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Background <p>In oncology, health-related quality of life (HRQoL) is along with overall survival a decisive clinical element in regulatory and clinical decision-making. Consequently, health authorities are increasingly interested in HRQoL data when evaluating new cancer treatments, as it promises to better capture patient-relevant outcomes. Although strongly encouraged by regulatory agencies such as the FDA, EMA and Swissmedic, the inclusion of HRQoL data in regulatory submissions by pharmaceutical companies remains sparse due to variability in data quality, reporting standards, methodological rigor, and operational constraints.</p> Methods <p>We retrospectively analysed 342 Swissmedic-approved oncology applications (2001–2020) to assess the volume, role, and trends of HRQoL data. For each application, we recorded HRQoL inclusion, domains based on the WHO framework, measurement instruments, and differences across document types and application categories.</p> Results <p>HRQoL data appeared in 216 of 342 reports (63.2%). Inclusion was higher for new active substances (69.4%) than indication extensions (59.6%). Physical health dominated (87.1% of applications), while psychological (20.2%), social (1.8%), and environmental (6.7%) domains were rarely reported. Of 216 applications including HRQoL, only 62 (28.7%) suggested a benefit, 8 (3.7%) demonstrated a clinically meaningful improvement, and just 2 (0.9%) were reflected in product labels.</p> Conclusions <p>Although HRQoL reporting in Swiss oncology submissions is increasing, it is still mostly limited to physical health. Our findings suggest underreporting of more universal HRQoL domains and limited regulatory utility mostly due to incomplete information and underuse of HRQoL integration in the statistical analysis plan by pharmaceutical companies, issues that align with challenges documented across international agencies.</p>

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Quality of life in oncology drug approvals: evidence from Swiss decisions in relation to global regulatory trends

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年2月12日

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  • Alexander Horst,
  • Nicolas Löffler-Perez,
  • Ulrich-Peter Rohr,
  • Katarzyna Wac

摘要

Background

In oncology, health-related quality of life (HRQoL) is along with overall survival a decisive clinical element in regulatory and clinical decision-making. Consequently, health authorities are increasingly interested in HRQoL data when evaluating new cancer treatments, as it promises to better capture patient-relevant outcomes. Although strongly encouraged by regulatory agencies such as the FDA, EMA and Swissmedic, the inclusion of HRQoL data in regulatory submissions by pharmaceutical companies remains sparse due to variability in data quality, reporting standards, methodological rigor, and operational constraints.

Methods

We retrospectively analysed 342 Swissmedic-approved oncology applications (2001–2020) to assess the volume, role, and trends of HRQoL data. For each application, we recorded HRQoL inclusion, domains based on the WHO framework, measurement instruments, and differences across document types and application categories.

Results

HRQoL data appeared in 216 of 342 reports (63.2%). Inclusion was higher for new active substances (69.4%) than indication extensions (59.6%). Physical health dominated (87.1% of applications), while psychological (20.2%), social (1.8%), and environmental (6.7%) domains were rarely reported. Of 216 applications including HRQoL, only 62 (28.7%) suggested a benefit, 8 (3.7%) demonstrated a clinically meaningful improvement, and just 2 (0.9%) were reflected in product labels.

Conclusions

Although HRQoL reporting in Swiss oncology submissions is increasing, it is still mostly limited to physical health. Our findings suggest underreporting of more universal HRQoL domains and limited regulatory utility mostly due to incomplete information and underuse of HRQoL integration in the statistical analysis plan by pharmaceutical companies, issues that align with challenges documented across international agencies.