TY - JOUR
T1 - A Framework for Extension Studies Using Real-World Data to Examine Long-Term Safety and Effectiveness
AU - Burcu, Mehmet
AU - Manzano-Salgado, Cyntia B.
AU - Butler, Anne M.
AU - Christian, Jennifer B.
N1 - Funding Information:
This manuscript received endorsement from the International Society for Pharmacoepidemiology (ISPE). No specific funding for this work was provided. This article does not contain any studies with human or animal subjects performed by any of the authors. The authors acknowledge Mary E. Ritchey for comments and suggestions in the earlier versions of this manuscript. This article reflects the views and opinions of the authors and should not be construed to represent the views and opinions of any public or private entity.
Publisher Copyright:
© 2021, The Author(s).
PY - 2022/1
Y1 - 2022/1
N2 - Understanding the long-term benefits and risks of treatments, devices, and vaccines is critically important for individual- and population-level healthcare decision-making. Extension studies, or ‘roll-over studies,’ are studies that allow for patients participating in a parent clinical trial to ‘roll-over’ into a subsequent related study to continue to observe and measure long-term safety, tolerability, and/or effectiveness. These designs are not new and are often used as an approach to satisfy regulatory post-approval safety requirements. However, designs using traditional clinical trial infrastructure can be expensive and burdensome to conduct, particularly, when following patients for many years post trial completion. Given the increasing availability and access of real-world data (RWD) sources, direct-to-patient technologies, and novel real-world study designs, there are more cost-efficient approaches to conducting extension studies while assessing important long-term outcomes. Here, we describe various fit-for-purpose design options for extension studies, discuss related methodological considerations, and provide scientific and operational guidance on practices when planning to conduct an extension study using RWD. This manuscript is endorsed by the International Society for Pharmacoepidemiology (ISPE).
AB - Understanding the long-term benefits and risks of treatments, devices, and vaccines is critically important for individual- and population-level healthcare decision-making. Extension studies, or ‘roll-over studies,’ are studies that allow for patients participating in a parent clinical trial to ‘roll-over’ into a subsequent related study to continue to observe and measure long-term safety, tolerability, and/or effectiveness. These designs are not new and are often used as an approach to satisfy regulatory post-approval safety requirements. However, designs using traditional clinical trial infrastructure can be expensive and burdensome to conduct, particularly, when following patients for many years post trial completion. Given the increasing availability and access of real-world data (RWD) sources, direct-to-patient technologies, and novel real-world study designs, there are more cost-efficient approaches to conducting extension studies while assessing important long-term outcomes. Here, we describe various fit-for-purpose design options for extension studies, discuss related methodological considerations, and provide scientific and operational guidance on practices when planning to conduct an extension study using RWD. This manuscript is endorsed by the International Society for Pharmacoepidemiology (ISPE).
KW - Direct-to-patient
KW - Enriched studies
KW - Extension studies
KW - Long-term outcomes
KW - Observational follow-up
KW - Patient-mediated data
KW - Pragmatic approaches
KW - Real-world data
KW - Real-world evidence
KW - Roll-over studies
UR - http://www.scopus.com/inward/record.url?scp=85110846321&partnerID=8YFLogxK
U2 - 10.1007/s43441-021-00322-8
DO - 10.1007/s43441-021-00322-8
M3 - Review article
C2 - 34251656
AN - SCOPUS:85110846321
SN - 2168-4790
VL - 56
SP - 15
EP - 22
JO - Therapeutic Innovation and Regulatory Science
JF - Therapeutic Innovation and Regulatory Science
IS - 1
ER -