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New EU group could ultimately end up handcuffing much-needed efforts to modernize drug authorization.
Europe is at risk, yet again, of falling victim to timidity born of its divisions. Its indecisive responses to the challenges to its single currency, or to Russia's erosion of international law, are mirrored in its approach to medicines innovation, too.
Everyone in the pharmaceutical sector is now starting to get to know about MAPPs, the acronym for a convenient concept-"medicines adaptive pathways to patients"-that is now being explored in many different ways by pioneering spirits across the world. Basically, it's a matter of getting good new medicines to patients more quickly-or, to put it another way, of modernizing regulatory systems to take account of advances in biology. The discussions now taking place in Europe, the US, Singapore, and Canada reflect a common concern to see valuable innovations put to use sooner, and more judiciously, than is permitted by the current frameworks for authorizing and paying for medicines.
On MAPPs, Europe has, as is also so often the case, generated much of the leading thinking on how this clash between new and old might be resolved. Most prominently, the European Medicines Agency (EMA) is running a pilot with a dozen real medicines in the early stages of development, to identify what procedures need to change-among regulators and drug sponsors-to stand a chance of making real improvements in the system. Several European countries are also experimenting at national level with their own approaches-the UK is running an early access to medicines scheme; France, Belgium, and Spain all offer some examples of early authorization.
But just as the term MAPPs attains currency in the world of medicine, the European Union (EU) has come up with STAMP-a new expert group ostensibly intended to assist the process, but which, in its current manifestation, appears just as likely to suffocate it. STAMP stands for "safe and timely access to medicines for patients"- which sounds comfortingly like the same concept. On paper, its mandate, to provide advice and expertise to the European Commission on the use that can be made of EU pharmaceutical rules and related programs and policies, looks constructive. Its planned activities appear broadly consistent with the objectives of MAPPs: "STAMP will exchange views and information about the experience of member states, examine national initiatives, and identify ways to use more effectively the existing EU regulatory tools, with the aim to further improve safe and timely access and availability of medicines for patients."
But take another look at that last sentence. Ignore the well-intentioned phrase about improving access, and focus on the preceding phrase: "to use more effectively the existing EU regulatory tools." Innocuous? Plausible? Perhaps at first glance. But in the context of what is at stake in the discussions on MAPPs, potentially ruinous. If the EU's most strategic response to this effervescence of thought about a fundamental process in healthcare is to set up a group to see how the existing rules and tools can be used, it is missing the point of the exercise.
There is a very obvious reason why a more open-minded response is called for. Alternative pathways are alternatives. And the need for alternatives has already been clearly established by work that is underway-and has been underway for at least half-a-decade now-on tackling the mismatch between new science and traditional regulation. This is how the EMA sees its own challenge: "The concept of adaptive pathways foresees either an initial approval in a well-defined patient subgroup with a high medical need and subsequent widening of the indication to a larger patient population, or an early regulatory approval (e.g., conditional approval) which is prospectively planned, and where uncertainty is reduced through the collection of post-approval data on the medicine's use in patients."
This is how the EU's own Innovative Medicines Initiative -the multi-billion public-private partnership on drug research-sees it: "MAPPs refers to a flexible development and access pathway within the current regulatory framework that maximizes the positive impact of new medicines on public health by balancing timely access for patients with the need to provide evolving information on benefits and risks. It requires the early marketing authorization of a product focused on a well-defined and targeted population identified by predictive preclinical and clinical evidence as well as various sources of real-world evidence. It implies a clear safety and efficacy profile and may integrate a number of elements such as adaptive clinical trial design, patient-centric benefit/risk assessments, and the continuous evaluation of a therapy as new evidence (including real-world evidence) becomes available. MAPPs, therefore, relate to the entire life cycle of a medicine from development, through licensing to patient access (pricing/reimbursement and healthcare delivery)."
At the heart of the discussion is how to shift away from the straitjacket created by 50 years of a defensive approach to drug regulation-that cumulative, but inevitably futile, attempt to eliminate all risk by multiplying safety testing-and accept that the performance of a medicine may be more effectively measured by other procedures. New science opens up the possibility for changing the focus from a confrontational system of extensive pre-release trials and a one-off decision on what a medicine will do. It permits a more collaborative relationship based on continuous assessment by all involved parties, using more predictive mechanisms and better post-release monitoring, to obtain better outcomes. But it presupposes a readiness to look again at the current arrangements for balancing the need for prior evidence with the need for early patient access.
That is why sticking to the rules as a precondition for reflection is not likely to impel the sort of radical thinking and constructive development that a better deal for patients depends on.
If the heart of the discussion is the balance of evidence and access, the muscle of the debate could be described as a new balance of access and affordability-which also has to be taken into consideration in advancing MAPPs. The fastest procedures for authorizing a medicine are no use unless patients can actually obtain the medicine-and that is a function of the distinct national systems for payment or reimbursement for prescribed drugs. So making a reality out of MAPPs requires radical thinking, too, about how to bridge that gap. Here, too, however, STAMP appears to stamp even more firmly on EU initiative.
"It was clarified that the role of the group," reads the record of the first STAMP meeting, held in early 2015, "is not to provide advice for the revision of the basic acts [of EU pharmaceutical legislation]. In addition, health technology assessment, pricing, and reimbursement will not be the focus of STAMP." At a stroke-or perhaps with a sudden exertion of downward pressure-the EU rejected out of hand any talk of a review of the pricing and reimbursement arrangements that most critically govern actual patient access. The reluctance-nay, the downright refusal-to countenance any discussion of the economics is a consequence of Europe's Achilles' heel in the health sector.
Each member state retains absolute autonomy over health spending-and by implication, drug pricing and reimbursement. This autonomy is a longstanding and stoutly defended position of the member states (as is also, by no small coincidence, the case for foreign policy and for national economic management). It satisfies their own sense of responsibility. But it may, at the same time, amount to irresponsibility in terms of pursuing common goals-as the respective fates of the Euro, Ukraine, and innovative medicines are likely to demonstrate.
Reflector is Pharmaceutical Executive's correspondent in Brussels.