Real-World Evidence
The new CBER director, once best known in the pharma world for criticizing accelerated approval, committed to expediting cancer drugs with surrogate endpoints.
The UK regulator’s draft guideline on the use of external control arms based on real-world data reflects concepts similar to those outlined by the US Food and Drug Administration.
The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.
In light of the UK’s MHRA announcing a major overhaul of its clinical trial legislation, the agency’s deputy director for clinical investigations and trials Andrea Manfrin tells the Pink Sheet what sponsors can expect from the new regulation.
The European Health Data Space framework will allow companies to accelerate R&D processes and identify new molecular targets faster by facilitating centralized access to certain types of high-quality data, Finland’s Orion Pharma says.
Pharmaceutical companies need to “pressure test” their clinical development strategies early for health technology assessment purposes, particularly in light of the new EU HTA Regulation, a life sciences consultant says.
Newly published insights from a series of European Medicines Agency workshops can guide drug developers in designing development plans that meet the needs of both regulators and health technology assessment bodies.
Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.
Canada’s new guidance on rare disease registries is based on international guidelines, but in some areas there remains work to ensure that implementation of some recommendations is feasible in the Canadian context.
A retrospective analysis does not support a claim that multiple myeloma patients are more adherent to Hemady than generic dexamethasone, OPDP said in an "untitled" letter suggesting increased enforcement focus on promotions leveraging the agency’s 2018 CFL guidance.
Legal heavyweights from Nishith Desai Associates, Anand and Anand, Corporate Law Group and EY partner and national cyber leader talk about compliance requirements that pharma and global capability centers may need to consider as India strengthens the framework for the protection of digital personal data.
The Council of the EU has formally adopted the European Health Data Space regulation, meaning that it will soon enter into force in the EU. Experts explain what this means for the pharmaceutical industry.
The MHRA’s new scientific dialog program will offer drug developers “closed-door meetings” that will offer “confidential, commercially sensitive discussion” with its staff to help them “refine” their real-world evidence generation strategies.
Incorporating pragmatic and decentralized approaches into postapproval studies presents less risk for data collection and reliability than in the premarket setting, an expert panel said at a Friends of Cancer Research meeting.
The Center for Real-World Evidence Innovation will promote consistency across the FDA drug center’s many initiatives while addressing regulatory and scientific barriers to real-world evidence in regulatory decisions.
Clene’s ALS drug CNM-Au8 and UniQure’s gene therapy for Huntington’s disease emerged from US FDA discussions with accelerated approval plans using real-world evidence from compassionate use and natural history data sources.
AZ’s DAPA-MI trial showed the feasibility of R-RCT designs to support new indications in drug development and more established uses to inform medical practice.
RWE is already transforming the US FDA’s active surveillance systems and could catalyze change in pregnancy postmarketing studies, but is advancing more slowly for use as efficacy evidence.
As RWE advances, a multiplicity of roles are emerging, but case studies show how it is more effective filling evidence gaps during drug development and improving surveillance than creating a randomized controlled trial alternative.
Draft guidance from the European Medicines Agency explores how data quality can impact the use of real-world data to generate real-world evidence for regulatory assessments.