EC suggests reforms to EU advanced therapy medicine rules

The European Commission has suggested reforms to the way that the European Union (EU) approves advanced therapy medicinal products (ATMPs).

Since a special approval system was introduced under 2007 regulation (EC) No 1394/2007, until June 2013, only 10 marketing authorisation applications for advanced therapy drugs were made to the European Medicines Agency (EMA); five concerned products previously on the EU market; and only four were ultimately approved.

As a result, the Commission has released a report suggesting changes, while 'reflecting on the appropriate regulatory framework for new innovative products that many not be captured by existing provisions'.

Authorisation procedures needed to be streamlined and ensure requirements are 'proportionate and well-adapted', with special consideration for autologous products.

Specific suggestions included:

• Fine-tuning the current definitions of ATMPs;
• Considering measures how to avoid disparities in the classification of ATMPs between EU member states;
• Clarification of when market approval requirements can be waived for products being used immediately in healthcare – the so-called ‘hospital exemption’;
• Stating how data obtained from such treatment can be used in later marketing authorisation applications;
• Extending the certification procedure and clarifying the link between certification and marketing authorisation procedures;
• Creating a more favourable environment for ATMP developers working in academia or non-for-profit organisations, including promoting early contacts with regulators, reducing fees for scientific advice, and extending the certification scheme to these developers; and
• Considering possible fee incentives to reduce the cost of post-marketing obligations.

The report noted: 'Too burdensome requirements could have detrimental consequences for public health as it could prevent the appearance of valid treatments for unmet medical needs.'