PIVOTAL STUDIES OF ONCOLOGY DRUGS APPROVED IN EUROPE, WHICH EVIDENCE BASED-MEDICINE?
Author(s)
Aissaoui A1, Turkistani F2, Bin Sawad A2, Aissaoui N3
1Paris Dauphine University, PSL, Paris, France, 2MCPHS University, Boston, MA, USA, 3Paris Dauphine University, paris, France
OBJECTIVES: To explore the pivot studies achieved for solid tumors treatment oncology drugs (STTODs) marketed in Europe. METHODS: All STTODs approved by the European Medicines Agency (EMA) during the period from 2000 to 2014 was identified. The Summary of Product Characteristics (SmPC) prepared to the regulatory agency was reviewed. Phase 3 clinical trials for STTODs were divided into: best supportive studies (STTOD compared to standard of care) and placebo studies (STTOD compared to placebo). Both types of phase 3 clinical trials (best supportive studies and placebo studies) were further categorized depend on their results into: no patients’ improvement, patients’ improvement (no statistical significant), and patients’ improvement (statistical significant). RESULTS: There were total of 38 oncology drugs approved by EMA during the period from 2000 to 2014. The total number of phase 3 clinical trials was 73 (50 best supportive studies and 23 placebo studies). Eighty-six percent of the best supportive studies were open labeled randomized controlled trials (43 studies). Out of 50 best supportive studies, 32% of the studies show no patients’ improvement result, 30% of the studies show patients’ improvement but without statistically significant results, and 38% of the studies show patients’ improvement with statistically significant results. On the other hand, 95.7 % placebo studies were blinded randomized controlled trials (22 studies). Out of 23 placebo studies, 21.8% studies show no patients’ improvement results, 52.1% of the studies show patients’ improvement but without statistically significant results, and 26.1% of the studies show patients’ improvement with statistically significant results. CONCLUSIONS: A shift has occurred over the past 10 years in the design of phase 3 trials in solid tumor. Comparing against active comparator in open labeled randomized controlled trials is increasing, but the result of patients’ improvement with statistically significant is decreasing.
Conference/Value in Health Info
2016-10, ISPOR Europe 2016, Vienna, Austria
Value in Health, Vol. 19, No. 7 (November 2016)
Code
PCN48
Topic
Clinical Outcomes
Topic Subcategory
Relating Intermediate to Long-term Outcomes
Disease
Oncology