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Selection of Endpoints in Clinical Trials: Trends in European Marketing Authorization Practice in Oncological
Anna Kordecka1, Ewa Walkiewicz-Żarek1, Joanna Łapa1
1HTA Registry, Krakow, Poland.
Objectives:
To determine the types of endpoints that were the basis for efficacy assessment of medicines used in particular groups of oncological indications. Changes in the endpoints applied in marketing authorization practice were also considered.
Methods:
The analysis included marketing authorization applications (MAAs) for medicines used in oncological indications that were first-time approved by the European Medicines Agency (EMA) between 2009 and 2017, and the extensions of the analyzed medicines.
Results:
The analysis covered 125 MAAs: first-time approved (62%) and extensions (38%). In the analyzed trials, the endpoints that were reported most frequently included overall survival (OS), progression-free survival (PFS), and overall response rate (in 94.4%, 92.8%, 87.2% of MAAs, respectively). The following trends were observed: decreased significance of OS as a primary endpoint and increased significance of PFS as a primary endpoint (hematological indications). An analysis of MAAs for which the OS results were immature confirms the increased significance of PFS and new efficacy indicators (ie, pathological complete response).
Conclusions:
An analysis of EMA's marketing authorization practice proves that the use of surrogate endpoints is becoming increasingly common in evaluating oncological health technologies. EMA's guidelines underline the role played by surrogates in the process of assessing efficacy of new therapies. Results of an analysis demonstrate that protocols of clinical trials define surrogates as primary endpoints more and more often. Furthermore, a positive decision on granting marketing authorization is possible also in situations when only such clinical data are available.
Insights
The European Medicines Agency is increasingly using surrogate endpoints for cancer drug approvals, with progression-free survival gaining importance over overall survival.
Area of Science:
- Oncology
- Clinical Pharmacology
- Regulatory Science
Background:
- Marketing authorization practice for oncological indications involves efficacy assessment.
- Endpoint selection is crucial for evaluating new cancer therapies.
Purpose of the Study:
- To identify efficacy endpoints used in oncological indications.
- To analyze changes in endpoints within marketing authorization practice.
Main Methods:
- Analysis of marketing authorization applications (MAAs) approved by the European Medicines Agency (EMA) between 2009 and 2017.
- Inclusion of first-time approvals and extensions for oncological medicines.
Main Results:
- Overall survival (OS), progression-free survival (PFS), and overall response rate were most frequent endpoints.
- Observed trend: decreased significance of OS, increased significance of PFS as primary endpoint, especially in hematological cancers.
- Increased use of PFS and pathological complete response as primary endpoints when OS data were immature.
Conclusions:
- The European Medicines Agency (EMA) increasingly utilizes surrogate endpoints for evaluating oncological health technologies.
- EMA guidelines support the role of surrogate endpoints in efficacy assessment.
- Clinical trial protocols increasingly define surrogate endpoints as primary, enabling marketing authorization with such data.
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