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.

 
 
A voz das pessoas com doenças raras na EuropaEURORDIS A voz internacional das pessoas com doenças raras, Rare Diseases InternacionalRare Disease International Reúne doentes, famílias e especialistas para partilhar experiências num fórum multilinguístico. RareConnect O programa Rare Barometer é uma iniciativa da EURORDIS para a realização de inquéritos para transformar a experiência das pessoas com doenças raras em números e factos que podem ser partilhados com os responsáveis pela tomada de decisões.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Adira ao maior encontro europeu das partes interessadas no âmbito das doenças raras na Conferência Bienal Europeia sobre Doenças Raras e Produtos Órfãos. A ECRD é uma iniciativa da EURORDISEuropean Conference on Rare Diseases