TO: MEMORANDUM
FROM: Nancy Goodman
RE: Notes on the PhRMA/BIO/Children’s Cause for Cancer Advocacy Conference of October 27, 2016
DATE: November 2, 2016
Participants/Venue/Organization
In attendance at the meeting were about 73 people, 2/3rds of whom were with the pharmaceutical industry. Some 47 people worked at PhRMA, BIO or pharmaceutical companies. 13 were academic pediatric oncologists, 9 were advocates, 4 were FDA or NCI officials.
The meeting was held in a beautiful room at Hotel Monaco. The meeting was run by Dan Smith of Advocacy Smith.
Morning presentations
The morning presentations were given by Dr Peter Adamson (COG), Dr Gilles Vassal (ITCC), Dr Malcolm Smith (CTEP NCI) and Dr Greg Reaman (FDA). The speakers noted the challenges of pediatric cancer research: the need for new drugs for children’s cancers when there are not corresponding adult indications, the challenges to pediatric cancer researchers of accessing drugs developed for adult cancer indications. They noted the near absence of novel, unapproved drugs for study in children’s cancers despite the many drugs in the pipeline for the variety of molecular targets. They noted the many kids who can’t get on trials because there are no trials for which they qualify. They referenced RACE for Children Act, which reforms PREA, as part of the solution. They noted the cooperation of European and American regulatory agencies and pediatric clinical networks. They expressed the hope that this meeting would result in a renewed commitment by the pharmaceutical industry to support pediatric drug development.
Dr Christine Bucci-Rechtweg presented the pharmaceutical industry point of view. She expressed some concern about how to prioritize the many unapproved targeted drugs on the small number of kids with cancer. However, good news – she did not express concern about the FDA’s current and historical prioritization of pediatric studies under the application of Best Pharmaceuticals for Children’s Act.
Afternoon case studies
Industry presented five case studies in the afternoon. The first speaker presented on the opportunities of using pediatric expansion cohorts for exciting expansion studies. He concluded by recommending that all adult expansion studies incorporate pediatric plans before the first IND meeting with the FDA
The second speaker presented about how, like adult cancers, pediatric cancers are heterogeneous. She applied lessons learned from adult drug development to address biomarkers and companion diagnostics for kids. While the process was challenging, the company was successful in developing a drugs for a pediatric cancer indication.
The third and fourth presentations were presented anonymously by a McKinsey consultant. The third case study talked about challenges in developing for kids drugs that are abandoned for study in adult indications. As we discussed, the Creating Hope Act pediatric PRV provides an incentive in these cases and in addition, apart from the Creating Hope Act, COG has a trial of Amgen’s abandoned IGR1R inhibitor at this time. This appears to be an excellent opportunity for pediatric drug development.
In the fourth case study, she offered an example of a drug in which a benefit/risk analysis resulted in a drug going to adult phase 3 but not being safe enough to proceed to pediatric phase 1. Academic oncologists said they couldn’t think of a fact pattern in which terminal kids would be barred from clinical trials of a drug based on safety when the drug was safe enough and promising enough for an adult phase 3 trial. FDA officers could not identify the drug. The McKinsey consultant said she could not respond to the questions because only knew what she read on the Powerpoint.
Conclusion discussion
After the next break, Dan Smith led a conversation to generate ideas of what this group could do next. The academic oncologists, advocates and government officials wanted to address access to drugs – both drugs in development for adult indications and drugs developed specifically for kids. The industry folks wanted to address prioritization of drugs, heterogeneity and companion diagnostics, and benefit/risk.
After break, Dan Smith proposed that the next step would be the establishment of a public/private entity to accelerate drug development. This is in line with the “VPOD” idea (virtual pediatric oncology drug consortium) that was discussed before the conference. Present and future funding commitments to the public/private partnership were not discussed. The mission of the public/private partnership was briefly considered but not agreed upon. Dan Smith suggested that Susan Weiner would convene an executive group and report back to the larger group on a proposed of a mission for the public/private entity on in February, 2017.