Market forces alone are not sufficient to produce new drugs needed for children with cancer, according to the Institute of Medicine (IOM) report, which recommends forming new partnerships among government, industry, researchers, advocacy groups and other parties to lead R&D.
The report, named “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 Dr Peter Adamson, 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% of children, and for children who are cured, the short-term and long-term side effects of current treatments are often too high.”
The absolute number of US 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 R&D 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 R&D might allow us to better target those abnormalities,” said 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,” said Adamson. 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.
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.