Real-World Evidence

Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.

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.  

Indegene’s CTO talks to Scrip about the state of GenAI implementations in pharma amid hype and limited clarity on ROI, the need to address foundational issues early on, promising pilots, the firm’s alliance with Microsoft and Google’s generalist LLM for therapeutics.

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.

Database review contradicts earlier findings with Novo Nordisk’s blockbuster incretin, but its small size and flawed design mean its commercial implications will be limited.    

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.

Together, Merck’s heads of oncology and head of digital health outline their cross-functional strategy to enhance care for cancer patients through digital tools.

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. 

The FDA has published final guidance to assist developers of medical devices designed to treat opioid use disorder, along with considerations for sponsors of clinical studies to evaluate those devices.

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.

Aspirin is celebrating its 125th anniversary this year. To find out what brand owner Bayer Consumer Health is doing to celebrate this occasion, and what it is doing to take the brand into the next 125 years, HBW Insight chats with the company’s senior vice president, Global Head Allergy, Cough/Cold, Pain and Heart Health, Reese Fitzpatrick, and head of R&D, North America and Therapeutics, Rachel Koontz.

Parexel’s chief strategy officer Kushal Gohil, Clinical EVP Stephen Pyke and India head Sanjay Vyas share thoughts on possible hurdles to, and the cascading impact of obesity treatments, GenAI progress and the opportunities and challenges of real-world data in India, in the second of this two-part interview with Scrip.

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. 

In this episode of HBW Insight's Over the Counter podcast, we chat with women’s health start-up The SABI about the power of herbal medicine for hormonal wellness.

Real-world data and evidence, for example generated by digital consumer health technology like apps and wearables, could provide crucial support for Rx-to-OTC switch applications, argues Sanofi Consumer Healthcare’s Penny Glover at the Association of the European Self-Care Industry's 60th Annual Meeting in Brussels, Belgium. Recent research by Sanofi indicates that regulators are actually already using RWD and RWE in many cases when assessing switch applications, even if they do not call it that.