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Optimizing Rwe Generation Fda's appliedclinicaltrialsonline.com Volume 28 Number 4 April 2019 YOUR PEER-REVIEWED GUIDE TO GLOBAL CLINICAL TRIALS MANAGEMENT 1992–2019 ACTAACCT 2727 e Year c of Servi STUDY DESIGN OPTIMIZING RWE GENERATION REGULATORY For personal, non-commercial use FDA’S ‘REAL-WORLD’ FRAMEWORK DATA SHARING ADOPTING GLOBAL DATA STANDARDS WASHINGTON REPORT EU REPORT CLOSING THOUGHT The Push to Broaden Clinical Translational Research Message Rise of the ‘Citizen Trial Eligibility Criteria Lost in Transmission Data Scientist’ FROM THE EDITOR Emerging Biopharma Deserve TLC from CROs arly this month, I presented on the topic was 26%, compared to 60% that went to firms that had never before E“Why Aren’t Lessons Learned from Big received an approval. Pharma/Big CROs Translating to Smaller The days of quickly selling a promising compound early on to a large Biopharma Relationships?” For years, Applied pharma company or launching a full-fledged IPO are dwindling. An Ernst Clinical Trials has been the operational re- & Young report showed 75 biotech IPOs in 2014 compared to nine in the source for clinical trials for sponsors, CROs, second half of 2016. These days, innovation hubs in major regions of the and academia. The transition of the contract country are home to a vast number of companies looking to enter clini- research industry from a small cottage to a cal trials, take their compound at least through Phase II, and some are LISA HENDERSON fully-matured one has seen numerous out- fully intending to stick it through Phase III and even make it to a com- Editor-in-Chief sourcing models from the low-touch, very fo- mercial stage company. cused and accountable transactional service, to the high-touch, highly This is all evidence that these emerging biopharma are CROs’ latest involved and complex strategic partnership. Another example has fea- boom of customers. But, as the new crop of uninitiated companies, they tured CROs taking on internal departments from pharma companies to are having similar angst with their CRO. They feel unimportant to large ensure employees still had jobs, as well as the knowledge transfer rela- CROs, costs get away quickly, oversight is difficult, projects don’t come tive to that pharma’s specific development needs and culture. in on time or budget…the same list that we’ve seen with outsourcing It may seem that by this point, sponsors of all shapes and sizes relationships over the years. The problem is that emerging biopharma would know how to respond to the challenges relative to working with companies face constraints that the large, traditional pharma never an outsourcer, no matter what type of relationship is involved. But in have. A single clinical trial could be the only thing the company has; basi- discussions I have with smaller biopharma, that does not seem to be cally, its whole survival. They are more financially-constrained and may the case. Why should CROs be concerned about their service offerings either make poor decisions or cut corners, not to mention taking advice for these emerging players? Because they are not an insignificant part from new executives or external stakeholders that deviate from the orig- of the drug development landscape. inal plan, which then results in out-of-scope charges or change orders. Consider the following: The future of drug development does not lightly rest on an out- • Of all companies sponsoring one or more clinical development pro- sourced expert, it almost solely exists in their confines. It wouldn’t be a grams, 61% now fall outside the ranks of the top 50 largest pharma. stretch to ask CROs to be more mindful of their smaller clients and take • The percentage of FDA approvals obtained by big pharma in 2018 extra attention in handling their needs. 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DIRECTOR OF ADVERTISING Wayne K. Blow MultiMedia Healthcare LLC provides certain customer contact data (such as customers’ names, addresses, phone numbers, and e-mail addresses) to third parties UK: +44 1244 629 304 fax: +44 1925 732 798, who wish to promote relevant products, services, and other opportunities that may be of interest to you. If you do not want MultiMedia Healthcare LLC to make your contact information available to third parties for marketing purposes, simply call toll-free 866-529-2922 between the hours of 7:30 a.m. and 5 p.m. CST and a [email protected] customer service representative will assist you in removing your name from MultiMedia Healthcare LLC’s lists. Outside the U.S., please phone 218-740-6477. NATIONAL SALES MANAGER Bill Campbell Applied Clinical Trials does not verify any claims or other information appearing in any of the advertisements contained in the +1 (847) 283-0129 fax: +1 (847) 282-1456, publication, and cannot take responsibility for any losses or other damages incurred by readers in reliance of such content. [email protected] To subscribe, call toll-free 888-527-7008. Outside the U.S. call 218-740-6477. 2 APPLIED CLINICAL TRIALS appliedclinicaltrialsonline.com April 2019 CONTENTS APPLIED CLINICAL TRIALS VOLUME 28, NUMBER 4 FEATURED 12 Optimizing Study Design in Real-World Evidence Generation Rob Sambrook With outcomes from “real life” a critical compliment to clinical trial data, the importance of involving an epidemiologist at study inception is explored. SOURCE/GETTY KJPARGETER/SHUTTERSTOCK.COMIMAGE IMAGES; 15 The Framework for FDA’s 18 Global Standardization NEWS AND ANALYSIS Real-World Evidence Program of Clinical Research Data 4 WASHINGTON REPORT Beth Schurman Barbara Jauregui, Lynn D. Hudson, 6 EU REPORT A look at the scope of the agency’s Lauren B. Becnel, Eileen Navarro Almario, Ronald Fitzmartin, Frank draft framework for evaluating the use 10 Q&A of RWE to support new drug approvals Pétavy, Nathalie Seigneuret, James and the implications for sponsors. K. Malone, Fang Liz Zhou, Jose Galvez, Tammy Jackson, Nicole Harmon, Rebecca D. Kush COMMENTARY Outlining the latest government, A CLOSING THOUGHT industry, and public health efforts to promote increased 25 Non-Data Scientists: The Evolving adoption of common standards Role of Clinical Data Management in data collection and sharing. Derek Lawrence For personal, non-commercial use EDITORIAL ADVISORY BOARD Moe Alsumidaie Srini Dagalur, PhD Michael R.
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