Industry raises concerns on the feasibility of the joint EU HTA initiative

In a joint industry statement released in April 2024, several leading organizations in the life science sector voiced their concerns about the ‘workability’ of the draft implementing act for the new EU HTA procedure.

Recently released for public consultation, the draft implementing act on Joint Clinical Assessment (JCA) of medicinal products aims to establish detailed procedural steps and timelines for the new EU HTA procedure. The European Regulation on Health Technology Assessment (HTAR), Regulation (EU) 2021/2282 aims to create a transparent and inclusive framework for quality HTA across the 27 Member States of the European Union, reduce duplication for national HTA authorities and industry, and enhance business predictability, expediting access to medicines for EU patients.

The proposed framework’s viability has been a source of shared and serious concerns among various organizations, including the Alliance for Regenerative Medicines (ARM), the European Federation of Pharmaceutical Industries and Associations (EFPIA), the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), the European Association for Bioindustries (EuropaBio), and Vaccines Europe.

Despite the industry’s appreciation for the regulation’s aims, their joint statement underscores concern regarding the adequacy of upfront information and preparation time for companies engaging in JCAs. They caution,

“We believe that the draft rules in their current format will create an unworkable framework for JCAs and consequently lead to duplication of work.”

The association members of the HTA Stakeholder Network, representing the European life science sector, stress the need for sufficient interaction between companies and assessors throughout the process to ensure transparency and quality input. Without early engagement, especially for smaller companies with limited resources, there is a risk of inadequate submissions, compromising JCA effectiveness.

Member states are allotted 140 days to develop the assessment scope, comprising the questions (PICOs) for companies to address in their submission. Companies are unable to interact with assessors during this time and are not able to view the draft questions. Companies then receive the scope and have less than 90 days to respond with their submission dossier. The proposed timelines might necessitate companies to delay regulatory steps to meet JCA requirements. The ‘unrealistic’ deadline outlined in the implementing act has previously been highlighted as a cause for concern.

The joint statement raises concerns about the adequacy of upfront information and preparation time for companies participating in JCAs,

“If the JCA procedure, which ultimately is the measure of success of joint EU HTA does not provide transparency, predictability and workability for companies, joint EU HTA will fail to deliver on its aim of ensuring better access for patients to innovative health technologies.”

Proactive engagement to improve efficiency is stressed by the industry, which advocates for earlier company involvement in the scoping phase, similar to existing practices in many national HTA procedures and past EUnetHTA Joint Actions. This approach allows companies to prepare for many PICO scenarios in advance, which improves the quality of the submission dossier and, eventually, the JCA report. Additionally, companies confirming the intention to file for Marketing Authorization in advance allows HTA authorities to start planning ahead of time, instead of waiting until the EMA filing.

In light of these concerns, the industry has urged Member States’ representatives to consider proposed solutions in finalizing the implementing act on JCA of Medicinal Products. The goal is to ensure that the EU HTA procedure aligns with its intended aims, including the timely access to innovative health technologies for European patients.