Uncertainties in the Benefit-Risk Balance of Oncology Medicines
Author Information
Author(s): Taams A.C., Herberts C.A., Egberts A.C.G., Zafiropoulos N., Pignatti F., Bloem L.T.
Primary Institution: Utrecht University, Utrecht, The Netherlands
Hypothesis
What types and frequencies of uncertainties are described in the benefit-risk assessments of oncology medicines by the European Medicines Agency?
Conclusion
Most uncertainties about the benefit-risk balance of oncology medicines at initial marketing authorisation relate to safety, particularly specific adverse events and efficacy.
Supporting Evidence
- 121 oncology medicines were assessed, revealing a median of 6 uncertainties per medicine.
- 51% of uncertainties were related to safety, while 40% were related to efficacy.
- Cell and gene therapies had the highest number of uncertainties, averaging 8 per medicine.
- Uncertainties increased for medicines granted conditional marketing authorisation compared to standard authorisation.
Takeaway
When new cancer medicines are approved, there are often many questions about how safe and effective they are, especially for new types of treatments.
Methodology
A retrospective cohort study was conducted, including all oncology medicines with a new active substance authorized by the EMA from 2011 to 2022.
Potential Biases
Potential biases may arise from the reliance on EPARs rather than more detailed internal assessment reports.
Limitations
The study only focused on initial marketing authorisations for oncology medicines and did not measure the relative importance of different uncertainties.
Statistical Information
P-Value
0.001
Statistical Significance
p<0.05
Digital Object Identifier (DOI)
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