BACKGROUND: Pharmaceutical pragmatic clinical trials (PCTs) are designed to provide the type of evidence that is desired by patients, clinicians and payers but too often missing from traditional regulatory trials. PURPOSE: This paper presents framework for designing pragmatic trials incorporating evidence desired by post-regulatory decision makers while remaining within acceptable standards for regulatory approval. METHODS: Following a stakeholder meeting convened in May of 2009 to identify gaps in information collected in Phase 3 trials, CMTP staff and the authors drafted recommendations for Pragmatic Phase 3 Pharmaceutical Trials. This draft was circulated first to technical working group members for their comments. After revising the document based on these comments, it was distributed electronically to other select experts and then made available for public comment. The final version of the EGD appears on the CMTP website. RESULTS: The process resulted in a set of 10 recommendations for conducting Phase 3 trials that met regulatory needs while addressing information important to physicians, patients, payers, and policy-makers. These recommendations encompassed three primary areas: generalizability from the trial participants to the clinical population of interest; effectiveness relative to active comparators; and consistently measured relevant outcomes for coverage and treatment decisions. LIMITATIONS: While stakeholders were involved throughout the process, not all recommendations will meet the needs of all stakeholders. CONCLUSIONS: Pragmatic trial design need not be deferred until a product is in widespread use. Incremental movement toward the more pragmatic design of Phase 3 trials is desirable.
BACKGROUND: Pharmaceutical pragmatic clinical trials (PCTs) are designed to provide the type of evidence that is desired by patients, clinicians and payers but too often missing from traditional regulatory trials. PURPOSE: This paper presents framework for designing pragmatic trials incorporating evidence desired by post-regulatory decision makers while remaining within acceptable standards for regulatory approval. METHODS: Following a stakeholder meeting convened in May of 2009 to identify gaps in information collected in Phase 3 trials, CMTP staff and the authors drafted recommendations for Pragmatic Phase 3 Pharmaceutical Trials. This draft was circulated first to technical working group members for their comments. After revising the document based on these comments, it was distributed electronically to other select experts and then made available for public comment. The final version of the EGD appears on the CMTP website. RESULTS: The process resulted in a set of 10 recommendations for conducting Phase 3 trials that met regulatory needs while addressing information important to physicians, patients, payers, and policy-makers. These recommendations encompassed three primary areas: generalizability from the trial participants to the clinical population of interest; effectiveness relative to active comparators; and consistently measured relevant outcomes for coverage and treatment decisions. LIMITATIONS: While stakeholders were involved throughout the process, not all recommendations will meet the needs of all stakeholders. CONCLUSIONS: Pragmatic trial design need not be deferred until a product is in widespread use. Incremental movement toward the more pragmatic design of Phase 3 trials is desirable.
Authors: Christine L M Joseph; Dennis R Ownby; Suzanne L Havstad; Jacqueline Saltzgaber; Shannon Considine; Dayna Johnson; Ed Peterson; Gwen Alexander; Mei Lu; Wanda Gibson-Scipio; Christine Cole Johnson Journal: J Adolesc Health Date: 2012-09-27 Impact factor: 5.012