Going Dutch in Search of Drug Regulation Solutions

The first week of March will be remembered in European health circles for a long time as the week when everyone who mattered bumped into one another in Amsterdam. The Netherlands, which is in charge of EU business for the first six months of this year, has convoked an intensive round of meetings to show that it really is taking health policy seriously during its turn in the rotating EU presidency. 

For readers of Applied Clinical Trials, some of the most interesting outcomes can be expected from a meeting starting on that curious extra day in February and running through to Wednesday March 2, on ‘Innovation for the benefit of the patient: early interaction between market authorization, health technology assessment and payer in order to optimize patient access to innovative medicines’ (European meeting titles are not written by people in a hurry!). It could also have been entitled just "adaptive pathways" because that is what the agenda deals with – and most of the participants have already distinguished themselves in the evolution of this concept over the last two or three years.

Tomas Salmonson, Chair of Committee for Medicinal Products for Human Use at the EMA and scientific advisor to Sweden's Medical Products Agency, and Nick Crabb, Program Director for Scientific Affairs at the UK's National Institute for Health and Care Excellence – better known as NICE – will be doing most of the heavy lifting, assisted by senior figures from the Italian and Norwegian drug agencies as well as from WHO.

And the lifting will be heavy, because the declared objectives of the meeting will present a real challenge. The discussions will aim, says the Dutch presidency, to clarify which products early access schemes and adaptive pathways are useful and advisable for, how to get early alignment of the requirements for market authorization and for health technology assessment, and what conditions payers would want (in terms of managed entry – and exit – schemes). 

So the questions to be answered will have to include issues such as who should select products for adaptive licensing – should it be regulators, payers, public health authorities, patients, or clinicians? And should the selection of products give priority to potential treatments for an untreatable disease, or for an untreatable future disease (such as an antibiotic that defeats a developing resistance), to products that promise a cost-reduction for society? Should  very expensive products be excluded from accelerated access schemes? Or should companies be shut out if they deliberately focus on only a limited set of assessments?

Even before that meeting ends, another one will start just down the road, to kick-off the latest attempt to get national authorities to make better use of health technology assessments conducted at the European level. Entitled ‘Towards Sustainable HTA Cooperation in Europe’, this conference will plot the course of the new phase of the European network of Health Technology Assessment (EUnetHTA), which is to start its three-year life at the beginning of May.

Here the challenge will be to define how to overcome the barriers that still stand in the way of a fully-functioning network of HTA organizations in Europe, and which have hampered progress despite all the efforts of the last 10 years. Some of the obstacles are methodological, some are resource-related and some are legal. But between them, they have reduced the uptake of joint assessment reports and other work carried out in common within the network.

There is a sense of urgency, since by 2020 this work – which is currently funded by the EU – will have to be taken over by regional and national HTA organizations. If these clients are not benefiting from the joint work, they are unlikely to be enthusiastic about continuing the investment of resources that will become essential to keep the network alive. The discussions, therefore, will focus on EUnetHTA’s goals for 2020, and on definitions of how joint work can benefit the payers and governments who are the ultimate end-users, as well as on the housekeeping tasks of how the new program should be run.

The same week and the same town sees other EU meetings hosted by the Dutch Medicines Evaluation Board, playing its part within the framework of the country's EU presidency. The Scientific Advice Working Party of the EMA, which coordinates the provision of scientific advice and protocol assistance and formulates scientific opinions for the Committee for Medicinal Products for Human Use, is meeting on Wednesday and Thursday. The Pharmacovigilance Risk Assessment Committee (PRAC) is responsible for assessing and monitoring safety issues for human medicines is meeting on Thursday and Friday. So too is the Co-ordination Group for Mutual Recognition and Decentralized Procedures – Human (another candidate for the snappiest title of the year competition!), which examines questions about medicines marketed through the mutual recognition or decentralized procedures.

There were malicious suggestions earlier in the year that the Dutch presidency had lost interest in pursuing EU issues relating to health and medicines. One busy week may just lay that perception to rest.