Let’s survey a handful of the most frequently asked questions (FAQs) that patients and their loved ones have regarding clinical trials, how they work, the benefits to participants, and why they matter.
What are clinical trials?
Clinical trials are designed to investigate the safety and effectiveness of new proposed diagnostic procedures or treatments for a health condition. These treatments under study can include drugs, supplements, medical procedures like surgeries, or lifestyle interventions like changes to diet and exercise.
The goal is to demonstrate a new treatment’s safety and efficacy before introducing it to market so as to document any potential negative side effects and to make sure it works as intended.
In the context of neurology, clinical trials seek new diagnostic procedures and treatments for the more than 400 neurological conditions identified by the National Institute of Neurological Disorders and Stroke.
What rights do clinical trial participants have?
The 1979 Belmont Report enumerates in detail the rights afforded to clinical trial participants. These include the right to informed consent (to know and understand the procedure or medicine under study and its potential risks before beginning the experiment) and how the subjects were selected.
The rights enumerated in the Belmont Report are based on three primary values, which are justice, respect, and beneficence.
Can clinic trial participants quit whenever they like?
Yes. Because clinical trial participation is entirely voluntary, patients in clinal trials always have the option to terminate their participation at any time for any reason. Patients should notify researchers before quitting so that appropriate measures can be taken to safeguard their safety and restart them on any prior healthcare regimens they may have had before the start of the trial.
Do patients get paid for their participation in clinical trials?
Payment arrangements can vary widely. As a general rule, there is no financial compensation for trial participation. However, in many trials, patient expenses are covered – including, potentially, travel to and from the trial site, childcare, etc. In some instances, research groups may offer payment for participation as well. All financial details will be thoroughly disclosed before the trial begins as a routine part of informed consent.
Where do clinical trials take place?
The two primary settings for clinical trials are general practitioners’ doctor offices or hospitals. Participation often requires regular visitation to the trial site for ongoing evaluation. As mentioned previously, many research groups offer financial compensation for travel.
What are the advantages to participants in clinical trials?
The advantages to participants are severalfold:
- Thorough, ongoing monitoring of disease and treatment progress by healthcare experts free at the point of care
- Access to potentially transformative new therapies unavailable to the wider population (particularly beneficial for patients who have not experienced benefits from conventional, existing therapies)
- Being an integral part of the search for breakthrough new treatments that can potentially save millions of lives
How do researchers protect clinical trial participants?
Oversight entities staffed by medical authorities such as Institutional Review Boards (IRB), Data and Safety Monitoring Boards (DSMB), and various government agencies such as the Food and Drug Administration vet and monitor every clinical trial undertaken in the United States. One of their mandates is to safeguard participants.
As discussed earlier in the context of informed consent, patients have a right to full disclosure of all known potential side effects before a clinical trial begins.
Researchers then do their best to mitigate any negative health consequences resulting from negative side effects. Participants are entitled to receive all possible care to treat any side effects that emerge in the course of the trial.
Furthermore, researchers are ethically required to fully disclose any side effects previously documented in earlier phases of the study so that participants are fully cognizant of the risks.
Learn more about the protocols regarding the protection of participants in clinical trials.
What are the five clinical trial phases?
Clinical trials go through a series of phases. As the trial moves through each phase, they involve progressively higher numbers of participants and higher doses of the prescribed treatment. The purpose of the tiered phase system is to identify any negative side effects early to minimize risk to participants and screen out treatments that cause unintended harm or don’t work.
The five phases are as follows:
- Phase 0: only a handful of participants (10-20) at an extremely low dose
- Phase 1: 20-50 participants at a slightly higher dose. Only 70% of drugs tested at this phase make it to phase 2.
- Phase 2: approximately 100 participants. This phase lasts up to two years. Only a third of treatments in phase to make the cut for phase 3.
- Phase 3. Usually thousands of participants. This phase lasts up to 4 years. Only 25% of drugs in phase 3 eventually end up approved for use in the general population.
- Phase 4: Monitoring of the treatment’s efficacy and safety after it has been approved by the FDA and introduced to the market. This phase is often labeled “post-marketing surveillance.”
Who is eligible for clinical trial participation?
Specific and strict rules dictate who and who is not eligible for clinical trial participation. Researchers carefully screen each applicant to vet their appropriateness for inclusion in the trial.
There are two sets of criteria: inclusion criteria and exclusion criteria. Inclusion criteria refer to conditions that an applicant must meet in order to qualify to participate. Exclusion criteria refer to conditions that disqualify candidates from participation.
Inclusion and exclusion criteria can include:
- Pre-existing conditions
- Treatment history
- Smoking status
- Current medications
Learn more about inclusion/exclusion criteria.
What is the placebo effect?
The placebo effect is a fascinating and confounding psychological phenomenon often seen in clinical trials. Essentially, patients or observers who believe that they are receiving or administering an effective treatment might report a real or perceived benefit, even when there is no actual treatment provided.
The placebo effect is accounted for by administering a fake treatment like a sugar pill to a subset of participants and then “blinding” the study. Blinding means that patients, researchers, or both groups are unaware of who received the placebo and who received the real treatment, thereby ensuring greater objectivity.
Where can interested patients find clinical trials to join?
If you live in the Northwest Florida region and are interested in participating in a clinical trial, visit our regularly updated Northwest Florida Clinical Research Group clinical trials page.
Contact Northwest Florida Clinical Research Group to learn more about clinical trials
An important aspect of the work we do is to educate members of our community about the value of clinical trials, and to encourage them to join if they qualify to contribute to the discovery of landmark, potentially life-changing treatments for patients affected by various neurological disorders.
Contact Northwest Florida Clinical Research Group for more information about clinical trials. Again, if you are interested, check out our rolling list of current NWFLCRG studies welcoming new volunteer participants.