With the upcoming implementation of the EU Health Technology Assessment Regulation (HTAR) in January 2024, Europe is set to witness significant shifts in how health technologies, new treatments, are evaluated. While this regulation aims to harmonize the Joint Clinical Assessment (JCA) of Health Technologies and Joint Scientific Consultations (JSC) at the European level, its upcoming implementation from January 2025 for oncology and Advanced Therapy Medicinal Products (ATMPs) will be faced with substantial challenges.
Patient Participation
A major concern here is the issue of patient participation. Although the regulation formalises individual patient involvement, it stops short of requiring patient associations to be included as stakeholders.
In an interview with Pharmaceutiques France, CPE’s Policy Officer, Josephine Mosset, was direct in her assessment of the regulation’s shortcomings:
“The regulation as drafted only systematizes patient participation on an individual basis. Involving associations as stakeholders is only a possibility, not an imperative.”
Mosset highlighted the risk that additional procedural layers could delay access to treatments, particularly for patients in urgent need. The potential for such delays is a serious concern, as timely interventions are critical in many advanced therapies.
Adapting Methodologies
Another pressing challenge is how evidence, especially non-randomised evidence, will be handled within Joint Clinical Assessments. Concerns were raised by Cancer Patients Europe in its alongside 39 signatory organisations as the methodological guidelines for the JCA could undervalue non-randomised evidence, risking the dismissal of innovative and lifesaving treatments such as ATMPs.
European Commission’s Stance and Concerns
In response to CPE’s concerns, the European Commission provided reassurrance that non-randomised evidence would be considered within JCA. However, while this European Commission’s willingness to allow flexibility in the use of non-randomised evidence is viewed as a positive step, Mosset cautioned that there are still areas of concern around the acceptability of such evidence within JCA.
“We are encouraged by the openness of the European Commission, which is a constructive step towards more flexible assessments of advanced therapies. But we remain vigilant as to the risk of a rigid interpretation [of the methodological guidelines for JCA] that could impact assessments in some member states,” CPE’s Policy Officer states.
In addition, Cancer Patients Europe (CPE) has voiced strong criticism of the conflict-of-interest framework which could “exclude the most qualified patients from participating in the assessments.”
This issue remains a sticking point, as it risks undermining the effectiveness of patient input in critical evaluations.
Joint Scientific Consultations: A Key Pillar of the HTAR
Another key pillar of the HTAR is the Joint Scientific Consultations (JSC), which are intended to assist developers by providing free consultations to facilitate JCAs. These consultations often align with the European Medicines Agency’s (EMA) scientific advice, helping to streamline expectations and provide harmonised guidance. Patient involvement in these consultations is essential, particularly to ensure that quality-of-life criteria are systematically integrated into clinical trial protocols.
“We hope that the involvement of patients and their representatives in JSC will take greater account of their expectations, such as the systematic inclusion of criteria quality of life in the test protocols,” Mosset emphasised.
Despite these hopes, there remain unanswered questions about how exactly patients will be involved. Mosset expressed concern that if patients are brought into the process too late, their input could become “only symbolic,” rather than making a meaningful impact.
Conclusion
While the EU HTAR offers the promise of a more harmonised approach to health technology assessments across Europe, its success will depend heavily on how flexibly it is implemented. Both the adaptation of methodological guidelines and the genuine, meaningful inclusion of patients in these processes will be crucial to ensuring that the regulation delivers on its promise without introducing unnecessary delays or barriers to access.
