EU Joint HTA: What’s going on and what does it mean for industry?

EUnetHTA and the EU Joint HTA Process

Originally set up in 2006, EUnetHTA was established to facilitate information sharing between (HTA) agencies across the European Union (EU). Since its inception, there has been a continued push for closer ties between the EU HTA agencies and today, the EUnetHTA21 workstream aims to establish an EU-wide joint assessment of clinical effectiveness, with an accompanying process for joint early scientific dialogue with EU HTA agencies.¹

The Big Changes

Currently, manufacturers launching a drug in the EU will seek central marketing authorisation from the EMA, but then proceed to seek reimbursement at an individual country level. This means developing and submitting different dossiers for every EU country with an HTA process where they would like to launch (Figure 1a). In some cases, multiple submissions may be needed for just one market – for example markets such as Greece, where price is determined separately, and prior to, the reimbursement dossier. This often involves huge amounts of commercial planning, including determining where to launch, and when.

With the introduction of a joint HTA process, following EMA marketing authorisation, it is hoped that a single clinical dossier will be sufficient for launch across the EU. Further dossiers may be needed for economic or pricing evaluation in some markets. (Figure 1b). If successful, this could reduce the volume of submissions needed for launching across Europe.

The purpose of introducing a joint HTA process is, therefore, two-fold²:

• Reduce duplication of work across multiple EU HTA agencies
• Speed up patient access to medicines

The goal is for all products newly approved by the European Medicines Agency (EMA), or products with newly licensed indications, to undergo a joint HTA evaluation for all EU countries. The joint evaluation will focus on clinical data only, with economic assessment left to the nation states.²

Timelines for implementation

The first use of joint dossiers is scheduled for 2025 and will include oncology medicines and Advanced Therapy Medicinal Products (ATMPs) before expanding to include orphan drugs in 2028 and finally all EMA centrally authorised medicinal products in 2030. Figure 2 details the planned implementation process.

In preparation for launch in 2025, several public consultations are scheduled or in progress, including one public consultation on the draft submission dossier template and another on the proposed assessment report template. Both deliverables are due to be published in November this year. A further consultation on the briefing book template for Joint Scientific Consultations will take place in 2023.³

How have the proposed changes been received by industry?

Industry opinion on the joint process is mixed, with some optimism about the upcoming changes but also some concern that the new process will not achieve its aims.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) has stated that they ‘strongly support’ a system that can pool the resources and expertise of HTA agencies across the EU.⁴ Similarly, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) have shown support for a new EU HTA Procedure that can address the issues of ‘divergent’ HTA process and methodologies and the subsequent duplication of work.⁵

However, if the joint reports do not align with the value drivers of HTA agencies in clinically driven markets, it is possible further clinical documentation will be requested to fulfil the extra requirements. HTA agencies in health-economic driven markets will also require additional submissions to cover budget-impact or cost-effectiveness analyses, which may in turn require further analysis of the clinical data presented in the joint report. If this is the case, the issue of duplication will not be addressed and could even become worse.

EUCOPE’s current position on the process is that it falls short of addressing access delays caused by the current divergent pricing and reimbursement process across the EU, although it praises the work done so far for establishing a framework that could increase ‘harmonisation’ if developed further.⁶ Similarly, EFPIA have stated their regret that European Parliament and Council negotiators have yet to ‘agree on a predictable, efficient and non-duplicative system’ calling it a ‘missed opportunity’.⁴

Both EUCOPE and EFPIA have praised the opportunity for companies to seek joint scientific advice,^4,6 building upon the existing system where parallel advice can be sought from the EMA and different HTA agencies.⁷ This is hoped to be another avenue where duplication of work, and unnecessary burden on manufacturers, can be avoided. The advice should be particularly helpful for the manufacturers of ATMPs and oncology products, who will be among the first to make joint submissions.

Unfortunately, this benefit may be limited. In May 2022, EUCOPE Secretary-General Dr Alexander Natz expressed his concern that slots for the joint advice will be limited, meaning some companies may miss out.⁶

What does the future hold?

As the process for achieving reimbursement in Europe changes, so might key stakeholders and their respective value drivers. Current EU launch strategy is segmented across market and payer archetypes, with some markets driven by clinical data standards and others by various health economic thresholds. This, of course, also feeds into pricing strategy and the balance between value-based and cost-based pricing. Even with the consolidation of HTA evaluation, it is unlikely we will see European payers become a homogenous group with identical value drivers and priorities, but it is possible we will see a shift in payer value drivers in at least some markets.

As development of the joint dossier will be mandated following centralised authorisation, the choice of where to launch, and when, may no longer be in the hands of manufacturers. This could take away some of the commercial flexibility currently available when developing the strategy for a successful European launch. On the other hand, this could mean patients in some markets getting access, and sooner, to certain medicines than they may otherwise get. This will align with the EFPIA commitment of March 2022, to file for pricing and reimbursement in all EU countries as soon as possible, and no later than two years from central authorisation, where local procedure allows.⁸

In conclusion

So, what does this all mean for industry, and for manufacturers planning to launch in the EU following the introduction of the new process? The short answer is that there is no answer. Yet. There is widespread support for the aims of EUnetHTA21, in particular the desire to reduce duplication of work and speed up patient access to medicines. However, concerns remain over whether these goals will be met, with the failure to make the joint assessment reports fully binding the main concern.

The current lack of clarity is concerning for those companies expected to make the first submissions for the joint process, in just three years’ time. ATMP and cancer medicines have been prioritised to be assessed first under the new process due to their importance to patient health and the issues seen previously in patient access to such products. Given their importance, manufacturers of these products should not be used as unwilling guinea-pigs, particularly if they are unable to access early scientific advice and risk misalignment between their submitted dossier and what is expected from the assessors. Further clarity on the content of the dossiers is anticipated by the end of the year, however, how the process plays out at a national level may only become apparent after the first submissions have been made.

Sources:

1. About EUnetHTA21. EUnetHTA. https://www.eunethta.eu/about-eunethta/. Accessed 20^th May 2022.
2. What are the 5 Pricing & Market Access trends that will shape 2022? Remap Consulting. https://remapconsulting.com/what-are-the-5-pricing-market-access-trends-that-will-shape-2022/ Accessed 20^th May 2022.
3. Joint HTA Work. EUNetHTA. https://www.eunethta.eu/jointhtawork/ Accessed 25^th May 2022.
4. EU HTA; Compromise but at what cost? EFPIA. https://www.efpia.eu/news-events/the-efpia-view/statements-press-releases/eu-hta-compromise-but-at-what-cost/ Accessed 20^th May 2022.
5. Five Priorities for a Successful EU HTA Procedure. EUCOPE. https://blog.eucope.org/2022/five-priorities-for-a-successful-eu-hta-procedure/ Accessed 20^th May 2022.
6. Natz, A. New EU HTA Procedure -Will it Reduce the Complexity and Burdens for Manufacturers? Pharma Boardroom. 2022.
7. Parellel Joint Scientific Consultantion with Regulators and Health Technoogy Assessment Bodies. EMA. https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance/parallel-joint-scientific-consultation-regulators-health-technology-assessment-bodies. Accessed 20^th May 2022.
8. Addressing Patient Access Inequalities in Europe. The Industry commitment to file pricing and reimbursement applications across Europe and the European Access Portal. EFPIA. Brussels, Belgium. March 2022.