Managing the challenges and embracing the opportunities of EU-wide HTA
The way healthcare technology assessments (HTAs) are conducted in Europe is changing. In less than a year, all new advanced therapeutic products and oncology drugs will be subject to a mandatory, EU-wide joint clinical assessment (JCA), with orphan drugs, class IIb or III medical devices, and class D in vitro diagnostic devices all following suit by 2030.
Aimed at expanding access to new products by streamlining the current nation-by-nation approach to post-regulatory approval adoption, the EU HTA regulation is one of the biggest shake-ups to the HTA processes in decades. And, if organisations act now to overcome the challenges, they could reap the rewards for years to come.
HTA Regulation
While the European Medicines Agency (EMA) assesses the efficacy and safety of a product, an HTA assesses clinical and non-clinical factors, such as added value, relative clinical effectiveness, costs, and broader impact. In short, the EMA asks if a product could be adopted, but HTAs ask if it should.
To decide on national adoption, an HTA asks questions such as whether the new medicine or device is more effective than the current standard of care, and whether it represents value for money when compared to other interventions.
Currently, HTAs are conducted at the member state level following EMA clinical assessment and regulatory approval. Yet, with each country having its own HTA body, processes, and timelines, this can result in disparities, with access being delayed or even blocked in some nations, and duplication of costs and efforts for all involved.
The Regulation on Health Technology Assessment (HTAR), passed in 2021 under the auspices of European Network for Health Technology Assessment (EUnetHTA), aims to harmonise the process by introducing a new, EU-wide strategy.
Joint Scientific Consultations (JSC) will facilitate consultation between EMA, EU HTAs, and industry on intended population, the intervention, the relevant comparators, and the outcomes (PICO). These questions will be developed by stakeholder groups from an individual member state, each of which submit a “PICO” request for information to the developer. The findings will then form the basis of an EU-wide Joint Clinical Assessment (JCA).
While individual countries will not be bound by the JCAs, and can still opt to follow their own HTA pathway, they will act as a useful starting point for speeding up adoption.
The regulation is being introduced in steps. It will become mandatory for advanced therapeutic medicinal products and oncology products in January 2025, for orphan drugs by January 2028, and for all other products by January 2030.
Opportunities and challenges
This new approach offers clear potential benefits for medicine and device developers. Streamlining the process to create a single JSA, rather than numerous HTAs, could create faster routes to market across the EU, while reducing the administrative burden and costs of duplicated efforts.
In addition, because the PICOs are developed by national stakeholder groups, including patients and clinicians, it can contribute to a greater understanding of the needs and preferences of target countries and patient groups. These invaluable insights could, for example, be used to inform the development of future products and “beyond the pill” interventions that truly meet medical need.
However, adapting to this new way of working will also present challenges, chief among them being managing timelines.
Under the new rules, a completed JSA is required six months after EMA approval. Yet, it can take three months just to receive all the PICOs, limiting the time developers have to respond. Additionally, organisations may need to provide evidence to support multiple PICOs at once, all of which may require information on different endpoints, subgroup analyses, sensitivity analyses, and indirect treatment comparisons, for example.
The process, then, is likely to require organisations to supply a large volume of information in a very short period of time.
Managing the transition
To overcome the challenges and seize the opportunity, pharmaceutical companies and prospective market authorisation holders (pMAHs) need to be prepared.
While the first tranche of changes will not come into force until January 2025, adapting internal processes as early as possible will help ensure organisations are ready to hit the ground running. This new way of working will require new standard operating procedures (SOP), reallocation of resources, and team training – all things that take time to implement and complete.
Pharma companies and pMAHs can also spend this time anticipating the likely content of PICOs, so as to help speed up response times when the regulation comes into effect. Pharma companies and pMAHs already hold a wealth of data on their products and on what matters to patients. They can use this to understand the kind of questions they may need to address in the dossiers, and plan how they will access it.
It is, of course, impossible to predict the full content of all PICO requests, so, teams will also need to understand how to use their data to answer unforeseen questions quickly and accurately. For some organisations, this will be relatively straightforward. Others, however, will not have adequate in-house data science capabilities, and may opt to partner with data-specialist contract research organisations (CROs), which possess the skills and methodologies needed to quickly uncover and access the relevant insights, no matter how unexpected the request.
Ahead of the curve
Gradually, JCAs will become mandatory across all products, so, pharmaceutical companies and pMAHs have no choice but to overcome the challenges. However, that’s not to say we should only do it because we have to.
Embracing the new EU HTA framework will not will only open the door to more streamlined market access, it will also bring us a greater understanding of target patient populations, and help companies to uncover and tackle unmet medical need in future endeavours.
