Are clinical trials for you?

A decade may seem like an eternity, but that’s often how long it takes for a pharmaceutical company to prove that its new drug works and is safe for human use. A large part of this painstaking process is the clinical trial phase, a costly scientific investigation that must be done before Health Canada will approve any new medication.

After scientists discover a chemical compound that may potentially cure or treat a condition (or at least improve on existing treatment), it’s analyzed in the lab and later tested on animals. Because lab and animal testing yields limited information, the new drug must then be tested on humans before doctors can begin prescribing it. This is the clinical trial phase. 

It’s easy to imagine clinical trials as experiments run by mad scientists with human beings used in place of guinea pigs. In fact, they are carefully designed investigations that allow scientists to safely test newly developed drugs on a group of people who volunteer in the hopes they’ll help pave the way for a new treatment, either for themselves or family members with the disease. Clinical testing is a billion dollar industry in Canada with pharmaceutical companieand Health Canada paying for most of the research. At any given time, there may be up to 2,000 trials researching and testing new drugs.

There are four phases of clinical trials. Phase I trials, which are carried out on a small group of healthy volunteers, are the first step toward proving the new treatment is effective – and safe – for human use. Phase II trials involve several hundred patients who suffer from the condition the drug is meant to treat and who are divided into two groups – one group receiving the experimental drug and the other a placebo – to discover if the drug is working as anticipated. Roughly, only one-third of drugs fail to make it past the first two stages. Phase III trials involve a larger test group (up to several thousand patients) and are designed to give researchers a more thorough understanding of the drug’s efficacy, possible side effects and effective dosages. This stage can take up to two years. Phase IV trials are conducted after the drug has hit the market and monitor its ongoing effectiveness and safety.

Volunteers are needed
One problem researchers face is finding enough patients to take part in the clinical trials, says Todd Georgieff, vice-president of strategic initiatives for Endpoint Research Ltd., a Guelph, Ont.-based company that makes sure clinical trials are conducted under Canadian and international ethical and safety guidelines. And while the shortage of volunteers certainly isn’t unique to Canada, there are reasons why clinical trials here often have trouble attracting participants.

Next page: Counting on altruism

“In Canada, only three to six percent of cancer patients participate in clinical trials,” says Georgieff. “Ideally, this number would be double.” More clinical trial participants would not only expand our knowledge of cancer research but also attract pharmaceutical companies to stage more clinical trials in Canada.

One of many reasons trials are short of patients is that the structure of our health system discourages participation. Usually, you can join a trial if your family doctor or specialist is involved and suggests you take part. Failing that, your physician must refer you to the doctor who has been hired to run the trial. Your doctor may balk at this if he’s unfamiliar with the trial or the specialist running it.

The location of most trials is another factor limiting enrolment. Most take place in major cities, such as Vancouver, Calgary, Toronto and Montreal. “The trials go where the patients are,” Georgieff explains. Although some middle-sized centres do have active trials, rural Canadians and those who live in less populated or isolated communities seldom have a chance to participate.

In order to attract more volunteers, some clinical testing companies offer financial compensation to the participants, often targeting students or unemployed people – a practice that raises ethical and safety questions. It’s believed that seven people have died as a result of their participation in clinical trials. A follow-up study found that each person who died had some serious pre-existing health condition or were on medication but withheld this information so they wouldn’t be rejected from the trial. Money may have been a motivating factor in their decision to hide their condition and take the risk of participating.

Depend on altruism
Most trials, however, draw on the altruism of volunteers instead of offering financial compensation. These trials refund out-of-pocket expenses such as bus fare, mileage and parking costs. Some pay a minimum wage for time lost at work. Conversely, doctors who run clinical trials are paid quite well, earning anywhere from $200 to $1,000 dollars a patient. The issue of physicians being paid by pharmaceuticals can be a thorny one, raising all sorts of conflict-of-interest concerns. But Georgieff is quick to point out that there’s a huge amount of work involved in running a clinical trial; usually, the physician must hire an assistant and sacrifice two or three days a week from a busy (and lucrative) practice to do a thorough job on the study.

Next page: Weigh the pros and cons

Weighing the pros and cons
If you do find a clinical trial that appeals to you, weigh the benefits against the drawbacks before joining. First, the benefits. By participating, you’ll be playing a large role in bringing a potentially life-saving treatment to market. Moreover, during your time in the trial, you’ll receive cutting-edge treatment, which you may not have had access to otherwise. A cancer patient, for instance, who may have exhausted all options, will often participate in a clinical trial as a hopeful new treatment alternative. Plus, you’ll have access to the top-notch specialists running the trial, a huge benefit in Canada, particularly at a time where waiting lists mean you may only be able to see a specialist once every three or six months.

The major drawback is obvious: taking untested medication means there will be unknown risks and side effects to deal with. Moreover, if you take part in a clinical trial, your lifestyle will be disrupted. You’ll be under intense scrutiny by the medical investigators, which could mean regular hospital visits for extensive batteries of tests, sometimes as often as every two to four weeks, depending on your condition and the nature of the treatment. Another drawback is there’s no guarantee that the drug will work, meaning all your efforts and expectations could go down the drain.

Understand your involvement
After weighing the pros and cons and deciding you’re a candidate for a clinical trial, you’ll sign the “Informed Consent” document. Completing this form signifies you understand the terms of the trial, which pharmaceutical company is sponsoring it, what the goals are and what will be done with the results. Plus, you’ll be told about the risks involved, the safeguards in place and an estimate of the number of hospital visits required.

“The Informed Consent form should make it clear that participation is always up to the patient as well as letting them know they have the right to withdraw whenever they want,” says Georgieff, emphasizing patients should never feel coerced into taking part. “Before signing, the patient should have a chance to sit down with a doctor or nurse to work out any concerns they may have.”

Once the results of the clinical trial are finalized, the findings are evaluated by medical experts whose conclusions are published in medical journals. Only then can drug manufacturers begin making their application to Health Canada for approval of their new medication.