Realising the potential of HTA Reform

The HTA Regulation was intended to streamline how the value of medicines is assessed, but by overburdening the process, it risks making patients wait for new therapies. Now is the time to come together to ensure we can realise the potential of the EU HTA reform for patients.

Nathalie Moll is Director General at EFPIA.

Health Technology Assessment (HTA) is a key step in the long journey that a new medicine makes on its way to patients who need it. From laboratory research and clinical trials to rigorous checks by regulators to ensure a medication is safe and effective, the path to the patient is long and uncertain.

One of the final steps, for the small proportion of potential products that overcome these hurdles, is an evaluation by HTA bodies to determine the value of a new medicine. This informs crucial decisions, taken at national level, about whether and how the new product will be used and by whom.

The process is complex and requires significant work by medicines developers to generate the data HTA bodies need to come to an evidence-based decision. In Europe, we have faced challenges arising from different approaches taken from one Member State to another.

This problem was acknowledged by policymakers and resulted in the approval of the EU HTA Regulation. It creates a legal framework for European countries to work together on a single, joint assessment of clinical evidence for new products, rather than having 27 separate processes. The focus of the EU-level work in this area has been on shared approaches to analysing clinical information that national authorities can use in their decisions about pricing and reimbursement.

The key objective is to reduce duplication and enhance efficiency through Joint Clinical Assessment (JCA). However, with the EU HTA Regulation applying to oncology medicines and advanced therapies from 12 January 2025, the European Federation of Pharmaceutical Industries & Associations (EFPIA) is increasingly concerned that the implementation could make a difficult situation worse.

The goal was the right one, but its execution is shaping up to be counterproductive. Here’s why.

Unresolved tension

JCAs will, as the name implies, be jointly produced with input from all countries. National HTA bodies are then expected to give due consideration to these JCAs when completing their assessments.

However, it is becoming increasingly clear that the tension between EU Joint HTA and the national HTA is not completely addressed by the new system.

JCAs were envisioned as a way for Member States to align on aspects of this complex process that are common to them, thus streamlining a significant part of clinical assessment. The appeal of this from the outset was to overcome fragmentation and duplication that has been a drag on HTA in Europe.

Alas, as we enter the implementation phase, there are clear signs that Member States prefer to make the new system fit existing processes rather than leaning into collaborative approaches that could be taken across Europe. This would mean that instead of the EU approach promised by HTA reform, national authorities are seeking to preserve the status quo indefinitely.

Adding to the administrative load

The HTA Regulation will be part of a complex landscape. Developers already provide relevant information to inform regulatory assessment and will now have to submit, at the same time, a dossier for the JCA process – all of which feeds into national HTA procedures.

When it comes to the EU HTA, rather than streamlining what is common to all Member States into one joint assessment, the new reality is likely to look more like an amalgamation of existing national HTA requirements.

The EU Regulation requires each new joint assessment to cover information and data under at least four parameters: Population, Intervention, Comparator and Outcomes – known as PICO, which defines a given assessment’s scope. However, rather than aligning on a harmonised PICO, it seems that Member States (at least in EUnetHTA21) have proposed the opposite approach: making the assessment scope a combination of their existing requirements, to be included into an enormous matrix of PICOs.

So, instead of a focused and streamlined assessment scope, developers will face a new and more complex combination of requests for data and analyses, with no element of predictability on how this will be composed.  This may sound complex (because it is) and the result will be an additional layer of approval for new medicines.

As the implementing act governing the process itself has become available, concern among research-driven pharmaceutical companies has only deepened. Developers will be expected to respond to all PICOs within 100 days, while navigating the regulatory process as foreseen in the implementing act. This will put a major strain on companies and may be too much for some small and medium-sized enterprises.

While the European Medicines Agency engages with the developers early in the submission process, the new EU HTA seems to take the opposite approach: leaving no visibility to the developer on what must be submitted, until the 100 days kick-in. No matter how complex the PICOs will be, the implementing act will give the developer no predictable opportunity to engage with assessors to understand their expectations or offer alternative answers.

Getting back on track

The success of the EU HTA Regulation depends on the delivery of high-quality JCA reports which provide added value to national decision-makers. It is essential that sufficient time is available to conduct the required analyses and finalise clinical evidence dossiers.

EFPIA and its members are open to making a constructive contribution to improve the operation of HTA in Europe.

We strongly believe that the HTA Regulation can set Europe on the right path if the future system recognises the role of companies as key stakeholders in the process and enables a rebalancing of workload in line with respective remits and obligations.

EU HTA cannot become a duplication of national HTA systems, funded by the EU. Nor can it introduce conditions that are difficult for the developer to meet.

Europe needs HTA reform. Our shared task now is to ensure we find a way that makes the new Regulation work as intended.