Give a lasting holiday gift of health: Volunteer for a medical research study
That toy the kids whined about for weeks may be broken hours after emerging from the box. The gift card you gave will be frittered away, probably on junk. That adorable new pet adorned with a bow? It may be a pain in the parents’ anatomy by the new year.
If you’re like millions of Americans, you want this festive holiday season to also have higher meaning, especially when it comes to sharing things of value. So here’s some “out-of-the-box” thinking. Consider giving a unique gift of health by participating in medical research.
This volunteerism can be done by young and old, the well and the sick. It may benefit you in many ways: You will know you helped others in a special way. You may assist in advancing new treatments. You also may get extra medical attention, even access to therapies unavailable to the public.
Just by joining a clinical study, you will gain insights into a critical component of contemporary medicine. And you will learn more about a vital element in your health care—your right to informed consent.
Participating in medical research has risks. It requires a commitment not made lightly. Don’t let your eyes go all aglow at the prospect. Keep them and your mind wide open as you unwrap a distinctive way to help others. And read on.
Clinical trials can benefit volunteers
The dramatic headlines almost daily detail medical-scientific progress, including: New treatments based on patients’ genes, cancer drugs that get the body’s own systems to attack disease, vaccines being developed for viral infections like Zika and HIV-AIDS, and improved ways to avert heart disease. Look closely at any of these, and they share something fundamental: They require human testing, medical research conducted in rigorous, tightly supervised, and ethical fashion in what broadly are termed clinical trials. These occur, as the figure above shows, relatively late in what can be a long scientific process. Only after experts have labored long in labs, in research and development (R&D) and especially with animal studies, do they progress to clinical, interventional, or observational studies with slowly increasing numbers of human subjects.
In these studies, investigators may seek to understand how a disease runs its course, how to prevent or test for it, as well as how best to diagnose and treat it. The research may determine patients’ quality of life with drugs, devices, or treatments. Health professionals (doctors, nurses, and Ph.Ds.) conduct these studies, backed by research institutions (including medical schools and universities), the federal government, and businesses (drug or device makers). It often occurs at universities and academic medical centers, because this kind of study can be costly, time- and resource-intensive. It adds to institutional prestige—and potentially profits. The Washington, D.C.-area is rich with clinical trial sites, including top hospitals, elite academic medical centers, and the various institutes of the National Institutes of Health (NIH) in Bethesda. That could make your role in medical research easier and more convenient.
Types of clinical trials
In interventional studies, Uncle Sam notes, patients may be treated with: “Medical products, such as drugs or devices; procedures; or changes to participants’ behavior, such as diet. [These] trials may compare a new medical approach to a standard one that is already available, to a placebo that contains no active ingredients, or to no intervention. Some … compare interventions that are already available to each other. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants.”
In contrast, in observational studies, experts “assess health outcomes in groups of participants according to a research plan or protocol. Participants may receive interventions (which can include medical products such as drugs or devices) or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator (as in a clinical trial).” Observational research can involve following and recording data on large groups of subjects over extensive time. This is how the decades-long Framingham heart study led experts to emphasize exercise, diet, and other factors like stress-reduction in preventing coronary disease.
Participants in clinical studies must meet stringent requirements to qualify. Researchers value highly specialized subjects, including those with certain illnesses, past or present. It’s misunderstood that clinical studies only involve the sick or those with close relatives with specific diseases or conditions. Studies are planned in phases (see figure above). Early on, in Phase One and even Phase Two, researchers may want younger, healthier subjects to get baseline, control data. Children, veterans, and seniors also are welcome participants, enrolling under strict protections.
Benefits of participation
Study volunteers can benefit. They can get the satisfaction of knowing their participation helps others and may advance medicine. They often receive some compensation for their time and trouble. Besides their regular care—and anyone considering clinical trials should consult their personal physician—they may receive added medical attention. They may undergo intensive exams to assess their qualification for or status during trials. If seasonal allergies, say, seem to tee you up for a test of prescription drops to help ease watery, hay fever-inflamed eyes, you may also undergo at no charge a fuller battery of expensive allergy tests. If you’re about to be a control subject for a new blood pressure medication, you might get a complete, no-cost physical.
More important, in later stage trials, patients with chronic or serious illness may receive novel or innovative drugs or treatments not yet available to the public. The costs of this therapy may be partially or fully covered by trial sponsors and participating research institutions. Some gravely ill cancer patients have enrolled in trials and received drugs costing more than $100,000 a year—and that made huge differences in their health. But the effectiveness of experimental drugs may not last or they may require them for longer than is covered in a clinical trial. All research outcomes aren’t optimal and cheery. Volunteers must understand this from the start.
Risks? Informed consent is critical
You’re upset after learning that a loved one is afflicted with a disease with few favorable treatments. You’re healthy and decide you want to do something to prevent this from happening to anyone else. Or maybe you discover that your family has a predisposition (like the BRCA gene mutation for cancer, for instance), and, though it hasn’t hit you yet, you want to assist investigators in attacking it before it does. Or you may have cancer, heart disease, or other conditions, and you have decided, at some stage in your illness, that you want to help others. No matter the motivation, when you choose to enroll in a clinical trial you will be a volunteer. And among the few.
This isn’t a mere formality. Laws require investigators to detail for you answers to dozens of questions about a clinical trial, including who will run it, who will benefit from it, as well as its goals, methods, duration, demands (time and travel), potential costs to you, and, most critically, your possible health benefits and harms. Who oversees patient safety? What kind of protections will you have during and after the trial? Will drugs or treatments have side effects, and will these be lasting? The voluminous flow of information starts at the beginning of the process—it gets prominent display in online resources about medical studies and signing up to be considered for them (see below). If you’re healthy and raring to go, this may be patience testing; if you’re sick and possibly failing, it may seem excruciating.
Patient involvement and transparency have become a major aspect of medical research. And this includes a critical affirmation of one of your basic, vital patient rights: informed consent. This isn’t something that gets handled with a lot of signatures and initials on piles of paperwork. It is the duty of anyone who provides you any kind of medical treatment. Informed consent expresses a concept at the core of any free society: Each person has a right to decide what to do with his or her own body, as long as you don’t hurt someone else. That includes medical care. But this is a right that many scared people are quick to give up. They want to surrender decisions to doctors, not realizing two critical facts: Doctors may have very different values. They may not, for example, put as much emphasis on whether a surgery disfigures the body if it offers a slightly higher chance of a patient’s living some uncertain additional amount of time. Doctors also may have conflicts of interest that make it hard to give objective advice. For example, they may make a lot of money if you opt for their surgery and no money at all if you make a different choice.
Because MDs are just as flawed and imperfect as everyone else but know a lot more medical facts than most lay people, the law and medical ethics place a simple duty on every doctor: Give patients the important facts so they can make intelligent decisions about what treatment to have and where to get it. That’s why informed comes before consent. Information comes first. Consent is meaningless without the facts. Informed consent is really about building a bond between doctor and patient through a candid dialog that doesn’t leave out anything important. Patients, medical ethicists say, want to make a leap of trust with professional caregivers. But trust is built on honest information. It’s not a leap of blind faith. This is especially true in clinical trials, where participation is voluntary.
IRBs, single- and double-blind studies, and placebos
To be sure, the idealistic wish to heal the sick and cure diseases drives and sustains many fine doctors, nurses, and Ph.Ds, as well as universities, academic medical centers, and hospitals where they labor. But health care is a big business with huge financial stakes. Institutions gain a giant reputation boost and competitive advantage by being leaders in medical-scientific research. Drug development, via clinical trials, can lead to major profits. Although many medical studies seek to and eventually produce modest advances, some research can be likened to oil wildcatting—and many institutions of note not only are out there digging, they have intellectual property teams ready to reap riches from the rare gusher. In 2014, just 15 major American universities produced 70 percent of all revenues among their peers from patent-license royalties. New York University got $650 million in such payments for the rheumatoid arthritis drug Remicade. Emory University received $525 million in royalties for Emtriva, an HIV drug. The University of California at Los Angeles has signed a deal for $520 million for Xtandi, a prostate cancer treatment. And Northwestern University says that as much as 18 percent of its $18 billion endowment, the eighth largest in the country, can be traced to pioneering work in campus labs that resulted in Lyrica, a hot-selling, non-opioid prescription drug to treat nerve pain and seizures.
Here’s hoping that you’re blessed with great holidays, and if you choose to give your time to enroll in a clinical trial, that it helps you, your loved ones, and all of us with some significant health benefits!
Intrigued? If you want to learn more about enrolling in a clinical trial, talk to your own doctor first. She may be able to offer counsel on suitable choices. If you’re volunteering because you want to help a friend or loved one, you may want to talk with them and their health professionals to see if they have insights about appropriate research opportunities for you. Advocacy or support groups for specific illnesses or conditions also may offer productive ideas.
Uncle Sam has important resources you can tap, starting with the web site clinicaltrials.gov, which can be accessed here. This is one of the major registries of trials, and officials have sought to provide abundant information about research studies, participating in them, and other pertinent resources available through this site. The search engine allows volunteers to search a database of more than 230,000 trials around the world. Type in “healthy volunteer,” if that fits you, and many options will pop up. Alas, the trial abstracts are replete with medical and scientific jargon and may not be the easiest for lay people to figure out. Researchers are still grappling with this issue. It is invaluable, though, for prospective trial participants to study thoroughly the available information, including from the federal Food and Drug Administration, about risks and benefits of trial enrollment.
Although volunteers play a crucial role in medical research, investigators struggle to recruit them. Few Americans participate in clinical trials. Doctors may need to step up their role in letting patients know about them. Ditto for advocacy groups that aim to help individuals with specific diseases.
Uncle Sam, too, has come under fire for not doing more to assist. In response, the U.S. National Institutes of Health has tried to improve its major web site resource on clinical trials, boosting the information it provides, as well as aiming to simplify and clarify how users search for appropriate research projects. Hospitals, academic medical centers, and other research institutions have stepped up their communications about their trials.