Novel and Experimental Therapies
Before any drug can be approved for use as a medical treatment, it must undergo a series of research studies, called clinical trials, to determine if it is safe and effective. During this period of testing, which can take as many as 15 years, the new product is considered “investigational” and is available only to volunteers who participate in the trials. Clinical trials are done in several phases numbered as 1, 2, 3, and 4. Sometimes, as part of the clinical trial design, not all of the volunteers receive the investigational medication being studied and instead are given already approved medications or a placebo (a chemically inactive compound, sometimes referred to as a “sugar pill”).
The medical teams involved in the research studies collect extensive data on the effects of the drug, how the drug is absorbed by the body, and both the short- and long-term side effects, as the trial proceeds. The study results are then gathered and analyzed, providing the basis for the decision to approve or not approve the investigational product as a medication for use by the medical community and their patients. People with serious illnesses who are not responding to already available treatments sometimes enroll in clinical trials to gain access to medical treatments that could be helpful. However, there is no guarantee that an investigational product will provide any benefit, and of course there are always risks with all drugs, whether investigational or well-known.
There is hope, however, that safer and more effective therapies for people with lupus will be available in the near future. A robust pipeline of potential new drugs is in various stages of clinical development, and nearly two dozen biotechnology and pharmaceutical companies are now engaged in developing new treatments for lupus. New information about how the immune system functions is providing insight into the progression of lupus and ways to impede or halt disease activity.