PT  - JOURNAL ARTICLE
AU  - Faiez Zannad
AU  - Marc A Pfeffer
AU  - Deepak L Bhatt
AU  - Denise E Bonds
AU  - Jeffrey S Borer
AU  - Gonzalo Calvo-Rojas
AU  - Louis Fiore
AU  - Lars H Lund
AU  - David Madigan
AU  - Aldo Pietro Maggioni
AU  - Catherine M Meyers
AU  - Yves Rosenberg
AU  - Tabassome Simon
AU  - Wendy Gattis Stough
AU  - Andrew Zalewski
AU  - Nevine Zariffa
AU  - Robert Temple
TI  - Streamlining cardiovascular clinical trials to improve efficiency and generalisability
AID  - 10.1136/heartjnl-2017-311191
DP  - 2017 Aug 01
TA  - Heart
PG  - 1156--1162
VI  - 103
IP  - 15
4099  - http://heart.bmj.com/content/103/15/1156.short
4100  - http://heart.bmj.com/content/103/15/1156.full
SO  - Heart2017 Aug 01; 103
AB  - Controlled trials provide the most valid determination of the efficacy and safety of an intervention, but large cardiovascular clinical trials have become extremely costly and complex, making it difficult to study many important clinical questions. A critical question, and the main objective of this review, is how trials might be simplified while maintaining randomisation to preserve scientific integrity and unbiased efficacy assessments. Experience with alternative approaches is accumulating, specifically with registry-based randomised controlled trials that make use of data already collected. This approach addresses bias concerns while still capitalising on the benefits and efficiencies of a registry. Several completed or ongoing trials illustrate the feasibility of using registry-based controlled trials to answer important questions relevant to daily clinical practice. Randomised trials within healthcare organisation databases may also represent streamlined solutions for some types of investigations, although data quality (endpoint assessment) is likely to be a greater concern in those settings. These approaches are not without challenges, and issues pertaining to informed consent, blinding, data quality and regulatory standards remain to be fully explored. Collaboration among stakeholders is necessary to achieve standards for data management and analysis, to validate large data sources for use in randomised trials, and to re-evaluate ethical standards to encourage research while also ensuring that patients are protected. The rapidly evolving efforts to streamline cardiovascular clinical trials have the potential to lead to major advances in promoting better care and outcomes for patients with cardiovascular disease.