Challenge

Obtain CHMP’s (EMA’s Committee for Medicinal Products for Human Use) agreement on a change in development plan previously agreed (noting that all scientific advice must be included in the future Marketing Authorisation Application (MAA)).

Approach

The client is developing a product for an orphan disease.  An initial scientific advice procedure (known as ‘Protocol Assistance’ for an orphan product) was undertaken however following further development it was clear that the advice could not be followed.  EUDRAC assisted the client to develop the strategy for justifying the change in approach.  EUDRAC proposed the outline and reviewed the briefing document and associated regulatory documents.

Conclusion

A robust package was prepared outlining clearly why the initial development plan could not be followed and why the alternative plan was scientifically valid.  SAWP/CHMP agreed to the change in development plan and thus the risk of regulatory non-acceptance at time of MAA was averted.