Orphan Drugs

CONTENTS

Abstract

Executive summary

Annex: bibliography

Section 1. Introduction

Section 2. Patients’needs/epidemiology

2.1 Epidemiological profile of selected countries

2.2 Rare disease definitions

2.3 Patients’needs/health-care delivery

Section 3. Stakeholders’strategies

3.1 Pharmaceutical sector

3.2 Biotechnology sector

3.3 Patient associations/Consumer associations

Section 4. Legal framework

4.1 Public policies on orphan drugs (definitions, results)

4.2 Orphan drug status initiatives in the different countries

4.3 Comparative results of the different policies and lessons for Europe

Section 5. Policy options

5.1 Current European Policy

5.2 R&D policy

5.3 Industrial and other market regulation policies

5.4 Development and cooperation policies

Section 6 – Recommendations

6.1 Orphan drugs definition

6.2 Orphan drugs legal status

6.3 Incentives to encourage efficient orphan drugs supply

6.4 Industrial policies

6.5 R&D policies

6.6 Development and cooperation policies