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 voix des patients atteints de maladies rares en EuropeEURORDIS Donnant la voix, à l'international, aux personnes vivant avec une maladie rare, Rare Diseases International est une initiative d'EURORDISRare Disease International Pour rapprocher patients, familles et experts sur un forum modéré multilingue, où partager leurs expériences. RareConnect est une initiative d'EURORDIS RareConnect Le programme Rare Barometer est une initiative d’EURORDIS qui vise à réaliser des sondages pour transformer les expériences des patients de maladie rare en faits et chiffres à communiquer aux décideurs politiques.Rare Barometer Campagne internationale de sensibilisation organisée chaque année le dernier jour de février, la Journée Internationale des Maladies Rares est une initiative d'EURORDISRare Disease Day Participez au plus grand événement réunissant tous les acteurs des maladies rares en Europe lors de Conférence européenne bisannuelle sur les maladies rares et les médicaments orphelins (European Conference on Rare Diseases and Orphan Products, ou ECRD). L'ECRD est une initiative d'EURORDISEuropean Conference on Rare Diseases