Proposed Methodologies For EU Joint Clinical Assessment Are Threat To ATMPs and Rare Disease Patients

Sixteen out of 18 EU-approved advanced therapy medicinal products would fail to pass an EU-wide joint clinical assessment because proposed methodologies are unsuitable for such products, according to the Alliance for Regenerative Medicine.

Gene therapy
Proposed Methodologies for new joint clinical assessments are not suitable for ATMPs • Source: Shutterstock

The proposed methodologies for conducting the EU-wide joint clinical assessments that will become applicable from 2025 under recent legislation on health technology assessments (HTAs) could be inappropriate for advanced therapy medicinal products (ATMPs) for rare diseases, warns the Alliance for Regenerative Medicines. The ARM claims that the Health Technology Assessment Coordination Group in charge of the assessments would have rejected some 90% of EU-approved ATMPS had the current proposals already been in place.

The legislation

EU-level joint clinical assessments (JCAs) are similar to relative efficacy assessments and are part of the HTA Regulation ((EU) 2021/2282) aimed at increasing cooperation on heath technology assessment across Europe and reducing duplication of work to improve access to medicines. The regulation was signed off in January 2022 but does not become applicable until January 2025, when advanced therapy products will become the first medicines to be subject to a JCA. (Also see "The Implications Of The New EU HTA Regulation For Companies" - Pink Sheet, 30 March, 2022.)

Companies will be paying close attention to how the JCAs take shape: although the outcomes are not binding, HTA or pricing and reimbursement bodies in the EU member states have to take them into account in their decision making

The EUnethHTA 21 Consortium has proposed methods for conducting the JCAs set out in a number of “deliverables,” These will influence the final methods to be selected by the HTA Coordination Group, which oversees the JCAs.

However, according to ARM, the proposals “are unable to capture the full added clinical benefit of ATMPs. "Sixteen out of 18 products approved for marketing in the EU, including all seven gene therapies so far authorized for rare genetic diseases such as Novartis’ Zolgensma (onasemnogene abeparvovec), would have been rejected, says ARM. This is because they were not studied as part of randomized control trials (RCTs) and/or because it was not possible to show durability of effect at the time of launch, according to the alliance, which represents small and large companies, academic research institutions, major medical centers and patient groups.

 

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