By Peter O’ Donnell.
So at last it’s happened. Adaptive licensing has leapt from the pages of learned journals into the real world of European regulation. Is this the beginning of the end of binary decision-making on the merits of a new medicine? Has a half-century of the “yes/no” authorization process peaked, to be replaced by staggered approval, or progressive licensing? After years of reflection on the theoretical merits of a rolling approach, the European Medicines Agency last week launched a pilot project to test how well the theory might work in practice, with real medicines in development.
The agency is inviting companies to submit experimental medicines still in the early stage of clinical development as guinea pigs for this new approach—the latest attempt to strike the right balance between speeding up patient access to new medicines and ensuring patient safety. But to avoid giving the impression that caution is being thrown to the winds in the search for speed, the agency’s emphasis is on how the new approach is “prospectively planned.”
Early authorization would be given for use of a medicine in only a restricted patient population, then a series of iterative phases of evidence-gathering would support subsequent adaptations of the marketing authorization, expanding access to the medicine to broader patient populations. But it goes further than the existing regulatory mechanisms of compassionate use or conditional authorization. Widening patient populations would have access to an innovative therapy in line with evolving information on the benefits and risks.
Hans-Georg Eichler, the agency’s Senior Medical Officer, and one of the prominent advocates of adaptive licensing, says this could the way to “maximize the positive impact of new medicines on public health.” And the move has won immediate and explicit applause from some industry organizations. The decision “is to be welcomed,” said the UK’s Biotech Industry Association, which has campaigned for the introduction of adaptive licensing for years. “We will encourage our members to respond to the agency’s call for participation in the pilot project,” said its Executive Director, Steve Bates.
Support from UK-based industry groupings is not surprising. Much of the pressure to move the adaptive licensing agenda forward has come from the UK, which has been mooting an early access scheme of its own for promising unlicensed medicines in areas of high unmet medical need. The UK regulatory authority has over recent months played a leading part in press for European-level action to make full use of all available legal flexibilities to get innovative products to patients.
How fast the revolution will come is not so clear. In time-honored European fashion, everyone is to get a voice—regulators, industry, health technology assessment bodies, organizations that issue clinical treatment guidelines, patient organizations… “All discussions will take place in a ‘safe harbor’ environment,” insists the agency, which is aiming at “free exploration of the strengths and weaknesses of all options”—in development, assessment, licensing, reimbursement, monitoring, and pathways for use. And these options will be explored “in a confidential manner and without commitment from either side.” Not a recipe for rapid action.
The evolution of the approach itself is going to be adaptive too. The pilot phase will cover “as many programs as necessary” to gather knowledge and experience, address technical and scientific questions, and refine the design of the adaptive pathway for different products and indications. And “as the project progresses, the European Commission will examine the legal and policy aspects related to adaptive licensing in collaboration with the EU member states and by consultation with relevant stakeholders, as necessary.”
So if it is the beginning of the end of binary decisions, the end looks as if it is still a long, long way away.