It can be disappointing and frustrating to discover that a therapy for your condition has been approved in Europe, and to hear that it cannot be offered to you. Below, we discuss some of the reasons why this might happen.
It can be disappointing and frustrating to discover that a therapy for your condition has been approved in Europe, and to hear that it cannot be offered to you. Below, we discuss some of the reasons why this might happen.
The regulatory body for approving medicines in Europe is the European Medicines Agency.
From laboratory to patient: the journey of a centrally authorised medicine EMA guide showing the timeline of a medicine's development
Compassionate use EMA guide providing further details of the compassionate use case.
These resources provide more technical detail about the medicines authorisation process.
Authorisation of medicines | European Medicines Agency (europa.eu)
About medicines - The European Federation of Pharmaceutical Industries and Associations (EFPIA)
Discover and Development of Medicines - EUPATI Toolbox
This resource does not cover the regulatory approval process. You can find further details here:
Medicine development - The Association of the British Pharmaceutical Industry (ABPI)
You can find more detailed information on the process of pricing medicines in Europe in the resources below.
Medicine Pricing and Access in Europe - Health Action International (haiweb.org)
Value of medicines - The European Federation of Pharmaceutical Industries and Associations (EFPIA)
Innovation - The European Federation of Pharmaceutical Industries and Associations (EFPIA)
The value and pricing of innovative medicines - European Patients' Forum (EPF)
Medicines pricing and reimbursement: EU - Practical Law (Thomson Reuters)
Getting Ready for Advanced Therapy Medicinal Products (ATMPs) in Europe - The Alliance for Regenerative Medicine (ARM)
Medicine pricing - The Association of the British Pharmaceutical Industry (ABPI)
Creating EU market access roadmaps - GCT Catapult
Gene and cell therapies are often presented in the media as the ultimate solution to rare or complex conditions. However, each case is unique, and each therapy carries different potential risks and benefits. A patient and their healthcare team should balance the risks and benefits in their specific case.