[EQ] Policies for Orphan Diseases and Orphan Drugs

Policies for Orphan Diseases and Orphan Drugs

ALAIN DENIS, STEVEN SIMOENS, CHRISTEL FOSTIER, LUT MERGAERT, IRINA CLEEMPUT

Federaal Kenniscentrum voor de Gezondheidszorg Centre fédéral d’expertise des soins de santé

The Belgian Health Care Knowledge Centre – 2009

KCE reports 112C - Health Technology Assessment (HTA)

Available online PDF [132p.] at: http://www.kce.fgov.be/Download.aspx?ID=1664

“….Due to low prevalence, rare diseases have traditionally been neglected by industry and by the scientific, medical and political communities. It is estimated that there are currently between 5000 and 7000 different rare or orphan diseases. Both in the United States (US) and in the European Union (EU), schemes were launched to stimulate the development of ‘orphan drugs’, drugs developed to treat these orphan diseases.

 

The rationale behind these legislations is to compensate industry for the risks and lower potential return on investment as a consequence of the inherently low number of patients. These schemes are considered as a success with an increasing number of Orphan Designation requests filed at the US Food and Drug Administration (FDA) and the European Medicines Agency (EMEA).

 

This study’s objectives are to:

1. provide an overview of the commonly used definitions for ‘orphan diseases’ and ‘orphan drugs’ and describe the particularities of orphan drugs as compared to drugs for regular diseases;

2. describe the regulatory processes for orphan drugs from Orphan Designation to reimbursement;

3. compare the Belgian orphan drug reimbursement policy with other countries;
4. estimate the current budget impact of orphan drugs and forecast the expected future budget impact;

5. formulate recommendations for policy makers concerning orphan drugs….”

 

Table of contents

 

1 Introduction

2 Orphan, Rare and Neglected Diseases and Drugs: definitions and particularities

3 Policy Description

4 International comparison of orphan disease and drug markets in Europe

5 Critical Assessment

5.1 introduction

5.2 methodology

5.2.1 Qualitative overview

5.2.2 In-depth analysis

5.3 qualitative overview of all reimbursement dossiers

5.4 in-depth analysis of 15 selected reimbursement dossiers

5.4.1 Comparison of the evaluations by EMEA

5.4.2 Comparison of the studies mentioned in the NIHDI file, the EMEA file and EPAR

6 Budget impact analysis

7 Discussion and conclusions

7.1 orphan drug designation as a tactical step

7.2 prevalence versus economic motives

7.3 assessing clinical added value

7.4 the need for a right balance between ethical and economic concerns

7.5 pricing

7.6 extension of indications

7.7 growth of the budget impact of orphan drugs

7.8 variations in access and use among member states

7.9 awareness raising

7.10 colleges and control of eligibility

7.11 use of registries

8  Appendices

 

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