Action Center

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Event Details

On July 13th, the Muscular Dystrophy Association (MDA) was pleased to convene the very first Pompe disease Patient-Focused Drug Development (PFDD) meeting. This virtual meeting offered individuals with Pompe and their loved ones an opportunity to inform the U.S. Food and Drug Administration (FDA), drug developers, and other key stakeholders on the daily experiences of living with Pompe disease and how they viewed the benefits and risks of treatments for Pompe. MDA was pleased to partner with the Acid Maltase Deficiency Association (AMDA) and United Pompe Foundation in this endeavor to ensure that the viewpoints of the community were well represented.

This Patient-Focused Drug Development meeting was critical to ensuring the FDA has an informed understanding of the Pompe patient experience. With the lessons and takeaways from the patient community in hand, FDA will be able to better review and approve therapies to meet the needs of the Pompe community desires. The online meeting also gave biopharmaceutical companies the information they need to help them develop therapies that will target the most burdensome symptoms of the disease while constructing more patient-friendly clinical trials.

During the course of this meeting, patients living with Pompe disease and their family members and caregivers shared their insights and experiences living with Pompe disease via moderated panel discussions and audience participation. The focus of the meeting was to primarily cover the symptoms and impact of Pompe disease on daily life, current experiences with treatment, and perspectives on and expectations for potential future treatments. Live polling was conducted to capture patient viewpoints on disease experience as well as potential benefits, risks, and preferences for treatment. Following the meeting, a “Voice of the Patient Report” will be published that articulates the takeaways of the day.