A fast-track approval for new medicines - Patient safety at risk?

In January 2014 the European Medicines Agency (EMA) launched a pilot project called “adaptive pathways”. The goal of the project was to improve timely access for patients to new medicines, by gathering evidence through real-life use to supplement clinical trials data. The eligible medicines would be put on the market for a subgroup of patients suffering from a disease for which there is no existing treatment. The use of such medicines would gradually be expanded based on additional data generated after the product has been placed on the market.

It is key for patients to have timely access to medicines, however BEUC considers that sidestepping the standard benefit-risk assessment for licensing a medicine should only be done for a very limited range of medicines and only when there is no other available alternative. In particular we are concerned about the following:

1. The scope of the project is not clear.
2. The monitoring of medicines’ safety and efficacy is difficult and not fully operational.
3. The information provided to patients needs to be adapted.
4. The safeguards for patients in case of harm need to be upgraded.
5. Cost and reimbursement conditions are not clear.
6. The lack of a transparent public debate.
7. The rationale of adaptive pathways needs to put patients first.
8. The added value of adaptive pathways compared to other schemes is unclear.