Medical institutions, federal agencies, pharmaceutical corporations and patient advocacy organizations conduct thousands of clinical trials annually, and according to John Schall, CEO of the Caregiver Action Network, family caregivers are the backbone of the clinical trial experience. "Caregivers predominantly act as care managers," he explains. Among other roles, "they make sure appointments are kept, and they manage medications and medication regimens -- both compliance and adherence." Learning about the world of clinical trials, however, is akin to learning to speak a new language, and it can be overwhelming to both caregiver and care recipient -- especially in the middle of a health crisis. So here is a simple breakdown of the top 10 facts you need to know about clinical trials:
1. What are Clinical Trials?
Clinical trials are a combination of interventional studies and observational studies conducted on volunteer patients. Interventional studies research how drugs, procedures, devices or lifestyle modifications affect a select group of patients with a common health condition. Observational studies research trends among select patients, through comparing released medical data and healthcare records or through monitoring a group with a common health condition. For example, investigators may observe a select group of women to learn about the effects of different lifestyles on adrenal health. Although observational study participants are not assigned an intervention as part of the study, they may continue with whatever interventions are part of their routine care, such as pharmaceutical prescriptions.
2. Why Conduct Clinical Trials?
The purpose of clinical trials is to develop and bring to the market new and effective treatments for disease.
3. How Common Are Clinical Trials?
In 2006 alone, there were about 15,000 drugs in the research and development (R&D) pipeline (1) and about 2.5 million clinical trial volunteers.(2)
4. Who are the Players in a Clinical Trial?
Key players are the trial sponsor, who pays for the study; the principle investigator, who oversees the research; medical staff including doctors, nurses and social workers, who work directly with patients and collect data; the caregivers, who oversee patient compliance with clinical trial protocol; and the patients, who are the subject of study. "Patients and caregivers are the heart and soul of this research," says Craig Lipset, Head of Clinical Innovation at Pfizer Worldwide Research & Development. "No new medical advances would be made without them."
5. Why Might My Loved One Choose to Participate in a Clinical Trial?
According to Ken Getz, founder and board chair of the Center for Information and Study on Clinical Research Participation (CISCRP), there are numerous reasons to participate in a clinical trial -- from access to new and alternative treatments, to personalized and expert medical attention, to free medicines and procedures, to the desire to leave a legacy by helping the advancement of medicine. "Participants may know in their heart of hearts that [a specific treatment] will not cure the progression of their disease," Getz elaborates on the latter reason, "but if they participate, they may be able to help future patients."
6. Where Do I Find Out about Clinical Trials for My Loved One's Health Condition?
First, contact a patient advocacy organization that is ailment-specific to the condition affecting your loved one, and ask about clinical trial options. For example, if your loved one has Alzheimer's, contact the Alzheimer's Association. A simple internet search using the formula of "[disease name]" + "association" should pull up some of the top national advocacy groups. Second, visit the National Institutes of Health website on clinical trials, to find a list of upcoming research studies looking for volunteers. Third, contact CISCRP for information about, plus the added bonus of support throughout, upcoming clinical trials.
7. Does Participating in a Clinical Trial Mean Stopping Other Medical Therapies?
In some cases, there may be contraindications between therapies already being used and the therapies that will be used in a clinical trial. It is imperative to let the clinical trial medical team know exactly what therapies a loved one is receiving, whether prescribed or over the counter. These therapies may include drugs, supplements, bodywork, dietary modifications and so on. The medical team, in some cases in combination with the care recipient's own doctor or holistic health provider, will determine which therapies can be continued and which may need to be stopped before participating in the clinical trial.
8. How Risky Are Clinical Trials?
According to Lipset, observational studies -- such as the sharing of medical files -- typically pose no added risk, and the risk of interventional studies depends on numerous factors. For example, if two known drugs are being compared to see which is more effective in the treatment of a particular illness, the risk is low. It is important to note that even if a medication is FDA approved, an individual may suffer adverse side effects. If an unknown drug is being tested, however, the risk depends on how far along that drug is in the R&D phase: The more it has been tested, the less risky it is. Even tried-and-true drugs or procedures may be risky, adds Rebecca McCue, division research manager of the Stanford Systems Neuroscience & Pain Laboratory (SNAPL), such as in the case where an FDA-approved drug is used to treat an illness other than that for which it was approved.
While holistic intervention such as cognitive behavioral therapy poses negligible risk, other alternative medicine therapies may pose some risk. "A number of alternative therapies don't go through approval with the FDA," explains Sean Mackey, MD, PhD, Chief of the Division of Pain Management at Stanford University. For example, he says, nutritional supplements are unregulated by the FDA and therefore "have not been through the close scrutiny that pharmaceuticals go through. The vast majority are not dangerous agents, but we simply don't know their long-term safety and efficacy...We haven't studied a lot of these over-the-counter supplements in large-scale trials, because there are not drug companies interested in doing that."
To determine the exact risks associated with a particular clinical trial, experts agree, it is important to speak directly with the principle investigator and medical team of the trial your loved one is considering.
9. What steps are involved in evaluating the safety and efficacy of drugs used in clinical trials?
Pharmaceutical trials begin with years of pre-clinical research in laboratories, followed by testing on animals and concluding with several phases of testing on people. Before moving from one stage of research to another, the clinical trial team must submit an Investigational New Drug application (IND) -- through which both the Food and Drug Administration (FDA) and independent ethics committees review results, prior to authorizing the continuation of the study. When a study begins working with people, it starts off with a small group in Phase I and steadily increases the number of people involved, until Phase IV -- where the research team begins exploring additional indications for that drug. After completing Phase IV, the clinical trial team submits a New Drug Application (NDA) for approval to go on the market. Of the 5,000-10,000 drugs that annually enter R&D, only 250 make it to pre-clinical trial testing; only five make it to clinical trial testing; and only one makes it to FDA approval.(3)
10. How involved should I be in my loved one's decision to participate in a clinical trial?
To ensure the safety and quality of life for your loved one, get actively involved in evaluating the pros and cons of participating in a clinical trial. For starters, your loved one "may struggle to understand and weigh the risks and benefits" of participating in a trial, says Lipset, so by gathering and sifting through all the relevant information ahead of time, you can explain exactly what is involved. In addition, agree Lipset and Schall, you can help your loved one recognize and evaluate the impact not only of the risks and benefits of the treatment itself, but also of the protocol demands of the trial -- including travel requirements, financial commitment, time constraints and additional caregiving duties. Be sure to discuss issues that affect not only your loved one, but also yourself, advises Schall, since you will be a central player on the clinical trial team.
Stay tuned formy next article, "Top 10 Questions to Ask When Considering a Clinical Trial."