Over the last year, the European Commission (EC) has been consulting on multiple scenarios for HTA harmonisation, as we discussed in Remap perspectives September 17. The output from the EC on Strengthening of the EU cooperation on Health Technology Assessment (HTA) and the subsequent EC proposals have recently been published. The proposals are for a mandatory joint European HTA assessment which may take effect from 2023.
Why is the EC proposing changes to HTAs?
The EC identified several challenges with the current system, specifically:
- Market access for innovative technologies is impeded and distorted due to differing national or regional processes and methodologies for HTA across Europe
- The current situation contributes to a lack of business predictability, higher costs for industry, delays in access to technologies, and negative effects on innovation
- It can result in duplication of work for national HTA bodies, inefficient use of resources and limited transparency for patients in different countries
The EC also identified that the current voluntary approach to joint HTAs (e.g. EUnetHTA) is not sustainable due to short-term funding and ongoing renegotiation.
What are the EC proposals?
The EC has proposed four areas of cooperation to address the challenges highlighted above:
- Joint clinical assessments (JCA)
- Joint scientific consultations (JSC)
- Identification of emerging health technologies
- Voluntary cooperation
These will cover all new and existing active substances seeking new indications that undergo EMA’s central marketing authorisation. Medical devices and in vitro diagnostics can also be selected for assessments.
The EC has been very clear that the centralised JCA will only focus on the Relative Efficacy Assessment (REA) and not on any economic parameters. Any assessment of value, economic impact, social considerations or pricing and reimbursement negotiations remain the remit of the individual member states.
What does this mean in practice?
A new permanent body will be established to conduct the assessments, including the JCA and JSC. The methodologies used in the JCA and JSC assessments are likely to be based on the EUnetHTA assessments for REA and parallel consultations. It will also have its own funding (probably coming from the EU budget), and an initial FTE count of approximately 35. Whether the body is associated with EMA or a separate entity is not yet clear.
The anticipated process for a joint clinical assessment will take place during the EMA assessment, with expression of interest discussions occurring ~180 days prior to CHMP opinion, followed by submission of a letter of intent from the manufacturer. At ~90 days prior to CHMP opinion, a scoping meeting will occur, following which the manufacturer will submit its evidence package. At the time of the CHMP opinion, a draft of the JCA will be developed, with the JCA sent for consultation (including fact checking by the manufacturer) at the time of marketing authorisation. The final JCA will contain an analysis of the relative effects of the new health technology on the selected patient-relevant health outcomes, and the degree of certainty of these relative effects based on the available evidence.
The aim is to have the final version of the JCA published and available 1 day after the EPAR. This is illustrated below in Figure 1.
Figure 1: An overview of the likely joint clinical assessment process
What are the timelines for implementation of the EC’s proposals?
This process is expected to continue where EUnetHTA’s joint Action 3 finishes in 2020. As such, legislation is likely to go through the European parliament during 2018/19 with expected adaptation of regulation in 2020. Next follows a 3-year development and adoption period between 2020 and 2022. From 2023-25 there will be a transition period, where a limited number of assessments will be completed, and participation will be voluntary for member states. From 2026 onwards, it will be mandatory for all member states to participate.
What are the implications for manufacturers and member states?
From a manufacturer perspective, this has the potential to be very beneficial. In theory, it should lead to faster market access as member states will have much earlier access to an HTA that details the added clinical benefit of new products. It should reduce costs by limiting the number of submissions which will be advantageous, particularly for small and mid-sized companies. It should also reduce uncertainty, as there is only one defined process as opposed to multiple individual country HTAs.
However, there are potential challenges for manufacturers. There is no understanding of how orphan drugs will be assessed and if there are modifiers that may be acceptable. Similarly, whilst it should lower the overall costs and resource burden on companies, a company that has been through the EUnetHTA REA process stated that it cost their organisation ~100 FTE days and ~€200,000 to complete the process. This initiative has the potential to be an additional hurdle to patient access as many EU countries will still want to conduct their own HTA assessments, incorporating economic considerations, at the country level. This would not result in any meaningful reductions in patient access.
Whilst several member states welcome this initiative, particularly the smaller ones (e.g. Austria), there is likely to be some resistance, particularly from Germany and France. The primary reason is that it may enable faster access to healthcare, and therefore larger healthcare spending by forcing earlier pricing and/or reimbursement decisions. It also removes a useful tool from the toolkit of some countries, in that the JCA may not be as robust or consider all the factors that its’ previous HTA process did. Arguments such as different comparators in each market and different healthcare systems have been put forward as to reasons why it should not be mandatory. This will leave countries with having to make decisions effectively based on price alone and the JCA.
In summary, there is a strong movement by the EC to put in place a formal process to harmonise HTA processes across the EU. The EC has identified that only by mandating an EU process will real change happen. It could also be argued that given the success of EMA, there should be no reason why a similar process should not be adopted with HTA, which would benefit all involved, except for some member states (e.g. Germany, France). However, there has also been strong resistance from some large member states, which could delay or prevent this initiative from being implemented. The next two years whilst the legislation goes through the European Parliament will be critical, but all indications currently point to a mandatory single EU HTA assessment by 2026.