“Clinical trials” is phrase that’s always bandied about for illnesses such as cancer, and you may wonder what it actually is and whether it’s worth taking part. The use of human volunteers to collect data is not always for everyone. It is wrapped in the enigma of experimentation and feeling exploited. But what is it all about?
“Fear is a greater barrier than the reality.”
Robert L. Comis, M.D
The National Heart, Lung and Blood Institute, run by the US Health Department, describes a clinical trial as research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. These studies enable medical professionals to follow the best path for certain illnesses or groups of people. The NHLBI say: “Clinical trials produce the best data available for health care decision-making.”
So why are people reluctant to take part?
A survey by Harris Interactive, Inc., showed that of the patients who knew about clinical trials, 71 per cent still chose not to participate.
- Uncertainty of treatment – Fear of being treated as a guinea pig and getting a placebo is the most common factor that pulls patients away from clinical trials. In fact, placebos are rarely used in cancer treatment trials, and never in lieu of standard treatment, according to Robert L. Comis, M.D., president of the Coalition of National Cancer Cooperative Groups and chairman of the Eastern Cooperative Oncology Group in Philadelphia. Ninety-seven percent said they were treated with dignity and respect, and received excellent or good quality care.
- Worry about insurance – For our US friends, one in five participants were concerned that their insurance companies may not pay out. But for those who enrolled in clinical trials, 79 per cent said their insurance company ultimately paid for the care they received.
- Uncertainty of results/adverse effects – People are afraid whether the new therapy will work to improve their health or worsen their disease. Although researchers cannot guarantee outcomes, patient safety is apparently top priority. Each trial has enforced oversight, and patients also have rights that help protect them. Currently participant’s right are protected by regulatory bodies like the Institutional Review Board (IRB) in the US, and Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. There will be an equivalent to your native country, usually through the government.
- Inconveniences. Some people can’t commit to the time required to participate in a clinical trial. Others may find the available hours incompatible with their schedule. Travel is an issue for some, whether they’re unable to transport themselves or find a geographic location close enough. Other social and economic disadvantages contribute to some populations being underrepresented. Some trials even pay the participants for enrolling in a trial. However, FDA suggests people must learn thoroughly about the risks involved in a study before enrolling, and not get lured by the free treatment or money being offered.
- Missed opportunities – Low enrolment is attributed to many factors and varies on a per study basis, but if more people knew about clinical trials there would certainly be more participation. One study found that only one in three US adults knew about clinical trials, of the 1,000 surveyed.
The NHLBI state: “The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results.”
But does that ease our fears?
To be fair, it’s unusual for clinical trials to try something completely experimental. They usually go through a lot of scientific research before getting to this stage. That being said it is important to check the credentials if you do wish to take part.
It is one of the final stages of a long and careful research process. The process often begins in a laboratory, where scientists first develop and test new ideas.
If an approach seems promising, the next step may involve animal testing. This shows how the approach affects a living body and whether it’s harmful. However, an approach that works well in the lab or animals doesn’t always work well in people. Hence, research in humans is needed. It may start off with only a few patients, in order to ensure safety, while in later phases of clinical trials, researchers learn more about the new approach’s risks and benefits.
Why do they need volunteers?
Volunteers can play an important role at this stage of development, which is crucial for new treatments to reach pharmacy shelves. Without sufficient numbers of trial participants, the drug development process stalls and a trial must be repeated, scaled back or, even worse, the potential new therapy is abandoned. This lengthens the time it takes for new treatments to come to market. No amount of funding or other resources can compensate for the lack of clinical research volunteers.
According to the Foundation of Peripheral Neuropathy, across all diseases, 85 per cent across of clinical trials finish late due to difficulties enrolling participants and nearly one-third of trials fail to recruit a single subject and cannot ever begin.
Phases of the trial
Clinical trials are conducted in phases designed to answer a separate research question.
- Phase 1 Safety testing of a small batch of healthy volunteers – recruiting only a few patients, usually less than 100. The first few patients to take part (called a cohort or group) are given a very small dose of the drug. If all goes well, the next group have a slightly higher dose. The dose is gradually increased with each group. The researchers monitor the effect of the drug until they find the best dose to give. Doctors/researchers look at how the drug affects the volunteer: How is it absorbed? How is it metabolised and eliminated from the body? Does it cause side effects?
- Phase 2 Testing effectiveness and safety on small batch of patients – doctors evaluate the candidate drug’s effectiveness in a larger group of patient volunteers. In this case, researchers compare the efficacy of the drug on the patient compared to a different treatment. Patients are either given a placebo or a drug that is already used for treatment. Some phase 2 trials are randomised. This means the researchers put the people taking part into treatment groups at random. Doctors/ also analyse optimal dose strength and possible side effects.
- Phase 3 Testing effectiveness and safety on a much larger group of patient volunteers – Phase 3 trials usually involve many more patients than phase 1 or 2. This is because differences in success rates may be small. So, the trial needs many patients to be able to show the difference. This phase of research is essential in determining whether the drug is safe and effective. Patient volunteers are divided into three groups: the group that gets the standard treatment; the group that gets the new treatment being tested; and if doctors do not know if the new treatment is better than the standard treatment, but they believe it is as good and may be better.
- Phase 4 Researching long-term side effects – Phase 4 trials are usually done after a drug has been shown to work and has been granted a licence. The goal of this phase is to continue studying side effects of a new treatment.
If you take part in a clinical trial, you may be one of the first people to benefit from a new treatment.
However, there is also a chance that the new treatment turns out to be no better, or worse, than the standard treatment. It’s a chance / risk, and ultimately it’s your choice if it really is for you.
Where to find clinical trials:
- Centerwatch.com – global database of clinical trials
- NHS.uk – UK-based clinical trials
- UK Clinical Trials Gateway – Educational side of the NHS
- Global Register for Inherited Neuropathies – collects clinical and genetic information on patients from around the world diagnosed with various forms of hereditary neuropathies
- ClinicalTrials.gov – US health department based clinical trials
- World Health Organization – International clinical trials registry platform