Assessment of Food and Drug Administration- and European Medicines Agency-Approved Systemic Oncology Therapies and Clinically Meaningful Improvements in Quality of Life: A Systematic Review
- Recent oncology therapies approved by the FDA and EMA often lack evidence of clinically meaningful improvements in quality of life (QOL).
- Only 40% of FDA-approved and 58% of EMA-approved oncology therapies had published QOL evidence.
- Clinically meaningful QOL improvements beyond minimal differences were noted in just 6% of FDA and 11% of EMA indications.
- Regulatory approvals may rely on survival measures or anti-tumor activities rather than QOL evidence, which is crucial for patients undergoing palliative treatment.
- The presence of QOL evidence has increased over time for EMA-approved therapies but not for FDA-approved ones.
- The study highlights a gap between therapeutic approval and evidence of QOL benefits, emphasizing the need for more comprehensive evaluation in oncology drug approvals.
For patients with cancer treated with palliative intent, quality of life (QOL) is a critical aspect of treatment decision-making, alongside survival. In some cases, patients with advanced cancer have even prioritized QOL over survival. However, submission of QOL data are not mandatory for oncology drugs seeking regulatory approval by the US Food and Drug Administration (FDA) or European Medicines Agency (EMA). Thus, despite the value of QOL as a constituent of clinical benefit, it often does not appear to be a considerable factor in drug approval.
Clinical trials with the aim of drug registration are designed with primary objectives focused on demonstrating traditional components of clinical benefit, such as overall survival (OS). As such, trials are therefore powered to capture the statistically significant differences in these traditional end points, often without consideration of QOL evidence. However, regulatory approval is frequently based on surrogate outcomes for survival or clinical effectiveness, such as progression-free survival (PFS) or response rate. While such methods may serve to expedite drug approval, a drug’s final efficacy (or the lack thereof) may not be apparent at the time of market authorization. Given an absence of clinically meaningful survival gains, any increase in length of life may not be associated with comparable increases in QOL....
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