Can real-world data really replace randomised clinical trials?

Ramagopalan, Sreeram V; Simpson, Alex; Sammon, Cormac

doi:10.1186/s12916-019-1481-8

Cited by 51 publications

(48 citation statements)

References 8 publications

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“…In order to achieve their implementation in clinical care, a re-assessment of the standards of evidence sufficient to prove the benefit of precision cancer therapies is needed [ 32 ]. New evidence suggests that appropriately conducted real-world data studies have the potential to support regulatory decisions in the absence of RCT data [ 33 ].…”

Section: Discussionmentioning

confidence: 99%

NGS-guided precision oncology in metastatic breast and gynecological cancer: first experiences at the CCC Munich LMU

Sultova

Westphalen²,

Jung

et al. 2020

Arch Gynecol Obstet

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Purpose Comprehensive genomic profiling identifying actionable molecular alterations aims to enable personalized treatment for cancer patients. The purpose of this analysis was to retrospectively assess the impact of personalized recommendations made by a multidisciplinary tumor board (MTB) on the outcome of patients with breast or gynecological cancers, who had progressed under standard treatment. Here, first experiences of our Comprehensive Cancer Center Molecular Tumor Board are reported. Methods All patients were part of a prospective local registry. 95 patients diagnosed with metastatic breast cancer or gynecological malignancies underwent extended molecular profiling. From May 2017 through March 2019, the MTB reviewed all clinical cases considering tumor profile and evaluated molecular alterations regarding further diagnostic and therapeutic recommendations. Results 95 patients with metastatic breast or gynecological cancers were discussed in the MTB (68% breast cancer, 20% ovarian cancer, 5% cervical cancer, 3% endometrial cancer and 4% others). Genes with highest mutation rate were PIK3CA and ERBB2. Overall, 34 patients (36%) received a biomarker-based targeted therapy recommendation. Therapeutic recommendations were implemented in nine cases; four patients experienced clinical benefit with a partial response or disease stabilization lasting over 4 months. Conclusion In the setting of a multidisciplinary molecular tumor board, a small but clinically meaningful group of breast and gynecological cancer patients benefits from comprehensive genomic profiling. Broad and successful implementation of precision medicine is complicated by patient referral at late stage disease and limited access to targeted agents and early clinical trials. Trial registration number 284-10 (03.05.2018).

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Section: Discussionmentioning

confidence: 99%

NGS-guided precision oncology in metastatic breast and gynecological cancer: first experiences at the CCC Munich LMU

Sultova

Westphalen²,

Jung

et al. 2020

Arch Gynecol Obstet

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“…Moreover, due to the country-size scale of this observational study it is easy to mimic the randomization element of an RTC and properly compare treatment groups, given the number of individuals available to properly adjust for all baseline cofounders [40]. Actually, the use of propensity scores provides additional adjustment to control for confounding variables [41]. Here, as shown in Table 3, confounding effects between the compared groups due to the known variables associated to the outcomes considered can be ruled out.…”

Section: Discussionmentioning

confidence: 99%

Real world evidence of calcifediol use and mortality rate of COVID-19 hospitalized in a large cohort of 16,401 Andalusian patients

Loucera

Peña‐Chilet

Esteban-Medina

et al. 2021

Preprint

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Background: COVID-19 is a major worldwide health problem because of acute respiratory distress syndrome, and mortality. Several lines of evidence have suggested a relationship between the vitamin D endocrine system and severity of COVID-19. Methods: We present a retrospective survival study that includes all Andalusian patients hospitalized between January and November 2020 because of COVID-19 infection. Based on a central registry of electronic health records (the Andalusian Population Health Database, BPS), prescription of vitamin D or its metabolites within 15-30 days before hospitalization were recorded. The effect of treatment with vitamin D metabolites for other indication previous to the hospitalization was studied with respect to patient survival by means of Kaplan-Meyer survival curves and Log Hazard Ratios, using a propensity score to compensate the disbalance of compared classes and the confounding factors. The availability of detailed patient data in the BPS allowed to obtain Real-World Evidence (RWE) of the effects of prior use of vitamin D or its metabolites on the mortality due to COVID-19 infection. Findings: A retrospective cohort of 16.401patients was extracted from the BPS, which includes all the patients hospitalized with COVID-19 diagnosis between January and November 2020 in Andalusia, one of the largest regions in Europe with the size of an average median country. A total of 358 patients were found with cholecalciferol, and 193 with calcifediol, prescriptions 15 days before hospitalization. For a period extended to 30 days before hospitalization, the numbers increase to 416 and 210 and, respectively. Kaplan-Meyer survival curves and hazard ratios support an association between consumption of these metabolites and patient survival. Such association was stronger in calcifediol (Log Hazard Ratio, LHR = -1.27±0.32) than in cholecalciferol (LHR= -0.56±0.15), when prescribed 15 days before hospitalization This effect decreases when a larger 30 days period is considered (calcifediol LHR= -1.01±0.27 and cholecalciferol LHR= -0.27±0.12), suggesting that the closer was the treatment to the hospitalization the stronger the association. Conclusions: A significant reduction in mortality in patients hospitalized with COVID-19 is associated with the prescription of vitamin D, especially calcifediol, within 15-30 days prior to hospitalization.

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“…This is also the case for other social media platforms such as Facebook, Instagram, and Reddit. However, social media data frequently lack demographic indicators and ground truth, possibly resulting in biased or poorly representative samples-particularly when compared to the precisely defined inclusion and exclusion criteria of randomized controlled trials (RCTs) (173). In this section we provide a short overview of the literature related to the challenges of deriving valid and reliable indicators of human behavior from social media data and how these challenges can be mitigated.…”

Section: Limitationsmentioning

confidence: 99%

“…Such subsamples may vary considerably in the degree to which they represent an unbiased sample. Future research using social media data must benefit from the large-scale nature of this real-world data, while specifying more precise inclusion and exclusion criteria, as used in RCTs, to avoid sample biases (173). Getting to that point requires the ability to stratify social media user cohorts using more fine-tuned ML, as well as via greater collaboration with and openness from social media platform providers.…”

Section: Limitationsmentioning

confidence: 99%

Mining Social Media Data for Biomedical Signals and Health-Related Behavior

Correia

Wood

Bollen

et al. 2020

Annu. Rev. Biomed. Data Sci.

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Social media data have been increasingly used to study biomedical and health-related phenomena. From cohort-level discussions of a condition to population-level analyses of sentiment, social media have provided scientists with unprecedented amounts of data to study human behavior associated with a variety of health conditions and medical treatments. Here we review recent work in mining social media for biomedical, epidemiological, and social phenomena information relevant to the multilevel complexity of human health. We pay particular attention to topics where social media data analysis has shown the most progress, including pharmacovigilance and sentiment analysis, especially for mental health. We also discuss a variety of innovative uses of social media data for health-related applications as well as important limitations of social media data access and use.

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Can real-world data really replace randomised clinical trials?

Cited by 51 publications

References 8 publications

NGS-guided precision oncology in metastatic breast and gynecological cancer: first experiences at the CCC Munich LMU

NGS-guided precision oncology in metastatic breast and gynecological cancer: first experiences at the CCC Munich LMU

Real world evidence of calcifediol use and mortality rate of COVID-19 hospitalized in a large cohort of 16,401 Andalusian patients

Mining Social Media Data for Biomedical Signals and Health-Related Behavior

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