by Brad H. Goodman, MD, CPI
Astronaut Neil Armstrong once said “Research is creating new knowledge.” Nowhere is that more true than in medical discovery. Indeed, advances in diagnosing and treating many debilitating illnesses have come from knowledge gained through the clinical research process. So what does the future of medicine hold? A cure for cancer, heart disease, asthma or diabetes? Earlier and better disease detection? And how will we get there?
The Role of Clinical Trials
Medical research studies involving humans are called clinical trials or studies. Every day, medical researchers uncover new information about diseases and their treatment through such clinical studies of new and existing therapies. These clinical trials are a critical, but often misunderstood, component of medical discovery.
Clinical studies examine new or current treatments to determine their safety, effectiveness, and potential in diagnosing, preventing or treating illness. Physicians often collaborate as principal investigators with pharmaceutical companies and other sponsors to facilitate clinical studies. Before clinical analysis can be initiated in humans, however, a drug or device must have gone through extensive testing in the laboratory and with animals.
Clinical trials are generally conducted in four phases. Phase I of a study is usually designed to determine dosage and effects of a new or existing drug and is done with a small number of healthy volunteers. About 70 percent of new drugs pass this phase of testing.
Once a drug has been shown safe, it moves to Phase II to determine its effectiveness. In the larger Phase II trial, researchers enlist volunteers to learn more about the proper dosing of a drug, how well it works in the treatment of disease, and how to manage any side effects. Phase II trials may be placebo controlled, which means participants may receive the new medication or a placebo (an inactive medicine). All participants are monitored closely by clinical study coordinators and supervising physicians to determine the relative effectiveness and safety of the new treatment. If the new therapy shows promise for being as good as or better than existing treatments, as about one third do, it moves to Phase III.
Phase III trials typically compare the new treatment with the best currently available treatment. Researchers may discover more effective treatments, better dosing guidelines, or improved ways of administering existing therapies. Phase III studies may involve several hundred to several thousand volunteers around the world. Seventy to ninety percent of studies that enter Phase III study successfully complete this phase of testing. Upon successful completion, a pharmaceutical company can seek FDA approval of the new treatment.
Some therapies may also enter a Phase IV or “post marketing” study. Here researchers may seek to determine the long term impact of an approved drug on a patient’s quality of life, whether an “improved” version of an existing treatment is truly better than the current treatment, or the relative cost effectiveness of various alternative treatments.
Should You Participate in Clinical Trials?
People choose to participate in clinical trials for various reasons. Volunteers may receive free, cutting-edge medicines or other treatments that would not otherwise be available to them. They may learn more about the treatment of their own or a loved one’s medical condition. And, study volunteers all receive study related medical care and compensation for their time.
A patient’s safety and rights are protected in many ways throughout the clinical trials process. First, an ethics committee called an Institutional Review Board (IRB) must review clinical studies before they begin. An IRB is made up of doctors, nurses and people from the community; it is their job to consider the risks and benefits of every clinical study before it can proceed. The Food & Drug Administration (FDA) sets requirements for drug testing and also reviews results from clinical research studies to determine if an investigational drug should be made available to the public.
Before you will be enrolled in study, a clinical study coordinator or other medical professional will look at your medical history and study requirements to determine if you are eligible for the study. The principal investigator or study coordinator will also explain what you can expect from the study, answer your questions, and obtain your consent before proceeding.
Through all phases of clinical trials, the patient volunteer is carefully monitored by one or more study coordinators and the physician principal investigator. You are always free to raise questions or concerns, and a supervising physician is always available to you.
How Do You Become a Study Volunteer?
Many local physicians have active research practices in addition to their work with patient care. Some have even undergone advanced training to become Certified Principal Investigators through the Association of Clinical Research Professionals. In addition, medical practices active in research often employ full time clinical study coordinators who can keep you informed of study opportunities within their practices. Information about ongoing studies may be available in the waiting or treatment rooms at your physician’s office. Research opportunities are also advertised in the newspaper or through other news media.
Clinical research gives us the knowledge we need to advance medicine; the resulting medical breakthroughs give us all hope that we will live longer, healthier lives. Volunteer participation in a clinical trial can be a rewarding experience, and it is an essential part of medical discovery. Talk to your physician or other qualified health professional about whether participation in a clinical trial is right for you.