Improving value of clinical research - an evidence-based approach

Evidence suggests that 85% of biomedical research spending, i.e. 200 billion US dollars every year, goes to waste. In 2014, The Lancet published a series of five reviews showing how dividends from the investment in research might be increased at five stages - from the relevance and priorities of the questions being asked, to how the research is designed, conducted, and reported. Value and waste have since then become buzzwords in the academic as well as public debate surrounding health research. Although academic institutions are the major driving force of patient-oriented clinical research receiving large proportions of public funding they have been slow responders to the Series’ recommendations. Some of the identified underlying reasons include a lack of a common understanding of “value” as a concept and sparse practical guidance for academia on how to improve it. This work represents the first effort to formulate an academic response to The Lancet series on increasing value in clinical research by investigating the two distinct concepts in the equation: “Quality” and “cost”.
In a first step, we systematically reviewed existing quality concepts, both in the medical literature and across international clinical research stakeholder groups. Precise definitions of quality were sparse, and stakeholder perspectives of crucial components of quality varied. Based on these findings, we then engaged international stakeholder representatives in the creation of a comprehensive, consensus-based framework for the quality of clinical research that is applicable to all study types and spans the entire lifecycle of a clinical study, i.e. from conceptualization of the research question to dissemination of study results. Primarily, it is designed to be operationalized in the academic setting and fully supports the REWARD Statement. This framework builds the foundation for a common understanding of the concept of “quality” and its practical assessment. At Swiss national level, the framework has triggered all stakeholders to convene in a first symposium on how to increase value of academic clinical research and serves as an agenda for future research on research.
In a second step, we systematically reviewed the current evidence on the costs and associated resource use of Randomized Controlled Trials (RCTs), which we found to be sparse. Based on this, we laid the foundation for future study cost assessments in academia by (i) developing a comprehensive list of items for the retrospective and prospective assessment of costs, and (ii) generating first empirical evidence on main cost drivers in a case report on two academic RCTs. Although these two RCTs were conducted in very different settings and resulted in vastly different costs, the main drivers, i.e. personnel costs during conduct phase, were the same. In addition, we investigated the added value of two innovative aspects that affect both study quality and cost, i.e. risk-based trial monitoring and remote data collection. We show that both concepts may increase the cost-effectiveness of trial conduct and thereby increase value, but only if the methodology is further investigated and then, rigorously implemented.
Although we did not take the initially envisioned cost-consequence approach, we have certainly created awareness on value and waste in the academic context and engaged the major stakeholders in fundamental discussions on how to improve the current situation. In the future, the costs occurred need to inform quality assessments of clinical studies in order to create a tool that creates “value”, rather than sole quality conformity. Furthermore, the willingness-to-pay of academic decision makers in resource-constrained settings will weigh into the value equation and needs further investigation in the future. The impact of this work - and whether it eventually increases value in the system - now critically depends on its rigorous implementation, evaluation, and refinement.