Aetion的动态

We have all heard the term "fit for purpose data" but how do you actually prove that? Particularly to Regulators? In a recent publication (https://hubs.ly/Q02sdCbC0) key members of the FDA highlighted how high that bar really is: "Transparency helps to promote both relevance and reliability of a RWD source to address a scientific question and is particularly important for studies intended to support regulatory decision making. A comprehensive pre-specified protocol that transparently describes study objectives and design elements can facilitate assessment of data source relevance; specifically, outlining how the minimum data elements needed to determine selection criteria, exposure assessment, outcome, and relevant covariates can inform RWD source selection and study feasibility." The Challenge: Real World Researchers have the challenge to show regulators how they defined EACH study design element and how this was assessed in EACH data source. The Solution: Navidence Sherpa Platform: A library of consistent, standards-based definitions for each design elements in a computable format, tailored to your area of research, and which you can consistently apply across your RWE projects and candidate RWD sources. Interested to hear more about how we are helping sponsors support their discussions with regulatory agencies on qualification of RWD sources? Click here https://hubs.ly/Q02sdnpd0 to schedule time with one of the Navidence team at ISPOR in Atlanta May 6th-8th. #ISPOR

I'm delighted to welcome Stuart McCully (RWR-Regs) as a Guest Columnist with The Evidence Base?. In this new series - 'What's in a Name?' - Stuart explores the #terminology we use when we talk about #RWE, the studies and analytics we conduct to generate #realworldresearch, and the current challenges we need to overcome so that we can move beyond the basics and begin to speak to same language. In this first article, Stuart explores the need for establishing clear and universally aligned definitions of #realworlddata and #realworldevidence as a ‘critical step’ towards advancing the field.?At present, the definitions vary considerably between major stakeholders, such as FDA, NICE - National Institute for Health and Care Excellence, Medicines and Healthcare products Regulatory Agency, CADTH and others. #rwd #healthdata #ehds

2024 State of Real-World Evidence Policy July 25, 2024 12 - 4:45 p.m. ET Location: Virtually via Zoom Register to Attend Virtually Convened with the Duke-Margolis Real-World Evidence Collaborative, this public convening will provide a venue to review recent RWE Collaborative activities, strategic real-world data and real-world evidence (RWD/E) policy developments, and promising future applications of RWD/E. This year, speakers will focus on a few key topics: Barriers to accessing high-quality, reliable, and relevant real-world health data sufficient for regulatory decisions Applications of artificial intelligence in RWE studies Opportunities to use RWD/E to inform payment and coverage decisions for novel technologies To register https://lnkd.in/e7wkR-7C

I attended a (really enjoyable) industry conference recently and have been reflecting on a couple of themes that emerged during the presentations & discussions. ? 'Real World Evidence' (RWE) is often claimed to be something that is not readily accepted by decision-makers in a cost-effectiveness analysis (CEA). This sentiment baffles me somewhat as RWE (i.e. observational evidence) is used extensively in almost every CEA (e.g. to inform resource utilisation rates, general population mortality, long-term OS and PFS and so on). I suspect some commentators may mean, specifically, that RWE is not so readily accepted as an evidence source for relative effectiveness. Such hesitance among decision-makers seems the height of reasonableness to me. An RCT is undoubtedly a more reliable basis for relative effectiveness estimates compared to RWE (e.g. as an external control arm) and it is important that decision-makers highlight this. In my experience, decision-makers do generally accept the realities of an evidence base even if relative effectiveness is ultimately based partially on RWE, but they quite rightly need to account for the (often significant) resultant uncertainty when making resource-allocation decisions. Relatedly, it is sometimes claimed that there is a reluctance among decision-makers to consider alternative pricing/payment models (IPMs) and that they just want to 'keep it simple' with price discounts. There may be truth in this, I can imagine for administrative reasons if nothing else. But the key point in my view is that whatever the mechanism for payment (and some of these IPMs indeed may help to address issues of uncertainty and upfront affordability), we still must, first and foremost, calculate an average cost per QALY gained and if this is not below a suitable threshold, then adopting the treatment would do more harm than good. There's nothing more simple than that.??

An unsurprising finding that there is a lack of coordination between regulatory and other guidance issuing organizations. Nevertheless, a very nice comprehensive overview for those looking for the most up to date status on the regulatory guidance landscape for RWE. Great work Grammati Sarri! My optimistic take is that despite the lack of alignment, RWE is getting the attention it needs to take its place alongside other established approaches. Additionally, just like other innovative industries, it is important for us to recognize our role in helping move the needle by collaborating with these bodies to bring forward well designed and executed RWE studies(clear justification for using RWD; high quality, fit-for-purpose, datasets; etc) that can help define standards and drive alignment. #RWD #realworlddata #RWE #realworldevidence #DrugDevelopment #EvidenceGeneration??#Regularory #HTA

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Great day 1 at the #DIA #RWE24 meeting. Key themes today - Novel approaches and considerations for integrating RWD earlier - during clinical trial development Many new regulatory guidances to guide RWD for RWE Clinical Trial Diversity- Look forward to Day 2- #RTIHealthSolutions

? RWE4Decisions is hosting a series of multi-stakeholder meetings on operationalising Post-launch evidence generation (PLEG), to generate cross-country learnings and support multi-country collaborations. ?? Our recent Roundtable discussed “When to Do PLEG?”. ? ?? We heard insightful presentations on Real-World Data PLEG national approaches from France ????, Spain #Valtermed ????, and England ??????????????, which fuelled dynamic interactions in the breakout rooms discussing whether an anticipatory approach for PLEG could be designed. ? ?? We are immensely thankful for all of our participants and their valuable contributions, and we look forward to continuing the conversations on PLEG with the RWE4Decisions Learning Network at the next meeting in September. ??? ? ?? Until then, we look forward to the publication of an important exhaustive retrospective case-control study by Haute Autorité de Santé in a paper by Judith Fernandez et al, studying the occurence of Post-Registration Study requests in a number of positive opinions in ????.? ? #PLEG #PostLaunchEvidenceGeneration #HTA #HealthData #EHDS #RealWorldEvidence #RWD #RealWorldData