A Framework for Extension Studies Using Real-World Data to Examine Long-Term Safety and Effectiveness.
Ther Innov Regul Sci
; 56(1): 15-22, 2022 01.
Article
in En
| MEDLINE
| ID: mdl-34251656
ABSTRACT
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).
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Research Design
/
Pharmacoepidemiology
Type of study:
Guideline
/
Prognostic_studies
Limits:
Humans
Language:
En
Journal:
Ther Innov Regul Sci
Year:
2022
Document type:
Article
Affiliation country:
United States