Authored by: Nancy Meyerson Hess, MPhil, and Artem Andrianov, PhD
If you have been invited to participate in a clinical trial or if you are thinking about joining a research study, then you might wonder about the safety risks you should be aware of before enrolling. Even though clinical trials explore the safety and effectiveness of medical strategies, treatments, or devices, no clinical trial is completely safe for everyone.
A clinical trial helps make sure the clinical benefits outweigh the possible risks for most people. With this in mind; what is a tolerable level of safety risk for a patient? What is done to identify potential risks for patients when participating in a clinical trial, in addition to those mentioned in the Informed Consent?
In clinical trials we identify two different groups of risks, i.e. objective and subjective risks. Objective risks are the ones that arise from the technical side of a clinical trial. Subjective risks, on the other hand, are raised by patients who assess the potential risk of participating in a clinical trial through critical questions like: “What are the possible risks, side effects, and benefits of the study treatment compared to my current treatment?”.
Six questions to ask before enrolling in a clinical trial
1. Is it possible to be 100% risk-free regarding patient safety?
Reaching 80% up to 90 % risk-prevention is feasible and therefore realistic. As mentioned earlier, there is no such thing as a 100% risk-free clinical trial.
Conducting a clinical trial is a complex and time-consuming activity, and in most of the cases the execution of it requires quite a large organization. Clinical research studies not only involve your doctor or clinical investigator but also drug companies, CROs (Contract Research Organizations) and specialty organizations. Especially the latter group is charged with the implementation of processes that support the data quality control, which plays a critically important role in patient safety.
Carefully designed clinical research studies produce different types of information – clinical data – by different types of sources. So-called quality control functions delivered by the specialty organizations help to set out sufficient risk controls within clinical trials. Risk controls are automatically triggered by digital “traffic lights” that respond to critical data. They subsequently start activities that address the potential risk(s). Only when these risk controls are in place, can proper risk management, to ensure data integrity and patient safety, be facilitated. At the same time this process helps to increase the predictability of clinical trial success for both the patient and pharmaceutical company.
Despite regulated pre-clinical testing, the interaction of many variables, such as the human organism, disease states and complex biochemical reactions always leaves residual risk. Residual risk – threats that remain after all efforts to identify and eliminate risks at an early stage – is very difficult to plan for, control, and forecast.
2. Does this mean that when I decide to join a clinical trial I should not be concerned about my safety or health but should focus on my disease state and treatment instead?
Today, in most cases “yes”. However, in addition to following the instructions in the trial protocol, you should always be cautious of potential dangers. In a clinical trial, the clinical investigator has the overall responsibility for safeguarding your safety and health as you are a clinical trial participant.
3. But what can I do as a patient?
Be and stay informed. There are numerous possibilities to access information; the internet, social media, and other sources of information. Do not be afraid to use it. When considering participation in a clinical trial, pro-actively search for information on previous clinical trials with the same medication. Information on specific clinical trials is published on various websites, such as Clinical Trials, EU Clinical Trials Register, and the conducting drug company’s website.
Although the information given by these sources does neither include patient participant feedback nor contact information of patient participants, due to data privacy restrictions. There are educational and peer sites which offer the possibility to contact other patients, who might have participated in previous clinical trials with the same medication. Such information can be found on sites, such as Patients Like Me, patient organization sites, and Facebook, to name a few. Here, you have the opportunity to ask your peers for their opinion on the treatment, clinical trial, and positive and negative experiences.
In addition, dive into the specifics of the clinical trial. What is the mechanism of action of the medication? How does it influence the disease? Has it been used for other diseases and what were the results? What are the expected adverse events? Most importantly, create a risk-benefit table for yourself: assess what is important to you and assign a value to it to help you decide upon the overall benefit to you and your health.
4. Can there be too much patient safety?
On the contrary. Patient safety, together with data integrity, builds the foundation of clinical trials, as described in the Guideline for Good Clinical Practice (ICH E6 (R2) – ICH.org). Compliance with the Good Clinical Practice Guideline is mandatory for today’s clinical trials.
5. What is done to ensure quality and compliance in clinical trials?
There are various approaches to assessing and confirming the quality and compliance in clinical trials. One of them is to utilize technological reviews on the large volume of data – big data – collected in the clinical trial by, for example, running safety and data consistency checks every night on the clinical trial data. Intelligent algorithms are used to analyze and evaluate all types of data inconsistencies and potential risks to patients. This can take up to 4 to 5 hours for a phase III clinical trial with about 200 to 400 patients. Following these checks, the data are reviewed and approved as “clean data” when all safety and integrity criteria have been met. This provides the basis for the patient safety and data integrity of the clinical trial but does not replace the patient’s own responsibilities.
6. What would these “patient’s own responsibilities” be? What can I do as a patient to avoid risks in a study?
A patient participating in a clinical trial is the most important part of the clinical trial. Patients have an obligation to receive as much information as they like, respecting data privacy and certain confidential information. An open relationship with your clinical investigator, using social media, websites, and regularly asking for updates on the clinical trial are a good start. Collect and assess facts on the pharmaceutical company manufacturing the medication, on the medication itself, and even on your clinical investigator. After all, it is your decision whether or not to participate in a clinical trial.