Personalized Medicines in Clinical Trial

Author: Diyo Thomas, Statistical Programmer at Genpro Research

Personalized medicine, stratified medicine, precision medicine, and P4 medicine are some terms that we often hear these days. With the advent of these concepts wherein the interventions can now be catered to a person as if one chooses his/her clothes perfect to their size and comfort, novel and transforming approaches are being developed in the healthcare industry. Whether it be preventing a disease, treating for an existing one, or for coping with the aftereffects of a disease, precision medicine comes in as a package for all. Employing predictive tools and designing healthcare plans suitable for the patient personalized medicine help them to effectively diagnose the diseases and treat them in a unique way.

How is it done?


The Human Genome Project and its successful completion have contributed way lots to personalized medicine and the information gathered through it becomes a major source of input. Genomics, which earlier was used only to address rare disorders is now viewed as a go-to-input for knowing more about the cause of any common disease. The knowledge acquired through this project is immeasurable. While genetics plays its role, there are other key factors that contribute to a person’s physical conditions. Thus lifestyle, environment, drug, and behavioral addictions become the key of study when it comes to allocating a treatment regimen to a subject. As of 19th September 2019, there are 108,263 registered clinical trials in the United States alone and 154,378 trials in other countries. After conducting a successful pre-clinical study, clinical studies begin wherein they test the Investigational New Product (INP) on humans. They check for safety and efficacy. Even after having completed all the phases of clinical trials, the INP might not work for most of the people. This is because everyone is unique in their own way and we need to dive deeper into one’s history.

Personalized Medicine and Clinical Trials

Several new approaches for clinical trials have been launched by precision medicine initiatives worldwide. To list a few there are Basket trials, Umbrella trials, N-of-1 study design, the Exceptional Responders Initiative, Adaptive Trials, and longitudinal cohort studies.


Basket trials evaluates the effect of a single drug on a single mutation on different tumor types all at the same time. The advantage of this trial over others is that it can greatly increase the number of subjects eligible to receive some drugs.

Umbrella studies are the ones in which there are different treatment arms. It is based on the type of cancer the subject has and their specific molecular structure that each subject is assigned to a treatment.

An N-of-1 trial is the clinical trial in which only a single patient is being studied in the entire trial. In this type random allocation can be used to decide the order of investigational and control intervention to be given to the patient in a randomised controlled trial. It can also be fixed by the researcher. It is majorly employed outside of oncology studies. Each subject acts as his or her own comparator as the efficacy is measured against different investigational agents that the subject receives.

Exceptional Responders Initiative is launched to understand the molecular substructure of rare responses to treatment. Through this specific case studies becomes possible wherein any dramatic or unusual long-lasting responses are seen in patients which were not seen in similar patients who got the same treatment.

Adaptive trials are more flexible than the conventional fixed design as they make better use of time, money and above all acquired results through the trial. Based on pre-specified rules the trial’s course is modified for best results.

Longitudinal studies use continuous or repeated measures to follow specific individuals over prolonged periods of time, often years or decades. This study type is useful for evaluating the relationship between risk factors, cause of the disease, and the outcomes of treatments over different lengths of time. These are generally observational but can also be experimental.

Cancer Genomics or Oncogenomics is a wide area of application of genomics and personalized medicine to cancer research and treatment. Just like everyone in this world are unique and different people’s cancers are also different. We may not find any two identical. This is because these are not just single disease but contain unique complexities of its own. Treatment strategies are given based on where the cancer developed and how tumours look under the microscope. But usually in cancer trials, patients who have cancer in the same body part are enrolled and are given same treatment. This does not address its uniqueness which is achieved by the above clinical trial designs.


Precision medicine approaches are soon to become part of routine healthcare wherein healthcare providers will need a constant update on molecular genetics and biochemistry. In the upcoming years researchers must have access to lifestyle and health information so that that data can help them design a clinical trial. So the question is how do they get this information. Various projects have been initiated worldwide to achieve this. Human Genome Project and National Institute of Health’s All of Us project are 2 among them. Along with various advantages there are several challenges to personalised medicine as well. These varies from lack of enough data to the feasibility of carrying out it in cancer centres or hospitals. Researchers, healthcare providers and policy-makers should work together to address the major challenges as personalized medicine continues to move forward.


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