Lupus Research: How it Works, What You Should Know to Participate

Medical research is conducted by standardized procedures -- so all patients are exposed to the same things -- to assure that the results are valid. The primary types are clinical and laboratory. Within clinical are outcomes, epidemiological and health services research.

• Clinical research, also known as patient-oriented research, involves whole patients - people like you - and answers questions about how the individuals are doing. It may consist of interviewing or examining a patient, having the patient complete questionnaires, reviewing patient charts. Patients who participate are called subjects.
• Laboratory research is conducted apart from the patient, but may still involve patients because samples of your tissue or blood may be studied. Other forms of laboratory research involve animals or chemicals.
• Epidemiology is the study of patterns of diseases among large groups or populations of subjects. For example, what "kinds" of lupus patients will get kidney disease? Those of a certain age group? Race? Those who take particular medications? Epidemiology tells us about patterns, not individuals.
• Health Services is the study of how particular services can change what happens to patients. Health services include things like doctor visits, medications, physical therapy, health education classes, books and other educational materials, home health aides, etc.

The results of clinical research are called outcomes. For example, what happens after a lupus patient is treated with a particular drug? Does the patient's skin rash improve or worsen? Can steroids be decreased? If so, how quickly? The answers to these questions are the outcomes.

Who decides what should be researched?

It all starts with a question. The research question is what the investigator wishes to answer. It can relate to what causes or is associated with a particular condition (risk factor), what makes an existing condition better or worse, what happens to groups of patients with the passage of time, or simply what "goes on" in particular types of patients. The research hypothesis is the investigator's best educated guess as to what the answer to the research question will be. This hypothesis is based on the researcher's personal experience and prior research of others.

When a researcher has a question and hypotheses, the investigator designs the "protocol" or procedures for the study. The protocol is then submitted to the Institutional Review Board (or IRB) of the hospital. This Board is a group of medical and non-medical personnel who are responsible for evaluating each protocol and making certain that this research will not violate the rights of the study participants, is legal, and ethical. The investigator may not begin the study until the IRB gives its approval. This approval must be renewed every year and if anything about the original study procedures changes.

How are studies designed?

The research design is the process by which the scientific question will be answered. It includes what kinds of patients will be studied, what will be evaluated and how, how often assessments will take place, and how the results will be analyzed. Further, studies may be:

• Cross-sectional, examining subjects at one point in time, vs. longitudinal, examining the subjects at different points over time;
• Retrospective, looking at the past (such as by reviewing medical charts) to determine what has already happened, or prospective, look forward to assess what happens over time.
• Observational, simply monitoring (through questionnaires, reviews of medical records, or blood tests) what is naturally happening to patients without altering the usual delivery of care or the subject's lifestyle; or interventional, requiring subjects to do something "extra," such as taking a medication, participating in educational or exercise programs (as well as having medical records monitored, blood or biopsy testing, etc.).
• Controlled, in which two groups are compared; in interventional studies, one group of subjects receives the medication or other intervention being evaluated and the "control" group does not; in observational studies, the control group usually doesn't have the same condition as the patients being studies.
• Blinded, which means that the subjects (and often the investigators) do not know which patients receive the intervention or treatment being tested and which are receiving a placebo or alternative treatment. To insure that there is no bias in the study, it is "unblinded" only after completion of the study.

Who gets to participate?

If you've heard about a research study that interests you, the first step is finding about eligibility. Not every study wants to examine all patients with a particular condition. Therefore, all studies have inclusion criteria (characteristics that the subject MUST have) and exclusion criteria (characteristics that the subject MUST NOT have) to participate. These criteria are necessary to keep the research standardized.

How are research participants' rights and privacy protected?

The IRB must approve all research to insure it is legal and ethical before each study is begun. A study is approved only if the IRB members agree that it will not infringe upon the rights of the patients and that it is not likely to bring harm to the patients.

All research is voluntary. You cannot be forced to participate in any study. You should never feel as if someone is coercing you to enroll in any research. If you choose not to participate in research, this will not affect the medical care you receive.

Before participating, researchers are obligated to get your informed consent. They must explain all the procedures and obligations of the study in a way you understand. This is your chance to ask questions about the study before you give your consent. If you agree to participate, you sign a consent form which completely explains the research procedures, your role in the study, and who is responsible for the study, and whom to contact if you have any other questions or in case of emergency. Keep a copy of the consent form for your records. Even after signing the consent, you may withdraw from any study at any time and for any reason.

All information collected about you and your condition is completely confidential. Any data used in public (such as in medical journals) will not identify you by name. You will be completely anonymous. Even your own doctor will not have access to the information you give to the researcher, unless you give your permission. (However, you should notify your physicians of any study you participate in.)

Why should a patient be interested in participating in research?

It may benefit you directly and immediately. But research also helps to improve the scientific understanding of your disease/condition so that other studies can be done and new ideas for treatment and improved quality of life can be developed. Even if the expected outcome doesn't occur, this still helps science. Negative results are still important results. It is just as important to know what doesn't work for whom as it is to know what does work.

What's the difference between a research study and a registry?

A Research Registry is just a collection of information on a group of patients who have something in common. It usually includes basic facts (like name, age, sex, race), medical history, and sometimes psychosocial information. This information is kept to assist with research. It is not necessarily for any particular study but might be used in the future to understand the epidemiology of this population, to identify patients for new studies, or to help design more studies based on what information is available.

The HSS SLE Registry is an ongoing collection of data on the lupus patients seen at the Hospital. It includes your demographic information, general medical information, medications, and lupus history (including how active your lupus is and how much damage it has done to your body). This information is updated every three to six months.

Some LUPUS research projects at HSS that you may wish to find out about:

• The SLE Registry and Repository - Contact Heidi Norbis at 212-774-2223
• Lupus and Cognitive Function - Contact Helene Jurgens or Kristen Abbott at 212-606-1867
• SELENA (Study of Estrogens in Lupus) - Contact Elizabeth at 212-774-2808
• Lupus and Atherosclerosis - Contact Heidi Norbis at 212-774-2223
• The Fibromyalgia Registry - Contact Jasmin Kwok at 212-774-2808

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posted 2/18/2001

Summary of a presentation given at The SLE Workshop, a free support and education group held monthly for people with lupus and their families/friends. Summary prepared by Diana Benzaia.