CER Daily Newsfeed

BioMarin Generates Patient Experience Data With Early And Often Engagement

(6/18, Clinical Leader) reports “...[Ebony Dashiell-Aje, Ph.D., executive director & head, patient centered outcomes science, BioMarin Pharmaceutical:] Without strong champions advocating for its importance, sponsors can struggle to integrate patient engagement into their workflows because it is seen as secondary to quantitative clinical outcomes and real-world evidence generation. Additionally, time and budget pressures often lead to shortcuts, with engagement efforts reduced or omitted entirely to save on resources –metrics and data to support rationale for the work are critical here for driving adoption.” Full

FDA to Speed Reviews for Drugs Supporting 'National Interests'

(6/18, Jonathan Gardner, BioPharma Dive) reports “...The new program, however, is a pilot that doesn't have congressional authority and, in its first year, will only grant a limited number of vouchers to companies ‘aligned with U.S. national priorities.’...To qualify, companies have to submit the portion of their application covering a drug's manufacturing process, as well as its proposed prescribing information, at least 60 days before their final submission, according to the FDA. They also have to be available for ‘ongoing communication with prompt responses.’ The agency could additionally extend a review if the application is ‘insufficient or incomplete,’ if study results are ‘ambiguous’ or the review is ‘particularly complex.’” Full

Does the FDA Commissioner's New Voucher Program Open a Pandora's Box?

(6/18, Matthew Herper, STAT+) comments “...The new process will convene experts for ‘a team-based review’ that Makary likens to the "tumor boards" that are used for difficult treatment decisions in oncology. Breaking down barriers is a good idea, but cutting down on the degree to which FDA staffers actually kick the tires on new data is not. And the FDA doesn't have the best record when it comes to ways in which it deviates from standard approval processes. One obvious example: The FDA convened experts from across the agency for the approval of the Alzheimer's drug Aduhelm, which was essentially rejected by the medical community and later withdrawn from the market.” Subscription Required

Advancing Clinical Trial Excellence and Real-World Evidence in the EU: Insights from EMA and HMA Leaders

(6/18, Katie McCool, The Evidence Base) reports “...[Chair of the HMA Management Group and Executive Director of the Spanish Agency of Medicines and Medical Devices María Jesús Lamas Díaz] outlined how the updated HMA-EMA strategy to 2028 sets priorities in digitalization and regulatory innovation. ‘We have the aim, among others, to foster the generation of high-quality evidence throughout the whole cycle life of medicines,’ she said. She highlighted work to modernize trial design, reduce reliance on animal testing, integrate AI tools, and better support researchers through early scientific advice.” Subscription Required

Guidance Creates Access Gap in UK for GPP Drug Spevigo

(6/18, Phil Taylor, PharmaPhorum) reports “...NICE has published new guidance recommending IL-36 inhibitor Spevigo (spesolimab) for the treatment of flares – devastating eruptions of painful blisters – in people with generalised pustular psoriasis (GPP). The decision does, however, create a disparity in access to Boehringer's drug within the UK, as the Scottish Medicines Consortium (SMC) turned down the drug, which has a UK list price of £15,000 (ex-VAT) for a pair of 450mg vials, in guidance published in March.” Full