Real-world Evidence Needs Careful Interpretation

Nash, Peter

doi:10.3899/jrheum.201004

Cited by 3 publications

(2 citation statements)

References 8 publications

(5 reference statements)

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“…Whereas "real-world studies," providing evidence on treatment outcomes in clinical practice are increasingly recognized as necessary complementary information sources for patients not included in clinical trials, 1,2 they are also criticized for their susceptibility to bias. 3 Comparative effectiveness studies using real-world data are the main target of such criticism because in clinical practice treatment decisions are mainly based on perceived predictors of treatment benefit, introducing confounding by indication. 4 In the treatment of rheumatoid arthritis (RA), for example, switching to a second conventional synthetic disease-modifying drug (csDMARD) or to a combination of csDMARDs is recommended for patients who have failed initial methotrexate (MTX) and who do not have indicators of a poor prognosis, whereas switching to a biological DMARD (bDMARD) is recommended for patients with poor prognosis.…”

Section: Articlementioning

confidence: 99%

“…Randomized controlled trials (RCTs) are the gold standard for evaluating the relative efficacy and safety of drugs, but practical constraints preclude well‐powered head‐to‐head RCTs of all therapeutic options in all clinically relevant patient groups. Whereas “real‐world studies,” providing evidence on treatment outcomes in clinical practice are increasingly recognized as necessary complementary information sources for patients not included in clinical trials, 1,2 they are also criticized for their susceptibility to bias 3 . Comparative effectiveness studies using real‐world data are the main target of such criticism because in clinical practice treatment decisions are mainly based on perceived predictors of treatment benefit, introducing confounding by indication 4 .…”

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confidence: 99%

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Combined Conventional Synthetic Disease Modifying Therapy vs. Infliximab for Rheumatoid Arthritis: Emulating a Randomized Trial in Observational Data

Barbulescu

Askling

Saevarsdóttir

et al. 2022

Clin Pharma and Therapeutics

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Observational studies are often considered unreliable for evaluating relative treatment effectiveness, but it has been suggested that following target trial protocols could reduce bias. Using observational data from patients with rheumatoid arthritis (RA) in the Swedish Rheumatology Quality Register (SRQ), between 2006 and 2020, we emulated the protocol of the Swedish Farmacotherapy trial (SWEFOT) and compared the results. SWEFOT was a pragmatic trial nested in SRQ, between 2002 and 2005, where methotrexate (MTX) insufficient responders were randomized to receive additional infliximab or sulfasalazine (SSZ) + hydroxychloroquine (HCQ). Patients with RA initiating infliximab (N = 313) or SSZ + HCQ (N = 196) after MTX were identified in SRQ and the Prescribed Drugs Register, mimicking the SWEFOT eligibility criteria. The primary outcome was the proportion of European Alliance of Associations for Rheumatology (EULAR) good responders at 9 months, classifying patients who discontinued treatment as "nonresponders." Through sensitivity analyses, we assessed the impact of relaxing eligibility criteria. The observed proportions reaching EULAR good response were close to those reported in SWEFOT: 39% (vs. 39% in SWEFOT) for infliximab and 28% (vs. 25%) for SSZ + HCQ. The crude observed response ratio was 1.39 (95% confidence interval (CI) 1.04-1.86), increasing to 1.48 (95% CI 0.98-2.24) after confounding adjustment, compared to 1.59 (95% CI 1.10-2.30) in SWEFOT. Results remained close to SWEFOT when relaxing eligibility criteria until allowing prior disease-modifying anti-rheumatic drug (DMARD) use which reduced the observed difference between treatments. By applying a prespecified trial emulation protocol to observational clinical registry data, we could replicate the results of SWEFOT, favoring infliximab over SSZ + HCQ combination therapy at 9 months.

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Section: Articlementioning

confidence: 99%

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confidence: 99%

Combined Conventional Synthetic Disease Modifying Therapy vs. Infliximab for Rheumatoid Arthritis: Emulating a Randomized Trial in Observational Data

Barbulescu

Askling

Saevarsdóttir

et al. 2022

Clin Pharma and Therapeutics

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Harnessing of real-world data and real-world evidence using digital tools: utility and potential models in rheumatology practice

Kataria

Ravindran²

2021

Rheumatology

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The diversity of diseases in rheumatology and variability in disease prevalence necessitates greater data parity in disease presentation, treatment responses including adverse events to drugs and various co-morbidities. Randomized Controlled Trials (RCTs) are the gold standard for drug development and performance evaluation. However, when the drug is applied outside the controlled environment the outcomes may differ in patient population. In this context, the need to understand the macro and micro changes involved in disease evolution and progression becomes important and so is the need for harvesting and harnessing the Real-World Data (RWD) from various resources to use them in generating Real World Evidence (RWE). Digital tools with potential relevance to rheumatology can be potentially leveraged to obtain greater patient insights, greater information on disease progression and disease micro processes and even in the early diagnosis of diseases. Since the patients spend only a minuscule proportion of their time in hospital or in a clinic, using the modern digital tools to generate realistic, bias proof RWD in non-invasive patient friendly manner becomes critical. In this review we have appraised different digital mediums and mechanisms for collecting RWD and proposed digital care models for generating RWE in rheumatology.

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Early experience of COVID-19 vaccination in adults with systemic rheumatic diseases: results from the COVID-19 Global Rheumatology Alliance Vaccine Survey

et al. 2021

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BackgroundWe describe the early experiences of adults with systemic rheumatic disease who received the COVID-19 vaccine.MethodsFrom 2 April to 30 April 2021, we conducted an online, international survey of adults with systemic rheumatic disease who received COVID-19 vaccination. We collected patient-reported data on clinician communication, beliefs and intent about discontinuing disease-modifying antirheumatic drugs (DMARDs) around the time of vaccination, and patient-reported adverse events after vaccination.ResultsWe analysed 2860 adults with systemic rheumatic diseases who received COVID-19 vaccination (mean age 55.3 years, 86.7% female, 86.3% white). Types of COVID-19 vaccines were Pfizer-BioNTech (53.2%), Oxford/AstraZeneca (22.6%), Moderna (21.3%), Janssen/Johnson & Johnson (1.7%) and others (1.2%). The most common rheumatic disease was rheumatoid arthritis (42.3%), and 81.2% of respondents were on a DMARD. The majority (81.9%) reported communicating with clinicians about vaccination. Most (66.9%) were willing to temporarily discontinue DMARDs to improve vaccine efficacy, although many (44.3%) were concerned about rheumatic disease flares. After vaccination, the most reported patient-reported adverse events were fatigue/somnolence (33.4%), headache (27.7%), muscle/joint pains (22.8%) and fever/chills (19.9%). Rheumatic disease flares that required medication changes occurred in 4.6%.ConclusionAmong adults with systemic rheumatic disease who received COVID-19 vaccination, patient-reported adverse events were typical of those reported in the general population. Most patients were willing to temporarily discontinue DMARDs to improve vaccine efficacy. The relatively low frequency of rheumatic disease flare requiring medications was reassuring.

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Real-world Evidence Needs Careful Interpretation

Cited by 3 publications

References 8 publications

Combined Conventional Synthetic Disease Modifying Therapy vs. Infliximab for Rheumatoid Arthritis: Emulating a Randomized Trial in Observational Data

Combined Conventional Synthetic Disease Modifying Therapy vs. Infliximab for Rheumatoid Arthritis: Emulating a Randomized Trial in Observational Data

Harnessing of real-world data and real-world evidence using digital tools: utility and potential models in rheumatology practice

Early experience of COVID-19 vaccination in adults with systemic rheumatic diseases: results from the COVID-19 Global Rheumatology Alliance Vaccine Survey

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