Thank you Miss New York Jamie Macchia for making Childhood Cancer your platform this past year in memory of your dear friend and for all children. We are grateful for the work you have accomplished and the amazing awareness you have spread. Your presence is DC last week was greatly appreciated. ... See MoreSee Less
Nancy Goodman spoke at FDA meeting on expanded access and described Kids v Cancer Compassionate Use Navigator. Here is an excerpt of Nancy's comments:
"Children with untreatable cancers turn to compassionate use because there are exceedingly few pediatric clinical trials that include investigational drugs. Compassionate use is their only option. However, compassionate use is a challenging and time consuming process. Given these concerns, we have designed a program, the first purpose of which is to alleviate the burden of physicians and families. By addressing this problem, we hope more physicians and families will apply for compassionate use and more children will have access to unapproved drugs. Our second goal is to collect data. We believe the data is valuable for at least three reasons. The data could lead to new research directions by identifying extreme responders. It could create opportunities for the FDA to step in and propose solutions to specific cases as it becomes aware of them. And, it would contribute to the policy discussion around compassionate use. One of my goals here is also to use this experience to encourage more pediatric trials of unapproved drugs so that children’s only access to these drugs isn't just through compassionate use.
Going forward, we are now informing the community of this program. Kids v Cancer’s compassionate use program was designed for pediatric cancer patients, however our content is not pediatric cancer specific and could be adopted for all other patients as well." www.kidsvcancer.org/compassionate-use/... See MoreSee Less