Report Claims Clinical Trials Miss Many Populations

TUESDAY April 1, 2008 -- A new analysis of the American clinical trial process suggests that the system for testing new drugs has routinely excluded or under-represented women, older people, minorities, disabled individuals and rural populations for decades.

"We've got a big problem," said Daniel S. Goldberg, chief policy adviser for the report. "And it's extremely urgent that we fix it. Because we're trying to figure out how to streamline health care and make people healthy, of course. And the fact that we have under-representation in clinical trials undermines both of these goals and undermines the quality of the evidence we come up with."

The report was conducted by the Chronic Disease Prevention & Control Research Center at Baylor College of Medicine in conjunction with the Intercultural Cancer Council, both based in Houston.

Part of a team of more than 300 analysts led by Armin D. Weinberg, Goldberg and his colleagues spent four years conducting an in-depth review of policy positions held by public, private and nonprofit clinical trial sponsors in the United States. Their research was funded by what is described as an "unrestricted educational grant" provided by Genentech Inc.

The team noted that about 80,000 clinical trials are conducted in this country each year, and that less than 1 percent of the American population -- 2.3 million men and women -- participate in such trials.

Additional trial problems, such as the lack of adequate training for members of institutional review boards, who are legally obligated to assess the structure of a proposed trial, were cited.

The report also admonished against the wasting of limited resources that results when government institutions and private industry duplicate each other's efforts in conducting trials focused on the same disease or treatment.

However, it is the continuing absence of specific constituencies in many trial populations that gets the lion's share of the criticism.

The research looked at cancer clinical trials and found that only 25 percent of patients in such trials were over the age of 65. In addition, older people were often excluded from studies focused on Alzheimer's, arthritis and incontinence, the researchers noted.

As evidence of the problem, Weinberg and his colleagues honed in on a study of clinical trial composition that found that, between 1995 and 1999, blacks, Asian-Pacific Islanders, Hispanics and Native Americans together made up for less than 10 percent of patients included in new cancer drug trials.

Under-representation of this sort, they say, leads to results that do not account for a host of factors -- genetic, cultural, racial, religious, linguistic, as well as variables related to age and gender -- that could have a huge impact on how well new drugs do in the real world.

To address these shortcomings, the team proposed nine policy solutions:

  • government regulatory changes;
  • increased collaboration between government and private industry on clinical trial design;
  • increased community involvement in patient participation;
  • scientific journal oversight of patient breakdowns;
  • new, specialized training for review boards;
  • reallocation of research funding to avoid duplication and address disparities;
  • increased public education;
  • increased focus on easing the patient participation process;
  • guaranteeing insurance coverage for all related costs.

"The bottom line is there has been a lot of discussion and attention paid in recent years to how clinical trials are put together, and, in particular, the need to account for differences in study patient populations," said Weinberg. "And that's the good news. But the bad news is that as a practical matter, it has yet to have a real impact on studies themselves."

"And the problem is that many people, when they take a pill, don't realize that it didn't come out of thin air," added Weinberg. "So, we hope that our work will help people appreciate how the process works, and that what we're talking about is the next generation and how we improve the products and type of therapies that we will have in the future."

Adil Shamoo, a professor of biochemistry and bioethics at the University of Maryland School of Medicine in Baltimore, agreed that trials must ensure proper patient representation. And he stressed that the problem stems from a lack of structural focus, rather than from any lack of potential volunteers.

"We have an extremely willing volunteer population in this country, so there is no question that proper trial representation is doable," said Shamoo, who is also the co-founder of the nonprofit Citizens for Responsible Care and Research, based in Columbia, Md. "And medicine is going to be given to millions of different people, so you do need that representation. Otherwise, you can have risks that you won't know about."

Other reports released this week buttress the findings of the Baylor team.

On Monday, Duke University researchers reported at the American College of Cardiology meeting in Chicago that although more women are being included in clinical cardiology trials, their numbers are still so low that it's questionable whether the results can actually be applied to women. And a commentary in the April issue of The Lancet Oncology stressed that more teens and young adults need to be included in trials of new cancer drugs, because they are currently under-represented.

More information

For additional information on clinical trials, visit the U.S. National Institutes of Health.

Posted: April 2008


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