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Reflector is Pharm Exec's Brussels correspondent.
The long-cherished dream of finding a European approach to assessing the value of new medicines seems to recede further with every step taken to pursue it, writes Reflector.
The long-cherished dream of finding a European approach to assessing the value of new medicines seems to recede further with every step taken to pursue it. The latest demonstration of the challenges comes in a detailed on-the-ground exposé of the differences in how European countries assess health technology. No two countries do it the same way, and the differences range from what constitutes permissible data to how long different authorities take to reach their subsequent reimbursement decisions or to the acceptability of indirect treatment comparisons.
This is no black propaganda by opponents of the greater collaboration that the EU is trying to promote. This analysis is instigated by the federation of European research-based drug firms, EFPIA, who are strong supporters of some harmonization in health technology assessment (HTA). They stand to gain from more standardisation of the approach taken by the EU's 28 member states, because, as they state clearly in a new year position paper, "the fragmentation of requests today makes it difficult for companies."
For pharmaceutical companies operating at a global level, the continuing divergences in national systems mean, they say, lost opportunities to integrate more harmonized European data requirements into global development plans. It is hard to design pivotal randomized controlled trials that reflect the evidentiary needs of the majority of HTA authorities across the EU, and sometimes even additional clinical trials are needed to satisfy national HTA requirements. For companies, the fragmentation means at the very least duplicative administrative work. For agencies it generates uncertainty. And for patients it can mean delays, says EFPIA.
The catalogue of discrepancies is long. France, for instance, conducts an HTA review of all medicines at launch, while the UK examines only a selection of products, while several member states have their own specific processes for vaccines or orphan products. France and Germany focus on the clinical aspects of HTA to support their reimbursement decisions, but others use a full HTA-with the Netherlands employing sequential assessments of clinical then economic aspects, in contrast to Sweden and England where the full HTA is done in one step.
France requires publication to support any data that are not included in the clinical study reports, while England accepts data on file, that is not necessarily published. The Netherlands, Sweden and England permit HTA to start as soon as a positive opinion emerges from the European Medicines Agency's scientific committee, but France, Germany, Poland, and Spain demand that a full marketing authorization has been granted first.
In terms of the methodologies used for clinical assessment, the divergences are often even more marked, says EFPIA. HTA agencies adopt different approaches to interpreting the same clinical data – on aspects including trial design, relevant endpoints, appropriateness of defined patient subgroups andtreatment comparators. The industry federation contrasts the strict validity criteria for surrogate endpoints that Germany's IQWIG insists on with the practice in the UK's NICE of following EMA opinions.
The objective of the EFPIA analysis is not to furnish an exercise in ritual lamentation about what isn't working. It is to argue for change, to make a better system that will work and will improve decision-making in the future. And the position paper-which is a response to a 2016 public consultation in which the European Commission has asked for views on how to move ahead-offers plenty of possible remedies.
EFPIA contends that solutions require much more than creating a community of HTA technicians. All the agencies involved in supporting decisions on access to pharmaceuticals should be involved in European assessment of relative efficacy at time of launch, it says. Otherwise any system will run the risk of being disconnected from the reality of decision-making. And where joint work among member states leads to technical agreement on a common HTA approach for a product or class of products, that approach has to be adopted nationally by the member states that took part in the joint work. "The existence of a joint report should remove some work currently conducted at national level," it insists. "We need an open discussion on barriers to national adoption, and a commitment and political willingness to address them."
To make any collaborative approach function, a permanent system is needed, argues EFPIA. This needs to provide capacity for a joint scientific advice process that involves regulators and HTA bodies, and it needs a permanent structure, including funding and secretarial and organizational support. Member states should conduct European assessments of the relative efficacy of pharmaceuticals at time of launch in a collaborative manner and on agreed methodologies, making use of a rapporteur and reviewer system, it proposes. Rapporteurs and reviewers should be part of a committee that would endorse the European report produced-and would have members and experts with direct links to national decision-making on reimbursement or access.
But the chances of a better system working in the future depend on a lot of changes in national practices and attitudes-changes that, the EFPIA analysis concludes, just aren't happening now. The coming months will show how wide the support is across Europe for closer cooperation on HTA. The Commission's consultation deadline falls in January, and many organizations are commenting-often with very different views to those of the research-based industry. By the end of this year, the Commission has promised that it will digest the many inputs, and make some firm proposals as to how a permanent system might be established and how it should operate. As with all European Union projects, success or failure will be determined not just by the quality of the proposed process, but by how much political interest there is in the member states. That is where anyone wanting to influence the outcome should be focusing their attentions between now and the autumn.