Reflections on Dr. Hans-Georg Eichler's Closing Keynote
Dr. Hans-Georg Eichler, Senior Medical Officer, EMA, provided the closing plenary keynote speech entitled “Data standardization: (how) important for tomorrow's knowledge generation?” The presentation discussed the efficiency of knowledge generation in medical research and the need for augmenting knowledge generated by randomized clinical trials (RCTs) with other sources of knowledge generation. RCTs are geared to provide safety and efficacy assessments, but must be augmented with data on effectiveness of medical products. Data on effectiveness can be harvested from an increasing number of non-traditional sources, such as patient-level data sharing, sharing of comparator data obtained from RCTs, observational data, mixed treatment comparisons.
Non-conventional meta-analysis can generate interesting findings via, for example, combining data from differently designed studies and findings may lead to more focused and efficient RCTs. This kind of work is not possible without data standardization. Dr. Eichler discussed the continuum of knowledge generation and how research design might utilize real-word data (RWD) to support RCTs, for example, augmenting the control arm of an RCT with RWD, resulting in fewer patients enrolled in the controlled arm. Approaches like this may gain more traction as data from past clinical trials are shared more widely, ideally in a standardized format.
There are many exciting opportunities ahead, as well as challenges. Fundamentally, the efficiency of knowledge generation will increase as RCT and RWD are shared. That knowledge will be generated from a diverse family of data sources and methodologies that complement RCTs, increasing the efficiency of RCTs. Data standardization is a necessary enabler to take advantage of these opportunities. Though standards alone aren’t sufficient, other initiatives must provide common data models for RWD, common outcome measures, accessibility, data protection processes, data governance.