Pragmatic Clinical Trials: Bridging the Gap Between Research and Real-World Practice

Definition and Purpose:
Pragmatic clinical trials are designed to test the effectiveness of interventions in real-life clinical practice settings, aiming to maximize applicability and generalizability of outcomes by minimizing modifications to routine care12.

Design Considerations:
These trials often involve randomization, may use cohort multiple randomized controlled trials (cmRCTs) for efficient recruitment and retention, and can employ adaptive trial designs to increase efficiency and conclusiveness12.

Outcomes and Analysis:
Pragmatic trials focus on major outcomes such as mortality and severe morbidity, assessed over adequate time intervals, and emphasize the importance of safety evaluations, including uncommon adverse effects12.

Real-World Application:
Unlike explanatory trials, which are conducted under optimal conditions to confirm physiological or clinical hypotheses, pragmatic trials aim to inform clinical or policy decisions by providing evidence for the adoption of interventions into real-world clinical practice124.

Tools and Guidelines:
The PRECIS and PRECIS-2 tools help trialists make design decisions consistent with the intended purpose of their trial, scoring nine domains from very explanatory to very pragmatic12.

Ethical and Regulatory Considerations:
Pragmatic trials must adhere to high methodological standards and ethical guidelines to ensure the reliability and applicability of their findings124.

Future Directions:
The integration of real-world data (RWD) and pragmatic clinical trials is expected to play a growing role in clinical, regulatory, and payer decision-making, complementing traditional randomized controlled trials (RCTs)34.

Sources:

1. https://academic.oup.com/ecco-jcc/article/18/8/1222/7609924?login=false

2. https://watermark.silverchair.com/jjae026.pdf?token=AQECAHi208BE49Ooan9kkhWErcy7Dm3ZL9Cf3qfKAc485ysgAAA14wggNaBgkqhkiG9w0BBwagggNLMIIDRwIBADCCA0AGCSqGSIb3DQEHATAeBglghkgBZQMEAS4wEQQMlG8qhzE0SKWlhpkCAgEQgIIDEQcRmE47l3KrczVNChyqjmQbmYSHFNN47p2qpZvrXj6Q4tGDsCizH7n3AHrXaCsmfBSzt-CHyShExYyyZsCFdY-yAlE8eey-vwfw3Y2b954kSIgdp2UwUNYySATt0iTCl47TmQ1qAV2CblZxzuzlsuFsliHmI-vclTq7-exir8oaDLrIbZRteeb8q4wP75ubApZdcQwH-3YYzF4yvlxI24NwAkxReiQngeBeY9SKwzVoE04rgCGtN9hy8trn7QGguI-p8-QwlYqQ3pVpef9KHvJebDgrHgB-8sgxuTOmWBVNTrfhjMOeXpSwgAYpiDv90uBk-v8lLXoOdBerAWKkVwkAOMCsrLx4Tx5fTGfxBFXFkl-iZdalVJ43Bl1EF83bUwG9V9wFjMvfsaMCMm962CvdtSbqO0mlWcIE3-tLCT3Onk6vMyEhcfJmhm1LF-63wdYYcX-S1SuFwxjRaV-JbOmIMs312yxsqd3RQdeJ0-SVhVvOUEvqX-PtYZOSe1mMcP2GNpeUl1OFywWQaTWuFfLo-sQyECtFNcOaJdQMlZJyKtxCgyGyraUFfhpJzB4WzYuCtBwazlpLcLAcDVMOdXvpBSQyJN9irTzpe2dF3b5q2wSbSoI58V7Mb06eaDeRQCmi8pmrlCnwqJFlxrD07UzsDUk9eWWLEAeVIsJwmKZglsWPooRgzO8FhQtgkAAxim6HXQvocKrVcdTJm1TGob2Wb7N7rJ22xzSSTnz9aMz4-x-B2AhVzXdL90D2cEJYjLx9UEkuMhbgtizqL0MidMSsBL8TOMIp2QfXDpf2ywQW5QktnKB2BXZpBILD4z5RGXJOTWdOkAdYAJzMA4xRm8dqigaxfst9ZGE07mxrebAT1PrDzZoZpPBEuIqkzLUYU7daZmCCdLF5EDSvut8lQOY0R9nvdoOBTXQCrB5DunLtcv2EreFdUgWdj1IhCAqHmJgND0jdWO9rafAeDmPOSYTsF09oUqdmFL52oNPYKGnnyuJJLC3ehCqz-wbfejO3zm22pfal0X_k

3. https://www.dovepress.com/tactical-considerations-for-designing-real-world-studies-fit-for-purpo-peer-reviewed-fulltext-article-POR

4. https://www.e-jcpp.org/journal/view.php?doi=10.36011%2Fcpp.2020.2.e12