With the new rules for the JCA of medicines and the HTA Coordination Group’s guidelines on comparing treatments, the JCA process under EU HTA regulation is at a crucial stage. The WFH and other stakeholders are concerned that patients in Europe with few treatment options will face more delays and restrictions in accessing potentially life-changing medicines. This goes against the goal of the EU HTA and JCA, which aim to improve and speed up patient access to treatments.
The concern is that the JCA assessment method considers the data used to authorize most ATMPs to be too unreliable for clinical assessment. By suggesting that single-arm or non-randomized studies might be insufficient to estimate treatment effectiveness, the methodology seems to contradict the HTA Regulation, which leaves such judgments to member states and limits the JCA to describing, not evaluating, the certainty of treatment effects. While it is true that randomized controlled trials (RCTs) are the optimal approach and should be done whenever possible, for many ATMPs—and especially for rare diseases—RCTs are not practical or ethical. As medicine advances rapidly with innovative therapies like ATMPs offering hope to patients with urgent needs, it is essential that HTA methods evolve to embrace this scientific progress.
The call to action is as follows:
We urge the members of the HTA Coordination Group and its relevant subgroups, and JCA assessors to recognize all types of available evidence including single-arm trials and real-world evidence (RWE), and to use the JCA report to describe, rather than judge, any resulting uncertainty as to the treatments’ benefits, as called for by the HTA Regulation. A significant portion of outstanding uncertainty can be addressed at the national level during the appraisal phase and through the collection of RWE.
The WFH and the other stakeholders feel that this approach will help develop a JCA system that meets healthcare needs without hindering patient access to transformative therapies.
To read the full call to action, along with background information, click here.