[Methodology for analysing quality-of-life data in the benefit assessment of pharmaceuticals].
Early benefit assessment aims to prove a benefit of a new pharmaceutical over the appropriate comparator based on patient-relevant endpoints. In addition to mortality and morbidity, quality of life is a patient-relevant endpoint. Thus, phase III clinical trials are the basis of evidence. But HTA and health authorities attach different importance to quality of life. Using the example of oncology, the challenges with study design and analysis will be discussed. A particular challenge to the analysis of quality-of-life data is varying observation times in treatment arms with different effectiveness. Based on the example of Crizotinib possible solutions will be presented.
Pfizer Deutschland GmbH, Berlin. Electronic address: Friedhelm.Leverkus@pfizer.com.
Friedhelm
F
Leverkus
D000241:Adenosine / Q000009:adverse effects / Q000031:analogs & derivatives / Q000627:therapeutic use; D017326:Clinical Trials, Phase III as Topic; D000077547:Crizotinib; D017277:Drug Approval; D023381:Endpoint Determination / Q000379:methods; D006801:Humans; D053208:Kaplan-Meier Estimate; D009369:Neoplasms / Q000188:drug therapy / Q000401:mortality; D063868:Patient Outcome Assessment; D017060:Patient Satisfaction; D055553:Prescription Drugs / Q000009:adverse effects* / Q000627:therapeutic use*; D047428:Protein Kinase Inhibitors / Q000009:adverse effects / Q000627:therapeutic use; D011720:Pyrazoles / Q000009:adverse effects / Q000627:therapeutic use; D011725:Pyridines / Q000009:adverse effects / Q000627:therapeutic use; D011788:Quality of Life / Q000523:psychology*; D019057:Quality-Adjusted Life Years; D018570:Risk Assessment / Q000379:methods; D000077486:Ticagrelor
D055553:Prescription Drugs; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011725:Pyridines; D000077547:Crizotinib; D000077486:Ticagrelor; D000241:Adenosine
Lebensqualität; Quality of life; Studienplanung; early benefit assessment; frühe Nutzenbewertung; patient-relevant endpoints; patientenrelevante Endpunkte; statistical analysis; statistische Auswertung; study planning