Real-World Evidence
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Draft guidance from the European Medicines Agency explores how data quality can impact the use of real-world data to generate real-world evidence for regulatory assessments.
The incoming EU Health Technology Assessment Regulation will see historical data move “more center stage” for advanced therapies, because directly comparing highly individualized therapies is often unfeasible, an advanced therapies expert says.
As drug companies continue to explore the use of social media data to support their submissions, a panel of EU regulators have drawn up a seven-point action plan to help evaluate the usefulness and impact of such data in regulatory decision-making.
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.
While they could speed enrollment and lead to more representative samples, based on what FDA says local health care providers would be allowed to do as part of trial conduct, there may only be limited opportunities to integrate clinical studies into routine clinical practice.
Obeticholic acid has not confirmed clinical benefit and the benefit-risk profile is not favorable in primary biliary cholangitis, the FDA advisory committee said. The agency must now decide whether to keep Ocaliva on the market with new study requirements or seek withdrawal.
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.
New rules on health technology assessments in Spain make room for real-world evidence and early dialogue.
The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.
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.