What R WE reading in the Journal of Comparative Effectiveness Research? A look at the top 6 most read articles from the journal in 2022
In this Editorial, we share the most read* content of 2022 published in our partner journal, the Journal of Comparative Effectiveness Research.
With comparative effectiveness research (CER) based on real-world data (RWD) providing complementary information to randomized controlled trials (RCTs), there are several methods in which this data is generated. Amongst these are propensity score matching and inverse probability of treatment weighting, which are used to control for confounding by indication in observational studies. In their review, Allan and co-authors describe the fundamentals of these two methods and their applicability in comparing the effectiveness and safety of oral anticoagulants.
This study was published in the journal in January 2022 and is also listed as one of the top 5 most-read articles on spinal muscular atrophy (SMA) in 2022. In the absence of head-to-head randomized trials comparing the efficacy of disease-modifying therapies in SMA, the authors carried out an indirect treatment comparison to estimate the relative effectiveness of risdiplam versus nusinersen and onasemnogene abeparvovec for the treatment of Types 1–3 SMA based on currently available published clinical trial evidence.
Nonrandomized studies (NRS) are becoming more frequently used in health technology assessment (HTA) evaluations, but unmeasured confounding can lead to biased treatment effect estimates, which is a concern for HTA agencies. To address this concern, quantitative bias analysis methods have been developed to quantify the impact of unmeasured confounding and adjust effect estimates from NRS. In this Special Report, the authors consider the importance of balance between methodological complexity and ease of use whilst ensuring that the methods fit within the existing frameworks used by HTA bodies.
Learn more about this publication in our ‘Peek Behind the Paper’ interview Seamus Kent of NICE here.
In their regular feature, Alex Simpson and Sreeram Ramagopalan provide an overview of developments in RWE relating to HTA. In part 8 of this series from April 2022, they summarize the draft NICE RWE framework, which has now been published in full. Not unsurprising, with the ever-increasing interest in how RWE can be used in regulatory decision-making, this was one of the top read articles from 2022.
Other industry updates by Sreeram Ramagopalan published on The Evidence Base can be found here.
The latest update of the Association of British Neurologists (ABN) guidelines on the use of disease-modifying therapies (DMTs) for people with multiple sclerosis (PwMS) took place in 2015. These guidelines classify DMTs according to their average relapse reduction. Several new DMTs have become available since, which have not yet been categorised in this way and the authors looked to address this in this article. They used two approaches to classify the new DMTs. The first approach aligns closely with ABN guidelines and considers the relative reduction in relapse risk by evaluating direct comparative results from relevant randomized controlled trials and using the reported annualized relapse rate (ARR) as a measure. The second approach uses classification probabilities based on comparisons against placebo from a network meta-analysis, also using ARR as the outcome. Overall, their study provides important evidence regarding the relative efficacy of contemporary therapies compared with older approved treatments for PwMS.
Originally published in February 2019, this narrative review stands the test of time. The authors present the advantages and disadvantages of the methods for criteria weighting, a key element of multicriteria decision analysis that is becoming extensively used in healthcare decision-making. In comparing these methods, they also provide guidance for those selecting the most appropriate methodology for a given decision problem in low- and middle-income settings.
Overall, the most read content from 2022 again highlights the broad range of interests of our readers. If you’re interested in contributing to the Journal of Comparative Effectiveness Research, please reach out to our Editor, Laura Dormer, with your pre-submission enquiry.
* All article views are correct as of the publication date of this Editorial.