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Introduction Clinical trials are always a lively area of biopharma regulation and the current moment isparticularly so. As the articles in this package show, there are new opportunities to design studies more efficientlyand effectively, as through master protocols and decentralized trials, and to use new types of datain marketing applications, such as real-world evidence. The Pink Sheet closely covers how theseapproaches are evolving, how industry peers are applying them in practice, and what regulatorsexpect from sponsors for determining that these approaches are fit for regulatory purpose. Ofcourse, we also will cover new requirements for sponsors, such as compliance with the EU clinicaltrials regulation and the US guidance on diversity and inclusion in research. With this knowledge, sponsors can avoid missteps in compliance, and also navigate the newresearch flexibilities with the confidence that they’ll meet regulatory standards. Master Protocols OfferAnother Avenue To AdvanceReal-World Evidence Brenda Sandburg09 Oct 2023 Experts discuss how master protocols can help produce morecomparable real-world evidence and draw more investigatorsinto studies. FDA CMO Hilary Marston says payers must bemore involved in discussion of the value of real-world data. Executive Summary In the ongoing quest to expand the use ofreal-world evidence in drug development,researchers are now focusing on the potentialuse of master protocols to support multiple RWEstudies under a single research protocol. Key Takeaways •Master protocols should allow for anexpansive number of investigators inRWE studies.•AI has the potential to transformcoding, reimbursement, and datageneration for physicians.•Health systems can struggle to usethe data they are amassing to adjustpatient care. The Duke-Margolis Center for Health Policy atDuke University focused on these efforts at ameeting last month on the state of real-worldevidence policy. Rachele Hendricks-Sturrup, research director ofreal-world evidence at the Duke-Margolis Center,said real-world master protocols aim to improveconsistency and efficiency in real-world dataand evidence studies. mechanisms that are inherent to masterprotocols may reduce variation and producemore reliable, comparable real-world evidence,” “Leveraging, as it is practical, the standardized Master Protocols Offer Another AvenueTo Advance Real-World Evidence she said. “And biomedical innovation and real-world evidence credibility will advance whenmultistakeholder-led actions coordinate sharedresources to address evidentiary gaps.” using a master protocol can help minimize that.In addition, he said a master protocol can alsotake some of the burden from individual trialsites and investigators and lower the overall costof the study. Master protocols provide a way tosystematically test multiple hypotheseswithin a single clinical trial. Hendricks-Sturruppointed out that FDA’s past master protocol-related work includes the Sentinel Initiative’sCOVID-19 natural history study and protocolfor distributed queries and the Reagan-UdalFoundation for the FDA’s COVID-19 EvidenceAccelerator. (Also see “Real-World Evidence OnCOVID-19: US FDA Approaching With ‘Sense OfUrgency’” - Pink Sheet, 21 Apr, 2020.) Josh Fessel, senior clinical advisor at theNational Institutes of Health’s National Centerfor Advancing Translational Sciences, also citedthe benefit of master protocols for investigators. “If we do this right, in terms of constructingthings like master protocols that get us allsinging from the same songbook, it does havethis effect of being an inclusive invitation tobring investigators in,’” he stated. “Every goodmaster protocol, in addition to producing highquality evidence that decisions can be madefrom, it also becomes an important playbookfor how somebody else might do somethingsimilar.” Elise Berliner, global senior principle for realworld evidence strategy at Cerner Enziva, saidmaster protocols for real-world evidence areneeded for multi-site studies to make sure datais collected the same way. Duke-Margolis issued a white paper, Harnessingthe Potential of Real-World Evidence MasterProtocols, the day of the meeting. It describesthe characteristics of RWE master protocols andhow they can support development of evidencefor medical products. “I would argue that we need harmonization ona broader scale, that having sort of commondefinitions for cohorts and for outcomemeasures will be beneficial. Because a lot oftimes the studies themselves for FDA approvalaren’t doing comparative effectiveness,” Berlinersaid. “But patients and clinicians are lookingfor that kind of comparative effectivenessinformation and it’s impossible to synthesizeacross studies.” The paper discusses innovative master protocoltemplates that are emerging to supportmore consistent structures and processes fordesigning and conducting multiple RWE studiesunder a single research protocol. It notes thatone recent example is the HARmonized Protoc