The U.S. pediatric cancer clinical trials programmes: international implications and the way forward

Abstract

The four national paediatric cancer clinical trials organisations in the United States--the Children's Cancer Group, the National Wilms' Tumor Study Group, the Intergroup Rhabdomyosarcoma Study Group and the Pediatric Oncology Group--were formed in 1955, 1969, 1972 and 1979, respectively. Together, the Children's Cancer Group and Pediatric Oncology Group serve as a national registry of nearly all childhood cancers in the United States, provide a national network of communication for researchers, care providers and families of paediatric patients with malignant disease and conduct laboratory investigations and clinical trials of new treatments of cancers in infants, children, adolescents and young adults. Nearly 95% of patients with cancer in the United States who are below 15 years of age are registered by the Children's Cancer Group and the Pediatric Oncology Group and more than half of American children with cancer are entered into at least one trial by a paediatric group. Improved survival of children receiving treatment according to well-defined protocols in specialised children's centres, in contrast to children who received treatment outside of these centres, has been shown for those with acute lymphoblastic leukaemia, lymphoma, Wilms' tumour, medulloblastoma, rhabdomyosarcoma and Ewing's sarcoma. By the year 2000, the overall cure rate for United States children and adolescents with cancer should exceed 85%. To reach this goal, the way forward will depend on international collaboration, implementation of global harmonisation, prevention of the erosion of biomedical research and clinical trials by the managed health care industry, increased public and private financial support and continued recruitment into paediatric oncology of brilliant and dedicated young investigators. The specific challenges ahead include: (1) transferring the knowledge, methodologies and technologies to countries that are less fortunate; (2) conducting multinational clinical trials in conjunction with paediatric cooperative groups in other countries; (3) accessing older adolescent patients who currently do not participate in cooperative group trials; (4) merging clinical trials by adult collaborative groups that overlap with the paediatric groups, as in acute lymphoblastic leukaemia, acute myelogenous leukaemia, Hodgkin's disease, osteosarcoma and germ cell tumours; (5) establishing a stable source of funding for national and international cooperative paediatric cancer clinical trials; (6) creating an informatics system that can link paediatric cooperative group operation centres around the world, and the institutions within each collaborative group; and (7) securing the support of the insurance industry and government in covering clinical trials.