Do innovative medicines against cancer always have a real added value? : Synthesis

45 p.ill.,FOREWORD 1 -- KEY MESSAGES 2 -- SUMMARY 4 -- 1. INTRODUCTION 6 -- 1.1. CANCER MEDICINE IS BECOMING MORE AND MORE EXPENSIVE 6 -- 1.2. ARE THE BENEFITS FOR THE PATIENT SUFFICIENTLY PROVEN? 6 -- 1.3. THE CONFIDENTIALITY OBLIGATION MAKES THE SYSTEM EVEN LESS TRANSPARENT 7 -- 2. RESEARCH QUESTIONS AND METHODS 8 -- 2.1. RESEARCH QUESTIONS 8 -- 2.2. HOW DID WE PROCEED? 9 -- 2.2.1. Observational data 9 -- 2.2.2. Literature study 10 -- 3. EVOLUTION OF THE OVERALL SURVIVAL AND BUDGETARY IMPACT OF THE INNOVATIVE -- MEDICINES 11 -- 3.1. PRELIMINARY REMARKS 11 -- 3.2. SUMMARY OF RESULTS 12 -- 4. RESTRICTIONS OF OBSERVATIONAL DATA 19 -- 5. COST EFFECTIVENESS 20 -- 6. WAYS TO BETTER ASSESS THE ADDED VALUE OF INNOVATIVE MEDICINES IN THE FUTURE 21 -- 6.1. UNCERTAINTIES IN CLINICAL STUDIES 22 -- 6.1.1. Randomised controlled studies remain the ‘golden standard’ 22 -- 6.1.2. Survival is not always the primary outcome measure 22 -- 6.1.3. New medicines are not always compared with the right treatment 23 -- 6.1.4. The outcome is measured by surrogate endpoints 24 -- 6.1.5. Quality of life is one thing that is often overlooked in clinical studies. 25 -- 6.2. UNCERTAINTIES WITH REGARD TO MEAS 28 -- 6.2.1. Refund mechanisms of the MEAs and taxes paid by the pharmaceutical industry 29 -- 6.2.2. The industry is not encouraged to provide the missing evidence 30 -- 6.2.3. Confidential pricing creates a lack of transparency with negative consequences 32 -- 7. ADDITIONAL STATEMENTS THAT DESERVE THE NECESSARY NUANCE 34 -- 8. CONCLUSION 37 -- RECOMMENDATIONS 38 -- REFERENCES 4