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Suppose you are ill with a serious or chronic condition — cancer, diabetes, psoriasis or any of a number of diseases - and your doctor suggests you consider taking part in a "clinical trial" of a new therapy that may or may not prove better than the current standard of care. How do you decide if this is a good idea for you or not?
Especially in Houston, where so much groundbreaking biomedical research is under way, such a choice is not uncommon. At M.D. Anderson, for example, more than 11,000 patients opt to take part each year in clinical trials of potential new cancer therapies.
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If the option of a clinical trial ever comes your way, there will be a lot to think about, so let's consider a few of the basics.
1. What is a clinical trial? A clinical trial is a research study with people. Trials involve the use of untested or partially tested new drugs, or novel combinations of previously tested drugs, or new pieces of medical equipment or diagnostic tests. Clinical trials are done in a carefully prescribed manner, with every step written down in a "protocol" that details what will be done in the trial and why.
2. When is a clinical trial called for? An orderly sequence of clinical trials determines whether a drug or device may eventually be used in routine medical practice. Phase I trials determine the initial safety, a preferred dose and schedule for a new drug; Phase II trials determine if the new drug works; and Phase III trials determine if the drug works better and is at least as safe as the current standard. Phase I trials involve small groups of patients; Phase III trials include many patients, often at several participating research centers.
3. What are some possible benefits to me in taking part? Many trials may offer clinical benefits that would not otherwise be available if the patient was receiving standard therapy that is not very effective. This is especially true in Phase III trials when a promising new treatment is compared to an older one. Even in early-phase clinical trials, patients may experience limited or temporary benefits while also contributing to knowledge that will help other patients in the future.
4. What are some possible risks to me? Risks of harm are usually more predictable in a Phase III trial that compares a new treatment to the current standard of care treatment. In contrast, risks can be less predictable in a first-in-humans clinical trial (Phase I) of a drug that has only undergone animal testing. Toxicities can be affected by the dose level and by how the drug is metabolized, so Phase I participants may receive a dose that is either more toxic or less effective than ideal. Phase I studies also may require more time by participants because of increased safety monitoring and pharmacology studies.
5. How do I decide whether or not to take part? That decision begins with a face-to-face discussion with the physician researcher, who should explain the purpose and details of the trial clearly and answer questions directly. The nurse assigned to the clinical trial also can be very helpful with information and explanations. A confidante or family member can help by taking notes or recording the conversation for later review. Part of the decision-making process involves the review and signing of an informed consent document, which includes detailed information about the design and purpose of the trial, its potential benefits and risks, known frequencies and severities of side effects, the rights of participants, and much more. The informed consent document should be signed only when the participant feels comfortable with the facts and can make a voluntary decision.
6. How are people selected for a clinical trial? Eligibility criteria are set in advance and all potential participants are screened to ensure they meet those criteria. For example, participants might all have "advanced disease" for which all known treatments have not worked. On the other hand, all participants may be newly diagnosed with "no prior therapy" for the illness. Setting such criteria helps rule out differences between participants and ensures an apples-to-apples evaluation of a potential treatment.
7. What if I change my mind before the treatment ends? This happens for many reasons - side effects are unacceptable to participants, or they move far away from the treatment center, or decide that trial requirements are too demanding. A participant may withdraw from a trial at any time and for any reason without any further obligation.
8. Who watches out for my best interests? Lots of people do, starting with an Institutional Review Board (IRB), which is required by the federal government at institutions conducting clinical research. An IRB is comprised of clinical experts, public members and others who review and approve the scientific basis for and details of each clinical trial. Federal agencies such as the Office for Human Research Protection and the Food and Drug Administration provide oversight and monitor and audit institutions that conduct clinical trials. Federal review procedures are rigorous.
9. Will I know which treatment option I am receiving? That depends on the type of trial. Sometimes the treatment is obvious. In certain randomized trials, knowing the type of treatment could undermine the trial's objectivity and integrity, so neither the doctor nor patient knows until the trial is "unblinded" at its conclusion. In other types of trials, both the physician and patient know from the beginning exactly what treatment is being received.
10. Will I ever know the results of the trial? Again, in most trials, clinical benefit, if any, will be apparent to individual participants. Data on the overall success of a treatment is known when the results are published in a research journal or presented at a scientific meeting, so that other physicians use the information for the benefit of patients. If you are interested in learning about the eventual outcome of a clinical trial, ask the physician to let you know. As a public service, the National Institutes of Health provides detailed information at clinicaltrials.gov.
Freedman is a professor of gynecologic oncology and clinical research authority at The University of Texas M.D. Anderson Cancer Center.
