Randomized and non-randomized designs for causal inference with longitudinal data in rare disorders

Abstract In the United States, approximately 7000 rare diseases affect 30 million patients, and only 10% of these diseases have existing therapies. Sound study design and causal inference methods are essential to demonstrate the therapeutic efficacy, safety, and effectiveness of new therapies. In th...

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Détails bibliographiques
Auteurs principaux: Rima Izem, Robert McCarter
Format: article
Langue:EN
Publié: BMC 2021
Sujets:
R
Accès en ligne:https://doaj.org/article/9253ab0790f24de99be7790223c1c8ff
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