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Imagine you are in charge of European legislation on pharmaceuticals. Imagine you've been told to draft rules on how to conduct clinical assessments at European level to help measure the value of a medicine. What would you go for?
Imagine you are in charge of European legislation on pharmaceuticals. Imagine you've been told to draft rules on how to conduct clinical assessments at European level to help measure the value of a medicine. What would you go for? The European Parliament has just agreed its list. In adopting a formal position on the EU plan for closer coordination of health technology assessment, MEPs voted to fix some of the conditions that they want to see imposed. And their opening bid (which they will shortly start to discuss with officials and diplomats from the EU countries to finalise the legislation) includes the following elements. The parliament wants the approach to be based on high standards of quality, and the best available scientific evidence. (They would hardly ask for anything less!) But the MEPs emphasise their insistence on quality: it must be "the most up-to-date and public data"-and "failure to comply with that requirement may trigger a penalty mechanism." They go on to recommend that the evidence should come-"where practically feasible and ethically justifiable" - primarily from double-blind randomized clinical trials, meta-analysis, and systematic reviews. The developer "conducts at least one randomized controlled clinical trial, comparing its health technology in terms of clinically relevant outcomes with an active comparator considered among the best current proven intervention at the time the trial was designed (standard treatment), or the most common intervention when no standard treatment exists." All the data and results of conducted comparative trials should be submitted. MEPs don’t want any old surrogate end-points, either. In their proposed system, "the assessments of relative effectiveness are based on end-points which are relevant to the patient with useful, relevant, tangible, and specific criteria suited to the clinical situation concerned." Similarly, they define the comparators that should be used: "The reference comparators for the clinical entity concerned and the best and/or most commonly used technological or process based comparator." And drug developers will have to provide the dossier they have already submitted to the European Medicines Agency in their marketing authorization application-which will include a clinical study report. There are always exceptions, the parliament position notes. So its methodologies take into account the specificities of new procedures and certain types of medicinal products with less clinical evidence available at the time of the marketing authorization (such as orphan medicinal products or conditional marketing authorizations). In the case of orphan medicines, the report should not re-assess the criteria of the orphan designation-although assessors should have full access to the data used by the authorities responsible for granting the marketing authorization, as well as the possibility of using or generating additional relevant data. And it may be justifiable to consider that there is no need for additional evidence beyond the significant benefit assessment already carried by the European Medicines Agency. But that doesn't amount to anything like a free pass. "Any such lack of evidence does not prevent the generation of additional evidence required to be post monitored and which may require post-assessment", says the parliament. And since clinical trials are "the studies par excellence", so the use of-say-epidemiological studies, or another type of study, may be carried out only in exceptional cases "and shall be fully justified." That's the parliament's take. What's yours? Now, while Parliament, Council, and Commission go into the three-way talks that will aim to turn the proposal into law, there is every chance for some inspired thinking to influence the discussion. In a few years time, what is decided now will govern the way that clinical assessment is conducted right across Europe for decades to come. Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium