title: Recommendations for the design of Phase 3 pharmaceutical trials that are more informative for patients, clinicians, and payers.
abstract: 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.
  
  
  OBJECTIVE
  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.
  
  
  CONCLUSIONS
  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.affiliation: Value Institute, Christiana Care Health System, United States. Electronic address: ssonnad@christianacare.org.
authors.forename: Seema S
authors.initials: SS
authors.lastname: Sonnad
authors.forename: C Daniel
authors.initials: CD
authors.lastname: Mullins
authors.forename: Danielle
authors.initials: D
authors.lastname: Whicher
authors.forename: Jennifer C
authors.initials: JC
authors.lastname: Goldsack
authors.forename: Penny E
authors.initials: PE
authors.lastname: Mohr
authors.forename: Sean R
authors.initials: SR
authors.lastname: Tunis
mesh_terms: D002985:Clinical Protocols; D017326:Clinical Trials, Phase III as Topic / Q000379:methods* / Q000592:standards; D057186:Comparative Effectiveness Research / Q000379:methods / Q000592:standards; D017277:Drug Approval; D004358:Drug Therapy; D015507:Drugs, Investigational / Q000627:therapeutic use*; D006801:Humans; D018579:Patient Selection; D064792:Pragmatic Clinical Trials as Topic / Q000379:methods / Q000592:standards; D016896:Treatment Outcome
chemical_list: D015507:Drugs, Investigational
keywords: Comparative effectiveness; Methods guidance; Pragmatic trials