The report in Wednesday's Journal of the American Medical Association comes amid heightened public attention to medical research because of liability lawsuits over the painkiller Vioxx, political debate over stem cell research and the untapped potential of curing or preventing disease through mapping the human genome.
"If we're soon going to be spending $100 billion a year, we'd better have treatments that work over a long period of time against diseases that are important today and will be more important tomorrow," said Dr. Hamilton Moses III, co-author of the study and chairman of the Alerion Institute, which conducts studies on research policy.
"If we don't know those conditions are satisfied, we can't judge whether we're getting our money's worth," he said.
The study is part of a special issue of JAMA devoted to the state of U.S. medical research. What emerges from the issue is a picture of an amorphous, mostly profit-driven system, where industry research focuses on existing drugs and lets discovery-stage research lag behind.
The authors call on the medical industry, government and foundations to do better at investing in research on diseases with fewer effective treatments, such as Alzheimer's, and at translating basic research into new treatments and cures.
The authors have ties to the industry, medical schools and health companies, doing consulting work and sitting on drug company boards, according to financial disclosures published with the study.
The imbalance between late-stage and early-stage research is growing, the authors wrote, and is due partly to lengthy clinical trials required for new drug approval and partly to pure marketing. Companies often run costly studies to show their drugs work better than competitors' drugs.
In their funding analysis, Moses and his colleagues found that the industry sponsors 57 percent of medical research and the National Institutes of Health pays for 28 percent. That proportion has remained unchanged over the past decade.
The analysis also found that the United States spends about six cents of every health care dollar on medical research. But the nation spends only one-tenth of a cent of every dollar on longer-term evaluation of which drugs and treatments work best at the lowest cost.
"The data in this article make it plain that we are spending huge amounts of money, more than any other country, to develop new drugs and devices and other treatments," said Dan Fox, president of the Milbank Memorial Fund, a philanthropic group that works on health policy issues. "But we are not spending as much as we could to disseminate the most effective treatments and practices throughout the health system."
In separate JAMA articles, National Institutes of Health Director Elias Zerhouni said genetics advancements and stem cell discoveries require teams of experts who haven't worked together before, such as biologists and computer programmers, to convert basic science into new therapies.
And Julie Gerberding, director of the Centers for Disease Control and Prevention, said threats such as terrorism and avian flu require more investment in health protection.