Global Genes recently published, “From Molecules to Medicines: How Patients Can Share Their Voices Through the Drug Development Process.” This toolkit was the product of a collaboration between Global Genes and leaders in advocacy and government, including Cure SMA. Work from Cure SMA was used as case studies in five different areas: encouraging research, funding research, providing data, participating in trials, and participating in committees.
The world of drug development is rapidly changing, and one of the most fundamental changes is the increased participation of patients, their families, and their caregivers throughout the process. Historically, pharmaceutical companies and regulatory agencies included participation from patients at limited points during drug development, most often when a drug was nearing approval. Patients’ and caregivers’ knowledge was not routinely included at key points such as targeting early research, assessing the benefits and risks of new therapies, developing regulatory guidelines, or designing clinical trials.
However, this is changing. This new toolkit, which is part of a series on drug development, highlights how the patients—including those in the SMA community—are using their voices to drive conversations, research and drug development to yield significant advances for their disease communities.
Thank you to our community, whose dedication and persistence have become an example for the rest of the advocacy world to emulate. Our thanks also to Global Genes and the other collaborators on this project, for inviting us to be part of the important work being done in rare disease advocacy.