Real-World Evidence
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
Pharmaceutical companies should only use AI in evidence generation and reporting where there is “demonstrable value from doing so,” according to England’s health technology assessment body, NICE.
Results from the first pilot real-world evidence study commissioned by the European Medicines Agency to help HTA bodies and payers characterize patients with multiple myeloma have drawn positive feedback.
Overreliance on “problematic” trials while overlooking the value of real-world evidence could affect the development of other rare disease treatments, according to Advanz’s CEO.
The selection of study variables and effort required for validation depends on the necessary level of certainty and the impact of potential misclassification on study inference, the US FDA’s final guidance says, a change from the September 2021 draft.
A new UK network, led by an expert who has also worked on high-level EU data projects, has been established to test whether a common data model could help to facilitate real-world evidence studies by increasing the “usability” of data.
From the US FDA’s ISTAND Program to EU’s Melloddy Initiative, and from the global challenges of real-world data to the opportunities in India, Parexel’s EVP for Clinical Data and Digital Services, chief strategy officer and India head speak on a range of topics in this interview with the Pink Sheet.
CDER real world evidence policy head John Concato said the FDA could approve a new labeled use based only on real-world evidence, but made clear the approach is challenging.
Europe’s recently launched electronic RWD catalogs as of June contained 216 registered data sources and 2,840 studies distributed across various regions of the world.
The former head of the European Medicines Agency’s Data Analytics and Methods Task Force says sponsors should engage early with regulators to get feedback on the suitability of real-world data for a regulatory application. However, industry suggests developing a fitness-for-purpose framework with specific criteria to determine when RWD may be appropriate.
The European Commission has invested in several projects on novel real-world evidence methodologies, either via its key funding program for research and innovation or via public-private partnership initiatives.
Insights from Day Four of the BIO International Convention include FDA commissioner Califf on the state of real-world evidence and how it impacts IRA negotiations, Roche's interest in cardiovascular/metabolic assets, Merck's plans in immunology, and industry's rising interest in neuropsychiatry.
The European Medicines Agency has encouraged companies to seek “guidance and direction” early in the development process after it reported a significant fall in the number of companies that sought scientific advice and protocol assistance compared with the three previous years.
Japan has been promoting a revised law that will allow the broader use of real world data by allowing the use of both pseudonymised and anonymised data.
The Center for Clinical Trial Innovation was created to be CDER’s hub for expertise and information on innovative clinical trial approaches. C3TI lead Kevin Bugin talks to the Pink Sheet about the program's genesis and what it hopes to accomplish.
A real-world data research partnership involving Genomics England is helping pharma companies to continuously draw “more insights” from genomic information to improve their regulatory submissions and optimize trials.
A study from the health technology assessment department of France’s national health agency, HAS, has identified which pharmaceutical products are most likely to receive a request for post-market studies. The agency hopes this information will aid in companies’ forward planning.
The European Medicines Agency has developed draft recommendations on the key methodological factors to consider when conducting and assessing observational studies with real-world data for regulatory decision-making throughout a medicine's lifecycle.
Real-world data collected from a wide range of sources, such as wearable devices and public databases, can be used by pharmaceutical companies to boost their marketing authorization and health technology assessment applications, a regulatory policy expert from Sanofi says.
Congratulations to the winners of the 2024 Citeline Awards, held 8 May in Boston.