September Events, Part 1: Advocacy Workshop and FDA Meeting
As we make our way through Childhood Cancer Awareness Month, we'll keep you updated on some important events taking place. Today, we want to share with you two events from this past week:
9/6: "Champions for Children with Cancer" Advocacy Workshop
Last weekend, we hosted a regional workshop in Durham, North Carolina, that brought together enthusiastic local advocates for a day of education and mobilization around national and local policy challenges facing children with cancer.
At the workshop, participants heard from research and policy experts, as well as a survivor active in raising funds for research. Advocates collaborated in brainstorming sessions throughout the day and worked on action plans for moving forward, which will include ongoing work with Children's Cause through webinars and action alert opportunities.
As a first step, advocates agreed to reach out to their Congressional Members after the workshop and request their co-sponsorship of the Caroline Pryce Walker Conquer Childhood Cancer Reauthorization Act. Their efforts have already paid off: Senator Kay Hagan of North Carolina signed on as a co-sponsor this week!
Below photos, l-r: Susan Weiner, CCCA's President and Founder, presents to the group; Advocates discussing issues; Small group brainstorming session
9/10: "Under the Microscope" - An FDA Pediatric Drug Development Meeting
On Wednesday, the FDA's Office of Health and Constituent Affairs (OHCA) held a day-long meeting devoted to challenges in pediatric drug development. The meeting brought together stakeholders from industry, patient advocacy groups, healthcare, academia, and the public to explore pediatric product development, clinical trial design, and the regulatory decision-making process.
Attendees participating in person and online heard from speakers and panelists with wide-ranging expertise and backgrounds.
"Over the past 20 years, we have evolved from a view that we must protect children from research to a view that we must protect children through research." - FDA
The day kicked off with FDA's Dr. Lynne Yao discussing the historical framework of pediatric drug development, focused on legislative measures throughout the decades that have shaped the FDA into what it is today -- including BPCA and PREA. Dr. Dianne Murphy, Director of the Office of Pediatric Therapeutics at FDA, shared this supporting evidence for the importance of legislation encouraging pediatric trials: In the 15 years after the original BPCA/PREA legislation was introduced in 1997, over 900 pediatric trials and 500 pediatric labeling changes were made. In the early '90s, up to 80% of product prescriptions for children were for off-label use. Today, that number is about 46%.
Dr. Christina Bucci-Rechtweg of Novartis Pharmaceuticals presented on pediatric drug development from an industry perspective, discussing big pharma's evolving efforts to overcome historical barriers and develop a business model that considers the unique needs of children. Dr. Bucci-Rechtweg used case studies to demonstrate success stories from Novartis and emphasized that legislative action in the U.S. has been an effective strategy for progress in pediatric drug development.
Panelists in an afternoon discussion on challenges in product development also agreed that creating a broader pipeline for pediatric drugs requires regulatory and legislative measures such as BPCA/PREA. Other speakers discussed ethical issues unique to the pediatric patient population, the need for awareness around childhood minority health disparities, and the importance of collaboration among stakeholders.
The full webcast of the meeting should be available here on October 1st, pending the quality of the recording.
Stay tuned to this blog and our other social media channels all next week for much more coverage of September events!