A new FDA legislative mechanism holds the promise of filling a well-known innovation gap in pediatric cancer drug development, according to speakers at a meeting on molecularly targeted cancer therapies for children and adults hosted by the Friends of Cancer Research (FOCR) in Washington, D.C. The organization produced a background paper on the topic (not a consensus document) with contributions from many individuals to explore the path forward in accelerating such development.

“There’s nothing more devastating than a child not getting the right treatment,” noted FOCR founder and Chair Ellen V. Sigal, PhD, in opening the meeting. For this reason, she considered the meeting’s topic “extraordinarily important.”

The new FDA legislative mechanism puts teeth into discovering effective new drugs for children with cancer. Specifically, the RACE for Children Act, Title V of the FDA Reauthorization Act of 2017, amended the Pediatric Research Equity Act (PREA) by mandating early pediatric evaluation of new drugs intended for adults with cancer. Recognizing the major contribution of molecularly targeted agents to precision oncology, Title V specifies that FDA may require by statute early pediatric investigations of molecularly targeted cancer drugs. Before this new amendment, PREA had not been considered an effective mechanism for developing drugs for pediatric cancers.

Title V directs the FDA, in collaboration with the NCI, to establish, publish, and regularly update a list of molecular targets the agency considers may be relevant enough to the growth or progression of pediatric cancers to trigger the requirement for a pediatric investigation. The amended law also directs the FDA to hold an initial public meeting for stakeholders and convene future public meetings to undergird the establishment of the molecular targets list. The FOCR background paper presents a framework of factors to guide the establishment, updating, and application of the molecular targets list.

Pediatric cancers differ from adult cancers in their etiology and biology, said Gregory Reaman, MD, FDA’s Associate Director for Oncology Sciences at the Center for Drug Evaluation Research (CDER), and Associate Director for Pediatric Oncology at the FDA’s Oncology Center of Excellence. Historically, treatment of children with cancer has been shaped by off-label use of adult drugs, and the diagnosis of cancer in children, especially metastatic cancer, “has been suboptimal,” added Reaman, inaugural and Immediate Past Chair of the Children’s Oncology Group (COG).

The new legislation will help fill the void in pediatric cancer drug development, Reaman stressed. “We must focus on the intent of this legislation,” he said, which is to trigger early pediatric investigations of agents with promising signals. “Ideally, defining a profound effect is what we’d all like to see.”

Asked by Oncology Times if the RACE for Children Act will truly be a motivator for pharmaceutical companies—given that cancers in children are rare—Reaman said, “Absolutely.” He noted that, for example, today pharmaceutical companies are developing drugs for adult cancers with smaller and smaller subsets of patients.

https://journals.lww.com/oncology-times/Fulltext/2018/04050/New_Opportu…