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Although only the beginning of an exploratory process, the EMA is being accused of colluding in a conspiracy to favor drug firms at the expense of patient protection.
As the European Medicines Agency prepares to deliver its first full report on its pilot project on adaptive pathways for medicines, it is making efforts to provide reassurance that it is not intending to dismantle the current regulatory procedures for authorizing medicines. The report is due out in March, a year after the agency started its experimental talks with companies on speeding access to innovative products by making greater use of real world evidence and carefully planned use in restricted and selected populations. But although this is still only the beginning of an exploratory process, the agency is already being accused of colluding in a conspiracy to favor drug firms at the expense of patient protection.
Health campaigners-and notably the French organization that champions rational drug prescribing, Prescrire-have been vigorous in their denunciations of the pilot. Prescrire recently told a meeting of EU and national regulatory officials that the system was going to be providing benefits for industry through earlier launch and longer patent protection, while "lowering the bar" with methodological shortcuts and lower evidence requirements. "EU citizens are to become guinea pigs," they said. Existing mechanisms such as conditional approvals and compassionate use provide sufficiently rapid channels for meeting unmet medical need, without subverting the regulatory system, campaigners claim.
The adaptive pathways approach is already here, retorted Francesca Cerreta of the EMA when she presented an initial report to the most recent meeting of the European commission's special committee on speeding up drug authorization. She offered the example of Lemtrada for multiple sclerosis, an expensive drug costing around $160,000 per treatment and with a difficult safety profile. Five-year follow-up of the limited population to receive this drug made it possible to determine that two-thirds did not require re-treatment. The additional data from follow-up opens up the possibility of a much clearer definition of a product's value in real world use, over and above what may be available from clinical trials in a selected population. This, she suggested, was a good example of the merits of the adaptive pathways approach. But, she insisted, the agency is not instituting new procedures. Instead it is making the best use of real world evidence.
Nor is the approach intended for or suitable for all products, the EMA has been insisting, to allay concerns about any risks of an impending revolution. Cerreta spelled out the criteria for selecting a drug as a candidate for this approach. There must be an iterative development plan starting in a well-defined population and expanding (or perhaps already having) a conditional marketing authorization with surrogate endpoints. And it must be possible to acquire real world data on safety and efficacy to supplement to clinical trials.
Of the 59 products that were submitted as candidates for the EMA pilot only 20 were selected for discussion with the company-and so far only 15 of those discussions have taken place. And of those, only 11 proposals were selected for further review through in-depth meetings. The main reasons for candidates being rejected were, she said, that the development was already too advanced, so it was too late to change anything, or there was limited learning potential in the product because there was no developed proposal for the use of real world data and limited scope for iteration.
The European jury is still out on how useful adaptive pathways will be. The EMA's current pilot is not going to provide all the answers-the best that can be hoped for is a clearer view of what will be needed to make such a scheme effective at European level. But one conclusion is certain: it is going to provoke a lot more debate.