AMSTERDAM – Europe's famously fragmented system – or systems – for pricing and reimbursement will lose a few rough edges in the years ahead, as coordinated clinical assessment of product dossiers becomes the norm for all therapeutic products that are currently subject to the EMA's centralized market authorization process.
Ioana Siska, one of the key European Commission officials involved in that process, updated delegates on the progress of the initiative at the Advanced Therapies Summit, during a panel discussion Wednesday, titled "The EMA and EUnetHTA Collaboration on Evidence Generation."
The European Union's 28 member states collectively have more than 50 health technology assessment (HTA) agencies between them, all of which are involved, in various ways, in assessing the relative clinical effectiveness and economic impacts of newly approved therapeutics. Those assessments form the basis of the pricing negotiations between drug firms and individual national health systems. While drug prices continue – and will continue – to be determined at the national level, all of the EU members states (except Luxembourg, which has observer status) are participating in what is now a decade-long initiative to coordinate aspects of the assessment process, in order to eliminate the painful country-by-country effort that drug firms have to engage in at present.
The process has been a relatively informal one up to now, conducted through EUnetHTA, a cooperation network among Europe's HTA agencies, which has been funded on a project basis. It is now evolving in a more formal direction, following the European Commission's (EC's) publication on Jan. 31 of a proposal for a regulation, which would provide a legal basis for a single, harmonized HTA assessment of the clinical and scientific aspects of new products. It will not involve any economic evaluation of their impacts.
"We are not going to enter into a competence which is a member state's competence," said Siska, who is policy officer in the EC's Directorate General for Health and Food Safety.
Its emergence will be gradual. It will take at least six to seven years before the process is in place. "Maybe I'm too optimistic. It depends on the negotiation process with the European Parliament and the European Council," she said. Once the regulation is passed, it will take about three more years to develop a legal framework around it, and a subsequent transition period to allow member states to join gradually and to ramp up the number of products to be assessed.
It is not yet clear what organizational shape this new HTA assessment process will take. Although EUnetHTA already cooperates closely with the EMA – they have been offering parallel consultations since July 2017 – whether it will mimic the EMA's structure is not yet clear. "It's too early to think like this," Siska told BioWorld. "The European Commission will provide the secretariat support," she added.
The Alliance for Regenerative Medicine (ARM) has welcomed the mandatory aspect of the harmonized HTA assessment process. The EUnetHTA initiative has had some successes, but "it's been a slow process," ARM senior director and European policy coordinator Annie Hubert told BioWorld.
Individual HTAs have not been bound by the outcomes of joint assessments undertaken on a pilot basis. "There was no specific commitment for them to buy into what was agreed previously," she said. In the future, they'll have no choice. "It will become mandatory for member states to use this. They cannot rechallenge it."
While change is coming, it will drop slowly – and it won't alter the fundamental requirement for pricing negotiations to be conducted at a national level. "It will not eliminate all the pain for sure," Hubert said. But it will reduce it. "The description of clinical value is an important component of an HTA application."
It should, moreover, reduce some delays and some uncertainties, which could help therapeutic developers when pitching for investment.
Also at the summit, the ARM released a position paper in response to the joint plan for fostering the development of Advanced Therapeutic Medicinal Products (ATMPs), which the EC and the EMA published last October. It includes recommendations on the adoption of a pragmatic and flexible approach to product licensing, the development of regulatory processes that recognize the specific nature of ATMPs, the responsible use of the hospital exemption pathway, and initiatives to support funding, investment and market access.