How Amicus Embedded Patient Advocacy into its Corporate DNA

Jayne Gershkowitz is one of the founding leaders of rare disease company Amicus Therapeutics and has served as Chief Patient Advocate since 2016. She’s responsible for developing and executing global strategies that ensure that the needs of people living with rare diseases and their families are integrated to all company operations and for keeping extraordinary patient dedication as the bedrock of Amicus. Gershkowitz founded the company’s Patient Advisory Boards program, through which patients, families and caregivers openly share their input and concerns with Amicus; leads Amicus’ Public Policy work to advocate for policies that advance innovation and expand access to satisfy unmet needs of those living with rare diseases; and guides advocacy-based evidence generation resulting in multiple publications co-authored with patient community thought leaders and medical key opinion leaders. She established Healing Beyond Disease™, an Amicus philanthropic and volunteer initiative to further serve the rare disease community in meaningful ways. She is a member of the company’s Executive Committee, reporting directly to the CEO, and sits on the board of directors. Gershkowitz is widely recognized as a trailblazer in Patient Advocacy. In 2023, she was the Imagine & Believe honoree of National Tay-Sachs & Allied Diseases Association. In 2019, she received the second annual “Heart of BioNJ” Award in honor of her selflessness and dedication to patients in the rare disease community. She is a 2018 PharmaVOICE 100 honoree, recognized for providing inspiration and innovation in the life sciences industry. She often speaks about the strategic function of Patient Advocacy at industry conferences and how it is the essence of patient- focused drug development. She is the treasurer of the Board of Trustees of BioNJ and a member of the Board of Trustees of the Healthcare Institute of New Jersey. Gershkowitz is the co- founder and vice chair of the board of Professional Patient Advocates in Life Sciences (PPALS), a non-profit educational organization committed to supporting the function of Patient Advocacy within the biotech and pharmaceutical industries. Additionally, she was the executive director of National Tay-Sachs & Allied Diseases Association from 1998 – 2006.

Diagnosed in her teens, Sabina Kineen is passionate about educating others how the diagnosis of an inherited, rare disease can impact an entire family and sharing the unique perspective of living as both a patient and a caregiver. Ms Kineen strives to use her own life experiences, including her involvement and participation in clinical trials, to support patients and industry in order to build bridges and encourage health equity and patient engagement.