Regulatory system for medicines could be used more effectively, says new report

The current regulatory system for medicines in Europe is missing opportunities to make medicines available to patients earlier, says a new report from Escher, the independent TI Pharma platform for regulatory innovation, based in the Netherlands.

'Overall, the EU regulatory system operates well, however important recent changes had not yet been evaluated,' said André Broekmans, Chairman of Escher. 'One of our conclusions is that pathways that were intended to bring important new medicines faster to the market are not used in the right way. We are therefore missing opportunities to make medicines available to patients earlier.'

Escher collaborated with researchers from Utrecht University to look into a number of areas that might be improved: pharmacovigilance, conditional approval, paediatric investigation plans and decentralised authorisation procedures.

The research showed that some parts of the regulations are not yet fully achieving the effects they were intended to have. For example, the conditional marketing authorisation pathway was intended to bring much-needed products, for example, for cancer treatment, to the market faster. But the researchers found that in practice it has often been used as a back-up for the ‘normal’ approval pathway, because requesting conditional approval had some perceived drawbacks (e.g. potential reimbursement issues and the level of post-marketing obligations).

The way in which formal assessments of regulatory instruments are conducted needs to be strengthened

The researchers also identified opportunities for making the regulatory system more efficient, without compromising public health, for example, by adjusting the timing of the submission and the level of detail of plans for the investigation of medicines in children.

Broekmans said these discrepancies between the initial objectives of legislation and the effects of regulatory instruments in real-world practice should be better monitored.

’The way in which formal assessments of regulatory instruments are conducted needs to be strengthened. Also, one of the main messages from the report is that the effectiveness of regulatory instruments can be increased by reflecting on the interpretation and implementation of the primary legislation,' he said.

'Companies and authorities need to learn together how to use better the opportunities that the current system provides. This is especially important as we are heading towards a future with more adaptive approaches to regulation and medicines with different profiles and characteristics.'

Richard Bergström, Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA), added: 'Our latest learning from genomics and our improved understanding of human biology have resulted in a record number of promising breakthrough medicines in development. Entering a new era of personalised medicine, the current model for medicines development and approval may not be appropriate. Against this background EFPIA decided to provide an unrestricted research grant to the Escher platform to explore if the model is fit for purpose.'

Hubertus Cranz, Director General of the Association of the European Self-Medication Industry (AESGP), who also funded the research, says that another important conclusion of the report is that 'the research from Escher shows that many areas can be improved without having to adapt the legislation itself, but by working on a better implementation'.