New forms of evidence are needed to complement evidence generated from randomised controlled trials (RCTs). Real-World Evidence (RWE) is a potential new form of evidence, but remains undefined. This paper sets to fill that gap by defining RWE as the output from a rigorous research process which: (1) includes a clear a priori statement of a hypothesis to be tested or research question to be answered; (2) defines the data sources that will be used and critically appraises their strengths and weaknesses; and (3) applies appropriate methods, including advanced analytics. These elements should be set out in advance of the study commencing, ideally in a published protocol.The strengths of RWE studies are that they are more inclusive than RCTs and can enable an evidence base to be developed around real-world effectiveness and to start to address the complications of managing other real-world problems such as multimorbidity. Computerised medical record systems and big data provide a rich source of data for RWE studies. However, guidance is needed to help assess the rigour of RWE studies so that the strength of recommendations based on their output can be determined. Additionally, RWE advanced analytics methods need better categorisation and validation.We predict that the core role of RCTs will shift towards assessing safety and achieving regulatory compliance. RWE studies, notwithstanding their limitations, may become established as the best vehicle to assess efficacy.

The Learning Health Community is an emergent global multistakeholder grassroots incipient movement bonded together by a set of consensus Core Values Underlying a National-Scale Person-Centered Continuous Learning Health System developed at the 2012 Learning Health System (LHS) Summit. The Learning Health Community's Second LHS Summit was convened on December 8 to 9, 2016 building upon LHS efforts taking shape in order to achieve consensus on actions that, if taken, will advance LHSs and the LHS vision from what remain appealing concepts to a working reality for improving the health of individuals and populations globally. An iterative half-year collaborative revision process following the Second LHS Summit led to the development of the Learning Health Systems Consensus Action Plan.This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

Patient-centered drug development (PCDD) is a shift in the way that drugs are developed, systematically incorporating patient participation in all stages of medicines development. The more the research sector understands the needs and values of patients, the more effective and efficient it can be in bringing meaningful drugs and evidence to patients and providers. In this paper, we describe PCDD, provide examples of PCDD work across the phases of drug development, and discuss the challenges to making PCDD systematic. We describe how the developing Learning Health System will enable PCCD: we believe that the Learning Health System will address PCDD barriers by connecting stakeholders, enabling the more efficient flow of data, information, and evidence in the health ecosystem, and by providing governance for the connected ecosystem. is also evolving. The LHS is defined as a system "in which progress in science, informatics, and care culture align to generate new knowledge as an ongoing, natural by-product of the care experience, and seamlessly refine and deliver best practices for continuous improvement in health and health care." 6 The LHS will enable PCDD by providing the technical architecture for more efficient flow of data, information, and evidence in the health ecosystem and defining policy and governance rules of the road for the health ecosystem. This paper will describe PCDD and provide examples of Lilly progress to date on PCDD; we will describe the challenges to PCDD and elucidate how the developing LHS will facilitate PCDD. | ABOUT PCDDThe traditional drug development model is intended to discover, develop, and market medications to satisfy regulatory requirements.Patient-centered drug development shifts this focus by putting the patient at the center of drug development phases, actively involving patients in all phases of development. The PCDD approach arose from two key FDA initiatives: FDA Safety and Innovation Act and Prescription Drug User Fee Act reauthorization. 7 FDA described patientfocused drug development as intending more systematic and expansive approach to obtaining the patient perspective on disease severity or the unmet medical need in a therapeutic area to benefit the drugThis is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.