A clinical research trial may be the only way you can get early access to a breakthrough arthritis treatment; however, there are pluses and minuses so it's important to understand the questions that need to be asked and answered.



It should be emphasized that a clinical trial is just another component of standard arthritis treatment. It should be considered as an option for patients receiving conventional care.

Clinical trials are research studies that evaluate a new drug or treatment in three separate phases, prior to FDA approval. In a Phase I trial, the safety and the dosing level of a new drug are tested in a small group of patients. The trial examines the pharmacologic action of the drug in humans and its side effects at different dosage levels.

Often, the earliest subjects in a phase I trial are normal volunteers.

In a Phase II trial, the drug is further tested for safety and for efficacy in a larger group of people who have the disease under study.

Once efficacy has been established, the drug is tested in a Phase III trial among several hundred to thousands of patients to further explore its safety and efficacy and whether or not it is better than conventional treatment for arthritis.

Phase III trials attract the most attention among arthritis patients; however, not everyone can be in a trial, nor are they guaranteed to get the drug being tested. To be in the trial, patients must first meet entrance criteria, which are very specific and which exclude many patients. If they are accepted, patients may be randomly assigned to different treatment groups, depending on the phase of the trial.

In a Phase III trial, the “test” group receives the new drug, while the “control” group receives either the standard treatment for that specific type of arthritis or a placebo (an inactive pill, liquid or powder). In some trials, called a “single-blind” study, researchers are aware of who is taking what drug, while in others, called “double-blind,” neither the participants nor the research team knows what the participants are receiving.

Sometimes trials are stopped early, for a variety of reasons. One possibility is that one treatment is found to be vastly superior to the other, while another is exactly the opposite -- a treatment is found to be dangerous. When trials end early because of positive results, the patients receiving the beneficial treatment will continue to get it, and also, the rest of the patients in the trial are offered the drug as well.

Obviously, if the results are negative, the drug is immediately stopped in all patients.

Make sure you see an expert rheumatologist. It may be worth traveling some distance to find the best expert for your arthritis condition.

Educate yourself. Read everything you can about the center and the trial.

Questions to ask:

• How long has the center and the rheumatologist been conducting research?

• Have there been other studies that evaluated this drug, and if so, what were the results?

• How do the risks, side effects and benefits compare with my current treatment?

• What are my odds for receiving a placebo if I participate in the study?

• Who is the research team that will take care of me during the study?

• Will involvement require an extra time or travel commitment on my part… and will I be reimbursed for expenses?

• How might the study affect my daily life?

• How will I be informed about new risks that may be identified during the trial?

• If the treatment is working for me, can I continue getting it even after the study ends?

• What are my costs associated with the trial? Will my insurance cover the costs? If not, what will it cost me?

• Are there other patients already taking part in the study whom I could speak to?

• Will I be able to find out about the results of the study at the end?

Research clinical trials are an option for getting first crack at new drugs, saving money, and also for helping to make a contribution to medical research and to the future health of others with arthritis.