Director of the Rehabilitation Clinical Trials Center
- Please Volunteer for Clinical Research!
Seems like we're always being asked to do something. Pay your taxes, donate to charity, do your homework, feed the dog. Well here's another "ask". I ask those of you who have been unfortunate enough to contract a lung disease to consider volunteering for a clinical research study. This generally involves being poked and prodded in the interests of advancing medical knowledge about your particular disease.
Why is this so important? I remember a time some 40 years ago when I was just getting started in research when it was thought that pretty soon studies involving human subjects would be replaced by computer simulations that a computer could be programmed to determine whether a new therapy would or would not work. Well, despite huge advances in computer technology, this hasn't worked out very well. Based on computer simulations, studies of cells in a dish and experiments in animals we can get hints about whether a given new therapy might work in people with a given disease. However, it has been shown time after time that the proof of effectiveness has to come from studies of people with the disease in question.
Who performs these studies? People like me, medical doctors who have chosen a career in medical research. Viewed from a certain perspective, people like me seem to have made a strange choice. For a physician, medical research is a radically different life style than clinical practice (treating patients). For one thing, the medical researcher must accept delayed gratification. For a physician treating patients, the goal is to make people feel better. A patient is seen, a diagnosis is made, a treatment is prescribed and the patient responds…this can take place over a period of a day or two. For a medical researcher, the goal is to obtain new knowledge about a disease (for example, whether a new treatment works) is a much more drawn out process. Here are the usual steps:
- Formulate a question
- Search previous research to be sure that the question hasn't been answered previously
- Design a study to answer the question
- Put this design into the format of a research grant
- Find an agency that might fund the project. This might be the federal government, local government, a charitable foundation or for-profit organizations (for example, the pharmaceutical industry).
- Be prepared to wait. Most applications require several months (maybe 4-6) to be reviewed. Often grants are rejected initially, but are approved after revisions suggested by the grant reviewers.
- Perform the study. Many studies require several years to organize and execute.
- Analyze the results.
- Compose a report of the study in the form of a manuscript.
- Submit the manuscript to a medical journal. These manuscripts are reviewed by "peers" (other medical researchers) who often suggest changes but may also vote that the manuscript not be accepted for publication. The process of getting a manuscript accepted usually takes 3-6 months.
- Wait the 4-6 months it usually takes an accepted manuscript to be published.
Medical researchers generally accept this process because finding out something new is really a kick! Also medical research yields a more varied life; most of us travel often to present our findings to audiences of other physicians. Most of us work quite hard (I work longer hours than most, but not all, of my clinical practice friends). I make less money than most of my clinical practice friends (but still do pretty well, thank you).
What is the most difficult part of doing medical research? Often it is finding people willing to participate in research studies. A given question may require a certain kind of participant; the study may have a number of "inclusions" and "exclusions". We (and our staffs) spend a good part of our time trying to cajole people to take part in our studies. One thing we are forbidden to do by the committees that oversee our work is to pay people taking part in our research studies lots of money. We are allowed to compensate for time and inconvenience entailed in participation, but not to pay enough to make people do what they would not otherwise agree to do…we can not financially "coerce" people to take part.
Many people think that volunteering for a clinical research project is a good way to get advanced treatment for their illness. Surprisingly, this is not a reliable expectation. First of all, many studies are "placebo controlled" that is, some portion of the participants receives an inactive substance (a "sugar pill"). This is often a necessary part of the design of the study. Further, we usually genuinely do not know whether a treatment will be effective; if we did, there would be no reason to do the study.
Risks of taking part in a clinical research project are another thing to consider. The clinical researcher is obligated to disclose all possible risks; the list is often long. In many (probably most) studies the risks are either rather minor or very unlikely; nothing approaching the risk of crossing the street in mid-town Manhattan.
So why should people consider volunteering for a clinical research? Well, if you are a COPD patient, let me ask you a question. Ever hear of Advair, or Symbicort, or Spiriva, or portable oxygen, or pulmonary rehabilitation, or antibiotics? If you've benefited from these therapies, thank those people who came before you who participated in the clinical research projects that demonstrated their value.
Being a volunteer in a clinical research project is often not very burdensome. Some time is involved. Usually there is some discomfort (drawing blood, puffing into a spirometer, sometimes more complex procedures). There is an "informed consent" procedure that is mandated: all of the procedures, time commitments and risks are laid out in writing in language you can understand. Before they can be initiated, all studies are evaluated and approved by an independent local committee with membership including non-medical people. Clinical investigators and their staffs are almost always friendly and approachable…a clinical research operation that behaved otherwise would not last long.
So, the next time you see a request for volunteers in these pages or elsewhere, please give it at least a second thought.
- Thanks and best wishes.