Where science leads, legislation tries to follow

EU efforts towards a single market for medicines are still facing national obstacles.

Medical technology has transformed healthcare over the last century – but rising expenditure on drugs is disquieting ministries in member states where funding decisions are made, while consumer concerns, amplified by a rising tide of scepticism over technology, are leading to ever-stricter demands for safety. This is the shifting background against which current European Union debates on access to treatment are increasingly taking place.

Successive rounds of EU legislation over the past 60 years have imposed an accumulation of requirements on drugs, devices and diagnostics – and continue to do so, in response to advances in science. The EU exerts almost total control, with authorisation decisions issued only after satisfactory completion of rigorous sequences of tests and trials. In contrast to decisions on whether a drug can safely be put on the European market, decisions as to how much it will cost and who should pay remain entirely a matter of national competence.

But both of these approaches are now under fundamental review.

The European Medicines Agency, at the heart of the authorisation process, has admitted that the safety system it governs may have become too rigid, and be impeding access to potentially valuable medicines. In March it formally launched a pilot programme to explore a more flexible authorisation procedure, in which the blunt “Yes/No” decision-making after perhaps ten years of testing and trials might be replaced by an earlier, gradual, and closely monitored exposure to progressively wider patient populations. Hans-Georg Eichler, the agency’s senior medical officer, who is one of the leading architects of this so-called ‘adaptive licensing’, describes it as an attempt to “maximise the positive impact of new drugs on public health by balancing timely access for patients with the need to provide adequate evolving information on benefits and harms”.

On pricing and reimbursement too, radical new thinking is evident, prompted by the inconsistency between a single marketing authorisation valid for as many as 30 countries, and the 30 different decisions on whether the medicine should be paid for, based on 30 different methodologies.

The current process fully respects national sovereignty, but in so doing it is – as many national authorities now recognise – wasteful of time and resources, and can lead to inequalities of access between member states. So pricing bodies in the member states – such as health ministries or insurance organisations – are now working more closely together to see if they can agree at least on the criteria and methodologies they should employ, even if they retain the right to make their own decisions at the end of the process.

The EU is promoting these moves in the interests of making progress towards more of a single market in medicines. And there is cautious backing from the drugs industry, which sees potential efficiencies in avoiding duplicative processes. But neither of these initiatives is likely to move fast. Every revelation about a new side-effect strengthens popular calls for tighter controls on safety and re-ignites anxieties about any suggestion of simplifying authorisation processes. The complexities of drug pricing and reimbursement continue to defeat even timid EU attempts to legislate on the matter. That was amply demonstrated by the loss without trace of the European Commission’s 2012 draft directive on drug pricing methods, in the face of hostility from the member states.

Within the drug industry, which is the source of nearly all candidate drugs, the concern is often expressed that tinkering with the details of the rules may not be enough. Drug firms say what they most need are assurances that Europe can offer a market to innovations that will incentivise further research.

Behind the scenes, even more radical approaches are now being considered and discussed that could lead to industry, regulators and payers working more closely together in a significantly different relationship, so that they share the risks and the benefits of creating new medicines and bringing them to the patient.

After decades of a confrontational approach to drug innovation, the next 12 months could see the start of some more co-operative thinking and action.