Real-World Evidence

The US FDA’s Office of Women’s Health provides a research roadmap to address health concerns specific to women. The FDA recently updated the roadmap, outlining areas in which further research is needed.

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.

The US FDA’s Office of Women’s Health provides a research roadmap to address health concerns specific to women. The FDA recently updated the roadmap, outlining areas in which further research is needed.

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The US FDA has issued a draft guidance document providing recommendations on how device firms can collect patient preference data to share with the agency as it reviews applications. The current draft supersedes final guidance the agency published in 2016.

Boston Consulting Group and senior pharma industry executives take a broad look at the challenges and opportunities for medical affairs evidence generation with multiple modalities.

The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked.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.