Sharpless Says Innovative Trial Designs Are FDA Priority
Innovative trial designs are a high priority in FDA plans, acting FDA Commissioner Ned Sharpless said in an agency-wide internal email sent Tuesday.
Sharpless told staffers the agency will continue to push for the use of platform trials, basket studies, adaptive trials and pragmatic randomized controlled trials that will save money and time on research and development. It also plans to promote the use of real-world evidence (RWE) in both pre- and postmarket adverse event detection and efficacy analyses in clinical situations that may not be able to use randomized trials.
“Enhanced use of RWE for regulatory decisionmaking will improve the generalizability of evidence to patient populations that may be excluded from traditional clinical trials,” he added.
The agency will continue to use novel approval pathways for drugs, biologics and devices — including RMAT, Accelerated Approval and Breakthrough — as they can make product development more efficient, Sharpless said.
Big Pharma Scores Well Overall on Data Transparency
Roche/Genentech and Novo Nordisk tied for top honors for data transparency on Bioethics International’s latest Good Pharma Scorecard.
The two companies both scored 100 percent on the nonprofit’s annual evaluation of big pharma’s track record on trial registration, results reporting, publication and data-sharing practices. Novartis was close behind at 99 percent.
The median score of the 12 companies evaluated – based on their sharing of data on 2015 FDA-approved drugs – was a respectable 92 percent, according to the study, but three companies fell below the 70 percent mark: Valeant at 63 percent, Amgen at 56 percent and Allergan, soon to be absorbed by AbbVie, at the bottom of the list with a poor transparency score of 46 percent.
Read the full report here: https://bit.ly/2OjcaXc.
EMA Studies Patient Registries’ Use in Rare Disease Trials and Postmarket Surveillance
Patient registries could provide valuable data for regulatory decisions, a new EMA drug safety study indicates.
Registries can be used when randomized controlled trials are not feasible, such as in the case of some rare diseases, or to meet postmarketing commitments.
The study further explains how registries are currently underused and explained how the absence or incompleteness of ten different factors impeded registry use. Factors include the use of common core data sets, common data coding terminologies, data access and sharing and data linkage capacity, among others.
“From a regulatory perspective, the ultimate requirement of patient registries is that they permit the conduct of high quality studies that evaluate the safety and effectiveness of medicines,” EMA says.
The agency plans to prepare a preliminary discussion paper and solicit public comments before issuing methodological and operational guidance on handling registry data in postmarket studies.
Industry, Academia Form Consortium to Develop Neurobehavioral Clinical Tools
Several big pharma companies have joined with European universities and an AI technology company to advance the development of clinical tools that measure neurobehavioral tasks.
Boehringer Ingelheim, Janssen, Lundbeck and Roche are participating in the Reward Task Optimization Consortium (RTOC), along with central nervous system experts from medical and mental health institutions in the Netherlands, Germany, Spain and Greece. BlackThorn Therapeutics, a clinical-stage neurobehavioral health company, will provide next-generation AI solutions to help build predictive models.
RTOC, which will be led by central nervous system research company P1vital, will develop three computerized tools that measure brain activity in trial participants and then test the tools in trials for schizophrenia and major depression therapies. RTOC expects to start recruiting participants in Europe in the third quarter of 2019.
“Invisible” Device Uses Radio Signals to Analyze Movement and Collect Patient Data
Novartis and MIT are collaborating on development of a wireless device that can measure physical activity in patients without requiring them to use wearable sensors or monitors.
The Emerald device analyzes surrounding radio signals, using machine learning algorithms to detect the pose and movement of a person, even through walls and in low light. It extracts various physiological metrics related to breathing, sleep stages, sleep apnea, mobility and gait, among others
The “invisible” device provides several benefits over traditional patient data collection methods, including improved biomarker development and the ability to detect changes in behavior. And it allows patients to go about their normal routines without any added burden, which provides a unique opportunity to support at-home clinical trials.