Sponsors of products for rare diseases must manage a complex environment and often in a much shorter timeframe to meet high unmet need. However, they have one significant advantage: access to free scientific advice from the European Medicines Agency (EMA).
Orphan Drug Designation, the PRIME (PRIority MEdicines) scheme, conditional marketing approval – these and other schemes exist to get drugs to patients with rare diseases faster.
The health authorities want to help sponsors streamline their processes, follow good practices with manufacturing quality and formulations and meet high regulatory standards. They want sponsors of orphan products to talk to them from early on — asking questions, getting help and working through problems collaboratively.
Untangling the issues
Scientific advisors can help sponsors untangle complex issues such as understanding and proving the mechanism of action for a product in an area that is not well-established, as is the case with rare disease development. They can also help to guide sponsors as they develop their CMC documentation to clearly explain how their product is being developed and built. The aim is to establish a logical connection between the drug’s quality, delivery system, mechanism of action, clinical trial design and desired clinical outcomes for patients.
The regulators want to help sponsors make sure their manufacturing quality and formulations are good and that standards have been met. They encourage early and frequent engagement, particularly with schemes such as PRIME. If you have data you weren’t quite expecting, they encourage you to go back to them, let them know what is happening, what approach you are considering and then ask them what their opinion is so they can help you figure it out.
Managing development in a shorter timeframe
When it comes to Orphan Designation products, the timeline is faster. Sponsors of products for rare diseases often are eligible for early access schemes and conditional approvals, which means the assessment process is accelerated. Having support to ensure your dossier, your pharmaceutical development and your quality controls are phase appropriate is key, and the scientific advice open to sponsors is vital.
But getting good scientific advice requires understanding the schemes, knowing how to leverage the opportunities as well as knowing the right questions to ask and how. It means being able to speak the often highly technical regulatory language and being able to communicate your position effectively to the EMA.
Knowing what to ask
All too often, we find orphan drug sponsors don’t understand or take full advantage of the advice and support on offer. That is why it often makes sense to work with a regulatory consultant who understands the EMA schemes, their expectations and how to take advantage of the advice on offer.
Getting impartial advice from consultants who have been through the process many times can help you prepare for scientific advice, making sure you have read and understand the regulations, know how they apply and can demonstrate to the authority that you have done what you can to tackle issues before going to them with your questions and suggestions.
The scientific advice is there to be leveraged. Being properly prepared will help you ensure you get the most out of the buffet on offer.
“All too often, we find non-EU sponsors don’t understand or take advantage of the advice on offer. That is why it often makes sense to work with a regulatory consultant who understands the EMA schemes.”