Reimbursement of orphan drugs in Belgium: what (else) matters?

Background: Most orphan drugs do not meet traditional standards of cost-effectiveness. Yet, most orphan drugs are reimbursed, which implies that other factors are taken into account at the time of reimbursement. To increase accountability of decision-makers, there is a need for more transparency in the factors that play a role in reimbursement decisions of orphan drugs.

Shining a light in the black box of orphan drug pricing.

Background: The pricing mechanism of orphan drugs appears arbitrary and has been referred to as a "black box". Therefore, the aim of this study is to investigate how drug- and disease-specific variables relate to orphan drug prices. Additionally, we aim to explore if certain country-specific pricing and reimbursement policies affect the price level of orphan drugs.

Ethical, legal and social implications of rare diseases and orphan drugs in Europe: meeting report of a Brocher symposium.

Ethical, legal and social implications of rare diseases and orphan drugs in Europe (Brocher symposium) Geneva, Switzerland 18-19 April 2013 As part of the Scientific Program of the Fondation Brocher, a two-day symposium on orphan drugs and rare diseases was held on the shores of Lac Léman in Geneva. Specific focus was on the ethical, legal and social implications of rare diseases and orphan drugs in Europe. The symposium gathered about 30 international stakeholders and experts, representing different scientific disciplines, the pharmaceutical industry and patient representatives.

Clinical evidence for orphan medicinal products-a cause for concern?

Background: The difficulties associated with organising clinical studies for orphan medicinal products (OMPs) are plentiful. Recent debate on the long-term effectiveness of some OMPs, led us to question whether the initial standards for clinical evidence for OMPs, set by the European Medicines Agency (EMA) at the time of marketing authorization, are too low. Therefore, the aim of this study was to quantitatively evaluate the characteristics and quality of clinical evidence that is presented for OMPs to obtain marketing authorization in Europe, using the new and validated COMPASS tool.

Development and validation of COMPASS: clinical evidence of orphan medicinal products - an assessment tool.

Background: Rare diseases are defined as life-threatening or chronically debilitating diseases with a prevalence of 50 out of 100,000 individuals or less. Orphan medicinal products (OMPs) are intended for the treatment of rare diseases. The assessment of quality of evidence in small populations is often complex.

Evaluating and improving orphan drug regulations in Europe: a Delphi policy study.

To encourage the development of orphan drugs, the European Union has implemented specific policies in 2000. However, the political, social, scientific and economic context has changed since the implementation of these policies. For that reason, the aim of this article is to evaluate orphan drug policies in Europe.

Orphan drugs for rare diseases: is it time to revisit their special market access status?

Orphan drugs are intended for diseases with a very low prevalence, and many countries have implemented legislation to support market access of orphan drugs. We argue that it is time to revisit the special market access status of orphan drugs. Indeed, evidence suggests that there is no societal preference for treating rare diseases.

Drugs for rare diseases: influence of orphan designation status on price.

The literature indicates that the expenditure on orphan drugs will be increasing over the coming years. The market for orphan drugs has inherent market characteristics that sometimes result in high prices. The aim of this study was to analyse whether awarding orphan designation status has an influence on the price setting of drugs for rare disease indications.