Exploring the Implications of Differing PICO Criteria for Oncology Drug Assessments in Europe
By AmerisourceBergen
A Review of Joint Action 3 and National HTA Requirements
The field of healthcare is constantly evolving, with regulatory frameworks playing a crucial role in ensuring patient access to safe and effective treatments. One such framework is the European Union’s Joint Clinical Assessment (JCA), which relies on the definition of consolidated population, intervention, comparators, and outcomes (PICO) criteria to guide assessments. However, as we discovered through our research presented at the ISPOR Europe 2023 conference, national differences in healthcare systems and treatment guidelines can significantly impact the application of PICO criteria in drug assessments.
Inspiration behind the Research
Our research was inspired by the need to examine how PICO criteria applied in Joint Action 3 (JA3) assessments differed from national health technology assessment (HTA) requirements to draw implications on the breadth of PICO criteria applied in the upcoming JCA process. We recognized that while the JCA aims to provide consolidated PICO requirements from all 27 European Union member states, variations across countries could have implications for drug assessments. To delve deeper into this issue, we focused our study on oncology drugs, given their upcoming evaluations starting January 2025.
Key Takeaways
Our research highlighted two essential takeaways. Firstly, national differences in healthcare systems and treatment guidelines may lead to extensive PICO criteria within the JCA process. This divergence creates challenges for manufacturers seeking market entry across multiple European countries. Secondly, additional data requirements from individual countries should be expected. These additional demands can further delay access to affected drugs within these nations.
Our hypothesis was confirmed through a comprehensive analysis of national HTA reports of three oncology drugs from Denmark, France, Germany, and Sweden. We found that these agencies often requested additional PICO evidence compared to what was required for JA3 assessments. For example, Germany exhibited more restrictive criteria for drug applications by requesting subgroup analyses. Denmark and Sweden included additional comparators beyond what was covered in JA3 reports for certain drugs. Additionally, France, Denmark, and Sweden considered evidence from studies other than randomized controlled trials (RCTs), such as observational studies or compassionate use data.
In addition to comparing PICOs between JA3 and national assessments, we explored whether countries utilized the JA3 report during their own evaluations. Surprisingly, we discovered that only Sweden had incorporated the report into one out of three drug assessments. This finding suggests that countries relied more heavily on their own national analyses rather than utilizing the consolidated JA3 report — even when they were involved in its evaluation process.
Our hypothesis was confirmed through a comprehensive analysis of national HTA reports of three oncology drugs from Denmark, France, Germany, and Sweden. We found that these agencies often requested additional PICO evidence compared to what was required for JA3 assessments. For example, Germany exhibited more restrictive criteria for drug applications by requesting subgroup analyses. Denmark and Sweden included additional comparators beyond what was covered in JA3 reports for certain drugs. Additionally, France, Denmark, and Sweden considered evidence from studies other than randomized controlled trials (RCTs), such as observational studies or compassionate use data.
In addition to comparing PICOs between JA3 and national assessments, we explored whether countries utilized the JA3 report during their own evaluations. Surprisingly, we discovered that only Sweden had incorporated the report into one out of three drug assessments. This finding suggests that countries relied more heavily on their own national analyses rather than utilizing the consolidated JA3 report — even when they were involved in its evaluation process.
What’s next?
Moving forward, it will be crucial to closely monitor the implementation of practical guidelines established by EUnetHTA 21 regarding PICO consolidation during the scoping process. By doing so, we can draw implications for biopharma companies and support their preparation for successful JCA processes and subsequent national HTA assessments.
Furthermore, as we approach 2025 when JCA processes commence fully, it becomes imperative to measure how final commissioning group secretaries manage key phases like scoping and PICO consolidation. Our teams at Cencora will continue monitoring this transition phase while offering our expertise to navigate uncertainties faced by manufacturers—particularly smaller biotech companies with limited product portfolios but significant stakes in these processes.
The discrepancies observed between JA3 and national HTA requirements emphasize the need for harmonization within European drug assessments. Consolidating PICO criteria across countries would streamline evaluations and facilitate timely access to innovative therapies for patients throughout Europe. As thought leaders committed to driving positive change within the pharmaceutical industry landscape, we will continue sharing our learnings while actively engaging with stakeholders such as EUCOPE (European Confederation of Pharmaceutical Entrepreneurs) and EFPIA (European Federation of Pharmaceutical Industries and Associations). Together with industry partners both big and small, we strive towards a future where broader learning perspectives help shape an effective JCA regulation that benefits patients across Europe.
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Authors: Mareike Konstanski, MSc; Jacob Gehrman, PhD; Jessica Schepis Martinez, MSc; Kim Joline Schmidt, MSc; Erika Wissinger, PhD; Herbert Altmann, PhD
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