BIO-Europe® special: Adaptive Licensing – A new approach in medicine market authorization

Written on 24 Oct 2014

Through ever stricter rules and regulations, the medical authorities have set increasingly high hurdles that must be taken before a medicine can receive market authorization. As a possible solution the European Medicines Agency (EMA) has identified the concept of adaptive licensing.

Stricter regulation serves the safety of the patients who can be certain that a medicine they have received has undergone some very vigorous reviews before it was allowed. On the other hand developers are faced with ever more dire prospects that their invention will eventually receive market authorization while at the same time the time and funds spent on the development increase steadily. And there are those patients that desperately hope for a medicinal breakthrough that would allow treatment of their illness rather sooner than later. To get all of these different interests aligned is one of the greatest challenges the participants in this game are facing.

Increased Risk, but earlier access

As a possible solution the European Medicines Agency (EMA) has identified the concept of so called adaptive licensing sometimes also known as staggering licensing. The idea behind this concept is simple and charming. Instead of waiting for the one moment where the industry has finally gathered all the necessary documentation to receive approval for its new medicine, earlier this year the EMA launched a pilot project wherein it wants to assess the possibility of granting medicines that are still in development an early market authorization restricted to certain groups of patients and from this point progress further (staggering) until full authorization.

The parties participating in this approach acknowledge the increased risk that comes with a medicine that has not yet met all qualifications for a full authorization but accept this risk in favour of an earlier access to that medicine by patients in particular need. For the industry this model has the great benefit that it can refine development of its medicines with experiences gathered directly in the field. Also, sales of the product commence earlier decreasing the risk of a total financial loss that would otherwise loom over it until full approval.

While the concept of adaptive marketing seems intriguing it is not a new one, not even on a European level. Already now the existing regulatory framework allows for compassionate use assessments and conditional marketing authorizations were already introduced under Art. 14.7 of Regulation 726/2004. Currently these existing tools have become more publicly known as possible options to obtain a treatment for the Ebola virus as soon as possible.

Specific regulation missing

While the existing regulatory tools are all aimed to be used in selected and exceptional cases the EMA maintains the opinion that from the current perspective these tools are sufficient to also provide a regulatory framework for a more general implementation of the adaptive licensing approach. Herein lays the danger of this promising concept. If the EMA truly wishes to establish an alternative to the existing route to market authorization it must do so with an accompanying set of regulations that specifically address the challenges of this alternative such as the management of responsibilities, the definition of the patient groups that have prioritized access to the medicine and what happens if the worst case scenario becomes reality. Borrowing from the existing set of regulations will only get you so far and would automatically restrict the new concept to a very narrow applicability. At the end of 2014 the EMA plans to evaluate the information gathered in the current pilot project. A first statement of the European Commission which currently assesses the need to implement additional regulations for adaptive licensing is also scheduled for that time.

Even if the European authorities should pave the way for adaptive licensing, another important factor for its success has yet to be addressed. How will medicines with restricted authorization be accepted and eventually reimbursed by the national healthcare systems? It remains to be seen whether adaptive licensing as an alternative to the traditional approach really has a future that goes beyond a mere niche existence.

Meet us on BIO-Europe® – Germany, booth 24. We look forward to you discussing this and other topics!