Millions of people around the world take medications that are ineffective and result only in unwanted side effects. Researchers are now turning to the model of Precision Medicine, where physicians and researchers take into account a person’s genes, lifestyle, and environment to develop a treatment plan and prescribe the right medication. While there have been many advances in the technology and theory, precision medicine is not currently in use for the majority of diseases. Presently, the method for prescription is essentially “one treatment fits all.” In 2015, President Obama announced that $215 million would be dedicated to the Precision Medicine Initiative, which has sparked a myriad of questions, most important of which is: How is this affecting clinical trials?1
What is Precision Medicine?
Precision medicine is the personalization of healthcare, and applies to medical devices, medical decisions, and medical treatments. It has been found that while some groups with similar genetic makeup respond well to a drug, other groups may not receive any positive benefits. In fact, the efficacy rate of the ten highest grossing drugs in the United States ranges from a high of about 25% to a low of about 3%.2 If so few patients are receiving benefits from the drugs that they are being prescribed, something must be done to increase the odds of success. Using pharmacogenomics, the study of how genes affect a person’s response to drugs, and big data, physicians and researchers are on a mission to compile a database of patient health information so that they can analyze genetic makeup, environmental elements, and lifestyle to prescribe medication and health treatment plans based on these combined factors.
What Does This Mean for Clinical Trials?
While there is still a long way to go, we have already seen changes happening to accommodate precision medicine. In July 2015, multiple oncology trials were launched that chose patients with advanced malignancies and tailored their treatments based on genomic sequencing of the patient and their malignancy.3 New methodologies, such as adaptive study design and biomarker-guided design, have developed to advance precision medicine, as well as reduce trial cost and shorten time to drug approval With an adaptive study design, researchers may adapt the parameters of a study according to what they observe; they may adjust dosing, sample size, schedule, and drug mixture, among other things. Studies have also started adopting a biomarker-guided design, recruiting patients with a particular biomarker status as opposed to all patients with a particular cancer type, in order to determine which patients, with which biomarkers, are more likely to benefit from which drugs.4
As precision medicine continues to move forward, clinical trial design will begin to change more dramatically in order to focus on finding a treatment option that works for an individual. With the current trial model, measurements are taken from thousands of people; with precision medicine, the goal will be to advance N-of-1 trials, in which one person makes up the entire trial. With individual trials, it is critical that enough data be collected over an extended period of time to positively identify a patient’s particular makeup as a responder to a particular drug.5 As researchers look ahead, they realize that understanding the unique characteristics of a person’s genetic makeup will help to advance medical research and treatments.
1 Levin Carter, MD., Jennifer. “New Clinical Trials Designed to Better Leverage Precision Medicine.” New Clinical Trials Designed to Better Leverage Precision Medicine. OBR Green, Mar. 2014. Web. 05 Jan. 2016.
2 Schork, Nicholas J. “Personalized Medicine: Time for One-Person Trials.” Nature.com. Nature Publishing Group, 29 Apr. 2015.
3 Healy, Melissa. “Cancer Trials Aim to Shore up ‘precision Medicine’s’ Base of Evidence.” Los Angeles Times. Los Angeles Times, 01 June 2015. Web.
4 Levin Carter, MD., Jennifer. “New Clinical Trials Designed to Better Leverage Precision Medicine .” New Clinical Trials Designed to Better Leverage Precision Medicine. OBR Green, Mar. 2014. Web. 05 Jan. 2016.
5 Schork, Nicholas J. “Personalized Medicine: Time for One-Person Trials.” Nature.com. Nature Publishing Group, 29 Apr. 2015.