Real-world evidence provides external controls for bluebird bio’s eli-cel and Ipsen’s palovarotene, while Reata used RWE to address FDA concerns about the pivotal single-arm study of omaveloxolone. In the post-market, Intercept is gearing up to seek full approval of Ocaliva relying in part on claims database.
Three candidates for US FDA approval are using real-world evidence as part of the efficacy argument, illustrating some of the most common ways to RWE in applications.
In keeping with FDA’s history of approvals incorporating RWE of efficacy, all of the applications target rare diseases: Friedreich’s ataxia...
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.
Companies dealing with commercial costings or contracts with the National Health Service for early phase clinical trials or trials of advanced therapy medicinal products should soon be able to conclude their negotiations more quickly.
The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.
As the planet heats up and sea levels rise, the need to reduce carbon emissions is becoming ever more urgent. The life sciences industry is just one of many that are looking to cut the carbon it produces.
