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Improving Value of Clinical Research – IMPROVING VALUE OF CLINICAL RESEARCH – AN EVIDENCE-BASED APPROACH Inaugural dissertation to be awarded the degree of Dr. sc. med. presented at the Faculty of Medicine of the University of Basel by Belinda von Niederhäusern from Grenchen, Solothurn Basel, 2017 Approved by the Faculty of Medicine On application of Prof. Dr. med. Christiane Pauli-Magnus (first supervisor) PD Dr. med. Matthias Briel (first supervisor) Prof. Matthias Schwenkglenks (second supervisor) Prof. Dr. med. Mirjam Christ-Crain (second supervisor) Prof. Christian Burri (external expert) Basel, September 18, 2017 Prof. Dr. Thomas C. Gasser, Dean TABLE OF CONTENTS ACKNOWLEDGMENTS ....................................................................................................... 1 PLAIN SUMMARY ............................................................................................................... 3 1 INTRODUCTION .............................................................................................................. 5 1.1 The need for improving value and reducing waste in clinical research .................... 5 1.2 The need for a common definition of the quality of research .................................... 7 1.3 The need for evidence on the cost of research ........................................................ 8 1.4 The need for cost-effective solutions ....................................................................... 8 1.4.1 Approach 1: Trial monitoring ................................................................ 8 1.4.2 Approach 2: High quality data .............................................................. 8 1.5 Main Objectives of this PhD .................................................................................. 10 1.6 Contributions by the PhD student .......................................................................... 10 2 FIRST AUTHOR PUBLICATIONS ................................................................................... 16 2.1 Manuscript I: Towards the development of a comprehensive framework: Qualitative systematic survey of definitions of clinical research quality ............ 16 2.2 Manuscript II: Towards increasing value and reducing waste in academic clinical research: Consensus on a comprehensive framework of clinical research quality ............................................................................................... 49 2.3 Manuscript III: Systematic review on costs and resource use of randomised clinical trials shows a lack of empirical data ..................................................... 92 2.4 Manuscript IV: Cost and resource use evaluation of randomised clinical trials: a case study exemplifying standardised assessment using a comprehensive cost item list .......................................................................... 116 2.5 Manuscript V: Generating evidence on a risk-based monitoring approach in the academic setting - Lessons learned ......................................................... 149 2.6 Manuscript VI: Validity of mobile electronic data capture in clinical studies: A pilot study in a pediatric population ................................................................ 179 3 FURTHER PUBLICATIONS .......................................................................................... 204 3.1 Published reviews (first author) ........................................................................... 204 3.2 Published original articles (co-author).................................................................. 205 4 DISCUSSION ................................................................................................................ 208 4.1 Improving value, reducing waste: Who’s listened? .............................................. 208 4.2 The academic response ...................................................................................... 209 4.3 Look into the future: The value equation .............................................................. 211 4.4 Future directions ................................................................................................. 212 4.5 Closing remarks .................................................................................................. 214 CURRICULUM VITAE ...................................................................................................... 216 ACKNOWLEDGMENTS Specifically, I would like to take the opportunity to thank Prof. Christiane Pauli-Magnus and PD Dr. Matthias Briel for their tremendous support at all stages of this thesis. It was a great pleasure to be supported by visionaries who are passionate and convinced in what they do. Both were highly supportive and allowed me to investigate, develop, and grow, both scientifically and personally, in an environment that endorsed trial, creativity, and error. At the same time, they shed light on paths that I would have never imagined to walk on during my PhD. I will forever be grateful for this opportunity and mostly, their trust in me as their first PhD student at the Clinical Trial Unit. Prof. Pauli-Magnus and PD. Dr. Briel have not only been supervisors, but became role models, and will be mentors for the future. I would also like to thank Prof. Matthias Schwenkglenks for his great support with this thesis, specifically all aspects related to costs. I very much enjoyed working for and with him due to his generous advice, tremendous knowledge, and his pleasant personality. I also thank Prof. Mirjam Christ-Crain who has always supported me in all aspects related to this PhD. Further, I would like to thank Prof. Christian Burri for accepting to be the external expert of this thesis. Whenever we met in person, he provoked new thoughts, was positive and supportive of the work I do, and finally, made me laugh. I want to thank all of you who devoted time, passion, and effort to any of the projects mentioned in here, including all co-authors. In particular, Prof. Gordon Guyatt for his stimulating advice, Dr. Benjamin Speich, Dr. Nadine Schur, and Dr. Priya Satalkar for fruitful discussions, fantastic team work, and their friendship. The colleagues at the Clinical Trial Unit deserve a special mention for their endless collaborative support, notably the statistics, data management, monitoring, and application development groups, but also all study nurses for a very cheerful working environment. A special gratitude goes out to the 52 experts who devoted their hours and thoughts to the development of our quality work until consensus was reached. Without them, our framework would have been a meaningless theory. Further, I would like to thank the Swiss Clinical Trial Organization for their support with everything related to quality and patient engagement, and the Swiss Office of Public Health that funded some of my research and was supportive in all aspects. 1 To my friends outside of University and those who crossed my path during the PhD, I am forever grateful for your friendship and support through good and rough times. Finally, I specifically thank my parents, Elisabeth and Hans-Rudolf von Niederhäusern, and my partner, Florian Stroh, who have always believed in my endeavors and never stopped supporting my life choices. You all provided me with the backbone I needed to be successful, celebrated each little step with joy and laughter, always stood by my side when I was in doubt, and made me the person I am today. 2 PLAIN SUMMARY 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
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