Today, March 7th sees the launch of the PRIME initiative by the European Medicines Agency. This scheme aims to strengthen support to medicines that have the potential to benefit patients who presently have no treatment options, or that may offer a major therapeutic advantage over existing treatments. These are considered PRIority MEdicines by EMA (hence PRIME).
It is important because if restores balance between the major regions. Until today Europe has been at a considerable disadvantage in that the US launched its Breakthrough Programme in 2014 and this has attracted more than 350 applications. This was followed by a similar initiative in Japan. The proposal driven by the Committee for Medicinal Products for Human Use (CHMP) is strongly supported and welcomed by industry. European regulators are to be congratulated on the speed at which they have introduced the proposal. It was only in mid-September that they announced the initiative and for European procedure to go through consultation and introduction in just six months is very impressive. It clearly shows willingness from the regulators to assist in bringing important priority medicines through the system and then make available to patients as early as possible.
Essentially the system is similar to that in the other regions and allows for the early designation of the priority medicine and then for frequent dialogue and discussion during development. Is the system perfect? Answer "no", there are additional features that industry would like to have seen, but recognised the resource constraints on the Agency. Nevertheless it is a very welcome start. PRIME should also be seen in the context of other initiatives. Within Europe we have seen a consultation on expanding and improving conditional authorisations. It is now expected that a new approach on conditional authorisations will be launched later this year. This discussion arose from the Escher report which highlighted that the conditional approval system within Europe was not being used as intended in the legislation. Rather it was being used as an exceptional rescue system. Additionally, there are moves to improve the accelerated review procedure within the European Union and very constructive discussions under Adapt Smart which is considering how to introduce the concepts of adaptive licensing within the regulatory process. It is recognised that these are complimentary and not mutually exclusive procedures. A product granted a priority designation is highly likely to be accepted for an accelerated review and perhaps conditional approval and the development would also employ adaptive designs.
Within the UK we have the EAMS (Early Access to Medicines Scheme) which has many similarities with PRIME but of course has stages 2 and 3 which involve the granting of a scientific opinion before the full authorisation and the access for UK patients through special commissioning in advance of the authorisation and consideration by NICE.
PRIME is helpful in the context of the Accelerated Access Review (AAR) which is due to deliver its final report in mid-April. The interim report of the AAR has raised the concept of the "pull through development". PRIME will therefore both assist and be complimentary to the expected recommendations from the Accelerated Access Review.
Clearly the regulatory landscape both in Europe and globally is changing with recognition that patients demanding early access to innovative medicines and that use of Real World and Big Data can be employed in the post marketing phase. Much of this agenda is being driven by the nature of the compounds which are coming forward. Arguably the previous and indeed the present regulatory system has been built for small relatively simply molecules treating large populations whereas today the majority of new medicines are large complex macro molecules targeting specific smaller populations. For those working in global regulatory strategy it is going to be important to decide how to access these various regional initiatives and bring them into a global early access strategy. PRIME is a significant development in making early access a reality in Europe.
Dr David Jefferys is Senior Vice President, Global Regulatory, Government Relations, Public Affairs and European Safety at Eisai Limited. He is also the Chair of the ABPI Regulatory Expert Network.