Prednisone may distort lab tests. Allergy tests and tuberculosis tests will be invalid. Pap smears may be misinterpreted as cervical dysplasia. Test results for tuberculosis and cancer may also be affected. Other medications may also interfere with medical test results. If you have questions consult your doctor or pharmacist.
While current therapies for some autoimmune diseases may be adequate to keep your disease in balance, advances in biotechnology research may revolutionize the future treatment of these potentially debilitating conditions by more specifically attacking the cause of the autoimmunity.
Drug development is a monumental team effort consisting of scientists, physicians, research professionals, and most importantly, the volunteers who participate in clinical studies.
Following is a summary of the clinical trial process which we hope you find useful in your journey towards understanding if clinical trial participation is right for you and/or someone you care about. It is our hope that by providing you with this information, you will become more familiar with the drug development process and explore further with your physician the possibility of clinical trial participation.
The process of developing new drugs is complex, time consuming, expensive and risky. Unfortunately, most drugs in development never make it to patients. The challenge is even greater for the development of drugs for diseases that affect limited numbers of people. Drug development is regulated by the Food and Drug Administration (FDA). The FDA estimates on average, it takes 8 years to study and test a new drug before it becomes available for patients.
The search for a new treatment begins in the laboratory with scientific research about a particular disease and to identify potential compounds that can be made into drugs to treat the disease. Potential compounds are analyzed for their physical and chemical properties, first in the laboratory and then in animals to determine their pharmacologic properties and toxic effects. Before a new drug can be approved for use in humans, it must be thoroughly tested in the laboratory.
Clinical trials are studies using the drug in humans, designed to identify new and better treatments for diseases. Clinical trials determine if a drug is safe and effective, at what doses it works best and what the side effects are. The organization Pharmaceutical Research and Manufacturers of America estimates that only five in 5,000 compounds that enter preclinical testing make it to human testing, and only one of those five may be safe and effective enough to eventually reach pharmacy shelves. The process has been likened to looking for a needle in a haystack!
Clinical trials are carried out in phases. Phase I determines how much of a new drug or treatment can be safely given to humans. A small number of patients (20-80) are given increasing dosages of the drug or treatment and carefully monitored for side effects. Phase II trials are conducted in even larger groups of people (100-300) to determine whether the new treatment actually destroys or slows progression of the disease and further evaluates the safety of the treatment. Phase III studies are conducted in even larger groups of patients to confirm the drug's effectiveness, monitor side effects, compare it to commonly used treatments and collect information that will allow the drug to be used safely. Following Phase III trials, FDA approval for marketing or rejection of the drug may occur. Following approval, Phase IV post-marketing studies are often initiated, which enable additional information about the drug's risks, benefits and optimal use to be gathered.
Clinical trials may be sponsored by pharmaceutical and/or biotechnology companies, but are administered at hospital and/or research institutions by physician experts in the disease field. Each site (hospital and/or research institution) is required to receive approval by an IRB (institutional review board) before clinical trials on patients can begin. An IRB is a panel of scientists, ethicists and non-scientists charged with overseeing clinical research where the clinical studies are conducted. It is the responsibility of the IRB to review the study protocol or research plan and ensure that risks to patients are minimized and that the study complies with all necessary guidelines.
As a volunteer in a clinical trial, you are participating in the development of new medical therapies – therapies that may offer better treatments and even cures for life-threatening and chronic diseases. The hope of personally benefiting from a new treatment or the desire to take part in research that might one day benefit other people is what motivates some people to take part in clinical trials. For patients suffering from a chronic disease, like pemphigus vulgaris, where a good treatment does not currently exist, the need for clinical trial participation is even more critical. Without patients as active clinical trial participants, it makes it more difficult and takes it much longer to make new treatments available.
So, is the risk of participating in a clinical trial worth a benefit that comes with no guarantee for the participant and/or others with the same disease? Ultimately, through a process of information gathering, weighing of the risks and potential benefits, and perhaps some soul searching, the answer will be one only you, the person considering trial participation, can provide.
Without individuals with these diseases willing to participate in clinical trials, safer, more effective therapies than existing ones would never make it to the clinic.
Although participation in a clinical trial may not benefit you directly, and is associated with a certain amount of risk, there is a possibility that information learned in the trial may contribute to better treatment of autoimmune diseases in the future. We understand that participation in clinical trials takes away time from work and home. However, individuals who participate in clinical trials generally receive the highest quality of medical care available and the frequent monitoring required may detect potential disease flares earlier, thus making them easier to treat with standard therapies.
A randomized, double-blind, placebo-controlled study of patients with PV using Infliximab
The purpose of this trial is to determine the safety of infliximab (Remicade) for the treatment of patients with pemphigus vulgaris. Secondary clinical endpoints include the number of days to cessation of new blister formation, the ability to reduce prednisone to 25% of the initial dose by week 18.