Patient’s Role in Drug Development

Quick Overview

In this presentation, David Meeker discusses the role of patients in drug development, specifically focusing on the Angelman Syndrome community. He emphasizes the importance of patients sharing their stories and advocating for themselves in order to drive progress in rare disease research. Meeker shares examples of patient-driven initiatives that have led to breakthroughs in treatment and highlights the power of organized patient communities in attracting industry attention and funding. He encourages patients to become more visible and studyable, as this increases the likelihood of finding effective therapies. Meeker concludes by emphasizing the importance of hope and the significant impact patients have in shaping their own healthcare outcomes.

Introduction

In this presentation at the 2020 FAST Science Summit, David Meeker discusses the important role that patients play in the development of drugs for rare diseases. He acknowledges the challenges faced by patients and their families and emphasizes the power of their stories in driving progress and finding solutions. Meeker shares his own experiences in the field of rare diseases and highlights the significance of trust in the healthcare system.

The Exciting Time for the Angelman Syndrome Community

Meeker recognizes the Angelman Syndrome community for their progress in attracting drug companies and advancing therapies through clinical trials. He commends their robust and connected community, as well as their determination to push forward and ultimately find a cure. He encourages them to appreciate how far they have come and the remarkable story they have already created.

The Fight and the Power of Storytelling

Meeker describes the fight that patients and their families face in navigating a healthcare system that often fails to recognize their needs. He emphasizes the power of storytelling as the most effective tool in this fight. Meeker encourages patients to share their stories, improve their storytelling skills, and understand the impact their stories can have.

Background and Experiences

As a physician and former executive in the biopharmaceutical industry, Meeker shares his background and experiences that have shaped his perspective on healthcare. He highlights the importance of trust in the system and the need for patients to hold the healthcare system accountable. Meeker also discusses his involvement in the cystic fibrosis gene therapy program and the lessons he learned from the Cystic Fibrosis Foundation.

Inspiring Stories

Meeker shares three inspiring stories that have impacted his thinking about healthcare and rare diseases. He discusses the Dent family’s journey with mucopolysaccharidosis and the transformative impact of enzyme replacement therapy. He also highlights the story of Mila, a young girl with Batten’s disease, and the collaborative efforts that led to potential treatments. Lastly, Meeker recounts a personal experience in South Korea, where he witnessed the resilience and talent of a young woman with MPS II.

Making Rare Diseases Visible

Meeker acknowledges the challenges faced by patients with rare diseases, including the difficulty of making a diagnosis and the limited availability of treatments. He emphasizes the importance of organizing and making rare diseases more studyable. Meeker encourages patient communities to support early researchers, collect data on natural history, and become more visible to attract industry attention.

Conclusion

In conclusion, Meeker urges patients not to underestimate the power of their collective voice and their ability to control their own destiny. He emphasizes the importance of hope and the exponential progress that can be achieved through organized efforts. Meeker expresses gratitude for being a part of the journey and wishes the Angelman Syndrome community the best in their pursuit of a cure.