2021
DOI: 10.1002/cpt.2399
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Lessons Learned Using Real‐World Data to Emulate Randomized Trials: A Case Study of Treatment Effectiveness for Newly Diagnosed Immune Thrombocytopenia

Abstract: Regulatory agencies are increasingly considering real-world evidence (RWE) to support label expansions of approved medicines. We conducted a comparative effectiveness study to emulate a proposed randomized trial of romiplostim vs. standard-of-care (SOC) therapy among patients with recently diagnosed (≤12 months) immune thrombocytopenia (ITP), that could support expansion of the romiplostim label. We discuss challenges that we encountered and solutions that were developed to address those challenges. Study size… Show more

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Cited by 3 publications
(16 citation statements)
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“…We retrieved 3133 unique records, of which 200 were included in the review (Figure 1). All reasons for excluding records after full-text review are given in eAppendix 6 in Supplement 1.…”
Section: Resultsmentioning
confidence: 99%
See 4 more Smart Citations
“…We retrieved 3133 unique records, of which 200 were included in the review (Figure 1). All reasons for excluding records after full-text review are given in eAppendix 6 in Supplement 1.…”
Section: Resultsmentioning
confidence: 99%
“… The included studies spanned 26 fields of medicine, predominately infectious disease (43 [22%]; 27 [14%] on COVID-19), cardiology (30 [15%]), and oncology (30 [15%]) . One hundred and thirty-one studies (66%)…”
Section: Resultsmentioning
confidence: 99%
See 3 more Smart Citations