The Clinical Trial Experience
Clinical trials are research studies that generate data to support FDA approval of a new medicine or a new indication for an existing medication. They also grant participants early access to new medicines, which are being developed to help combat chronic and serious diseases. By volunteering for a clinical trial, patients take an active role in their health care by helping researchers test new treatments. In Oregon, 4,067 clinical trials since 2004 have targeted diseases and conditions like asthma, arthritis, cancer, diabetes, cardiovascular disease and Alzheimer’s disease.
What to Expect
Since clinical trials are often conducted in a doctor’s office, patients may need to devote more time to physician visits and physical examinations. They may also have additional responsibilities, like keeping a daily log of their health. All prospective participants must sign an informed consent document saying they understand that the clinical trial is research, and that they can leave the trial at any time. After consulting with their health care providers, patients can volunteer to participate, leading to a prescreening interview. If they fit the criteria and requirements of the trial, they can be enrolled.
Patient Expenses
Patients should ask during pre-screening interviews what it will cost them to participate in a clinical trial. Clinical trial sponsors usually pay for all research-related expenses and additional testing or physician visits required by the trial. Patients or their insurance companies may be asked to pay for any routine treatments of their disease. And it’s important to know some health plans do not pay for clinical trials. Patients should make it a point to learn if they or their insurance company will be assessed any fees and should determine if their insurance company will cover the expense of routine examinations. Patients who live a distance from the trial site should learn the clinic’s policy for covering travel costs and living expenses. The National Cancer Institute, for example, makes patients responsible for their own travel costs for the initial screening visits. Once a patient is enrolled, the Institute will pay for transportation costs for all subsequent trial-related visits. These patients will receive a small per diem for food and lodging.
Expanded Access
Successful completion of the clinical trials is required to demonstrate to the FDA that an investigational drug is safe and effective, so that it can be approved and made available to a broad patient population. Clinical trials are the primary route by which patients can participate in the drug development process, receive access to unapproved investigational drugs and contribute to the collection of safety and efficacy data necessary for FDA approval. For patients with a serious or life-threatening disease who are ineligible or unable to participate in a clinical trial, use of an unapproved investigational drug through an expanded access program may be an option. The current FDA process for a patient to gain access to an investigational drug through expanded access was established in 2009 in close consultation with patients, physicians and the biopharmaceutical industry. Expanded access programs are part of many biopharmaceutical companies’ commitment to patients.
Phases of Clinical Trials
There are three phases of clinical testing used to evaluate potential new medicines:
PHASE I—Researchers test the drug in a small group of people, usually between 20 and 100 healthy adult volunteers, to evaluate its initial safety and tolerability profile, determine a safe dosage range and identify potential side effects.
PHASE II—The drug is given to volunteer patients, usually between 100 and 500 people, to study its efficacy, identify an optimal dose and to further evaluate its short-term safety.
PHASE III—The drug is provided to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients (but sometimes many more thousands), to generate statistically significant evidence to confirm its safety and effectiveness. They are the longest studies and usually take place in multiple sites around the world.
Frequently Asked Questions
Clinical trials are conducted by physicians, investigators and researchers at various organizations including local research institutions, hospitals and universities.
Participation in a clinical trial often provides patients access to potential new treatments and to expert health care at leading institutions. By participating in a clinical trial, you play an active role in your health and the health of others by contributing to scientific knowledge that may lead to potential new medical treatments for future patients.
As with any medical treatment, participation in a clinical trial offers benefits and risks. Researchers conducting these trials will clearly explain the potential benefits and risks so you can feel confident about making the best decision.
Clinical trials are tightly regulated to ensure maximum safety for participants. All clinical trials involving human participants are monitored by an Institutional Review Board (IRB) to ensure high safety standards and processes are followed. IRBs are comprised of at least five members with a wide range of backgrounds, including at least one scientist, at least one non-scientist (such as a lawyer or professor) and at least one member of the community who is not connected to the institution or sponsor of the trial. Additionally, federal agencies such as the Office of Human Subjects Research Protection and Food and Drug Administration oversee clinical trials to ensure patient safety. By the time a potential new treatment is allowed to be tested in patients, it has been in development for at least three to six years, meaning it’s already been through rigorous preclinical testing. A treatment must be backed by valid scientific evidence that shows it is likely to be safe before it can be administered to patients in a clinical trial. Researchers conducting clinical trials will clearly explain the potential benefits and risks, so you can feel confident about making the best decision in consultation with your doctor. Participants always have the right to end their participation at any time, even if the study is not over. According to the Center for Information and Study on Clinical Research Participation, most clinical trials participants indicate they would participate again.
It’s always best to discuss with your physician whether a clinical trial is right for you.
Some clinical trials are meant for those who have exhausted all treatment options, while others are meant to test ways to prevent recurrence. Some are designed for healthy participants to test ways to prevent the illness in the first place. Clinical trials need participants such as:
- Healthy participants
- Patients in remission
- Newly diagnosed patients
- Patients with no other treatment options
It’s always best to discuss with your physician whether a clinical trial is the right choice for you.
Some clinical trials are meant to determine how a treatment works for a specific condition. Others are meant to track disease prevention and therefore require healthy participants. Most individuals can find a clinical trial in which they are eligible to participate.