Clinical Research in Hydrocephalus
by John Kestle, M.D.
This article is published with permission of the Hydrocephalus Association. It first appeared in the Hydrocephalus Association Newsletter, Fall 2000.
Research has always been an essential component of neurosurgery, but traditionally 'research' has been equated with laboratory experiments. 'Clinical research' is different - it involves patients. Clinical research is important because things that work in the lab might not work in people, and some of the things that we already do in people might not work.
Clinical research can be done in a way that gives accurate, useful results if certain principles are followed: (1) The data should be collected prospectively, i.e., when the patient is seen. This is more accurate than collecting the data by going back and 'reviewing the charts.' (2) A control group is essential. Patients who receive a new therapy should be compared to a control group of patients who receive the old therapy or no therapy. If all the patients in a study receive the new therapy, no comparison is possible. (3) Randomized trials are the best studies for assessing new treatments. In a randomized trial a patient's treatment is determined by the flip of a coin (or something similar). This results in two groups of patients that are equal prior to treatment and allows a valid comparison of the two treatments.
Obviously, none of this is possible unless patients are willing to participate in such studies - but why participate? What's in it for you? The answer is, 'it depends.' If you are in a study and you are randomized to the treatment that turns out to be the better one, then you have benefited from being in the study. If there is no difference between the treatments or if you received the treatment that turned out be worse, then you have not received a direct tangible benefit from participating. The most compelling reason to participate is to help get the answer as soon as possible. This means that beneficial treatments will be available to you and other patients sooner and useless (or harmful) treatments will be stopped.
If you are considering participation in a clinical trial, there are a number of things to be aware of and/or ask:
1. Will the doctor ask you if it's OK to include in the study? Probably, but not always. Retrospective studies based on chart reviews usually do not require approval of the IRB (Institutional Review Board - the hospital/university committee that reviews and approves research). Prospective studies: IRB approval and consent is usually required. Randomized trials: IRB approval and consent definitely required.
2. What can you expect? Someone - the surgeon or resident, a study nurse, a research assistant or other person working on the study - will come and talk to you. It should not be a representative of a company if the research is industry-sponsored. She or he should explain the study, answer your questions, ask for your written consent and assure you that your personal and medical information will be kept confidential.
3. What questions should you ask? What is the usual standard of care; how does the study treatment differ? What are the risks of the treatments? If I'm in the studydo I have to come for extra visits/tests? Will it cost me time/money to be a study subject?
4. Randomization: What, exactly, happens? You must decide if you are willing to participate without knowing which treatment you will receive. If you agree, your treatment will be determined at random - this means that you will be assigned one of the two study treatments by a process similar to the flip of the coin.
5. What if you don't like your Rx? Be prepared ahead of time to accept either treatment - don't enter the study if one of the treatments would be unacceptable to you. It is very difficult to interpret the study results if patients enter and then change their minds. You are certainly free to leave a study after you enter it, but it's best if you start with a reasonable willingness to stick with it.
6. What not to expect. The surgeon to tell you which treatment is better - if they know the answer, they shouldn't be participating. A quick answer. Trials usually last for several years, the time generally required to accumulate enough participants and follow them for an adequate time after treatment. At the end of several years, at least we have an answer based on solid evidence, ratehr than repeating the same old arguments based on non-scientific data and opinion.
Clinical research is a partnership among patients, surgeons, researchers and, in some cases, industry. All parties benefit from well-designed clinical trials. If you are asked to participate, give it some consideration - clinical research can be an essential component in our fight against hydrocephalus.