Empowering Patients Through Collaboration: Astellas’ Commitment to Pompe Disease Awareness
This article shares real patient stories to raise awareness about Pompe disease. Individual experiences may differ. This content is for educational purposes only and should not be considered medical advice. Please consult your healthcare provider for guidance. This article was developed by Astellas. Speakers featured have received compensation from Astellas for their contributions
Partnering With Patients to Drive Better Outcome
Understanding the real-life experiences of patients is crucial in developing meaningful and effective treatments. At Astellas, engaging with patient advocacy groups and global key opinion leaders helps bridge the gap between science and the daily challenges faced by people living with rare conditions like Pompe disease.
This article highlights how Astellas fosters collaboration between patients and researchers, aiming to create more patient-focused clinical trials and ultimately, better health outcomes.
A Reliable Ally in the Fight Against Pompe Disease
Pompe disease is a rare genetic disorder that leads to progressive muscle weakness and breathing difficulties due to a deficiency or low levels of a vital enzyme. For those affected, daily life can be significantly impacted.
In developing new treatments, it’s vital to collaborate directly with patients to ensure clinical trials reflect their true needs. Their firsthand perspectives help shape research priorities, refine trial protocols, and define outcomes that truly matter to the community.
Christine Brown, Patient Partnerships Lead for Rare Diseases at Astellas, emphasizes this approach:
“It’s essential to understand the burden of the disease from the patient’s perspective, including how they view the potential benefits and risks of any new therapy.”
Listening to the Community: The Patient Insight Panel
To deepen its understanding of patient needs, Astellas created the Patient Insight Panel—an initiative that invites patients, caregivers, and families to share their insights with the company’s global research teams throughout the drug development process.
Over the past year, the panel has contributed through virtual meetings, email feedback, and surveys. Their insights have been instrumental in guiding Astellas’ Medical Affairs team on a genetic testing program and informing the Health Economics and Outcomes Research team’s evaluation of disease burden and treatment impact.
One significant outcome of this collaboration was the addition of a fatigue assessment to clinical trial criteria, addressing a key concern expressed by patients. These adjustments not only reflect patient priorities but may also enhance trial recruitment and participant retention moving forward.
“The feedback we receive from our Patient Insight Panel is vital. It helps us design better trials, reduce protocol amendments, improve recruitment and retention, and accelerate access to potential treatments.”
A Long-Term Commitment to the Pompe Communit
Astellas has been actively engaging with the Pompe disease community since 2015, supporting a variety of initiatives beyond the Patient Insight Panel. These include co-hosted events, educational webinars, and advocacy-driven collaborations—all focused on listening to and addressing patient concerns.
