OBJECTIVES : To identify, describe and classify MEAs for orphan drugs in selected European countries. The objective of this study is to find the different types of MEAs used for rare diseases and analyze their application.

METHODS For Identification, description, and comparison of MEAs, the regulatory requirements by HTA bodies in the following countries were reviewed and their main characteristics extracted: Bulgaria, France, Germany, Hungary, Romania, Slovakia, Sweden, UK. Identified MEAs were analyzed and compared by focusing on four key domains: special process/application of MEAs for rare diseases, type of MEA applied, inclusion of MEAs in the HTA process, confidential list of MEAs published.

RESULTS : There is a special application of MEAs for ultra-rare diseases & orphan drugs in only two of the countries – Romania, and Sweden. Only the UK have a highly specialized technology program for orphan drugs & rare diseases. The other countries do not have a separated, special process for orphan drugs, but the MEAs for both non-orphan & orphan drugs are the same. The most common type of MEA applications are financial based. No MEAs are identified for France, and Germany, due to data unavailability. The only country identified publishing confidential list of MEAs is Hungary.

CONCLUSIONS : This study confirmed that in the selected countries there is an increasing application of MEAs for rare diseases. European payers use MEAs to manage aspects of uncertainty associated with the introduction of orphan drugs in the health care system. The most common type of MEA application is still financially oriented. However, most of the countries do not have special HTA process for rare diseases, including application of MEAs for orphan drugs especially. Referring of the HTA decisions among countries is not always suitable due to the different types of used MEAs.