Participating in a Clinical Trial
Clinical trials test potential treatments in human volunteers to see if they should be used in the general population. They're an important part of product discovery and development and are required by the FDA before a new product can be brought to the market. The federal government has established strict regulations and guidelines for clinical research to protect participants from unreasonable risks. Although efforts are made to control risks, they can't be completely eliminated, so it's important to get a handle on the process and the risks that may be involved. Before deciding whether or not to participate in a clinical trial, it's useful to understand the process that a potential new drug or therapy undergoes before it is approved and marketed.
Only 1 in every 5,000 compounds survives the process of FDA approval in the United States, while the cost of developing a drug averages $897 million in a ten-to fifteen-year period. Clearly, all new therapies must weave their way through a stringent process before they are brought to market. Here's a brief outline of the process:
1. Preclinical trials. In this phase, potential therapies are tested in the laboratory and on animals to determine toxicity and side effects. After each stage, the sponsor of the new therapy meets with the FDA to determine their next steps and establish the parameters for future trials.
2. Clinical evaluation. In this phase, the sponsoring company approaches the FDA with the results from the preclinical studies and files an Investigational New Drug application. They also submit a proposal to study the dosage and safety in humans.
3. Phase I — Safety. Dosing for future trials and safety is assessed. In Phase I treatment trials, a small number of volunteer patients (usually between fifteen and thirty) are given the experimental treatment in gradually larger doses to test for any side effects or complications. The researchers conducting the trial will also try to determine what a safe dose would be and how it should be given.
4. Phase II — Safety and Efficacy. In this phase, studies are conducted to test for therapeutic effect as well as safety. The studies are usually larger at this point, with 40 to 300 participants. Several studies may be carried out in this phase so that the therapy can be studied in a variety of patient populations.
5. Phase III — Controlled Safety and Efficacy. In this phase, larger patient populations are used to confirm the beneficial effect from Phase II trials and to continue to monitor side effects. These trials can involve hundreds to thousands of people. The product is also compared to other treatment options or used in combination with other treatment approaches.
6. Regulatory filing. In this stage, the company submits an application for marketing approval, along with all the data collected in the trials. The FDA has at least six months to review a “priority” application (drugs for a life-threatening need) and ten months for a standard application. Final approval can take up to two years.
7. Phase IV — FDA Approval and Post-Approval Monitoring. Doctors can now prescribe the new drug, although the sponsoring company must continue to monitor any adverse side effects and report them to the FDA.
8. Manufacturing. The FDA regulates the manufacturing of drugs by, in some cases, testing several lots of drugs prior to marketing as well as inspecting manufacturing facilities and requiring companies to keep manufacturing records.
Which treatment each participant receives in these studies is determined by a process called randomization, where the treatments are randomly selected by a computer.
Phase II and III of a clinical trial often involve a control group, where one group is given the test drug and another is given a standardized treatment or a placebo, depending on the study. The control group provides the basis of comparison for the new treatment.
Studies are often blind studies or double-blind studies. A double-blind study is one in which neither the researcher nor the participants know who is receiving the experimental treatment and who is receiving the standard treatment or placebo. Blinding is done to ensure that neither the participant nor the researchers' expectations can influence the results.
Who Can Participate?
Each clinical trial has a set of inclusion and exclusion rules. These rules are designed to safely test the effectiveness of a treatment and to ensure the safety of the participants. In a trial for MS, for example, inclusion might be limited to people with a certain form of the disease, such as relapsing-remitting. A clinical trial may exclude people with certain medical conditions or those taking specific medications.
Certain tests and procedures are carried out on participants before the trial begins. These tests determine eligibility and establish the overall health of the patient and can include blood tests or neurological examinations, for example. Tests are also used to measure the effect of the treatment. These tests are often given before, during, and after the treatment to determine if the treatment improves the condition being studied compared to the control group. Clinical trials often require more tests and visits to doctors than would be required with standard treatment. For example, participants may be required to have regular MRIs, neurological exams, or blood tests.
All trials must follow a protocol, which is a set of rules on which the study is based. The protocol describes who is eligible to participate, the length of the study, details of the schedule and procedures, and any medications that will be involved, including the dosage prescribed.
The term informed consent refers to the guidelines that companies must follow to protect the participants in the trial. All of the key facts concerning the trial must be described to the volunteer within informed consent documents. The information will include the following:
Why the research is being done
What the researchers wish to accomplish
What will be done during the trial and the timeline involved
What risks are involved in the trial
What benefits can be expected from the trial
What other treatments are available
These documents should be studied carefully, and any questions should be asked before a decision is made. If a person decides to join a clinical trial, she will be asked to sign a consent form. Under informed consent, participants may ask questions or withdraw at any time before, during, or after the trial.
Every clinical trial in the United States must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected.
Should You Participate?
There are a variety of reasons people choose to join a clinical trial. The prospect of receiving a new treatment before it is available to the public is alluring to many people who are excited about a treatment's potential benefit. Contributing to research and society is another reason people want to participate.
In general, people who volunteer for clinical trials report a positive experience. A recent survey found that 54 percent of the people polled rated their experience as excellent. Still, it is important to understand the pros and cons of this volunteer opportunity. Here are some questions you should ask before making a decision:
Why is the research being done?
Has the study been reviewed and approved?
Why does the research team think the experimental medical intervention will work?
Where will the clinical trial take place?
How often will I have to go to the study site?
How long is the clinical trial?
What type of therapies, tests, and procedures will the participants have?
Will the therapies, tests, and procedures be painful?
Will participants have medical care after the study ends? If so, who provides it?
How will the therapies, tests, and procedures compare to those for the regular treatment?
Will participants be able to take their regular treatment and drugs during the clinical trial?
What happens if I am harmed by the experimental medical intervention?
As you can see, there is quite a bit to consider before deciding whether or not to participate in a clinical trial. Here are the pros to consider:
You may be among the first to benefit from a new treatment.
You'll be helping others by contributing to medical research.
You'll be closely monitored and receive high-quality medical care.
Weigh those against the cons:
Experimental treatments may bring unpleasant or serious side effects.
The treatment may not work for you, or it may end up being less effective than the available treatment.
You may receive a placebo.
Participating may require more of your time and energy than a normal treatment regimen.
There may be more tests and doctor visits.
Your health plan may not cover all your costs.
You may have to change doctors.
Before deciding to participate in a trial, it is imperative that you speak with your MS specialist first. She knows you best and will be able to help you make an informed decision.
Are people paid for their time in clinical trials?
Some, but not all, studies pay their participants. Somewhere between 33 and 40 percent of the 80,000 industry-and publicly funded human studies under way pay their subjects. Each year, 2.5 million people participate in research studies involving human subjects.