NBIA Disorders Association Research Registry
NBIA has a research registry
to enable NBIA individuals to participate in current and future research
projects. If you would like to participate in the research registry, but
have questions regarding it, please contact Patricia Wood at pwood@NBIAdisorders.org
or call at (619) 588-2315. Otherwise, you can print the registry
mail it to the address indicated and we will keep your
information on file for applicable research projects. Please
note that Adobe™ Acrobat Reader® is needed to view and print this
form. It may be downloaded
free of charge.
for Clinical Research
Most of us want a cure for our disease or a disease that may be affecting someone we love. Many of us would be happy just to settle for a safe and effective treatment, such as insulin for diabetes. However, neither cures nor treatments will become available without medical research.
When scientists are developing a new treatment for a common disease, it is relatively easy to find enough volunteers for a clinical trial. In that case, people have the luxury of saying, “Let somebody else be the guinea pig,” while we await a new allergy medicine or arthritis drug that has been proven to be safe and effective. However, people with rare diseases do not have that luxury because there may be very few patients on whom to test a new treatment, or few who meet certain eligibility requirements . Thus, the burden falls on us to volunteer for clinical trials if we are ever to have improved treatments or cures.
Stages of Research
“Clinical research” is research that is performed on humans, usually in a hospital clinic or a doctor’s office. There are four stages of clinical research: Phase I, Phase II, Phase III, and Phase IV.
The goal of Phase I testing is solely for safety. Volunteers are told they should not have any expectation that the drug will be therapeutic. Since this will be the first time the drug is used on humans, scientists do not know the proper dosage so they may start very low and escalate the dosage with each successive group of several patients until side effects appear.
Throughout Phase I, II, and III, volunteers are put through extensive tests to determine how the medication is impacting them, both in terms of effectiveness and side effects. These tests are specific to the proposed use of the medication, and may include blood and urine tests, x-rays, MRI or CAT scans, spinal taps, EKGs, and EEGs.
Placebo-Controlled Clinical Trials
The FDA also allows “crossover” studies so that no one will be denied the active compound. In crossover studies, volunteers may take a placebo for several weeks or months, and then cross over to the real drug for an equal amount of time. Conversely, a patient might begin with the active treatment, and several weeks later cross over to placebo. In this way, not only can groups of patients be compared, but individual patient results can be measured when on each “treatment arm”.
FDA does not require that half of the people in a clinical study take placebos. In some studies, only one-third or one-fourth of the volunteers will take a placebo and the rest may take the real treatment. Thus, your chance of getting a placebo may be relatively small. In other studies involving a disease that already has an accepted treatment, no placebos may be warranted because patients on the new drug can be compared to a “control group” of patients taking the standard treatment. In this case, the new drug has to be proven as safe and effective as the old drug, or safer, or more effective.
What Protections Do Research Volunteers Have?
The basic tenet of research involving humans is “informed consent.” This means no person is permitted to participate in research without his or her knowledge about the risks and possible benefits of the experiment, and his or her consent must be obtained. If a person is incapacitated and cannot give voluntary consent, or is a minor, there are specific rules applying to family members and legal guardians who can, under certain circumstances, provide consent.
Informed consent for a person who volunteers for clinical research must be provided in writing and orally. There must be no coercion or undue influence, and the experiment must be explained in language that is understandable to the volunteer. All possible risks and discomforts must be explained, as well as possible benefits. The researcher must also tell the volunteer about other possible treatments or procedures that might help them, as an alternative to the treatment being offered in the experiment.
The informed consent must also explain how the research team will keep the patient’s records confidential, and whether hospital staff, the FDA, or a company may have access to those records. If a volunteer is injured by an experiment, the informed consent must explain that the institution will provide treatment, and whether there may be costs to the patient for such treatment.
An informed consent document must provide contact information, both for someone who can answer questions about the clinical trial and for the person to be contacted if the volunteer should be injured. It must clearly explain that a person can refuse to participate in the trial without any objections from his or her doctor or hospital. If a person decides to participate, but later wants to withdraw from the trial, he is free to do so without any loss of benefits and no penalty. The document must also explain that the doctor can terminate a person’s participation, even if the patient objects, under certain circumstances (e.g., if you don’t comply with your doctor’s instructions, if there is evidence that the drug is not safe, or if harmful side effects may be experienced).
The FDA does not permit manufacturers to charge a fee for experimental drugs or medical devices (except under certain very unusual circumstances which FDA must individually approve), so you should not have to pay anything to participate in a clinical trial. In the case of medications for rare disorders, some small drug companies are forced to charge and have obtained approval from the FDA to do so, but they often have services to assist with insurance reimbursement or other financial assistance should you require it. Some hospitals, however, may try to recoup costs by charging your insurance company. It is therefore important to clarify all financial matters in advance, and in writing, before you decide whether to volunteer.
Benefit Vs. No Benefit
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