Pharmaceutical industry bodies voice concern over limited advice opportunities under EU HTA Regulation
With the imminent implementation of the EU Health Technology Assessment Regulation (HTAR), pharmaceutical industry groups have raised serious concerns about the limited availability of joint scientific consultations (JSCs) scheduled for 2025. They warn that this shortage could hinder the rollout of joint clinical assessments (JCAs) and delay patient access to innovative treatments across the European Union.
While the pharmaceutical sector broadly supports the EU HTAR's goals of providing a transparent and inclusive framework for high-quality health technology assessment (HTA), reducing duplication across national HTA bodies, and accelerating patient access to medicines, there is significant apprehension about operational hurdles. In particular, the low number of planned JSCs—the HTA Coordination Group has only planned for 5-7 JSCs for medicinal products in 2025—threatens the regulation's success.
Industry groups such as the Alliance for Regenerative Medicines (ARM), the European Federation of Pharmaceutical Industries and Associations (EFPIA), the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), EuropaBio, and Vaccines Europe voiced their concerns in a joint statement released on December 4, 2024. "We are concerned that only a fraction of companies submitting for a JCA will be able to discuss their clinical trial plans at the critical planning stage," the statement notes. "If the EU HTAR is to successfully deliver on its aims of reducing burdens for companies and speeding up access for patients, it is critical to offer more JSC slots, starting in 2025."
Effective from January 12, 2025, EU HTAR mandates JCAs to evaluate the relative clinical effectiveness of new cancer therapies and advanced therapy medicinal products (ATMPs) approved by the European Medicines Agency (EMA). The HTA Coordination Group has scheduled only 5–7 JSCs for medicinal products in 2025, a figure that, the industry bodies note, does not align with the number of products in development. A survey from the JSC subgroup earlier this year revealed that companies identified over 50 products likely to require advice, indicating a high demand for JSCs. Furthermore, developers of orphan drugs—which become subject to JCA requirements in January 2028—will need guidance to prepare for the start of Phase 3 trials in 2025. Without adequate opportunities for early advice, misunderstandings may arise, increasing the risk of delayed assessments and impacting patient access to innovative treatments.
“The risk is too high that future JCA’s will either be delayed due to perceived evidence gaps or that national HTA procedures will be protracted. While the JCA is meant to speed up access decisions, Member States might have to request additional evidence or studies from companies after the JCA, which would cause avoidable delays in access for patients to new treatments.”
The statement highlights that small- to mid-sized companies face unique challenges, as they often have fewer opportunities to navigate the European market due to their smaller product pipelines and constrained resources. For these companies, engaging with assessors to discuss evidence generation plans is vital to ensure clinical trial designs adhere to required standards and that comprehensive quality dossiers are prepared.
The industry bodies argue that all companies requesting a JSC should have access to this advice. Currently, all products within the scope of the centralized EMA process are eligible for scientific advice on regulatory questions, and the same level of support should be extended to JCAs.
To address these concerns, stakeholders propose several solutions, including introducing a fee-paying system to provide sufficient resources for additional advice meetings and extending the current interim scientific advice model coordinated by the German HTA body G-BA.