What is the Joint European HTA?

Pricing and reimbursement in the European Union (EU) can be daunting for emerging biopharmaceutical companies (EBPs; i.e. companies with less than $200 million in estimated annual spending on R&D or less than $500 million in global revenue). There are 27 countries each with their own requirements for applications, languages, dossiers, and processes. Meeting the needs of all these stakeholders in the hectic period around launch can be a significant challenge.

In the white paper The Future of EU HTA, IQVIA experts describe the development of an EU-wide health technology assessment (HTA) for new drugs. The EU HTA will be implemented in 2025 and aims to harmonize HTA processes and requirements across Europe to reduce duplication of effort and accelerate patient access to treatments. The EU HTA also provides the chance to seek early engagement and guidance on pivotal trial design. If the implementation of the EU HTA is successful, then accelerated access is a key advantage; however, the shift to this new process is likely to result in challenges for industry. The white paper asks if these changes constitute an opportunity or a threat for biopharmaceutical companies, and what actions should companies take to benefit from EU HTA? This blog considers these questions from the EBP perspective.

Will the new EU HTA process impact EBPs differently?

The new EU HTA process may have different or greater impacts on EBPs compared to larger companies for several reasons:

Therapeutic focus: The EU HTA initially applies to oncology drugs and advanced therapy medical products (ATMPs). EBPs are often engaged in developing these technologies and will be among the first to go through the new process, so many early reviews will be for EBP products. EBPs need to follow the guidance developed by EUnetHTA 21, an EU-wide initiative on HTA harmonization, to prepare for the new process. However, this guidance uses EUnetHTA 21 methods which have not yet been tested on ATMPs and which are unlikely to be tested on them by 2025. Assessment of ATMPs at the national level is still challenging and HTA recommendations in Europe often do not agree on their therapeutic value (Gozzo et al., 2021).

Evidence demand: A key part of the EU HTA process is understanding the clinical requirements of all 27 member states. Addressing all of these will require more evidence and more indirect comparisons than a single-country HTA. Generating the required evidence in small patient populations (e.g. for rare diseases) may create additional uncertainties, which could be particularly challenging for EBPs.

Company resources: It is estimated that EBPs have 30% slower launch times for new products than larger companies (IQVIA, Emerging Biopharma’s Contribution to Innovation). This may be partly due to EBPs having fewer resources to support commercial activities. This discrepancy may be exacerbated by the new EU HTA process. As it is currently envisioned, EU HTA will involve almost simultaneous regulatory and HTA/HEOR processes while still requiring local market access activities. EBPs do not necessarily have in-house teams of access experts or deep pockets to fund non-core activities, so may lack the resources to conduct all these activities concurrently. In addition, EU HTA requires deep knowledge of local requirements and evidence needs but EBPs may not have the resources in all the markets of interest.

What are the opportunities and challenges of the EU HTA for EBPs?

The EU HTA offers various opportunities to EBPs:

• Market access may be accelerated for EBP products, especially in small and mid-size EU markets which are not currently prioritized, resulting in earlier access for patients.
• Joint scientific advice may de-risk EU access for EBPs by providing structured and early guidance on the evidence requirements needed to achieve successful access.
• Engaging with joint scientific advice may also improve long-term success by increasing the importance of advance planning, which may avoid later failures that have widespread consequences.
• In the long term, streamlined and accelerated pan-EU HTA will enhance the confidence of EBPs from the US and other countries in tackling EU access without the need to partner with other companies in Europe.

The EU HTA also brings a number of potential challenges for EBPs:

• EBPs will need to dramatically increase their understanding and capacity for evidence synthesis to submit their EU HTA documentation (particularly their surge capacity at the point of both regulatory and EU HTA dossier submission).
• Uncertainty remains about the practical implementation of EU HTA (IQVIA, The Future of EU HTA). It is reasonable to expect launch problems as the new process is introduced, including the impact of EU HTA on local HTA timelines and operations. Disruption or delay to launch is a potential risk during this time, regardless of whether an asset is initially in or out of scope of the EU HTA.
• The number of available slots for joint scientific advice is expected to be limited and the prioritization process for this advice remains unclear, meaning that EBPs’ pivotal trial development may be impacted.
• As EBPs mature beyond their initial planning for near-term EU HTA adaptation, the upstream strategic considerations for EU HTA success need to be considered, as well as how to align  these with their market access strategy (IQVIA, EU HTA: Business as usual for market access strategy or time for change?).

What actions should EBPs take now?

Know where you stand: An implementation timeline for EU HTA is now available. EBPs should evaluate which of their assets will be subject to the new process in 2025 and determine how the timelines align with their development plans and evidence generation activities. Companies should understand the competitive landscape for all assets (in and out of scope of EU HTA) and how it may affect planning processes and launch strategy.

Be informed: Changes to the EU HTA may occur before and during implementation. EBPs should assess the details of the new process to clearly identify priority actions and stay informed of updates to avoid surprises. Where possible, companies should get involved in local and regional pharmaceutical association discussions.

Be prepared: Companies that engage with scientific advice where available and deliver the right evidence are more likely to benefit from the EU HTA. Successful engagement with scientific advice will require earlier investment in HTA planning; this cost will be small compared to the potential gains from earlier access for patients.

Partnering for success

As EBPs are at the forefront of developing breakthrough therapies and will be impacted differently than larger companies, the help of a trusted partner is key to navigating the dynamic EU HTA landscape and its associated uncertainties. IQVIA can support critical elements of planning and development, including evidence generation, market access strategy and optimizing pricing and reimbursement decisions. If you would like to learn more about managing the potential implications of EU HTA implementation, contact us today!