Publicly funded Practice-oriented Clinical Trials

98 p.ill.,1 INTRODUCTION 9 -- 1.1 BACKGROUND 9 -- 1.1.1 The importance of RCTs for evidence-based medicine and health technology assessment.9 -- 1.1.2 Publicly funded clinical trials.13 -- 1.2 SCOPE 16 -- 1.3 RESEARCH QUESTIONS.16 -- 1.4 METHODOLOGY 17 -- 2 EXAMPLES OF RESEARCH IMPACT 18 -- 2.1 INDIVIDUAL CASES 20 -- 2.2 RESEARCH PROGRAMS.22 -- 2.3 SOME REMARKS.24 -- 2.3.1 Methodological issues 24 -- 2.3.2 Bias.24 -- 2.3.3 Real-world impact versus projections 24 -- 2.3.4 Problem of attribution and spillover effect 25 -- 2.3.5 Conclusion on return on investment 25 -- 3 WHY DO WE NEED NON-COMMERCIAL CLINICAL TRIALS .26 -- 3.1 COMPARATIVE EFFECTIVENESS TRIALS WITH MEDICINAL PRODUCTS 26 -- 3.2 TRIALS WITH MEDICINAL PRODUCTS IN CHILDREN AND IN RARE DISEASES 28 -- 3.3 NON-COMMERCIAL TRIALS TO COUNTERBALANCE POSSIBLE PUBLICATION BIAS 29 -- 3.4 TRIALS WITH MEDICAL DEVICES 29 -- 3.5 TRIALS ON DIAGNOSTICS AND SCREENING 30 -- 3.6 TRIALS IN MEDICAL AREAS NOT OWNED BY PRIVATE COMPANIES.30 -- 4 HURDLES TO PERFORM PUBLIC FUNDED RCTS 32 -- 4.1 ADVANTAGES AND DISADVANTAGES OF TRIAL PARTICIPATION 32 -- 4.2 COMPETITION BETWEEN TRIALS FOR INCLUSION OF PATIENTS.33 -- 4.3 LACK OF RESEARCH INFRASTRUCTURE 33 -- 4.4 FREE-RIDER BEHAVIOUR OR INTERNATIONAL COLLABORATION 33 -- 4.5 THE DESIGN, INITIATION AND CONDUCT OF TRIALS TAKES TIME, REALISTIC PLANNING NEEDED ..34 -- 4.6 ACCESS TO AND PRICE OF THE COMPARATOR 35 -- 4.6.1 Placebo .35 -- 4.6.2 Off-label drugs 36 -- 4.6.3 Expensive drugs 36 -- 5 THE FRAMEWORK OF CLINICAL TRIALS 37 -- 5.1 QUALITY ASSURANCE.37 -- 5.1.1 GCP guidelines and SOPs.37 -- 5.1.2 Quality of non-commercial trials 38 -- 5.2 REGULATORY REQUIREMENTS 38 -- 5.3 IMPACT OF THE REGULATORY REQUIREMENTS 39 -- 6 NON-COMMERCIAL CLINICAL TRIALS IN EUROPE.42 -- 6.1 EXISTING REPORTS 42 -- 6.2 INTERNATIONAL CLINICAL RESEARCH ORGANIZATIONS 43 -- 6.2.1 European Clinical Research Infrastructures Network (ECRIN) 43 -- 6.2.2 European Organisation for Research and Treatment of Cancer (EORTC) 46 -- 6.2.3 Trials funded by the EU .46 -- 6.3 NATIONAL ORGANIZATIONS .46 -- 6.3.1 Belgium .46 -- 6.3.2 The Netherlands 51 -- 6.3.3 France 51 -- 6.3.4 Germany52 -- 6.3.5 UK 54 -- 6.3.6 The Nordic Countries .58 -- 6.3.7 Italy.58 -- 6.3.8 Spain.59 -- 7 DISCUSSION 61 -- 7.1 IDENTIFICATION AND SELECTION OF PUBLICLY FUNDED TRIALS.61 -- 7.1.1 NIHR 61 -- 7.1.2 Other reflections 63 -- 7.1.3 When can we start with public funding of trials 64 -- 7.2 EVALUATION OF PUBLICLY FUNDED RCTS.64 -- .2.1 Government-funded trials and the Declaration of Helsinki: a prime example 64 -- 7.2.2 A good investment? 65 -- 7.2.3 Research on research: improving processes.65 -- 7.3 FINANCING OF TRIALS WITH PUBLIC MONEY.67 -- 7.3.1 Public money used to fund public-private partnerships 67 -- 7.3.2 The start of a self-sustaining system?.67 -- 7.3.3 The necessary budget and expertise to set up a good trial 68 -- 7.4 EFFICIENCY IN CONDUCTING CLINICAL TRIALS AND COST CONTAINMENT 68 -- 7.4.1 Risk proportionality 69 -- 7.4.2 Research infrastructure and national collaboration 72 -- 7.4.3 International collaboration 73 -- 7.5 SPEED OF THE TOTAL PROCESS UP TO THE IMPLEMENTATION OF RESEARCH FINDINGS 7