Abstract
“Evidence based medicine is the conscientious, explicit, and judicious use of current best evidence in making decisions about the care of individual patients.”
If data are the new oil, then the precision medicine engine isn't going to seize up any time soon. We are at a point in time where the use of real-world data and the evidence generated from those data is gaining momentum in the healthcare industry, but it is critical that we build the tools to extrapolate accurate meaning while identifying any potential for bias. Importantly, this includes continuously interrogating the provenance and relevance of the data and how they relate to specific diseases or individual patients. Real-world data are generated from many sources, including observational studies, patient-generated health information, insurance claims, and electronic health records, and oversight of privacy and compliance issues will be of critical importance when these different databases are linked together. Investing resources to ensure the data are trustworthy and reliable before we put the algorithms to work cannot be overstated—we need to fill in the gaps before harmonizing the data and deriving meaningful interpretation.
In addition to improving healthcare for individuals, real-world evidence (RWE) is being used to improve how scientists run clinical trials, by selecting the most appropriate participants and trial sites. For some cancers where there is no approved treatment and rare diseases, RWE can offer a better grasp of the natural history of the disease and more effectively measure drug efficacy. A large randomized, controlled approach may not be viable either because of a limited population or ethical concerns surrounding the denial of an effective therapy for the subjects in the placebo group. Moreover, in rare conditions such as Duchenne muscular dystrophy, where there is a great deal of variability in the progression of the disease and where trials have historically been set up based on stratifying patients solely by age, smarter and smaller trial designs should be the focus moving forward.
Another area that RWE is being used is in health equity. What matters here are the nonclinical parameters that affect equitable access to healthcare—what is more commonly termed social determinants of health. We need to work harder to help disadvantaged groups. The pandemic has brought this into even sharper focus as underrepresented groups have been further marginalized and their access to healthcare severely hindered. We have to do a better job of understanding who our patients are. Digital technologies can play a part in helping health systems build a better picture of the socioeconomic and clinical profiles of all patients in the community, so that ultimately we can close the health equity gap. Let's make this our new reality in our pursuit to democratize precision medicine for all.
Damian Doherty, Editor in Chief
