Allocation of R&D resources for Rare Diseases

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Table of contents

I THE DIAGNOSTIC QUEST AND ITS PREJUDICE TO PATIENTS AND CAREGIVERS 
1 Social Determinants of Time to Diagnosis
1 Introduction
2 Literature Review
3 Data and Methods
4 Results
5 Discussion and Conclusion
6 Appendix
2 Children’s health shock externalities on mothers’ health
1 Introduction
2 Background Literature
3 Data and Descriptive Statistics
4 Empirical Strategy
5 Results
6 Discussion and Conclusion
7 Appendix
II FIRMS’ DECISIONS TO ALLOCATE R&D TO RARE DISEASES
Introductory Section
3 European Initiatives to Foster R&D on Rare Diseases
1 Introduction
2 Context
3 Data and Empirical Strategy
4 Results
5 Discussion
6 Appendix
4 Allocation of R&D resources for Rare Diseases
1 Introduction
2 A Conceptual Framework
3 Data
4 Methods
5 Results
6 Discussion and Conclusion
7 Appendix
III REGULATORS’ CHALLENGES IN DEFINING THE CONDITIONS OF ACCESS TO INNOVATIVE DRUGS 
5 Orphan Drugs and Longevity in the US, Revisited (1999-2015) 201
1 Introduction
2 Data and Empirical Strategy
3 Results
4 Discussion and Conclusion
5 Appendix
6 Cost-eectiveness threshold for health care technologies
1 Introduction
2 L’utilisation du seuil d’acceptabilite des technologies de sante
3 Le paradigme ecience-equite
4 Conclusion