Jeff Rodgers sits in his doctor’s office pouring over patient consent forms one last time before “signing my life away,” he jokes. His signature will bring to a close months of preparation, a process that began by replying to an advertisement in local newspaper. The sponsor of the ad was looking for participants for a study evaluating a potential new treatment in the fight against diabetes.
As a 40-year veteran in his own personal fight against Type1 diabetes, Jeff consented to numerous interviews conducted by telephone screeners, attended several appointments where both doctors and diabetes educators evaluated his general health and well-being, and was grilled with a series of written tests designed to explore his ethical outlook on life in general as well as to assay his willingness to adhere to strictly prescribed protocols.
Have I done my homework, Jeff asks himself as he puts his pen to the consent form. Myriad last minute thoughts rush through his mind. Am I entering this study as informed as I can be? Have I asked all right questions? These and many questions are quite common among participants enrolling themselves into research studies and clinical trials. And while some trepidation is both expected and helpful, being as informed as possible by asking the right questions before signing the consent form, can help to alleviate your fears and allow you to get the most out of the experience upon which you are about to embark.
WHAT IS A CLINICAL TRIAL?
Deploying new medicines and treatment therapies to the frontlines of the fights against disease begins with extensive theory and planning. For those theories that appear to hold promise, the process moves forward to laboratory testing. Here, extensive testing is conducted on animals, generally on rats and mice, though this can vary depending on the nature of both the disease and the treatment.
Finally, if the treatment under study makes it through the animal testing phase, it will be brought to clinical trial, which is generally the final phase in what is usually a very lengthy process. In the article, Clinical Trials: A Chance To Try Evolving Therapies, the Mayo Clinic writes:
…clinical trials are used to evaluate potential treatments that have had some positive effect against disease in the test tube, in animal experiments or in a limited number of people. The goal of clinical trials is to determine if a treatment both works and is safe.
According to research protocols cited and observed by the Mayo Clinic, clinical trials are typically conducted in three (sometimes four) phases:
- Phase 1: This initial phase is primarily concerned with the safety of the drug or therapy in question. During this phase, researches are seeking answers to questions like: how do the participants react to the drug, what are the potential side effects, what are the correct and optimal dosages, and so forth. This phase can last a few weeks to several months.
- Phase 2: During phase 2, researchers are concerned with whether the drug is performing as intended, and whether it is as good as — or better than — current available treatments. This phase is sometimes split among groups who are testing the drug or therapy itself while another group is tested against a placebo. There are many factors in play at this time that can have an effect on the duration of this second phase, and it is not uncommon for Phase 2 research to last as long as two years.
- Phase 3: If the results of phases 1 and 2 show promise, phase 3 trials involve the rollout of the therapy in question to a much larger group of participants. Because this is a much larger scaled operation, previously undiscovered side-effects are often revealed. Also, during this phase, participants are split into groups where some receive the current conventional therapy and others receive the potential new therapy under study. This final phase usually lasts one to four years.
Data concerning the duration of each phase is provided by the National Institutes of Health and the FDA Center for Drug Evaluation and Research.
PROS AND CONS OF PARTICIPATION
There are a variety of issues to consider when thinking about participating in a clinical study. While there are a plethora of resources on the internet outlining reasons both for and against, the Susan G. Komen Breast Cancer Foundation has developed an online pamphlet entitled, FACTS FOR LIFE — CLINICAL TRIALS, that list the pros and cons in an easy to understand format.
- Opportunities to try new medicines and therapies not yet available to the public
- Contributing to research that may (hopefully) benefit others who suffer
- Opportunities to receive top-of-the-line medical care regardless during trials
- New treatments may not work as well as standard or current treatments
- In randomized trials, you may not receive the treatment under study
- Potential for side effects is very real
- Unexpected costs may arise that are not covered by your insurance or by the study
IMPORTANT QUESTIONS TO ASK
With an understanding of what clinical trials are and how they are conducted, it is important to know what questions to ask yourself, your personal physician, and the team of physicians and researches conducting the study. It is imperative that you consider the following questions prior to enrolling in any clinical study:
- Who is conducting the study, and why? What is the scope and goal of the study?
- What are the known risks and potential side effects (if known) both during and after the study? What care is available in the event that side effects do occur?
- Will there be any pain or discomfort, and if so, what is the protocol for addressing these events?
- How will the study affect daily life? It’s important to know where your participation required (geographic location), how often are you expected to be at that location, will you be seen by the physician in charge, and do you have the time required to fully participate?
- Can I drop out of the study at will and of my own volition? Are there any official terms and conditions of regarding early withdrawal?
- What kind of follow-up care is available upon completion of the trial?
- Who is the point of contact for any questions or concerns? How readily available are they?
- What is the reimbursement schedule (if any) for costs such as travel to and from the study, participation time, etc.
For anyone considering participating in clinical trials, it is important to ensure that you are comfortable putting your questions to those in charge. Their willingness to listen and to address your questions should act as an indicator as to the basis of your future working relationship. You should be completely satisfied with the responses you receive; after all, when you become the subject of the study, your health and well-being are at stake.
FOR THE GREATER GOOD
“It is no exaggeration to say that a large share of the credit for current treatment successes belongs to the people who participated in the clinical trials conducted over the last decade,” writes Kevin Armington on his website, The Body. Whether or not the drug or therapy under study ever makes it to the level of “standard of care,” participating in clinical trials contributes to the accumulation of knowledge about the disease and treatments. Knowing what doesn’t work is as important as knowing what does.
As for Jeff Rodgers, who finally pens his name on the consent form in his lap, the opportunity to fight on the frontlines against a common enemy (in his case, Diabetes Mellitus), is all the incentive he needed to enroll in a clinical trial. That, and knowing that he has done his due diligence with respect to asking the right questions and feeling confident with the answers, provides a surprising (though not uncommon) psychological boost knowing that you’ve joined the fight against a particular disease. Be comfortable, be assured, and be aware and know that you are contributing to the greater good of humankind.