Understanding the Role of Clinical Trials and How They Might Help You

• Simple lack of awareness among potential volunteers
  
• Restrictive inclusion criteria — for example, being ineligible because of prior chemotherapy
• Mistrust of the process
• Fear of side effects of the new drugs
• Fear of not receiving the new therapy, but rather being given the standard-of-care comparison therapy
• Fear of being a guinea pig

The history of clinical trials is said to date back to 1747, when British physician James Lind conducted a systematic trial among British sailors with scurvy — a disease now known to be caused by a vitamin C deficiency. He dosed some sailors aboard a long sea voyage with a combination of lemon and orange juice to see if it helped cure the condition.

This trial also featured two other advances in experimentation: It was double blind (neither the researcher nor the participants knew who was getting the active drug) and placebo controlled (some participants received an inactive substance).

Today, most large clinical trials of drugs en route to U.S Food and Drug Administration (FDA) approval are randomized, double blind, and placebo controlled — the gold standard. Still, trials of medical devices and surgeries frequently are not randomized, and many, if not most, devices aren’t subject to the same clinical rigor as drugs.

Phase 1: Researchers test a treatment in a small group of people (about 20 to 80), for several months. The main purpose is to learn about safety and identify side effects, often as dosage levels are increased. About 70 percent of drugs pass this phase of testing.

Phase 2: Usually (but not always) a larger group of people (100 to 300) receive the treatment to further assess how effective it is, as well as its safety and side effects. About one-third of experimental drugs successfully complete phase 2.

Phase 3: The treatment is given to still larger numbers (1,000 to 3,000) and can last several years. This phase is intended to provide researchers — and, ultimately, the FDA — with more complete information on effectiveness and possible adverse reactions. Between 70 and 90 percent of drugs entering phase 3 studies succeed. After a successful phase 3 trial, a pharmaceutical company can request FDA approval to market the drug.

Phase 4: This phase, also referred to as postmarketing surveillance, occurs after a treatment has been approved by the FDA and made available to the public. At this stage, researchers track safety in the general population and gather information on benefits and optimal use. If problems occur, restrictions may be placed on the drug or it may be taken off the market.

Others are cognizant of past trials that resulted in products or treatments that subsequently turned out to be problematic, such as thalidomide, the sedative marketed in the late 1950s to pregnant women for morning sickness that ended up causing thousands of severe birth defects. Another example is the Dalkon Shield, an intrauterine device (IUD) used in the early 1970s that prompted more than 200,000 lawsuits by women harmed by it.

Over the years, and often in response to mistakes from the past, practices have been put in place to protect and inform people. For example, the Tuskegee tragedy led to a process known as informed consent, which requires informing prospective participants about the intervention in question and potential risks involved. Informed consent is now considered a critical component of both patients’ rights and clinical trials themselves.

Trials can be found for virtually every condition imaginable. While many of the most publicized trials often involve high-stakes therapies, such as those for late-stage cancer, lower-stakes trials are plentiful. Registered trials include treatments for everything from acne to insomnia or anxiety.

And they’re not all about pharmaceuticals. Many clinical trials involve interventions with therapies such as acupuncture, botanicals, mineral supplements, and even aromatherapy and yoga for hot flashes, for example.

People with a preference for lifestyle interventions to prevent or treat illness can help play a role in moving those disciplines forward as well. A number of trials involving diet and exercise have been registered.

Completed studies in this area include one assessing the effects of a low-fat vegan diet versus a Mediterranean diet on body weight and insulin sensitivity (run by the Physicians Committee for Responsible Medicine) and one researching the effects of intermittent fasting on the prevention and treatment of age-related diseases in participants who consumed a Mediterranean or Western diet (done by the Washington University School of Medicine).

In the end, whether to participate in a trial is a personal decision, but perhaps the most important takeaway is that the decision should be an informed one. Whether the thinking is “buyer beware” or “well informed is well armed,” the consensus is that it’s best to gather as much information and input as possible from trusted and valued sources, including doctors, other health professionals, family, and friends.

“Our motto is ‘Education before participation,’” says Getz. “We encourage patients and their families and friends to gather the facts, speak with other patients and professionals, and ask a lot of questions.”

There are several searchable databases that list available clinical trials, including:

• ClinialTrials.gov
• CISCRP.org

Additionally, you may find searchable listings at local healthcare centers, on disease-specific organizations’ websites, and even advertised in local newspapers.

In most cases, participating will be at no cost to you. Sometimes, insurance is billed for some devices, drugs, or services. This should be clarified in the informed consent process.

What to ask: Can you spell out what the cost will be to me?

Some clinical trials will not involve travel. Others will require regular check-ins. Find out what your obligations will be beforehand.

What to ask: Will my travel costs be covered?

A treatment trial may be comparing a new treatment to a standard treatment or to a placebo. You have no control over which you will receive.

What to ask yourself: Am I willing to participate if I’m not getting the new treatment?

You can opt out of a trial at any time.

What to ask: How should I handle the situation if I decide to opt out partway through the trial?

Some trials involve years of follow-up.

What to ask: When does the trial end, and how long will my obligation last?

• The number of clinical trials worldwide is growing. But participation and awareness remain low, despite the potential benefits, particularly among women and minorities.
• People who have participated in trials have reported satisfactory outcomes. Experts say that while there are some risks to joining a trial, most of the fears of harm or being used as a guinea pig are unfounded.
• There are trials for almost any condition and even trials on lifestyle interventions. There are many resources available to find a trial that could be right for you.