Clinical Studies Can Differ by Funding Source, Type of Study

Breast cancer treatment trials that are funded by drug companies are more likely to show positive results than studies sponsored by other sources, say researchers in the journal Cancer.

There are also major differences in trial design when the pharmaceutical industry pays the costs, the researchers add.

"This doesn't have implications tomorrow for patients, but it does have implications for the kind of questions we address and the kind of answers that we have for future patients," says study author Dr. Jeffrey Peppercorn, at the University of North Carolina School of Medicine.

Making Sure All Questions Are Asked

The drug industry now spends more money on research and development than the National Institutes of Health (NIH).

With collaborations between industry and academic medical centers more common, some experts are raising questions about the quality of research and potential conflicts of interest.

Previous research has shown that clinical trials of medications funded by pharmaceutical companies and other for-profit entities were more likely to report positive findings than similar trials funded by nonprofit groups.

Only one of those studies looked at cancer trials, however. Meanwhile, half of all pharmaceutical company-sponsored medications currently in the pipeline are for cancer.

Breast cancer is the most common cancer affecting American women, striking more than 200,000 women in the US, and more than 1 million worldwide, each year.

Until now, no one had undertaken a systematic study of pharmaceutical sponsorship of clinical research in breast cancer.

For this study, Dr. Peppercorn and his colleagues reviewed 140 studies in 10 English-language journals that reported breast cancer treatment results over the past decade.

Of the 140 studies, almost half (48 percent) reported some form of drug company involvement. Drug company participation increased over the period in question, from 44 percent in 1993 to 58 percent in 2003.

Eighty-four percent of studies published in 2003 that reported drug company involvement were positive, vs. 54 percent of studies with other funding sources.

Clinical trials with ties to industry were also more likely to have a single-arm study design, meaning the medication's effectiveness was not tested against a comparison group.

Of the 2003 studies, 66 percent were single-arm studies while only 33 percent of studies funded in other ways used this design.

Industry-sponsored trials were also more likely to involve patients with advanced disease.

"We found significant differences in the types of studies by pharmaceutical and non-pharmaceutical funders," notes Dr. Peppercorn.

"Companies may be very good at drug development but, as they play a larger and larger role in financing clinical research, we need to think carefully about what other clinical questions need to be addressed that drug companies may not be exploring," notes Dr. Peppercorn.

Drug Companies Can Bring Expertise

One expert tried to put the finding into perspective.

"This doesn't surprise me because most of the new drugs being discovered are being discovered by the pharmaceutical industry," says Dr. Jay Brooks, at Ochsner Health System in Baton Rouge, La.

In contrast, "in the 1980s, most of the new compounds were discovered by the National Institutes of Health and they went to the pharmaceutical industry with interesting compounds and had them develop products," explains Dr. Brooks.

Also, 20 years ago, many people did not feel cancer was a disease they could actually affect so there were not as many medications in the pipeline, says Dr. Brooks.

"The pharmaceutical industry knows more about the drugs it's bringing to trial so when they design a trial, they have a higher chance of a positive trial," says Dr. Brooks. "In terms of a single-arm trial, if the trial demonstrates a dramatic improvement in survival, do you really need to do a large study?"

For now, breast cancer patients need to keep the dialogue open with their physicians.

"Systemic issues in cancer trials or other medical trials will never be more important than patients discussing with their own physician options available, including clinical trials," says Dr. Peppercorn.

"For the individual patient, the specifics of a particular clinical trial are more important than the funding," he says.

Always consult your physician for more information.

What are Clinical Trials?

Clinical trials are studies, managed by government agencies, educational institutions, private not-for-profit organizations, or commercial businesses, to develop, produce, and evaluate the effectiveness of new treatments and therapies for diseases.

According to the National Cancer Institute (NCI), there are different types of cancer clinical trials:

• prevention trials designed to keep cancer from developing in people who have not previously had cancer.

• prevention trials designed to prevent a new type of cancer from developing in people who have had cancer.

• early detection trials to find cancer, especially in its early stages.

• treatment trials to test new therapies in people who have cancer.

• quality of life studies to improve comfort and quality of life for people who have cancer.

• behavioral studies to evaluate ways of modifying cancer-causing behaviors, such as tobacco use.

• genetic studies address how genetic makeup affects detection, diagnosis, and treatment of cancer.

Most clinical research progresses in an orderly series of steps or phases.

Phase I trials are the first studies to evaluate how a new therapy should be administered - how often, and in what dosage. The focus is to determine safety (drug dose, device safety, or other therapy's safety) for the next phase of testing.

Phase II trials provide preliminary information about how well the new therapy works, and generates more information about its safety and benefits.

A Phase II study usually focuses on a particular type of cancer.

Phase III trials compare a promising new therapy, combination of treatments, or procedure with a current standard of treatment.

Phase IV trials include the continuing evaluation that takes place after FDA (US Food and Drug Administration) approval, when the therapy is already available for general use.

Large cancer centers, university hospitals, local medical centers, or physician offices may all be included in managing clinical trials. Informed consent is a process designed to protect potential participants through detailed description of important facts about a specific clinical trial.

There may be only one or two locations involved in a particular study, or hundreds around the country.

Each protocol in a trial defines specific characteristics, called eligibility criteria, that participants should have in order to participate in the study. Characteristics may include type of disease and its stage, as well as the participant's age and general health.

Eligibility criteria help assure that the study results answer the research question and identify who will benefit in the future from the approach being studied.

Always consult your physician for more information.