The US FDA hosts an annual meeting for Patient Advocates in the field of Regenerative Medicine. There isn’t a current meeting planned for this year, but last year’s meeting, held on 24 May 2022, is worth revisiting as it had the theme of how natural history studies are important to the development of future therapies.
From the FDA.gov website
What’s new?
Video recording, transcript and slide deck are all available for download. The files are not small, as the event was 4 hours long, but are useful for understanding the FDA’s perspective. Dr. Wilson Bryan laid out the groundwork for the day, defining the types of studies as well as the purpose of studies and why they are important for propelling clinical trials forward. A panel of patient advocates, moderated by Karen Jackler, MPH, demonstrated how many individuals are affected by various diseases and the range in severity of their symptoms.
Why should you care?
Patient advocacy is critical for the advancement in rare and orphan diseases. I’m sure that you’ve heard that “the squeaky wheel gets the grease”. In this case, it’s absolutely true. Advancing the field requires an intimate understanding of the needs of the affected individual, not just what a pharmaceutical company thinks is needed to treat a patient group. Patient Advocacy groups can push for real change through informing the US FDA as to what measurements and improvements are important to the affected individuals, not just what is being researched in an IND-based clinical trial initiated by a large company.
What I find most compelling about patient advocacy groups is their focus on improvement of management of symptoms and a better Quality of Life. While a cure is definitely a desirable outcome, typically the groups are focused on how to make day-to-day lives more manageable. For example, a panel member noted that a decrease in seizures leads to fewer emergency episodes, less disruption in daily activities and a more productive life, both for the affected individual and their caregivers. Slides 37 - 39 are especially informative regarding individual abilities and the types of medical conditions that the individual reports.
What can you do?
If you have a family member or a friend with a rare disease, consider getting involved with the national or international patient organization. You can search for that organization if you know the name of the disease. “Getting involved” can mean many things, from donating money to organizing events or grant writing or advocating in legislation and regulation to social media exposure. Patient organizations are usually grateful for whatever assistance is offered.
If you’re more interested in the governmental side, the FDA has a great page that explains the FDA’s role in encouraging product development for rare diseases.
Definitions
CBER Center for Biologics Evaluation and Research
US FDA United States Food and Drug Administration
IND Investigational New Drug
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