TY - JOUR
T1 - Clinical Meaningfulness in Alzheimer’s Disease Clinical Trials. A Report from the EU-US CTAD Task Force
AU - EU/US CTAD Task Force
AU - Angioni, D.
AU - Cummings, J.
AU - Lansdall, C. J.
AU - Middleton, L.
AU - Sampaio, C.
AU - Gauthier, S.
AU - Cohen, S.
AU - Petersen, R. C.
AU - Rentz, D. M.
AU - Wessels, A. M.
AU - Hendrix, S. B.
AU - Jessen, F.
AU - Carrillo, M. C.
AU - Doody, R. S.
AU - Irizarry, M.
AU - Andrews, J. S.
AU - Vellas, B.
AU - Aisen, P.
AU - Andrieu, Sandrine
AU - Bateman, Randall
AU - Batrla, Richard
AU - Bell, Joanne
AU - Bosson, Oskar
AU - Bozeat, Sasha
AU - Brooks, Dawn
AU - Haeberlein, Samantha Budd
AU - Buracchio, Teresa
AU - Cho, Min
AU - Choung, Matthew
AU - Cook, Gavin
AU - Crisitello, Darrin
AU - Dangond, Fernando
AU - de Santi, Susan
AU - Dennehy, Ellen
AU - Dhadda, Shobha
AU - Dhillon, Harjeet
AU - Dunn, Billy
AU - Egan, Michael
AU - Elwood, Fiona
AU - Eriksson, Sven
AU - Fagan, Tom
AU - Fillit, Howard
AU - Freskgard, Per Ola
AU - Gallagher, Diana
AU - Gangi, Gopi
AU - Granda, Carlos
AU - Greeley, David
AU - Gronblad, Anna Kaija
AU - Hampel, Harald
AU - Hawthorne, Paul
AU - Henley, David
AU - Herring, Joe
AU - Hersch, Steve
AU - Holt, Bill
AU - Iwatsubo, Takeshi
AU - Jones, Daryl
AU - Kahl, Anja
AU - Kinney, Gene
AU - Kolb, Hartmuth
AU - Kramer, Lynn
AU - Kulic, Luka
AU - Kumar, Sanjay
AU - Lannfelt, Lars
AU - Lawson, John
AU - Legrand, Valérie
AU - Lenington, Rachel
AU - Longo, Frank
AU - Matthews, Brandy
AU - Masterman, Donna
AU - McLinden, Kristina
AU - Mikels, Sarah
AU - Miller, Michael
AU - Mintun, Mark
AU - Moebius, Hans
AU - Monteiro, Cecilia
AU - Morken, Mario
AU - Murphy, Jennifer
AU - Odergren, Tomas
AU - Osswald, Gunilla
AU - Parnas, Laura
AU - Patru, Maria Magdalena
AU - Prazma, Charlene
AU - Raman, Rema
AU - Reyderman, Larisa
AU - Rogers, Sharon
AU - Roman, Lise
AU - Romano, Gary
AU - Roskey, Mark
AU - Rubino, Ivana
AU - Ryan, Laurie
AU - Salloway, Stephen
AU - Schindler, Rachel
AU - Schneider, Lon
AU - Scott, David
AU - Sims, John
AU - Skovronsky, Daniel
AU - Soto, Marion
AU - Sperling, Reisa
AU - Steukers, Lennert
AU - Stoops, Erik
AU - Strittmatter, Stephen
AU - Tahami, Amir
AU - Tamagnan, Gilles
AU - Tariot, Gilles
AU - Toloue, Masoud
AU - Touchon, Jacques
AU - Vanmechelen, Eugeen
AU - Walt, Len
AU - Weinberg, Mark
AU - Weiner, Michael
AU - White, Anne
AU - Wiesel, Iris
AU - Wilson, David
AU - Yarenis, Lisa
AU - Zago, Wagner
AU - Zhou, Jin
N1 - Publisher Copyright:
© Serdi 2024.
PY - 2024/10
Y1 - 2024/10
N2 - Recent positive results of three phase III anti-amyloid monoclonal antibody trials are transforming the landscape of disease-modifying therapeutics for Alzheimer’s disease, following several decades of failures. Indeed, all three trials have met their primary endpoints. However, the absolute size of the benefit measured in these trials has generated a debate on whether the change scores observed on clinical outcome assessments represent a clinically meaningful benefit to patients. An evidence-based conclusion is urgently required to inform decision-making related to the approval, reimbursement, and ultimately, the management of emerging therapies in clinical practice. The EU-US CTAD Task Force met in Boston to address this important question. The current state-of-the-art knowledge for interpreting clinical meaningfulness of AD clinical trial results, including the point of view of patients and study partners on what is clinically meaningful, was discussed and is summarized here. A combination of methodologies to address the challenges emerged. There remain gaps in the understanding of clinical meaningfulness that only long-term longitudinal studies will be able to address.
AB - Recent positive results of three phase III anti-amyloid monoclonal antibody trials are transforming the landscape of disease-modifying therapeutics for Alzheimer’s disease, following several decades of failures. Indeed, all three trials have met their primary endpoints. However, the absolute size of the benefit measured in these trials has generated a debate on whether the change scores observed on clinical outcome assessments represent a clinically meaningful benefit to patients. An evidence-based conclusion is urgently required to inform decision-making related to the approval, reimbursement, and ultimately, the management of emerging therapies in clinical practice. The EU-US CTAD Task Force met in Boston to address this important question. The current state-of-the-art knowledge for interpreting clinical meaningfulness of AD clinical trial results, including the point of view of patients and study partners on what is clinically meaningful, was discussed and is summarized here. A combination of methodologies to address the challenges emerged. There remain gaps in the understanding of clinical meaningfulness that only long-term longitudinal studies will be able to address.
KW - CDR
KW - RCTs
KW - clinical outcome assessments
KW - iADRS
UR - http://www.scopus.com/inward/record.url?scp=85195541961&partnerID=8YFLogxK
U2 - 10.14283/jpad.2024.112
DO - 10.14283/jpad.2024.112
M3 - Article
C2 - 39350367
AN - SCOPUS:85195541961
SN - 2274-5807
VL - 11
SP - 1219
EP - 1227
JO - The journal of prevention of Alzheimer's disease
JF - The journal of prevention of Alzheimer's disease
IS - 5
ER -