It has been suggested that medical research is facing a “replication crisis.” This article shows that, by using simple mathematics, it is possible to predict that, using a significance level of 0.05, up to 35% of published clinical research findings may be false. One solution to reducing the high false positive rate is to raise the bar of significance from 5% to 0.5%. (castor)
Can Flatiron succeed in changing the clinical trial model?
Flatiron Health, a small tech company acquired by Roche, is trying to use real-world data from patient electronic medical records to replace more traditional clinical trial data. One of their major goals is to use data from health records to mimic a control group. However, FDA is not fully on board with the prospect of replacing control groups. (STAT)
New research ‘supergroup’ looks to reduce the number of required clinical trials
The MI-based CRO MMS Holdings is among the team at the Health Analytics Collective, a research group hoping to reduce the need for new clinical trials by using readily available data – and an MIT-incubated programming language. (outsourcing-pharma.com)
Real-world survival rates with medical device much worse than in trials
Survival rates in the post-approval study for the Abiomed Impella RP right ventricular assist device were dramatically worse relative to the device’s clinical trial data. While 73% of the device’s users survived at least 30 days in the premarket study, just 17% have met this primary survival endpoint since the device went on the market in 2017. (MEDPAGETODAY)
FDA publishes framework on real-world data (RWD) and real world evidence (RWE)
The U.S. FDA has published a new framework discussing how the agency will use real-world data (RWD) and real world evidence (RWE) to help companies win new indications for approved drugs and biologics, expand labeling, and satisfy post-approval study requirements. The impetus for the creation of this framework is the 21st Century Cures Act, which directed FDA to build an RWE program. (Regulatory Focus)