A year ago, U.S. researchers launched a high-priority study to determine if women with an often-fatal type of breast cancer could live longer by taking a specific combination of drugs. If the study found that to be true, the average rate of survival — four years — could be significantly extended.

A worthy question to address? It would seem so. But the answer may be a long time coming. The U.S. part of the international trial got under way nine months behind other countries. And researchers now expect to enroll only 350 U.S. patients of the original 3,500 sought.

Before drugs and therapies to save lives or reduce suffering can reach the market, they must be tested to ensure they’re both safe and effective. But the reluctance of Americans to participate in clinical trials has been a serious drag on medical research.

Enrollment problems delay more than 70 percent of all clinical trials from one to six months, according to a 2007 survey by CenterWatch, a Boston company that publishes information on clinical trials.

In cancer care, less than 5 percent of all patients enter clinical trials, even though more than 700 cancer therapies — many that appear highly promising — clog the research pipeline.

“It’s a major issue,” said Dr. E. Ray Dorsey, a neurologist at the University of Rochester Medical Center who has studied the issue. “Many trials are started and never finished because they can’t complete enrollment. A lot of money is wasted.”

To test a treatment — whether a pill, injection or procedure — researchers usually need large groups of people who have a specific illness and who are, for comparison purposes, of a certain age, ethnicity or gender.

Sometimes they need patients with a particular gene or complication. Then they must track the patients for several months or years to see if the therapy works and how it compares to standard treatment.

It’s no small task — and with 2,800 drugs and medical therapies in development and awaiting data on efficacy and outcome — doctors, researchers and drug companies are taking a hard look at how to get more people to participate.

To that end, initiatives to boost clinical trial participation are under way across the country.

Some medical centers offer simple perquisites, such as arranging transportation for participants; others are using computer programs that notify doctors if a particular patient might benefit from a trial.

Researchers, meanwhile, avoid the word “subject” and even agree to meet with trial participants after the study to answer questions. Everyone, it seems, wants to convince potential participants that they will be cared for and valued.

Although cancer research receives the most publicity, the need for participants plagues almost every area of medical research.

Earlier in March, the Huntington’s Disease Society of America e-mailed its members asking people afflicted with the fatal genetic neurological disease to consider participation in clinical trials.

The letter, from the group’s executive director, Barbara Boyle, said that a recent three-month trial on a potential drug treatment took more than nine months to enroll, while another promising trial may take longer.

“Our scientists and clinicians have done what they can to reach this point,” Boyle wrote. “Only you can make the final steps possible.”

Part of the blame for limited participation in clinical trials lies with researchers.

They acknowledge that some trials have been poorly conducted, risking patient health and dimming the public’s view of medical research.

Researchers also say they have failed to explain to patients that clinical trials often present two choices of care: the best established treatment or that treatment plus an experimental therapy.

“I think there is a misperception by the majority of people that it will take away from their treatment and won’t do them any good,” said Dr. Raymond DuBois, president of the American Association for Cancer Research and provost of M.D. Anderson Cancer Center in Houston. “At a bare minimum, we would offer the standard treatment option.”

Patients may fear they will get placebos (dummy pills or treatments), but those are used primarily in trials that assess prevention, rarely in treatment trials.

Nor are clinical trials necessarily a desperate attempt to stay alive after all other treatment has failed.

Said Dr. Robert Comis, president of the Coalition of Cancer Cooperative Groups: “Nowadays there are trials for all stages of disease. It’s a myth that this is something of a last resort.”

The one area of medicine in which clinical-trial enrollment is common has positive results to show for it. In pediatric cancer centers, more than 60 percent of children receive experimental therapies.

That decades-long practice, which began out of desperation to treat kids who would otherwise die, is now credited with vastly improved rates of survival, such as the astonishingly high cure rate associated with childhood leukemia.

“I think the real issue here is that in adult medicine, the clinical trial process is not an integral component of the entire treatment paradigm,” Comis said.

“What we’re trying to do is make it more mainstream.”

How to find a drug-trial option

Many people are unaware they are candidates for clinical trials, said Dr. E. Ray Dorsey, a neurologist at the University of Rochester Medical Center in New York.

A survey presented in 2008 at the annual meeting of the American Society for Clinical Oncology found that 60 percent of newly diagnosed cancer patients hadn’t been told about clinical trials as a treatment option.

But researchers are trying to bring clinical trials to the masses — via computer databases — instead of forcing people to search for them.

Government Web site:

clinicaltrials.gov

The largest database, clinicaltrials.gov, sponsored by the National Institutes of Health, lists federally and privately supported clinical trials for a wide range of diseases and conditions. The site was founded in 2000, but a 2007 federal law expanded its trials to include a wider range of conditions. Further, the law requires researchers listing their studies to report their results — including negative ones.

Cancer trials:

cancertrialshelp.org

Another database, TrialCheck at cancertrialshelp.org, was launched in 2003 and lists all industry- or government-sponsored cancer trials currently recruiting. The service uses information from clinicaltrials.gov to ensure the studies have met government safeguards. Besides trying to avoid dense medical terminology, TrialCheck offers a service that allows users to call a clinical trial specialist with the American Cancer Society for questions about a particular study or clinical trials in general. The site has seen a quadrupling of visits to the site in the past year.

Breast cancer:

breastcancertrials.org

Another type of matching service, for breast cancer patients, was launched in November. Breastcancertrials.org allows patients to enter information about their cancer history into a secure online database. The record is used to match the patient with breast cancer trials across the country.

The databases are only a first step. As electronic medical records become the norm, researchers hope to tie the records into an automated clinical trial matching system that will alert doctors and patients alike.

Dr. Robert Comis, president of the Coalition of Cancer Cooperative Groups, describes the goal for patients: “They will have access to their own health records, and they can say to their doctor, ‘It says here I’m eligible for three studies. Why didn’t you talk to me about that?’”

Researchers and patient advocates in various states are also fighting for federal legislation that would require insurance companies to pay for the patient care involved in clinical trials, following 23 states that already have such laws.

The federal bill was developed specifically to address enrollment in cancer trials, which make up a majority of clinical research. Called the Access to Cancer Clinical Trials law, it was introduced by Sen. Sherrod Brown, D-Ohio, last year and is before a House committee.

While the research sponsor, such as a drug company, usually provides the drug or therapy free of charge, some insurance policies don’t pay for care, such as blood tests, scans or examinations linked to the treatment, says Dr. James Thomas, director of clinical trials at the Ohio State University Comprehensive Cancer Center.

Other researchers are simply extending basic courtesies to boost enrollment. Some trial steering committees include a patient advocate to make sure patients’ concerns and views are represented, Dorsey says.

And more people will enroll, studies have found, if researchers assure them they’ll take the time to brief the participants when the study is over.

A study published in December by Dorsey reported on the results of a nationwide conference call between investigators and patients (or their caregivers) who had participated in a study on Huntington’s disease. For 90 minutes, the experts answered questions, explaining that the drug had failed to improve symptoms.

“They were disappointed,” Dorsey said of the participants. “But I think they felt appreciated for their efforts.”

— Los Angeles Times