A subcommittee of the Oncologic Drugs Advisory Committee (ODAC) recently spent a day discussing the similarities and differences between pediatric and adult hematologic malignancies. It was the second session in a series intended to advise the Food and Drug Administration (FDA) on how to apply its 1998 Pediatric Rule regarding the testing of cancer drugs in children. The rule states that "there is a federal mandate to perform pediatric studies if the indication under review for a marketing license exists in children."
Subcommittee members and a panel of cancer specialists addressed application of the rule in three areas: myeloid leukemia and myelodysplastic syndrome, lymphoblastic leukemia, and lymphomas.
Ethical Dilemma
In his introductory comments, David G. Poplack, MD, professor of pediatric oncology at Baylor College of Medicine, touched on a fundamental issue of testing new drugs in children that are intended for adult use. He cited acute lymphocytic leukemia (ALL), for which conventional cytotoxic therapy has a good survival record in youngsters. If a new drug shows promise in adults, it can be a challenge for pediatric clinical researchers to decide whether to test it in children, he said.
"On the one hand, you can say, how is it ethical to change the regimen?" said Dr. Poplack. "However, you can make a strong argument to the contrary. We know that many curative therapies are associated with toxicity. If you have something that is more specific—that may have fewer toxicities—you may be ethically bound to study that drug vs the combination therapies that may cure ALL but have high toxicities."
Although the FDA relies on ODAC for advice and guidance in approving the marketing of cancer drugs and biologic agents, as well as other issues relating to its regulatory responsibilities, the agency is under no obligation to accept any ODAC recommendation.
Questions Posed by the FDA
A 70-year-old man with a history of localized prostate cancer treated with whole-pelvis radiation therapy with a boost to the prostate, in conjunction with androgen deprivation therapy 7 years prior, presented with lower back pain. A bone scan revealed an area of activity in the sacrum. What is the most likely diagnosis?
