BACKGROUND: Evaluation of the clinical importance of outcomes in research studies is an essential element of clinical decision making. OBJECTIVE: To understand how clinicians and trainees weigh the importance of different types of clinical outcomes in drug trials. METHODS: A self-administered paper survey contained 4 scenarios asking participants to rate (1, "no proof" to 10, "good proof") the extent to which 4 study outcomes provided "proof that the new drug might help people." Outcomes included (1) a surrogate outcome; (2) a surrogate-enriched composite outcome; (3) stroke mortality; and (4) all-cause mortality. The primary study metrics were mean ratings for each of the 4 outcome types, and the proportion ranking outcome importance of all-cause mortality > stroke mortality > surrogate-enriched composite or surrogate alone. RESULTS: A convenience sample of 549 clinicians and trainees at 2 medical centers completed the survey (response rate: 87% medical students, 80% internal medicine residents, 69% general medicine faculty, and 41% physician experts). The surrogate-enriched composite outcome and stroke mortality were rated the most important evidence for benefit (6.6 and 6.4, respectively), with all-cause mortality and a surrogate outcome being rated significantly lower (5.2 and 4.6, respectively). In addition, 48% of clinicians rated improvement in all-cause mortality as more valuable than an improvement in a surrogate marker. Only 21% rated all-cause mortality as more valuable than a surrogate-enriched composite outcome. CONCLUSIONS: These findings raise concerns that clinicians and trainees may not interpret trial evidence in a way that promotes the best care for patients.
BACKGROUND: Evaluation of the clinical importance of outcomes in research studies is an essential element of clinical decision making. OBJECTIVE: To understand how clinicians and trainees weigh the importance of different types of clinical outcomes in drug trials. METHODS: A self-administered paper survey contained 4 scenarios asking participants to rate (1, "no proof" to 10, "good proof") the extent to which 4 study outcomes provided "proof that the new drug might help people." Outcomes included (1) a surrogate outcome; (2) a surrogate-enriched composite outcome; (3) stroke mortality; and (4) all-cause mortality. The primary study metrics were mean ratings for each of the 4 outcome types, and the proportion ranking outcome importance of all-cause mortality > stroke mortality > surrogate-enriched composite or surrogate alone. RESULTS: A convenience sample of 549 clinicians and trainees at 2 medical centers completed the survey (response rate: 87% medical students, 80% internal medicine residents, 69% general medicine faculty, and 41% physician experts). The surrogate-enriched composite outcome and stroke mortality were rated the most important evidence for benefit (6.6 and 6.4, respectively), with all-cause mortality and a surrogate outcome being rated significantly lower (5.2 and 4.6, respectively). In addition, 48% of clinicians rated improvement in all-cause mortality as more valuable than an improvement in a surrogate marker. Only 21% rated all-cause mortality as more valuable than a surrogate-enriched composite outcome. CONCLUSIONS: These findings raise concerns that clinicians and trainees may not interpret trial evidence in a way that promotes the best care for patients.
Authors: Ignacio Ferreira-González; Jason W Busse; Diane Heels-Ansdell; Victor M Montori; Elie A Akl; Dianne M Bryant; Pablo Alonso-Coello; Jordi Alonso; Andrew Worster; Suneel Upadhye; Roman Jaeschke; Holger J Schünemann; Gaietà Permanyer-Miralda; Valeria Pacheco-Huergo; Antònia Domingo-Salvany; Ping Wu; Edward J Mills; Gordon H Guyatt Journal: BMJ Date: 2007-04-02
Authors: Tanner J Caverly; Allan V Prochazka; Brandon P Combs; Brian P Lucas; Shane R Mueller; Jean S Kutner; Ingrid Binswanger; Angela Fagerlin; Jacqueline McCormick; Shirley Pfister; Daniel D Matlock Journal: Med Decis Making Date: 2014-11-05 Impact factor: 2.583
Authors: Alexandra Woodbridge; Ann Abraham; Rosa Ahn; Susan Saba; Deborah Korenstein; Erin Madden; Salomeh Keyhani Journal: J Gen Intern Med Date: 2018-03 Impact factor: 5.128