Market forces alone are not sufficient to produce new drugs needed for children with cancer

Market forces alone are not sufficient to produce new drugs needed for children with cancer, according to a new report by the Institute of Medicine (IOM). Faced with "a near absence of research in pediatric cancer drug discovery," the IOM report recommends forming new public-private partnerships among government, industry, researchers, advocacy groups and other parties to lead research and development.

The report, "Making Better Drugs for Children with Cancer," analyzes childhood cancer treatment in the light of historic advances. "Over the past 40 years, researchers and clinicians have achieved long-term survival for most children and adolescents with cancer," said pediatric oncologist Peter C. Adamson, M.D., chief of the Division of Clinical Pharmacology and Therapeutics at The Children's Hospital of Philadelphia, and an editor of the report. "However, our therapies are not curative for 30 percent of children, and for children who are cured, the short-term and long-term side effects of current treatments are often too high."

Dr. Adamson is a member of the IOM's Committee on Shortening the Time Line for New Cancer Treatments, which issued the report on April 18. As Chair of the Developmental Therapeutics program of the Children's Oncology Group, a nationwide consortium of pediatric oncology centers, Dr. Adamson took a leadership role in drafting the report. The Institute of Medicine is part of the National Academy of Sciences, a private, nonprofit organization of scholars chartered by the U.S. Congress.

The absolute number of U.S. children with cancer is relatively small (about 12,000 cases diagnosed annually, compared to 200,000 new cases of breast cancer alone), and pharmaceutical companies do not consider it profitable to invest in research and development for pediatric cancer drugs. Oncologists have used many existing adult cancer drugs to treat children, but many of those drugs have toxic side effects. On the other hand, say the authors, some new drugs being developed for adult cancers may still prove useful for children with less common cancers.

"The major childhood cancers are often distinct from adult cancers at the level of molecular abnormalities, and more focused research and development might allow us to better target those abnormalities," said Dr. Adamson. "More targeted drugs might not only allow us to better attack the most difficult childhood cancers, but also cause fewer toxic side effects, by sparing healthy cells."

The IOM report says that select components of a pediatric drug pipeline already exist in academic medical centers, industry, universities, and in federal centers such as the National Cancer Institute. Those resources include repositories of synthetic and natural products, services to screen compounds for anticancer activity, drug discovery databases, and programs to support drug assays and clinical trials.

"What is needed is a comprehensive mechanism to put all these pieces together into a pediatric drug pipeline," says Dr. Adamson, emphasizing one of the major recommendations of the IOM report. The IOM proposes a partnership among government, industry and academia to produce new pediatric anticancer drugs. It suggests that in cases where companies do not proceed to full development of promising pediatric drugs, the federally sponsored National Cancer Institute should assume responsibility as the developer of last resort.

As one model of a public-private partnership focused on a health condition, the IOM report describes the Therapeutics Development Program established by the Cystic Fibrosis Foundation to support the research and development of new drugs for cystic fibrosis. That program, begun in 1997, has forged agreements with government and industry to form a "virtual" research and development pipeline.

In addition to proposing a coordinated pediatric oncology drug pipeline, the IOM proposes to speed up the drug development process by setting priorities to determine which pediatric drugs should be tested among the limited pool of children with cancer. It also recommends initiating pediatric trials earlier than they typically begin.