If you're eyeing clinical studies as possible medical treatment, here's the 411
May 18, 2016 11:30AM
● By Cate Reynolds
Try MeClinical studies of new medicines and medical procedures could offer cutting edge cure-alls to rare and previously untreatable diseases (yes, Zika is on our minds). For some patients, clinical studies offer intrigue. For others, they may offer hope and treatment. A typical scenario (or atypical for that matter) plays out thusly: A patient diagnosis is made for which there is little known cure and only manageable treatment; patient worries and starts web researching the condition; clicks through many links before landing on a clinical study that may offer hope; hits print and brings the information to his/her specialist…where to go from here?
We’ve put together a primer of need-to-know basics if you’re exploring how to become a participant. Though a web query is likely where you’ll start researching options, our disclaimer is that the conversation should always begin with your primary care physician or specialist.
According to Clinicaltrials.gov, the federal National Institutes of Health’s face of information for clinical studies, “A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies.”
For starters, there are basically four types of bio-medical organizations through which clinical trials and observational studies are performed:
- Hospitals/doctors/health care providers
- Academic medical centers/universities (think Harvard, Johns Hopkins)
- Private pharmaceutical companies (think Pfizer, Novartis)
- Public sector agencies (think National Institutes of Health)
The questions on most patients’ minds are: is there a clinical study examining my condition? If so, by who? And, how can I participate?
Chances are there is a clinical study, either actively occurring or in the planning stage, of your specific condition. There are thousands of studies conducted in the United States each year investigating new drugs, devices, procedures, diet, or behaviors. (Clinicaltrials.gov offers a database search of studies on its homepage.) Clinical studies used in drug development are sometimes described by phase. These phases are defined by the Food and Drug Administration as follows:
- Phase 0: Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies)
- Phase 1: Studies that are usually conducted with healthy volunteers and that emphasize safety. The goal is to find out what the drug’s most frequent and serious adverse events are and, often, how the drug is metabolized and excreted.
- Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition). For example, participants receiving the drug may be compared to similar participants receiving a different treatment, usually an inactive substance, called a placebo, or a different drug. Safety continues to be evaluated, and short-term adverse events are studied.
- Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
- Phase 4: Studies occurring after FDA has approved a drug for marketing. These including post-market requirement and commitment studies that are required of or agreed to by the study sponsor. These studies gather additional information about a drug’s safety, efficacy, or optimal use.
To accurately pinpoint a study and its principal investigator (the lead doctor/researcher conducting the study), it’s recommended that a patient explore options with a medical specialist first, who can access information and cross-reference with other specialists in the field about such opportunities.
The reality, however, is that the criteria one needs to meet in order to participate in a study is very specific. Only the principal investigator and the study’s protocol will determine eligibility and acceptance. You may or may not meet that criteria.
According to Clinicaltrials.gov, a typical study protocol will outline the following:
- The reason for conducting the study
- Who may participate in the study (the eligibility criteria)
- The number of participants needed
- The schedule of tests, procedures, or drugs and their dosages
- The length of the study
- What information will be gathered about the participants
Additionally, before a patient commits to participation, he/she should fully understand how their medical health and information is protected, known as the “informed consent process.” “The informed consent process is intended to protect participants and should provide enough information for a person to understand the risks of, potential benefits of, and alternatives to the study,” states the NIH. And there are many questions and answers to explore with the principal investor. Just a few could be:
- Why do researchers believe the intervention being tested might or might not be effective?
- What are the possible interventions that I might receive during the trial?
- How often will I have to visit the hospital or clinic?
- How long will the study last?
- Who will pay for my participation?
- Will I be reimbursed for other expenses?
- What type of long-term follow-up care is part of this trial?
Perhaps, the single most important question of all could be: Does this clinical study feel right for me? A question that only you can answer.
For more detailed information about clinical studies, visit Clinicaltrials.gov.