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Purpose <p>To analyze national-level evaluations conducted by selected Health Technology Assessment (HTA) bodies and compare them with the methodological framework outlined in the European HTA Regulation for Joint Clinical Assessment (JCA), in order to assess whether JCA would improve alignment among national practices.</p> Methods <p>We used onco-hematological medicines as a case model. From the European Medicine Agency (EMA) website, we identified onco-hematological medicines licensed from January 2023 to April 2025 and the pivotal trials supporting their marketing authorization (MA). We retrieved the reports of seven national HTA agencies and examined the rationale and evidence underlying the agencies’ evaluation and their final recommendations. To estimate how the JCA opinion would have changed compared to those taken by the national HTA authorities, we developed a set of possible Population, Intervention, Comparators, and Outcomes (PICOs) and applied JCA criteria to selected medicines.</p> Results <p>We assessed ten trials supporting the MA of eight medicines. All except momelotinib were approved based on one single pivotal trial. Six were single-arm studies. By June 2025, 22 national HTA reports were available for six medicines, incorporating pivotal trial data and indirect treatment comparisons for five medicines. Overall, recommendations from HTA agencies varied. The PICO scoping process generated at least four PICOs per medicine, reflecting high heterogeneity across countries. The limited evidence at the time of MA was insufficient to address all the PICOs.</p> Conclusion <p>Although JCA strengthens procedural harmonization, the current HTA fragmentation may persist due to the proliferation of PICOs and divergent interpretations of evidence.</p>

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Comparing National Health Technology Assessment approaches and the new European Joint Clinical Assessment framework: The case of onco-hematological medicines

  • 期刊论文

  • 发表日期：

    2026年6月29日

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  • Benedetta Starinieri,
  • Eleonora Allocati,
  • Chiara Gerardi,
  • Roberta Joppi,
  • Rita Banzi

摘要

Purpose

To analyze national-level evaluations conducted by selected Health Technology Assessment (HTA) bodies and compare them with the methodological framework outlined in the European HTA Regulation for Joint Clinical Assessment (JCA), in order to assess whether JCA would improve alignment among national practices.

Methods

We used onco-hematological medicines as a case model. From the European Medicine Agency (EMA) website, we identified onco-hematological medicines licensed from January 2023 to April 2025 and the pivotal trials supporting their marketing authorization (MA). We retrieved the reports of seven national HTA agencies and examined the rationale and evidence underlying the agencies’ evaluation and their final recommendations. To estimate how the JCA opinion would have changed compared to those taken by the national HTA authorities, we developed a set of possible Population, Intervention, Comparators, and Outcomes (PICOs) and applied JCA criteria to selected medicines.

Results

We assessed ten trials supporting the MA of eight medicines. All except momelotinib were approved based on one single pivotal trial. Six were single-arm studies. By June 2025, 22 national HTA reports were available for six medicines, incorporating pivotal trial data and indirect treatment comparisons for five medicines. Overall, recommendations from HTA agencies varied. The PICO scoping process generated at least four PICOs per medicine, reflecting high heterogeneity across countries. The limited evidence at the time of MA was insufficient to address all the PICOs.

Conclusion

Although JCA strengthens procedural harmonization, the current HTA fragmentation may persist due to the proliferation of PICOs and divergent interpretations of evidence.