Causal inferences and real-world evidence: A comparative effectiveness evaluation of abiraterone acetate against enzalutamide

Johansson, PerUppsala universitet,Institutet för arbetsmarknads- och utbildningspolitisk utvärdering (IFAU),Statistiska institutionen,Hälsoekonomi,Tsinghua University, Beijing, China (författare)

Causal inferences and real-world evidence : A comparative effectiveness evaluation of abiraterone acetate against enzalutamide

Regulatory authorities are recognizing the need for real-world evidence (RWE) as a complement to randomized controlled trials in the approval of drugs. However, RWE needs to be fit for regulatory purposes. There is an ongoing discussion regarding whether pre-publication of a protocol on appropriate repositories, e.g. ClinicalTrials.gov, would increase the quality of RWE or not. This paper illustrates that an observational study based on a pre-published protocol can entail the same level of detail as a protocol for a randomized experiment. The strategy is exemplified by designing a comparative effectiveness evaluation of abiraterone acetate against enzalutamide in clinical practice. These two cancer drugs are prescribed to patients with advanced prostate cancer. Two complementary designs, including pre-analysis plans, were published before data on outcomes and proxy-outcomes were obtained. The underlying assumptions are assessed and both analyses show an increased mortality risk from being prescribed abiraterone acetate compared to enzalutamide.

Jonéus, PaulinaUppsala universitet,Statistiska institutionen(Swepub:uu)paujo877 (författare)
Langenskiöld, Sophie,1968-Uppsala universitet,Hälsoekonomi(Swepub:uu)sopla565 (författare)
Uppsala universitetInstitutet för arbetsmarknads- och utbildningspolitisk utvärdering (IFAU) (creator_code:org_t)

Av författaren/redakt...: Johansson, Per; Jonéus, Paulina; Langenskiöld, So ...

Om ämnet

MEDICIN OCH HÄLSOVETENSKAP: MEDICIN OCH HÄLS ...; och Klinisk medicin; och Cancer och onkol ...

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