Real-world propensity score comparison of treatment effectiveness of peginterferon beta-1a vs. subcutaneous interferon beta-1a, glatiramer acetate, and teriflunomide in patients with relapsing-remitting multiple sclerosis

Reder, Anthony T.; Arndt, Nancy; Roman, Cortnee; Geremakis, Caroline; Mendoza, Jason P.; Su, Ray; Makin, Charles; Avila, Robin; Vignos, Megan

doi:10.1016/j.msard.2021.102935

Cited by 4 publications

(6 citation statements)

References 20 publications

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“…The systematic search identified 8048 records (Data S1). Based on assessment of 242 full‐text articles, we considered 48 studies eligible for analysis: 22 randomized controlled trials and 26 observational studies 17,33–79 …”

Section: Resultsmentioning

confidence: 99%

Do efficacy results obtained from randomized controlled trials translate to effectiveness data from observational studies for relapsing–remitting multiple sclerosis?

Verweij,

Ahmed,

Zhou

et al. 2024

Pharmacoepidemiology and Drug

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BackgroundRandomized controlled trials are considered the gold standard in regulatory decision making, as observational studies are known to have important methodological limitations. However, real‐world evidence may be helpful in specific situations. This review investigates how the effect estimates obtained from randomized controlled trials compare to those obtained from observational studies, using drug therapy for relapsing–remitting multiple sclerosis as an example.Study Design and SettingA systematic review of randomized controlled trials and observational studies was conducted. The primary outcome was the annualized relapse rate. Using (network) meta‐analysis together with posterior predictive distributions, the drug‐specific rate ratios from the network of randomized controlled trials were compared with those from the network of observational studies.ResultsEffect estimates from 26 observational studies showed greater magnitudes and were less precise compared to estimates obtained from 21 randomized controlled trials. Twenty of the 28 treatment comparisons between designs had similar rate ratios. Seven inconsistencies in observed rate ratios could be attributed to two specific disease‐modifying therapies.ConclusionIn this case study, estimates from observational studies predominantly agreed with estimates from randomized controlled trials given their posterior predictive distributions. Multiple observational studies together may therefore supplement additional pivotal randomized controlled trials in relapsing–remitting multiple sclerosis, for instance facilitating the extrapolation of trial results to the broader patient population.

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Section: Resultsmentioning

confidence: 99%

Do efficacy results obtained from randomized controlled trials translate to effectiveness data from observational studies for relapsing–remitting multiple sclerosis?

Verweij,

Ahmed,

Zhou

et al. 2024

Pharmacoepidemiology and Drug

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“…In the 1990s, recombinant versions of IFN-β replaced crude extracts of purified natural fibroblast IFN-β such as Frone®, which was administered via subcutaneous (sc) injection [ 23 , 24 ]. Recombinant IFN-β, administered parenterally, was the first treatment to demonstrate measurable clinical benefit in improving the natural history of MS in patients with relapsing MS [ 11 , 13 ]; it prevented and shortened relapses, limited the formation of new brain lesions seen on magnetic resonance imaging (MRI), and slowed disability progression [ 13 , 25 ]. In 1993, IFN-β-1b (Betaseron®, delivered by sc injection) [ 26 ] became the first recombinant IFN-β and the first DMT to be approved by the FDA for MS treatment [ 11 , 13 ].…”

Section: Ifn In Msmentioning

confidence: 99%

“…The hallmark of MS is autoimmune inflammation that targets the CNS. While the exact cause of MS remains unknown, adaptive immunity appears to play a prominent role in the pathogenesis of RRMS, whereas innate immune responses contribute to SPMS and are likely to contribute to CNS damage throughout different phases of MS [ 11 , 13 , 25 ]. MS is thought to be triggered and perpetuated by overactivity and/or regulatory/homeostatic imbalance of the immune system [ 13 ].…”

Section: Mechanism Of Action Of Interferons In Msmentioning

confidence: 99%

See 1 more Smart Citation

Twenty Years of Subcutaneous Interferon-Beta-1a for Multiple Sclerosis: Contemporary Perspectives

Freedman,

Coyle,

Hellwig

et al. 2024

Neurol Ther

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Multiple sclerosis (MS) is a chronic, progressive, inflammatory disorder of the central nervous system. Relapsing–remitting MS (RRMS), the most common form of the disease, is characterized by transient neurological dysfunction with concurrent accumulation of disability. Over the past three decades, disease-modifying therapies (DMTs) capable of reducing the frequency of relapses and slowing disability worsening have been studied and approved for use in patients with RRMS. The first DMTs were interferon-betas (IFN-βs), which were approved in the 1990s. Among them was IFN-β-1a for subcutaneous (sc) injection (Rebif ® ), which was approved for the treatment of MS in Europe and Canada in 1998 and in the USA in 2002. Twenty years of clinical data and experience have supported the efficacy and safety of IFN-β-1a sc in the treatment of RRMS, including pivotal trials, real-world data, and extension studies lasting up to 15 years past initial treatment. Today, IFN-β-1a sc remains an important therapeutic option in clinical use, especially around pregnancy planning and lactation, and may also be considered for aging patients, in which MS activity declines and long-term immunosuppression associated with some alternative therapies is a concern. In addition, IFN-β-1a sc is used as a comparator in many clinical studies and provides a framework for research into the mechanisms by which MS begins and progresses.

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“…A multi-center, randomized head-to-head comparison of subcutaneous interferon beta vs. GA (REGARD study) did not show significant differences between the two drugs in time to first relapse (37). A real-world study of a large United States healthcare claims database using propensity score matching showed mildly lower annualized relapse rate comparing pegylated interferon 1a and GA (least square means ratio 0.809, 95% CI 0.67-0.97, p = 0.027) but no difference in healthcare resource utilization (inpatient stays p=0.83, durable medical equipment p = 0.29) (38). Another comparison using MSBase registry data using propensity-score matching demonstrated slightly lower relapse incidence in patients treated with GA and subcutaneous interferon beta-1a compared to intramuscular IFN beta-1a and IFN beta-1b (p < 0.001) though no differences in 12-month disability progression (39).…”

Section: Interferons Vs Gamentioning

confidence: 99%

Therapeutic Advances in Multiple Sclerosis

et al. 2022

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Multiple sclerosis (MS) is an autoimmune disease affecting the central nervous system that causes significant disability and healthcare burden. The treatment of MS has evolved over the past three decades with development of new, high efficacy disease modifying therapies targeting various mechanisms including immune modulation, immune cell suppression or depletion and enhanced immune cell sequestration. Emerging therapies include CNS-penetrant Bruton's tyrosine kinase inhibitors and autologous hematopoietic stem cell transplantation as well as therapies aimed at remyelination or neuroprotection. Therapy development for progressive MS has been more challenging with limited efficacy of current approved agents for inactive disease and older patients with MS. The aim of this review is to provide a broad overview of the current therapeutic landscape for MS.

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Real-world propensity score comparison of treatment effectiveness of peginterferon beta-1a vs. subcutaneous interferon beta-1a, glatiramer acetate, and teriflunomide in patients with relapsing-remitting multiple sclerosis

Abstract: Real-world propensity score comparison of treatment effectiveness of peginterferon beta-1a vs. subcutaneous interferon beta-1a, glatiramer acetate, and teriflunomide in patients with relapsing-remitting multiple sclerosis

Cited by 4 publications

References 20 publications

Do efficacy results obtained from randomized controlled trials translate to effectiveness data from observational studies for relapsing–remitting multiple sclerosis?

Do efficacy results obtained from randomized controlled trials translate to effectiveness data from observational studies for relapsing–remitting multiple sclerosis?

Twenty Years of Subcutaneous Interferon-Beta-1a for Multiple Sclerosis: Contemporary Perspectives

Therapeutic Advances in Multiple Sclerosis

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