Speaker
\ nBiography
\nAbstr act
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\n David Wright
(AstraZeneca)
Dr Wright became the Head of Statist ical Innovation at AstraZeneca in September 2016. David leads a team of ex pert statistical methodologists who advise colleagues within AstraZeneca o n novel trial design and analysis issues. Between 1999 and 2016 David work ed for the Medicines and Healthcare products Regulatory Agency (MHRA) (for merly the Medicines Control Agency (MCA)) as a Statistical Assessor. David was Chair of the Biostatistics Working Party at the European Medicines Ag ency from 2011-2016.
\nWhat are Decentralised Tri als? What are the key opportunities and risks with their use?
\nThis talk with give an introduction to Decentralised Tria ls and outline some of the opportunities and risks they bring if conducted instead of site based/centralized trials.
\n
\n Magnus Jö\;r
nten-Karlsson (AstraZeneca)
Magnus is an Execu tive Product Director at AstraZeneca responsible for development and deplo yment of Digital Patient Solutions. He is accountable for development of U nify\, an internal AstraZeneca solution to meet the needs of patients and HCPs participating in AstraZeneca Clinical trials.
\n \ nTo successful ly support decentralized clinical trials we need to think holistically abo ut designing a toolkit for patients and sites. In this talk I will touch o n how we define\, evaluate and select technology (medical devices)\; curre nt progress on the tools\, moving away from isolated silo tools that only deliver one thing to broader platforms\; and the impact that is having on our sites and patients.
\n
\n Magalie Hilton
\n
(Roche)
Magalie Hilton is a senior principa l statistical scientist at Roche\, Switzerland. She joined the company in 2009 and previously worked at a CRO in England. She has worked across seve ral indications in oncology including in glioblastoma\, pediatric and lung driver mutations drug development. Magalie has been involved in the setup \, conduct and analysis of platform trials and this decentralised trial. S he received her M.Sc. in Applied Statistics\, Health and Social Sciences\, at the University of Bordeaux (France).
\nALpha- T: a Pre-Pandemic Decentralized Trial in Oncology.
\nALpha-T is a Phase II\, Open-Label\, Single Arm trial with a decentr alized (DCT) home-based approach. Patients with locally-advanced or metast atic ALK-positive non-NSCLC are enrolled. The collaborations involving the patients\, the caregivers\, the sequencing partner\, the telemedicine com pany\, the regulators and the Sponsor are key for the implementation of ho me-based solutions. Based on this case study example\, top challenges and opportunities of a DCT will be presented.
\n\n Yashoda Sharma
\n (DiMe)
\n \n
Founded in 2019\, the Digital Medicine Society (DiMe) is a global non-profit and the profes sional home for all members of the digital medicine community. Together\, we drive scientific progress and broad acceptance of digital medicine to e nhance public health. Digital medicine offers the potential to redefine he althcare\, solving some of the most pressing and persistent challenges to good health for all. The digitization of healthcare presents a fleeting op portunity to move away from a system designed to treat the sick and toward one that is defined by each individual&rsquo\;s health. Core to our work of driving the development and adoption of digital health measurements is advancing health equity\, to fuel the effectiveness of healthcare.
\nChallenges to effective and equitable clinical trials cannot be solved with individual entities working in isolation\; multi-stakeholde r\, interdisciplinary expertise is required. DiMe partnered with the FDA a nd organizations across the healthcare continuum to prioritize inclusion i n digital health measurement product development and deployment\, and dive rsity\, equity\, and inclusion in digitized clinical trials. With this wor k we developed toolkits that healthcare and research organizations can inc orporate into their current workflows to drive the adoption and implementa tion of digital and decentralized clinical trials\, and best practices to ensure health resources are delivered with a health equity lens.
\n
\n Khadija Rantell
\n (MHRA)
At the MHRA\, she writes assessment reports on methodological aspects of licensing applications\, advises comp anies on methodological aspects of their clinical development programme\, and contributes to the assessment of clinical trial applications and clini cal investigations of medical devices.
\nPrior to joinin g the agency\, Khadija worked in academia for over 10 years\, where she ta ught statistics\, both at undergraduate and postgraduate levels\, and was involved in research across a wide range of therapeutic areas. She has a P hD in Applied Statistics to Healthcare Research from Sheffield University.
\nKhadija has a particular interest in early phase clin ical trial designs\, innovative trial designs\, patient reported outcomes\ , real-word-data\, and rare diseases. She is a member of the Patient Repor ted Outcomes Specialist Interest Group at the MHRA\, the EFSPI (European F ederation for Statisticians in the Pharmaceutical Industry) Scientific Com mittee\, and the EFPIA/EFSPI Estimand Implementation Working Group (EIWG).
\nTowards more patient-centric trials: opportuni ties and challenges of decentralized clinical trials.
\nThe COVID-19 pandemic has necessitated flexibility in trial condu ct and accelerated changes in the clinical trial landscape. Decentralised clinical trials (DCTs) introduce new approaches to the conduct of clinical trials that aim to make clinical trials more easily accessible and conven ient for participants to take part in. Moving forward\, it will be importa nt to leverage the use of more innovative tools and approaches to trial de sign\, and conduct\, including adoption of remote approaches such as elect ronic patient-reported outcomes\, home visits by health staff\, follow-ups over phone\, telemedicine etc. This talk will cover key considerations fo r suitability of use of decentralised clinical trial model\, reflecting on the opportunities and challenges of implementing decentralised elements i n clinical trials.
\n
\n Rima Izem
\n (Novartis)
Dr. Rima Izem joined Novartis in 2021 as an Associa te Director in the Statistical Methodology group in Analytics. There\, she supports development and implementation of novel hybrid designs and stati stical methods using real-world-data across therapeutic areas\, and primar ily in development and post-marketing. In her prior roles as faculty at Ha rvard University\, reviewer and team leader at the US Food and Drug Admini stration\, and faculty at Children&rsquo\;s National Research Institute sh e acquired a broad experience in clinical research and regulatory practice . This experience includes leading or contributing to several pioneering p rojects in comparative safety and effectiveness in the FDA Sentinel Distri buted System\, and comparative effectiveness research using rare disease r egistries.
\nDecentralized clinical trials: scien tific considerations through the lens of the estimand framework.< /p>\n
While the industry and regulators&rsquo\; interest in decentralized methods is long-standing\, the Covid-19 pandemic accelerated or broadened the adoption and the experience with these methods in clinic al trials. Potential benefits of decentralizing studies include broadening access of clinical trials to a more diverse population\, reducing the bur den of participation in trials\, or using innovative endpoints. As decentr alization is moving from a necessity to a choice\, researchers need to con sider the actual value added and implications of these designs beyond the operational aspects of their implementation
\nOur presen tation illustrates how the estimand framework can clarify our thinking abo ut those strategic decisions around decentralization. Those questions go b eyond whether or not to decentralize\, to how much and what to decentraliz e. That is\, going through questions related to each attribute of the esti mand\, and especially population\, treatment and variable\, can guide the teams through a systematic thought process about the scientific opportunit ies\, assumptions and potential risks associated with a possible use of de centralized components in the design of a trial. Illustrations from decent ralized trial case studies will support the presentation and discussion of various considerations from this framework. Using this approach\, teams c an more easily calibrate the design decentralization complexity (from hybr id to fully decentralized) to the value added in answering scientific ques tions that could not be solely answered by an all on-site approach.
\n
\n Rachel Will
iams
\n (GSK)
\n
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\n Luis Garcia-Gan
cedo \;
\n (GSK)
\n
\n
Luis Garcia-Gancedo. \;A Digital Health Leader in the pharmaceutical industry\, Luis is pa ssionate about applying technology to improve healthcare. His mission is t o use digital technologies and platforms to improve the lives of patients\ , helping them do more\, feel better and live longer. Currently leading GS K's efforts to develop novel Digital Biomarkers to support their pipeline of innovative medicines. Luis has a unique combination of skills balancing in-depth technical expertise in Engineering and Data Science with Leaders hip\, Digital Health and Clinical Development. Prior to joining GSK\, Luis spent several years in academia\, most recently as a Research Associate a t the Engineering Department\, University of Cambridge and as a College Le cturer in Engineering at Newnham College\, University of Cambridge. He gai ned a BSc/MSc in Physics from the University of Oviedo (Spain)\, a PhD in Electronic Engineering from the University of Brighton (UK) and completed an \;Executive MBA at the University of Cambridge (UK).
\nDeveloping Digital Biomarkers: Application to Rheumatoid Arth ritis.
\nData collected in clinical trials pres ent an incomplete view of disease activity due to daily symptoms fluctuati on and heterogeneity. Digital technology can enable remote continuous moni toring and the addition of objective of patient assessments. Rheumatoid ar thritis (RA) is a chronic condition with fluctuating symptoms allowing for feasibility assessment of digital biomarkers. First\, we performed the PA RADE study as the first in pharma to use the Apple Researchkit®\; for f ully remote enrollment and data collection via the iPhone\, including base line information\, symptoms\, wrist movement\, and a walk test. Based on t his experience\, we performed the weaRAble PRO study\,enrolling patients t hrough a clinic\, and adding an Apple Watch as well as the iPhone for data collection. These studies indicate that digital technology allows for rem ote data collection\, increased measurement frequency\, a richer picture o f disease activity\, and a focus on patient centricity.
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