Within a year, Europe will implement a uniform, pan-EU framework under the new Health Technology Assessment Regulation (HTAR). Concerns remain about stakeholders’ readiness and administrative complexities.

The HTAR introduces a Joint Clinical Assessment (JCA) across the continent, addressing disparities in evaluating the clinical value of pharmaceuticals and medical devices. This framework mandates that all 27 Member States adopt standardised procedures for evaluating the clinical evidence of newly submitted products or devices.

At DIA Europe 2024 in Brussels, where science, healthcare, patients, and regulatory professionals convened, Maya Matthews of the European Commission underscored the unprecedented nature of the endeavour: “We are all here to recognise that what we are doing has never been done before. So the unique nature is the fact that we are coming together to look at where can we act as one Europe.”

Matthews highlighted the challenges ahead while emphasizing the ultimate goal: creating evidence-based reports to assist Member States in expediting their reimbursement decisions.

Stakeholders at a crossroads

Looking ahead, the new joint procedure is scheduled to launch in Q1 2025 for oncology medicines and other advanced therapy medicinal products, with orphan drugs to follow in 2028. By 2030, JCAs will encompass all medicinal products with an EMA-issued Marketing Authorization (MA).

An audience poll at the event revealed that while many stakeholders have made significant strides, a considerable number remain unprepared.

“The next critical step is to gear up for the commencement in January 2025. Considering that the presubmission phase begins approximately 6-7 months earlier than the letter of intent from an EMA perspective for an initial marketing authorisation submitted post-January 12, 2025, it is essential to start preparing for this phase without delay,” stressed Michael Berntgen, Head of the Scientific Evidence Generation Department at the European Medicines Agency (EMA).

Inka Heikkinen of the EFPIA HTA Working Group echoes this urgency: “This is a huge effort, if you haven’t started, you need to begin now. Even if you are filing in 2028, the time to act is now.”

Berntgen reveals that currently, four products are undergoing this process, all within the field of oncology: “And we can already say there is an uptake, but there is space for more.”

He emphasizes the importance of identifying products within scope early on: “Take a close look at your portfolio, assess your scope, and identify relevant products promptly.”

Balancing perspectives

The draft act is currently open for feedback until April 2, 2024, with input being considered for finalising this critical initiative.

From an EFPIA perspective, Heikkinen emphasizes that this represents the first opportunity to comment. While there are several positive aspects, there are also significant concerns, particularly regarding the JCA procedure.

The JCA serves as a cornerstone of this regulation, aiming to establish a transparent and streamlined process. Its goal is to eliminate redundancies and expedite the delivery of life science innovations to patients across Europe, regardless of their location. Some attendees have praised it as a significant step towards a unified approach.

However, others have voiced concerns about potential duplication of efforts and administrative complexities.

National reimbursement decisions

It’s essential to recognise that while these assessments are informative, they do not predetermine national reimbursement decisions and are not legally binding. National HTA bodies retain autonomy in drawing their conclusions. They foster a nuanced approach to evaluating the clinical value of new medicines and may request further evidence during the decision-making process.

Isabelle Stoeckert, representing Bayer, raised an important question: “If we receive national questions, will additional assessment parameters or frameworks be introduced?” This inquiry underscores concerns about maintaining consistency and clarity across Europe’s evaluation landscape.

Timeline issue

Industry stakeholders find themselves at a crossroads. Their voices resonate in shaping forthcoming regulations, yet concerns persist. One critical issue looms large: timelines. Specifically, the 90-day window allocated for dossier preparation raises industry eyebrows.

Heikkinen encapsulated this challenge succinctly: “Ninety days won’t suffice for us. We need at least 135 days to craft a high-quality dossier.”

Bayer, too, raised its voice. “Ninety days to prepare a value dossier is not a lot,” asserted Stoeckert.

Patient perspective

Valentina Strammiello, representing the European Patients’ Forum, emphasized the need for an unwavering commitment from Member States in implementing the regulation: “… the main reason why we are doing it, [is to] avoid duplication of efforts, provide faster access to medicinal products and medical devices, make sure that also the quality of joint clinical assessment is as high as possible.”

Simplified access system for Europe

Advocacy for streamlining Europe’s access system gains momentum, with the goal of reorienting investments towards Europe and promoting patient-centred innovation.

Stoeckert adds, “It’s imperative that we collectively strive to simplify Europe’s access system, ensuring sufficient predictability to attract investments back to our region.”

Urgent action is needed to address concerns and ensure a smooth transition to the new framework. With careful consideration and collaboration, Europe can navigate these challenges and realise the potential of the HTAR to enhance healthcare accessibility and innovation in the region.

[By Nicole Verbeeck I Edited by Brian Maguire | Euractiv’s Advocacy Lab ]

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