Pediatric Product Development for The Treatment of Childhood Cancer

Hi, I’m Dr. Skip Nelson Deputy Director of the Office of Pediatric Therapeutics Food and Drug Administration I’m here today talking with Dr. Greg Reman Associate Director of the Office of Hematology and Oncology Products about pediatric product development for the treatment of childhood cancer. Welcome, Greg. Thanks, Skip. Good to be here. Well Greg lets start out with by talking about the importance of safety and ethnicity information about products for the use in children with cancer. Well I think it’s important given that cancer is a life threatening disease, despite that there really is a mandate for the agency to provide evaluation, through evaluation, of both efficacy and safety for these drugs which have significant potential toxicities, when used incorrectly or inappropriately. We’ve had legislation in place for the last fifteen or so years that have tried to stimulate pediatric product development called the Best Pharmaceuticals for Children Act where companies can get six months of marketing exclusivity for doing pediatric trials. How well has that worked for pediatric cancer? Well BPCA has worked and has worked well in contributing to information, pediatric specific information, for product labeling. It’s also resulted in the approval of several drugs, and I think we now have opportunities to make BPCA work even better for children with cancer. Now, I think you are referring to some of the changes that were made in the FDA Safety and Innovation Act. That is correct. Could you talk a little about those changes? Sure. As part of FDASIA, the [FDA] Safety and Innovation Act there actually a requirement now for companies to early in the development of a product for an adult cancer indication to think about and to communicate with the agency about a pediatric plan for evaluation, and also a requirement for the agency to respond within a specific time period. And that is now at the end of phase two which comes much earlier. That is correct. Now FDASIA also involved a new voucher program which was modeled after the Creating Hope Act. Exactly. What impact do you think that may have? I think that has potential for impact particularly for products that might be developed exclusively or nearly exclusively for childhood cancers. It a rare condition for children fortunately, it’s a small market, and therefore special incentives are required. So I think in that setting the priority voucher system resulting from the Creating Hope Act could have a real impact. So this voucher gives companies the ability to ask for a six month review instead of a nine month review so it’s effectively shortening the time line. The Act also allowed for the sale of those vouchers which may help in its value. Correct. How important is that? I think that’s very important because generally the companies that would develop these products for a small market are very small companies and these priority vouchers would probably be of much greater importance for a larger company with a big blockbuster drug and therefore bring some financial benefit to the small companies who sell them. Now in your position are you beginning to see interest on the part of industry for developing products for pediatric cancer? Yes, we are. There’s actually two significant products in development now that are exclusively for pediatric cancer. More exciting I think is the fact that drugs, new targeted drugs, that are being developed for adult cancer indications companies are coming in with pediatric plans, or at least proposed pediatric plans to evaluate them much earlier in the timeline of development. Great. Thank you, Greg. Thank you, Skip. And thank you for listening. Again, the FDA is supportive of September is National Childhood Cancer Awareness Month, and we look forward to continuing to work on and improving products and developing products for the treatment of pediatric cancer.