Drugmakers are brimming these days with news about new cancer drugs offering advances in fighting the disease.
But cancers in children, which usually differ from adult versions, have been all but ignored.
Drugmakers and biotech companies are largely dismissive when it comes to developing cancer drugs for youngsters because they represent small markets that cannot make a large profit and pump up stock prices for the companies, doctors and analysts say. And U.S. government incentives have done little to facilitate the development of new treatments for children.
“The pediatric market is minuscule compared with the adult market, so there really is no incentive to drug companies,” said Dr. Gregory Reaman, chairman of the Children’s Oncology Group.
The group is a large network of pediatric oncologists, many of whom are treating children with drugs already approved for adults in hopes they will have similar effects in kids.
Reaman said the network does not get much financial or logistical help from the drug companies. And he said the lag time between development of cancer treatments for adults and getting those same drugs approved for children is unacceptable.
About 9,200 American children are likely to be diagnosed with cancer this year -- about one third from various forms of leukemia -- out of a total of 1.37 million total new U.S. cancer cases, according to the National Cancer Institute.
Some of the world’s largest makers of cancer drugs say they provide the medicines to organizations and academic institutions that want to test them on children. Some of the most prominent research institutions include the Dana Farber Institute in Boston and the St. Jude Children’s Research Hospital in Memphis, Tennessee.
For instance, AstraZeneca Plc, one of the world’s largest makers of cancer treatments, sometimes provides free drugs to organizations or to U.S. government researchers, said spokeswoman Mary Lynn Carver.
“Not having the pediatric indication does not mean there is not a lot of research being done with pediatric use on the compound,” she said.
But pediatric cancer doctors are frustrated that decades have passed since a drug was successfully developed specifically for a children’s cancer first -- especially since few adult drugs have gotten quick clearance for children.
“While there has certainly been more activity in recent years, it still is a struggle to convince these companies that it is in the best interests of children,” said Dr. Mitchell Cairo, a pediatric oncologist at the Columbia Presbyterian Children’s hospital in New York.
Drug companies, namely Novartis AG, have made some progress in developing cancer treatments designed for pediatric use.
Novartis was granted regulatory approval in 2001 to treat chronic myeloid leukemia (CML) in adults with new drug Gleevec, which U.S. regulators approved in May last year for use in children with CML who have a certain genetic marker.
Ilex Oncology is seeking U.S. approval to sell a new drug for children whose leukemia has worsened and has not significantly responded to other treatments.
U.S. government incentives
Some analysts suggest more progress could be made if Washington was more proactive in encouraging the development of such drugs.
The FDA oversees the orphan drug law, which gives tax breaks and seven years of marketing exclusivity to companies developing drugs for unmet medical needs affecting relatively small amounts of people. But the law is often used by biotech companies to treat rare genetic disorders.
Another federal law gives companies six months of extra U.S. market exclusivity when a company agrees to test one of its drugs in children. And Congress recently mandated that companies test drugs in children that would reasonably be expected to work for them -- changing it from a voluntary program to a required one.
That law, however, does not ensure that the clinical testing on children will take place soon after the adult testing, leaving the potential for significant lag time.
“The ideal situation would be that the companies submit the pediatric studies at the same time as the studies that support the adult indication,” said Dr. Ramzi Dagher, a top drug evaluation supervisor at the FDA.
But it doesn’t always behoove drugmakers to do so, he noted.
“Some companies don’t have the resources to do all the studies at once. Some companies may want approval to be able to get more resources and then finish the pediatric studies.”