients are counting on us and our wins change lives.
BioMarin’s story began with parents who imagined better outcomes and brighter futures for their children. Nearly three decades later, that legacy continues to inspire our commitment to developing medicines that make a profound impact on people’s lives. Delivering that level of impact requires listening to and learning from patients, caregivers and advocates to develop a robust understanding of the complex needs of the rare disease communities we aim to serve.
We work to cultivate deep and genuine relationships with patient communities and advocates to ensure BioMarin incorporates their needs and perspectives into every stage of our work, from preclinical research to clinical development and commercialization.
Working with colleagues across BioMarin and beyond, our Patient Advocacy team serves as a bridge, ensuring the company understands the perspectives and needs of the communities we serve.
Our goal is to bring forward treatments that improve lives in real, measurable ways. Patients’ needs and perspectives serve as our guiding star in this work.
We cultivate deep and genuine relationships with patient communities, advocates and colleagues built on trust and respect.
We work collaboratively across BioMarin and in partnership with patient communities, recognizing that diverse perspectives and expertise lead to faster progress and the best outcomes for patients and our company.
We are constantly evolving our understanding of community needs to inform business priorities. We are agile, responsive and always ready to adapt our approach to best serve patients and support BioMarin’s objectives.
We measure our success by the positive, sustainable impact we deliver for patient communities.
One of the greatest privileges of working in rare disease is having the opportunity to get to know some of the people who make up this incredible community: the children, adults, parents and caregivers who generously share their stories and perspectives. It is an honor to learn about their lives, and work to incorporate their views and lived experiences into every aspect of BioMarin’s business.”
Marni Kottle, Chief Corporate Affairs & Patient Advocacy Officer
BioMarin partners with patient communities to meaningfully inform decision-making across all areas of our business. We strive to build authentic, sustainable relationships that elevate lived experiences and integrate patient perspectives into clinical research, regulatory engagement and evidence generation. We also help advance awareness through collaborative education and storytelling, while supporting patient organizations as they evolve their capabilities. Together, these efforts reinforce our commitment to patient-focused practices across our global operations.
We focus on building authentic, sustainable engagement with patient communities to inform decision-making in all areas of our business. This includes mapping the needs and lived experiences of patient communities, providing designated community liaisons, and funding strategic initiatives designed to support patient-led programs and events.
We work to integrate patient voices and experiences early to help shape and accelerate our research and development efforts.
We help advance awareness and informed decision-making through educational programs across a number of channels. We seek to amplify community voices, bring people together to share their lived experiences with our employees, co-create campaigns and tools with advocacy groups, and participate in conferences to help drive awareness.
We advocate for patient-focused policies and equitable access to medicines. From leading legislative briefings and spearheading coalition-building to engaging with Health Technology Assessment bodies, our efforts help ensure patient perspectives and real-world evidence inform the decisions that impact rare disease communities.
Rare genetic conditions often come with uncommon challenges, and we’re here to help. Learn more about the programs and practices we have in place to help support patient communities, access and safety.
Our RARE Scholars program provides financial support to U.S. college students living with, or caring for someone living with, one of several rare genetic conditions – achondroplasia, Batten disease, mucopolysaccharidosis (MPS) or phenylketonuria (PKU).
We support expanded access programs when feasible, and when we have substantial scientific evidence to support both the safety and the efficacy of an investigational product for an indication.
