OBJECTIVES: The use of surrogate endpoints (SEs) for cancer drug approval in health systems is common. The objectives of this study were to identify systematic reviews (SRs) that evaluated the correlation of SEs with overall survival (OS) in cancer drugs to analyze the applications of approved cancer drugs with SEs in Mexico and to apply the validation framework proposed by the Institute for Quality and Efficiency in Health Care (IQWiG).
METHODS: An overview of SRs was conducted according to Cochrane Collaboration methodology. Applications for approved cancer drugs with SEs in Mexico were analyzed. The IQWiG validation framework was applied to evaluate the SEs identified in the overview and in the applications in Mexico.
RESULTS: A total of 85 SRs that assessed 192 SEs for different types of cancer were selected. According to the IQWiG model, only 2.5% of the SEs analyzed in the overview and only one of the applications in Mexico could be used as surrogates for OS because the reliability (methodological quality) of the SRs and the strength of the correlation of SEs with OS was mostly low (92%) and low (correlation coefficient r ≤ 0.7; 50.5%), respectively. Of the total number of cancer drugs approved in Mexico, 19.4% used SEs.
CONCLUSIONS: Most SEs for different types of cancer could not be used as surrogates for OS according to the IQWiG model, and their use for the approval of cancer drugs in Mexico is generally not justified.
Síntesis amplia/ Revisión panorámica de revisiones sistemáticas
This overview summarizes evidence for the efficacy and safety of bortezomib, thalidomide, and lenalidomide in patients with multiple myeloma. We searched the Medline, Scopus, and LILACS databases through August 2016, including systematic reviews with meta-analyses of randomized controlled trials assessing the efficacy and/or safety of bortezomib, thalidomide, or lenalidomide in patients with multiple myeloma. Two authors performed study selection, data extraction, and quality assessment using AMSTAR and GRADE instruments. Twenty-nine studies satisfied the inclusion criteria. All three drugs significantly improved overall response and progression-free survival; however, only bortezomib showed significantly greater overall survival compared with the control arm (induction therapy, continuous therapy, or at any phase of treatment). The main concerns on adverse events were thrombosis/embolism events, peripheral neuropathy, and second primary malignancies. The most common problems detected in systematic reviews were non-registration of the study protocol and conflicts of interest not clearly acknowledged. Future research should adhere to quality assessment tools so that best evidence can be used in decision-making. Protocol PROSPERO registration number: CRD42016036062.
The use of surrogate endpoints (SEs) for cancer drug approval in health systems is common. The objectives of this study were to identify systematic reviews (SRs) that evaluated the correlation of SEs with overall survival (OS) in cancer drugs to analyze the applications of approved cancer drugs with SEs in Mexico and to apply the validation framework proposed by the Institute for Quality and Efficiency in Health Care (IQWiG).
METHODS:
An overview of SRs was conducted according to Cochrane Collaboration methodology. Applications for approved cancer drugs with SEs in Mexico were analyzed. The IQWiG validation framework was applied to evaluate the SEs identified in the overview and in the applications in Mexico.
RESULTS:
A total of 85 SRs that assessed 192 SEs for different types of cancer were selected. According to the IQWiG model, only 2.5% of the SEs analyzed in the overview and only one of the applications in Mexico could be used as surrogates for OS because the reliability (methodological quality) of the SRs and the strength of the correlation of SEs with OS was mostly low (92%) and low (correlation coefficient r ≤ 0.7; 50.5%), respectively. Of the total number of cancer drugs approved in Mexico, 19.4% used SEs.
CONCLUSIONS:
Most SEs for different types of cancer could not be used as surrogates for OS according to the IQWiG model, and their use for the approval of cancer drugs in Mexico is generally not justified.
