In March 2018, PHASTAR attended mSquared - Medrio’s annual user conference in San Francisco. The conference hosted CROs, sponsors and other organisations from around the globe. It was an opportunity to learn first-hand about Medrio’s new products and features and to get a preview of the work Medrio are doing to further accelerate and enhance processes. There was plenty of opportunity to exchange ideas and user hints and tips at workshops and personalised training sessions.
Personalised medicine is a move away from the ‘one size fits all’ approach to the treatment and care of patients, to one which uses new approaches to better manage patients’ health and targets therapies to achieve the best outcomes in the management of a patient’s disease or predisposition to disease. With substantial and increasing medication use globally comes a growing risk of harm associated with such medication. Medication error can be defined as a reduction in the probability of treatment being timely and effective, or an increase in the risk of harm relating to medicines and prescribing compared with generally accepted practice. The Institute for Healthcare Improvement (IHI) has described the ‘five rights’ of medication administration in order to try and reduce medication error. These are; the right patient, the right drug, the right dose, the right route and the right time. Patient centred care, along with this long awaited personalised approach, is now more relevant and necessary than ever as the complexities of the human body are increasingly understood.
One standard request in larger clinical trials is to assess whether the observed treatment effect in the primary endpoint is consistent across subgroups. Analyses on subgroups is a widely discussed topic: searching the internet for “subgroup analysis in clinical trials” gives over 700,000 results. The discussion in the majority of these articles revolves around potential pitfalls of subgroup analysis. The idea of subgroup analyses is to test for homogeneity similarity or heterogeneity across the subgroups.
However, there is another type of “subgroup analysis” - where the interest lies in targeting certain patient populations that would benefit from a particular treatment. This is one goal of precision medicine; to effectively screen patients to ensure they are given the most appropriate treatment, based on genetic data (for example, pre-screening patients to identify those that are “biomarker positive” prior to treatment, or to enrol them onto a clinical trial).
Although the first published results relating genetics to treatment outcomes was in 1902, we are still learning so much about the field of "precision medicine" that there is ongoing debate about its definition and name (e.g. personalised/stratified/P4 - Predictive, Preventive, Personalized and Participatory). The basic premise is that the relationship between how a patient responds to a treatment can be better explained by taking into account data on the patients' molecular profile, environment and lifestyle. Traditional assessment of treatments using randomized clinical trials have generally assumed that there is an average effect across all the patients allocated to that treatment in a trial, a "one size fits all" approach. Of course, we are all unique, and there is no doubt that our health is determined by genetics as well as lifestyle and environmental factors.
PHASTAR will be attending the BioTrinity conference for the first time which is taking place from 23-25 April in London. We are looking forward to meeting with companies that have need for statistical consulting, data analysis and data collection for clinical studies.