Can you provide an overview of Amicus Therapeutics and detail your role within the organization?
I am Jayne Gershkowitz, Chief Patient Advocate at Amicus Therapeutics for the past eight years, and leading Patient Advocacy and Public Policy at the company for almost 18 years. From the inception of Amicus over 20 years ago, we've embedded the ethos of supporting disease communities and their families into our organization’s belief system. This foundational principle has guided our approach to patient advocacy, placing it at the forefront of our mission. My role involves leading a global team dedicated to advocating for individuals living with rare diseases, including people with a diagnosis, their families, and the healthcare professionals who support them. At Amicus, Patient Advocacy is the essence of our operations, distinguishing our efforts as genuinely authentic and deeply rooted in our company's values.
How does Amicus integrate its patient-first philosophy into daily operations and strategic planning?
Our patient-first philosophy is not just a concept, but a strategy infused into every aspect of our operations. Advocacy at Amicus transcends traditional boundaries, influencing all functions and shaping our short- and long-term plans. Members of my Patient & Professional Advocacy team -with their range of healthcare, communications, nonprofit management, and policy specialization - are integral to cross-functional teams across the company, ensuring that patient and caregiver insights and needs are considered in all our endeavors. This comprehensive approach ensures that our actions remain aligned with our mission to improve patients' lives, guiding our drug discovery, development, and delivery to respond effectively to the disease communities' needs. By maintaining a relentless focus on patient advocacy, we embed a culture of empathy and understanding, enabling us to develop treatments that genuinely reflect what is meaningful and needed by those we aim to help.
What recent developments at Amicus are particularly exciting?
Our journey at Amicus has been marked by continuous learning and excitement, particularly in our work with rare lysosomal diseases such as Fabry and Pompe. The innovation in our therapeutic approaches highlights our commitment to push the boundaries of science for the betterment of patient care. Beyond these advancements, the evolution of patient communities worldwide and their increasing sophistication, active participation, and collaboration in advocacy work are truly inspiring. Initiatives to support and empower women and adolescents affected by Fabry disease, for example, have led to significant progress in how this condition is perceived and treated. By facilitating educational programs and creating innovative forums for dialogue, we've seen remarkable strides in patient empowerment, contributing to a broader understanding of these diseases and enhancing the quality of life for those affected.
How does Amicus approach the challenges of drug pricing and ensuring accessibility?
Addressing accessibility is a critical aspect of our work at Amicus, with patient advocacy and public policy playing a vital role.
Our commitment to ensuring that treatments are not only fairly priced but also broadly accessible is a cornerstone of our approach.
We are instrumental in obtaining and conveying the patient perspective – the lived experience - throughout the reimbursement submission process. By integrating patient experiences and the tangible value of having treatment options into our communications with payers, Amicus Market Access can aim to underscore the impact our medications could have on individuals' lives. This approach helps us navigate the complexities of the healthcare environment, as we strive to ensure that our treatments reach those in need under the fairest and most equitable terms possible.
Looking ahead, what do you see as the primary challenges for patient advocacy and Amicus in the battle against rare diseases?
As the landscape of rare disease treatment becomes increasingly interesting with new entrants and intensified research efforts, the challenge lies in managing the growing demands on patient organizations and ensuring efficient collaboration across the sector. The proliferation of companies entering the rare disease space brings valuable new insights and potential treatments but also strains the limited resources of patient advocacy organizations. The community seeks to foster a collaborative environment where information and research efforts are shared more effectively, reducing redundancy and accelerating progress. This requires a concerted effort to recognize bandwidth limitations of small nonprofits, streamline communication, integrate patient insights into research and development more seamlessly, and work towards common goals. As we move forward, the challenge will be to balance the drive for innovation with the need for cooperation and shared learning, ensuring that advancements in treatment translate into tangible benefits for the rare disease community.
