If you have a programme under development, you need to make an informed decision about where to take it. When approaching different regulatory agencies in parallel you may end up with contradictory advice even though both assessments are based on the same data. How do you make the most of what you have?

According to the panel it’s achievable with the right adaptations. The discussion took off with some general thoughts about the major jurisdictions they have been interacting with during the pandemic; the US Food and Drug Agency (FDA), the European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

The pandemic has clearly taken a toll on agency resources which has impacted responsiveness and timelines. The panellists described some frustration originating from almost exclusively written feedback in response to meeting requests. Written feedback much more easily leads to misunderstandings, inadequate questions, and answers which in turn may put the programme and timelines at risk.

As a member of the EU, the UK assessors performed around 30% of the EMA assessments. With the Brexit this has created a meaningful resource constraint on the European system. The system has also experienced challenges with regrowth which has an impact on assessor quality and response time. The panel recognised that the European and national agencies need more resources, a strong focus on the development of their assessors and for imbalances in the contribution of different member states to be addressed. The rapporteurs need to continue to support and train each other.

The UK approach was perceived as pragmatic and proactive. The agency was responding according to timelines and the interactions was viewed as pleasant and proactive. The assessors are qualified, but their numbers limited, which has made the agency take a more selective approach regarding which programmes to work with.

Different streamlined approaches incorporating Health Technology Assessment – pathways with an incentive to aid developers to do the right thing early on to put products in the hands of the patients – were also discussed. The general opinion was that reimbursement is a part of the market access strategy and it is essential to start collecting the necessary data early to demonstrate the product value, leverage development synergies and avoid rework in later phases.

The UK Innovative Licensing and Access Pathway (ILAP) provides early appointment of rapporteur and regular face to face meetings which is very helpful.

Until recently, EMA’s PRIority Medicines (PRIME) scheme accepted a lot of early programmes, possibly too early according to the panel. There seem to be an ongoing shift towards having some clinical data before PRIME designation which is preferable and will allow the agency to put more effort and resources against more mature products and development programmes.

A less known pathway that was discussed the different Named Patient Programs (NPP) in place in many countries and under many different names. This was perceived not as an expedited pathway but primarily part of a market access strategy. The NPP requires standard regulatory preparations, it is however, an opportunity to take market shares in other countries before launch. From the panel’s experience, the evidence requirements are no less onerous for these kinds of programmes than the traditional pathways, so although it may be easy to get lured in this direction, the effort is not perceived as lighter than other options. There are rarely any shortcuts to demonstrating the product’s benefit / risk profile, nor its value.

• Which pathway/s/ are right for your product? There are multiple routes for approval and each pathway has unique eligibility criteria and benefits. Make sure to choose the one that suits your programme best.
• Take multiple regions into account early when you structure your dossier, and make sure you understand the different approaches across jurisdictions. Much of the international assessments are based on the ICH guidelines which are the same across jurisdictions but used differently they can generate contractionary advice. Find the common denominators in your dossier to make your multi-jurisdictional approach cost- and resource efficient.
• Start early! Always have reimbursement on top of your mind already during the study design. If you can collect additional data to demonstrate value during your programme, this will be a major cost saver compared to collecting it in discreet exercises later.