CER Daily Newsfeed

The Growing Role of Real-World Evidence in Clinical Trials

(5/14, Jeffrey Keefer, Jaclyn Bosco, IQVIA Blog) comments “...The Global R&D Trends 2026 Report highlights the growing regulatory acceptance of RWE, particularly in situations where randomization is not feasible or unethical and where there is a high unmet medical need. In 2025, real world evidence contributed to seven FDA approvals, using a range of approaches that included observational studies, external controls, and contextual safety and effectiveness data...Importantly, RWE is not limited to initial approvals. It is increasingly used to support label expansions, new populations, and indication modifications, allowing sponsors to extend the value of approved therapies more efficiently.” Full

FDA RCT-DUPLICATE Webinar Explores the Evolving Role of Real-World Evidence in Complementing Randomized Trials

(5/14, Katie McCool, The Evidence Base) reports “...[FDA’s Marie Bradley] noted that rigorous, pre-specified RWD studies can reach conclusions similar to those of RCTs when trials are closely emulated, and key variables are well measured. She also emphasized that the initiative highlighted important limitations of RWE, particularly in situations where elements of trial design cannot be adequately replicated using observational data.” Full

Trump 'Most Favored Nation' Drug Policy Risks China R&D Takeover

(5/14, Seeking Alpha) reports "...Speaking at the FT US Pharma and Biotech Summit in New York on Thursday, Pharmaceutical Research and Manufacturers of America (PhRMA) COO Lori Reilly indicated that depressed prices, in both the US and Europe, could negatively impact US drug R&D. She said that drug policies outside the US ‘undervalue innovation and [include] discriminatory practices.’ If we ‘import those types of policies and values and do nothing…that is an invitation to China to overtake the US in biopharma innovation.’" Full

The New EU HTA Reality: JCA Preparation Requires Deep Early Planning

(5/15, Eliza Slawther, Pink Sheet) reports “...Pharmaceutical companies should ensure they have the right operational model to address the challenges of preparing for joint clinical assessments under the EU Health Technology Assessment Regulation, and perform a ‘dry run’ ahead of time, a market access consultancy CEO advises.” Subscription Required