High-quality and compliant Phase I to Phase III clinical trials are critical for obtaining market approval of a drug or biologic. After the product is approved for market, post-marketing surveillance and post-marketing studies are performed in order to gain a deeper understanding of its safety and efficacy profile.
Phase I clinical trials
Are you ready to test your research idea in a proof-of-concept (POC) Phase I clinical trial? Is it time for a first-in-human trial to investigate the pharmacokinetics (PK) of your IMP in healthy, paid volunteers? Phase I clinical trials typically consist of a small number of subjects to test safety, pharmacokinetics, and possibly multiple doses. In oncology studies, a small number of patients are treated and the study may be a combined Phase I/Phase II clinical trial to investigate both efficacy and safety.
Phase II clinical trials
Phase II clinical trials investigate the efficacy and safety of the investigational drug or biologic, typically in up to a few hundred patients. Phase II clinical trials may be randomized, and the choice of whether to use a comparator or placebo for the control group is critical and can make or break the study. Indeed, the choices made regarding study design, blinding, control group, sample size and endpoints are all crucial and need to be based on previous experience, published data, and regulatory recommendations.
Phase III clinical trials
Phase III clinical trials usually include hundreds or even a few thousand patients in a randomized trial. The Phase III clinical trial is referred to as a pivotal study if the trial intends to provide unambiguous, evidence-based data confirming efficacy and safety that will be used to submit an application for market approval to the regulatory authorities. Because Phase III clinical trials are of pivotal importance for gaining market approval, it is crucial to have a clear clinical development strategy based on the market and regulatory landscape in order to get it right from the start.
Phase IV clinical trials
Phase IV clinical trials, or post-marketing studies, are performed after the product has been approved for market and can identify serious and/or unexpected signals of risk that may have been previously undetectable during clinical development due to a relatively small number of treated patients. Post-marketing studies may even be recommended by regulatory authorities to study a specific subpopulation of patients or to further assess product quality. Ultimately, the knowledge gained from post-marketing studies is used to increase patient safety and achieve a better understanding of the product’s benefit-risk profile.
Observational trial or non-interventional trial (NIS): no new intervention (e.g., treatment, procedure, medicine, etc.) is introduced into the subject’s care. During this type of study, the subject’s routine care is observed and the real-world data is collected and analyzed.
Randomized clinical trial: participants are randomized into different treatment groups, such as a control group and a test group. The control group may receive placebo or a comparator (i.e., standard treatment). There may be several test groups, for example to test different doses of the investigational product.
Registry-based randomized clinical trial (R-RCT): Sweden is a pioneer in this area, which combines the power of national clinical registries with the rigor of randomized clinical trials.
Types of investigational products
Pharmaceutical drug: a substance intended to diagnose, prevent, or treat a disease, or modify physiological functions, through pharmacological action.
Medical device: a device or reagent intended for diagnosis, monitoring, treatment or prevention of a disease or condition. Medical devices can also support, modify or replace parts of the anatomy or physiological processes. In Europe, there are four classes of medical devices.
In vitro diagnostic (IVD) medical device: a medical device (i.e., device or reagent) that is intended to diagnose, monitor, prevent or treat a disease or condition by in vitro testing of human specimens (e.g., urine, blood, tissue, etc.). In Europe, there are four classes of IVDs.