Real-world evidence and product development: Opportunities, challenges and risk mitigation

Naidoo, Poobalan; Bouharati, Célia; Rambiritch, Virendra; Jose, Nadina; Karamchand, Sumanth; Chilton, Robert; Leisegang, Rory

doi:10.1007/s00508-021-01851-w

Cited by 30 publications

(17 citation statements)

References 22 publications

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“…It is based on larger data than research using primary data collection, with a relatively low cost, once the platform is established. Demands of rapid RWE in post-marketing safety surveillance are increasing considerably after the COVID-19 pandemic (Naidoo et al, 2021 ). For example, a study to explore the frequency and severity of myocarditis after COVID-19 vaccination used the data until May 24, 2021 from the database of Clalit Health Services in Israel, and the results were published in the New England Journal of Medicine just after about 4.5 months from the data period (Barda et al, 2021 ).…”

Section: Discussionmentioning

confidence: 99%

Real world data and data science in medical research: present and future

Togo

Yonemoto

2022

Jpn J Stat Data Sci

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Real world data (RWD) are generating greater interest in recent times despite being not new. There are various purposes of the RWD analytics in medical research as follows: effectiveness and safety of medical treatment, epidemiology such as incidence and prevalence of disease, burden of disease, quality of life and activity of daily living, medical costs, etc. The RWD research in medicine is a mixture of digital transformation, statistics or data science, public health, and regulatory science. Most of the articles describing the RWD or real-world evidence (RWE) in medical research cover only a portion of these specializations, which might lead to an incomplete understanding of the RWD. This article summarizes the overview and challenges of the RWD analysis in medical fields from methodological perspectives. As the first step for the RWD analysis, data source of the RWD should be comprehended. The progress of the RWD is closely related to the digitization, especially of medical administrative data and medical records. Second, the selection of appropriate statistical and epidemiological methods is highly critical for an RWD analysis than those for randomized clinical trials. This is because it contains greater varieties of bias, which should be controlled by balancing the underlying risk between treatment groups. Last, the future of the RWD is discussed in terms of overcoming limited data by proxy confounders, using unstructured text data, linking of multiple databases, using the RWD or RWE for a regulatory purpose, and evaluating values and new aspects in medical research brought by the RWD.

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Section: Discussionmentioning

confidence: 99%

Real world data and data science in medical research: present and future

Togo

Yonemoto

2022

Jpn J Stat Data Sci

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“…Drugs commercialization is based on clinical trials, which are developed with that molecule in a population with speci c characteristics. However, it is important to develop real-world evidence (RWE) studies because their results can improve patient care by complementing information obtained from traditional clinical trial programs 9 .…”

Section: Discussionmentioning

confidence: 99%

Real-World Study of HIV Patients Starting Antiretroviral Treatment With Boosted Protease Inhibitors in the Integrase Inhibitors Era

Pardo¹,

Salillas²,

Jiménez-Nácher³

et al. 2022

Preprint

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Background Boosted-protease inhibitors (b-PIs) are not recommended for initial therapy in HIV patients, but there is still a cohort of patients who could benefit from this antiretroviral treatment (ART). The purpose of this study was to analyze the profile of HIV patients who receive for the first time an ART containing two nucleoside reverse transcriptase inhibitors (NRTIs) and one b-PI. Efficacy and safety of this treatment, as well as the reasons for starting and changing it, were evaluated. Methods An observational, retrospective study was performed on the use of b-PIs in adult HIV patients. Individuals who started treatment with two NRTIs (emtricitabine and tenofovir alafenamide) and one b-PI (darunavir combined with cobicistat or ritonavir) between October 2019 and September 2021 were eligible for our study. This study was retrospectively registered by local Ethics Committee of our hospital (protocol number: PI-5066) on January 13th 2022. Results Overall, 52 patients started ART with two NRTIs plus one b-PIs. Only 8% of them were naïve, 21% received already two NRTIs plus a b-PI and 71% received other regimens. Reasons to start PIs were adverse effects to previous treatment and previous treatment failure. Most patients continued PIs treatment during the 2 years follow-up period and only six of them withdrew it. Conclusions This study analyzes the subgroup of patients that could benefit from treatment with b-PIs in the current era of integrase inhibitors. Even though its use is minority, its efficacy is high especially in patients with toxicity, therapeutic failure or poor adherence to other ARTs.

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“…Real-world data (RWD) consist of information collected during routine clinical, counseling, or coaching practice [ 59 - 61 ]. Real-world evidence (RWE) originates from studies based on valid RWD collected outside traditional controlled clinical trial programs.…”

Section: Discussionmentioning

confidence: 99%

“…RWD, however, involves inherent biases and must be fit for the purpose of generating RWE. Challenges of RWD include data quality, reproducibility, replicability, and accuracy, which may affect validity [ 59 ].…”

Section: Discussionmentioning

confidence: 99%

Success4life Youth Empowerment for Promoting Well-being and Boosting Mental Health: Protocol for an Experimental Study

Setia¹,

Furtner²,

Bendahmane³

et al. 2022

JMIR Res Protoc

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Background There is an increasingly alarming worsening of mental health among the youth. There remain significant unmet needs for developing innovative, evidence-based technology–enhanced, positive psychology interventions (PPIs) all-inclusive in targeting psychological distress and risk factors related to high-risk behavior commonly encountered in adolescents. Objective We aim to assess the effectiveness of a hybrid (incorporating both synchronous and asynchronous learning) and holistic (targeting social and emotional learning and tackling risk factors unique for this age group) PPI, “success4life youth empowerment,” in improving well-being in the youth. Methods Students’ well-being will be assessed by the 5-item World Health Organization Well-Being Index, and hope will be assessed by the 6-item Children’s Hope Scale at week 0, week 8, and week 10, month 6, and month 12. Any improvement in well-being and hope will be measured, estimating the difference in postintervention (week 8 and week 10) and preintervention (week 0) scores by determining the P value and effect size using appropriate statistical tests. Results This study includes 2 phases: pilot phase 1, delivered by the creators of the succcess4life youth empowerment modules and platform, and phase 2, which will consist of the estimation of scalability through the recruitment of trainers. We hope to start student recruitment by 2022 and aim to complete the results for phase 1 pilot testing by 2023. Conclusions We anticipate that a primarily web-based, 10-week holistic PPI can support improvement in the mental wellness of the youth and has the potential for effective scalability. International Registered Report Identifier (IRRID) PRR1-10.2196/38463

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Real-world evidence and product development: Opportunities, challenges and risk mitigation

Cited by 30 publications

References 22 publications

Real world data and data science in medical research: present and future

Real world data and data science in medical research: present and future

Real-World Study of HIV Patients Starting Antiretroviral Treatment With Boosted Protease Inhibitors in the Integrase Inhibitors Era

Success4life Youth Empowerment for Promoting Well-being and Boosting Mental Health: Protocol for an Experimental Study

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