Wendy Erler | Senior Vice President, Patient Affairs
Sarepta Therapeutics

Wendy Erler is a seasoned healthcare executive with a passion for improving the lives of patients with rare diseases. As the Vice President and Global Head of Patient Experience & Insights at Alexion – AstraZeneca Rare Disease, she brings a wealth of experience in developing patient-centric strategies that drive positive outcomes. Wendy is known for her innovative approach to understanding the unique needs of rare disease patients and translating those insights into impactful solutions.

With a background in healthcare marketing and patient advocacy, Wendy has a deep understanding of the challenges faced by individuals living with rare diseases and their families. She is dedicated to fostering collaboration between patients, healthcare providers, and industry stakeholders to ensure that patient voices are heard and valued in the drug development process. She has led critical projects focused on the sibling experience in rare disease, Women in Rare, and mental health intervention for a neurological rare disease.

Wendy has a deep commitment to driving innovation and improving outcomes for rare disease patients to make a difference in the lives of those affected by rare diseases. This only happens with true listening, collaboration and information sharing.

Appearances:

Day 1- World Orphan Drug Congress USA 2025 @ 09:45

Keynote: Vision 2030: Charting a bright future for rare medicine

Discussion to focus on immediate outcomes of Rare Disease Innovation Hub as a new model of collaboration between CDER and CBER. While the extent to which this initiative will translate into influencing policy or decision making is unknown, it has the potential significance on the scale of Patient Focused Drug Development 2.0 – representing an opportunity for a systematic approach to prioritize and streamline rare disease drug development. with. Discussion sub-topics:

Vision for Rare Disease Innovation Hub and advice for the RD community
Impacts to endpoint assessments, trial design, patient recruitment and development efficiency

Moderator: Rachel Smith, Executive Director, Global Head of Rare Disease, CoE, Parexel

Amy Comstock Rick, Director of Strategic Coalitions, Rare Disease Innovation Hub, FDA

Jennifer McNary, Co-Founder, Canary Advisors

Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics

Susan Winckler, Chief Executive Officer, Reagan Udall Foundation for the F.D.A.

Day 2 - World Orphan Drug Congress USA 2025 @ 13:40

Panel: Bridging the gap: The rare disease media landscape and industry partners

Industry partners recognize the need to learn from patient and caregiver's stories of their lived experience. Patient and caregiver stories are the heart of rare disease insights. Patients and caregivers have created authentic, compelling media - from blogs to podcasts to films - offering an invaluable window into the rare disease experience. Tapping into the emerging and increasingly robust media environment specific to rare disease stories can be a first step for patient engagement. Storytelling isn't just advocacy and it can be a key asset to industry supporting and developing meaningful relationships with these creators - who have the pulse of the community. Publicly available and searchable sources of rare disease patient stories represent an under-utilized opportunity for industry. The panel will outline ways industry can make use of these media outlets as part of their social listening and pre-engagement plans to set themselves up for more meaningful connection, and authentic engagement.

Moderator: Daniel DeFabio, Global Genes Director of Education & Co-Founder,, Disorder: The Rare Disease Film Festival