Critical assessment of belgian reimbursement dossiers of orphan drugs.

Background: Orphan medicinal products are designed to diagnose or treat rare diseases that are serious, life threatening or chronically debilitating and that affect 50 or fewer people in every 100 000 in the EU. In Belgium, the Drug Reimbursement Committee (DRC) evaluates reimbursement requests for orphan drugs based on multiple criteria: the therapeutic value, price and proposed reimbursement tariff; the importance of the drug in clinical practice; and the budget impact of the drug.

Objectives: This study aimed to assess reimbursement dossiers of orphan drugs in Belgium and to compare them with the clinical evidence submitted to the European Medicines Agency (EMA).

Issues surrounding orphan disease and orphan drug policies in Europe.

An orphan disease is a disease with a very low prevalence. Although there are 5000-7000 orphan diseases, only 50 orphan drugs (i.e.

A comparative study of European rare disease and orphan drug markets.

Objectives: This article aims to compare regulatory aspects of rare disease and orphan drug markets in Belgium, France, Italy, the Netherlands, Sweden and the United Kingdom.

Methods: Information was derived from the international literature, analysis of legal texts, and a survey completed by national experts.

Results: These countries adopted varying approaches towards regulating rare disease and orphan drug markets and, hence, the availability, pricing and reimbursement of orphan drugs vary between countries.

Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013.

Objective: This article aims to calculate the impact of orphan drugs on the Belgian drug budget in 2008 and to forecast its impact over the following 5 years.

Method: The 2008 budget impact was calculated by triangulating information derived from multiple Belgian data sources. The 2008-2013 budget impact analysis was based on three scenarios reflecting different levels of growth in the number of registered orphan drugs in the European Union, the number of drugs reimbursed in Belgium, and the average annual cost per patient per drug in Belgium.

[Orphan diseases and orphan medicines: a Belgian and European study].

The objective of this study is to analyze policies concerning orphan medicines, used to treat patients suffering from a rare disease. The decisions about orphan designation and marketing authorization of orphan medicines are taken at European level, but each Member State is responsible for decisions regarding reimbursement. The European measures to encourage the development of orphan medicines, such as market exclusivity for a period of ten years, seem to be successful.