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Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera.

ABSTRACT: The first commercially approved human gene therapy in the Western world is Glybera (alipogene tiparvovec), which is an adenoassociated viral vector encoding the lipoprotein lipase gene. Glybera was recommended for marketing authorization by the European Medicines Agency in 2012. The European Medicines Agency had only ever reviewed three marketing authorization applications for gene therapy medicinal products. Unlike in the case of Glybera, the applications of the first two products, Cerepro and Contusugene Ladenovec Gendux/Advexin, both of which were for cancer diseases, were withdrawn. In this report, we studied the European public assessment reports of the three gene therapy products. During the assessment process, Glybera was re-examined and reviewed for a fourth time. We therefore researched the re-examination procedure of the European Union regulatory process. Approximately 25% of the new medicinal products initially given negative opinions from the Committee for Medicinal Products for Human Use were ultimately approved after re-examination from 2009 to 2013. The indications of most medicines were changed during the re-examination procedure, and the products were later approved with a mode of approval. These results suggested that the re-examination system in the European Union contributed to the approval of both several new drugs and the first gene therapy product.

SUBMITTER: Watanabe N

PROVIDER: S-EPMC4449026 | biostudies-other | 2015

REPOSITORIES: biostudies-other

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Project description:BackgroundSecond medical opinions have become commonplace and even mandatory in some health-care systems, as variations in diagnosis, treatment or prognosis may emerge among physicians.ObjectiveTo evaluate whether physicians' judgment is affected by another medical opinion given to a patient.DesignOrthopedic surgeons and neurologists filled out questionnaires presenting eight hypothetical clinical scenarios with suggested treatments. One group of physicians (in each specialty) was told what the other physician's opinion was (study group), and the other group was not told what it was (control group).ParticipantsA convenience sample of 332 physicians in Israel: 172 orthopedic surgeons (45.9% of their population) and 160 neurologists (64.0% of their population).MeasurementsScoring was by choice of less or more interventional treatment in the scenarios. We used ?(2) tests and repeated measures ANOVA to compare these scores between the two groups. We also fitted a cumulative ordinal regression to account for the dependence within each physician's responses.ResultsOrthopedic surgeons in the study group chose a more interventionist treatment when the other physician suggested an intervention than those in the control group [F (1, 170)?=4.6, p=0.03; OR=1.437, 95% CI 1.115-1.852]. Evaluating this effect separately in each scenario showed that in four out of the eight scenarios, they chose a more interventional treatment when the other physician suggested an intervention (scenario 1, p=0.039; scenario 2, p<0.001; scenario 3, p=0.033; scenario 6, p<0.001). These effects were insignificant among the neurologists [F (1,158)?=0.44, p=0.51; OR=1.087, 95% CI 0.811-1.458]. In both specialties there were no differences in responses by level of clinical experience [orthopedic surgeons: F (2, 166)?=0.752, p=0.473; neurologists: F (2,154)?=1.951, p=0.146].ConclusionsThe exploratory survey showed that in some cases physicians' judgments may be affected by other physicians' opinions, but unaffected in other cases. Weighing previous opinions may yield a more informed clinical decision, yet physicians may be unintentionally influenced by previous opinions. Second opinion has the potential to improve the clinical decision-making processes, and mechanisms are needed to reconcile discrepant opinions.

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Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera.

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