Centralised Procedure for the Scientific Assessment of the Therapeutic Added Value of Orphan Drugs

EURORDIS underlines the fundamental dichotomy between, on the one hand, the scientific and economic evaluation of orphan drugs (designation and authorisation) carried out at a European level and, on the other hand, the evaluation of the Therapeutic Added Value (TAV) and other pharmaco-economic aspects used to determine the pricing and reimbursement of orphan drugs, carried out at a national level.

 

Eurordis observes that patients do not have real and equitable access to the orphan drugs they need. Access is not in line with the legal timeframe, it differs according to the country in which the patient lives and is unrelated to domestic GDP. This is a major issue for rare disease patients and their families and requires coordinated action.

 

A specific EU approach for orphan drugs is feasible. EURORDIS proposes that scientific assessment of the TAV of Orphan Drugs is performed through an EU centralised procedure. A COMP Working Party could deliver an expert opinion on the scientific assessment of the TAV, given that data and expertise is gathered within EMEA rather than at Member State level. National pricing and reimbursement decisions would thus be facilitated and accelerated, leading to improved access for patients.

 

This Contribution is one of four specific topics addressed in Eurordis’ response to the European Commission’s Public Consultation for the first Commission Communication on Rare Diseases.

 
 
La voce dei malati rari in EuropaEURORDIS Rare Diseases International, la voce internazionale dei malati rariRare Disease International RareConnect è un'iniziativa di EURORDIS che mette in contatto malati, famiglie ed esperti per condividere esperienze in un forum multilingua e moderato RareConnect Il Programma Rare Barometer è un’iniziativa di EURORDIS volta a condurre indagini con l’obiettivo di trasformare le esperienze delle persone affette da malattie rare in cifre e fatti che possono essere condivisi con i decisori politici.Rare Barometer La Giornata delle Malattie Rare, una campagna di sensibilizzazione internazionale che si svolge l'ultimo giorno di febbraio di ogni anno.Rare Disease Day La Conferenza Europea biennale sulle Malattie Rare è il più grande incontro di soggetti interessati al tema delle Malattie Rare in Europa. L'ECRD è un'iniziativa di EURORDISEuropean Conference on Rare Diseases