April 13, 2004 -- Widespread fears over ethics and safety are causing cancer patients to avoid participation in clinical trials needed to discover new treatments, experts and researchers tell lawmakers.
Government officials complain that enrollment in clinical studies is abysmally low and that the shortage is contributing to an overall slowdown in the number of innovative new drugs under development. While many cite a collective unease among patients as the main factor, others blame a lack of interest among doctors, who may lack the incentive to steer patients toward experimental drugs.
Only 3% of adult cancer patients sign up for trials even though 20% typically meet eligibility requirements, according to a 1999 study by the American Society of Clinical Oncology.
The number of innovative cancer drugs in development "has decreased significantly," partly because of the patient shortage, Richard Pazdur, MD, director of division of oncology drug products at the FDA, tells members of the House Committee on Government Reform.
Researchers say part of the problem is that patients often don't find out about available trials. Those that do are often afraid to participate, largely due to publicity surrounding ethical lapses in research or even deaths in studies. One widely-reported case involved a patient named Jesse Gelsinger, an 18-year-old who died while participating in a gene therapy trial at the University of Pennsylvania in 1999.
Such casualties are extremely rare, though they continue to frighten even patients with difficult-to-treat cancers, says Andrew Pecora, MD, director of the Cancer Center at Hackensack University Medical Center in New Jersey.
"The minute you start talking about clinical trials, alarm bells start to go off," he says.
Scientists have long worried about reluctance among blacks. Rep. Elijah Cummings, D-Md., says that the reticence for many owes to distrust born by the Tuskegee experiments of the 1920s, where researchers observed the effects of syphilis in black men rather then treat them.
"They have seen, or heard rumors at least, of African Americans that have been experimented upon. It causes them not to want to be apart of any experiment," says Cummings, who leads the Congressional Black Caucus.
'It Starts and Ends with the Doctor'
Others stress that fearful patients are only half the problem.
"Of primary concern is that so few doctors recommend a clinical trial as a viable treatment option," says Ellen Stovall, president of the National Coalition for Cancer Survivorship.
Rep. Henry Waxman, D-Calif., criticizes drug manufacturers for failing to participate fully in a web site database, www.clinicaltrials.gov. Federal law requires companies to post on the site details of every cancer trial they conduct, though the site lists only about half of all ongoing trials, says Waxman, referring to a 2003 FDA study of the site.
"This lack of participation by the drug industry in an important resource for patients is inexcusable," says Waxman, the committee's senior Democrat.
The Pharmaceutical Research and Manufacturers of America (PhRMA), the largest drug industry group, did not immediately respond to requests for comment.
Stovall says the National Cancer Institute (NCI), which funds about 70% of the 1,700 ongoing cancer trials, pays clinics about $2,000 per patient for conducting trials and reporting results back to researchers. The figure is about half of what conducting the studies actually costs, she says.
Michaele Christian, MD, associate director of the NCI's division of cancer treatment and diagnosis, tells lawmakers that inadequate payment "has been a subject of great interest and concern to us."
Even doctors who don't conduct studies often don't tell patients about available trials or bother enrolling them, experts say. That is a particular worry for federal agencies, who along with drug companies rely on community doctors for about 80% of patients participating in studies.
Committee Chairman Rep. Tom Davis, R-Va., says Clinton-era Medicare reforms authorizing the program to pay for the care of patients who participate in cancer trials do not go far enough. He urges the NCI to do more to address physicians' unwillingness to participate and suggests that professional societies make it part of ongoing medical education.
"It really starts and ends with the physician," he says.