Key considerations in the design of real-world studies

Fang, Yixin; He, Weili; Wang, Hongwei; Wu, Meijing

doi:10.1016/j.cct.2020.106091

Cited by 16 publications

(6 citation statements)

References 22 publications

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“…Discrepancies in inclusion criteria between the two studies were expected to lead to differences in the distribution of some covariates between the two studies; such imbalances were typically accommodated by weighting [24, 25], as discussed below; the remaining discrepancies could not be resolved but were expected to have little effect (see the Discussion section and the Supplement).…”

Section: Methodsmentioning

confidence: 99%

Estimating the Effect of PrEP in Black Men Who Have Sex with Men: A Framework to Utilize Data from Multiple Non-Randomized Studies to Estimate Causal Effects

Meisner,

Xia,

Chan

et al. 2024

Preprint

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Black men who have sex with men (MSM) are disproportionately burdened by the HIV epidemic in the US. The effectiveness of pre-exposure prophylaxis (PrEP) in preventing HIV infection has been demonstrated through randomized placebo-controlled clinical trials in several populations. Importantly, no such trial has been conducted exclusively among Black MSM in the US, and it would be unethical and infeasible to do so now. To estimate the causal effects of PrEP access, initiation, and adherence on HIV risk, we utilized causal inference methods to combine data from two non-randomized studies that exclusively enrolled Black MSM. The estimated relative risks of HIV were: (i) 0.52 (95% confidence interval: 0.21, 1.22) for individuals with versus without PrEP access, (ii) 0.48 (0.12, 0.89) for individuals who initiated PrEP but were not adherent versus those who did not initiate, and (iii) 0.23 (0.02, 0.80) for individuals who were adherent to PrEP versus those who did not initiate. Beyond addressing the knowledge gap around the effect of PrEP in Black MSM in the US, which may have ramifications for public health, we have provided a framework to combine data from multiple non-randomized studies to estimate causal effects, which has broad utility.

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Section: Methodsmentioning

confidence: 99%

Estimating the Effect of PrEP in Black Men Who Have Sex with Men: A Framework to Utilize Data from Multiple Non-Randomized Studies to Estimate Causal Effects

Meisner,

Xia,

Chan

et al. 2024

Preprint

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“…Selection of which method to be used can vary based on the study question, sample size, and study design. 28 …”

Section: How Do You Determine High-quality Rwe?mentioning

confidence: 99%

Old dog with new tricks: An introduction to real-world evidence for pharmacists

Rahmatian

Tadrous

2021

American Journal of Health-System Pharmacy

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Disclaimer In an effort to expedite the publication of articles related to the COVID-19 pandemic, AJHP is posting these manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time.

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“…The only way to assess treatment effectiveness is through real-world studies even though they are not designed to evaluate comparative effectiveness and suffer from bias and confounding factors ( 12 ). Real-world studies are nevertheless important for the following:…”

Section: Introductionmentioning

confidence: 99%

A real-world retrospective, observational study of first-line pembrolizumab plus chemotherapy for metastatic non-squamous non-small cell lung cancer with PD-L1 tumor proportion score < 50% (PEMBROREAL)

Cafaro,

Foca,

Nanni

et al. 2024

Front. Oncol.

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IntroductionThe phase III Keynote-189 trial established a first-line treatment combining pembrolizumab with pemetrexed and platinum as a standard treatment for patients with stage IV non-small cell lung cancer (NSCLC) without known EGFR and ALK driver mutations and independent of programmed cell death ligand 1 (PD-L1) expression. However, in Italy, eligibility for the National Health Service payment program is limited to patients with PD-L1 <50%. The PEMBROREAL study assesses the real-world effectiveness and safety of pembrolizumab in patients eligible for the National Health Service payment program.MethodsPEMBROREAL is a retrospective, observational study on patients with NSCLC who started pembrolizumab combined with pemetrexed and platinum within the reimbursability time window, considered as December 2019 to December 2020. The primary endpoints were to assess progression-free survival (PFS) and overall survival (OS; using the Kaplan–Meier method), response to therapy, and tolerability.ResultsUntil February 2022, 279 patients (median follow-up: 19.7 months) have been observed. The median PFS was 8.0 months (95% confidence interval: 6.5–9.2). OS was not reached, but we can estimate a 12- to 24-month survival rate for the combined treatment: 66.1% and 52.5%, respectively. PD-L1 expression and Eastern Cooperative Group (ECOG) Performance Status were both associated with PFS and OS. Overall, only 44.4% of patients reported an adverse event, whereas toxicity led to a 5.4% discontinuation rate.ConclusionThe results of the PEMBROREAL study have shown that the combined treatment of pembrolizumab with pemetrexed and platinum is effective for metastatic non-squamous NSCLC, even for patients with PD-L1 levels below 50%, despite the differences in patient demographics and pathological features compared to the Keynote-189 study. The adverse events reported during the study were more typical of chemotherapy treatment rather than immunotherapy, and physicians were able to manage them easily.

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Key considerations in the design of real-world studies

Cited by 16 publications

References 22 publications

Estimating the Effect of PrEP in Black Men Who Have Sex with Men: A Framework to Utilize Data from Multiple Non-Randomized Studies to Estimate Causal Effects

Estimating the Effect of PrEP in Black Men Who Have Sex with Men: A Framework to Utilize Data from Multiple Non-Randomized Studies to Estimate Causal Effects

Old dog with new tricks: An introduction to real-world evidence for pharmacists

A real-world retrospective, observational study of first-line pembrolizumab plus chemotherapy for metastatic non-squamous non-small cell lung cancer with PD-L1 tumor proportion score < 50% (PEMBROREAL)

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