Changing Regulation Around ATMPs in Europe

Starting in January 2025, companies looking to bring advanced therapy medicinal products (ATMPs) and oncology products to market in Europe will be required to adhere to the Joint Clinical Assessment process (JCA), as part of the EU’s new health technology assessment (HTA) regulation. The assessment aims to harmonize processes and evidence requirements for manufacturers through a single clinical test – intentions revealed by the European Commission in the recent JCA framework. However, questions remain over how the process should run, what companies need to do to prepare for it, and how ATMP developers can prioritize joint scientific consultations.

We spoke with Herbert Altmann, vice president, pan-european market access and healthcare consulting, and Lung-I Cheng, vice president and head of cell & gene therapy service line, both at Cencora (formerly AmerisourceBergen), to learn more.

What do ATMP developers need to understand about the EU’s new HTA regulation and, more specifically, the JCA process? 
 

Health technology assessments — which mandate formal documentation and evidentiary requirements — play a vital role in determining whether a product approved by the EMA is reimbursed and accessible in the market. Assessing the clinical benefit of a product is a critical part of the HTA process, but the criteria used to assess new pharmaceuticals varies widely across Europe. 

As part of the EU’s Regulation on Health Assessment Technology, the JCA wants to streamline the clinical assessment of new pharmaceuticals and medical devices across member states to reduce redundant practices and bring life science innovation faster to patients in Europe.  

The implementation of the JCA process will occur in phases, with the first phase commencing on January 12 2025. Initially focusing on oncology drugs and ATMPs, this new process will be mandatory. And, given the tight timeline, it is imperative for developers of ATMPs to grasp the intricacies of the regulation and its procedures. 

How early should ATMP developers start preparing for the new regulation and the JCA process? 
 

Developers should initiate their internal market access planning for JCA at least 10 months before expected filing with EMA. This timeline allows ample preparation for meeting JCA dossier requirements and developing high-quality package evidence. Ideally, alignment on internal processes should occur prior to designing phase III clinical trials.

Companies need to be familiar with the JCA and Joint Scientific Consultation (JSC) templates, evidence requirements, and timeline. As part of the planning process, developers should prioritize building cross-functional capacity and capabilities. While questions about local HTA processes remain, developers can leverage key learnings from the Joint Action 3 (JA3) pilot assessments to inform their strategy. They can also work with partners to conduct scenario testing, PICO simulation, and consolidation workshops to prepare for the new requirements.

In particular, we believe that the JSC serves as a great opportunity for companies to gain valuable scientific advice, which can help them develop the best possible evidence package for future HTA assessments — including pan-EU and the individual member states. By participating in a JSC, companies will receive advice from national HTA bodies and the EMA before they finalize their pivotal clinical trial designs.

Engaging with the national HTA bodies is particularly important for ATMP developers, many of which are emerging companies that may not have well-established local market teams.

How will the new HTA impact ATMP approvals?
 

Though it’s still an emerging market, the ATMP sector is entering a new era – with new modalities reaching the market and an influx of new therapies on the horizon, including those that target larger patient populations. There may be as many as six regulatory approvals of ATMPs in Europe in 2024!

The new regulation could optimize patient access to innovative therapies, but this vast undertaking also presents significant complexities to overcome and there remains skepticism and concern that the methodologies won’t be fit-for-purpose for ATMPs. The Alliance for Regenerative Medicine (ARM) recently issued a statement, warning the approach – specifically the need for randomised controlled trials – could result in inconclusive JCA reports for many ATMPs.

The lingering questions and concerns, coupled with the tight timeline, underscore the importance of proactive planning. Developers should involve cross-functional perspectives during strategy development and engage partners as early as possible. Through early planning and collaboration, developers can successfully navigate the requirements brought by the new regulation and unlock the potential of their product.