Patient Perceptions of FDA Approval: Gaps in Education or Variation in Values?

2021 ◽  
pp. 10.1212/CPJ.0000000000001034
Author(s):  
Paul J. Ford ◽  
Robert J. Fox ◽  
Mary Beth Mercer ◽  
Stacey S. Cofield
Keyword(s):  
Drug Approval ◽  
Patient Perceptions ◽  
Approval Process ◽  
Inclusion Criteria ◽  
Web Based ◽  
Risks And Benefits ◽  
Disease Modifying Therapies ◽  
Fda Approval ◽  
Disease Modifying

Abstract:Objective:To assess perceptions and opinions about the FDA approval process for disease modifying therapies (DMT) in people living with multiple sclerosis (MS).Methods:People living with MS were invited to complete a web-based survey of their perceptions of the FDA role and process for approval of MS medications. The survey asked about the role of the FDA, factors involved in the approval process, which voices should represent those with MS in deliberations about drug approval, and the level of comfort with uncertain safety of newly-approved therapies.Results:3533 respondents met inclusion criteria for data analysis. Most respondents appeared to understand the role of the FDA, although only half understood a fundamental FDA role: balancing the risks and benefits when considering drug approval. Significant differences were observed in many areas between those who have and have not tried DMTs. Comfort with uncertainty was associated with several factors relating to side effects and benefits thought important for the FDA to consider. Most respondents reported that people who participated in the medication’s clinical trial were particularly able to represent people living with MS.Conclusion:Perceptions regarding the FDA and views of who should represent people living with MS varied between those who have and have not tried DMT. There is variability in personal values that should be recognized and taken into account when considering regulatory responsibilities. Interventions are needed to address educational gaps regarding the mission and trustworthiness of the FDA as an oversight body.

Perspectives of individuals with multiple sclerosis on discontinuation of disease-modifying therapies

2019 ◽  
Vol 26 (12) ◽  
pp. 1581-1589
Author(s):  
Marisa P McGinley ◽  
Philip A Cola ◽  
Robert J Fox ◽  
Jeffrey A Cohen ◽  
John J Corboy ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Decision Making ◽  
Structural Equation ◽  
Descriptive Analysis ◽  
Equation Modeling ◽  
Decision Making Process ◽  
Inclusion Criteria ◽  
Research Committee ◽  
Disease Modifying Therapies ◽  
Disease Modifying

Background: Therapeutic research in multiple sclerosis (MS) has focused on the development of treatments with little investigation regarding the possibility of discontinuation of disease-modifying therapies (DMTs). Objective: To understand the opinion of individuals with MS concerning stopping DMTs and the factors that influence the decision-making process. Methods: A mixed method approach was used starting with three focus groups from which a survey was developed. This survey was sent to 1000 participants in the North American Research Committee on Multiple Sclerosis registry who met inclusion criteria (age ⩾45 years; on most recent DMT for ⩾5 years). Descriptive analysis and structural equation modeling were used. Results: Of 1000 participants receiving the survey, 377 provided complete responses and met inclusion criteria. Only 11.9% of participants reported that if their disease was considered stable, they would consider coming off medications. A high level of external locus of control in influential others such as physicians significantly decreased the likelihood of considering discontinuation. Conclusions: Most individuals with MS report being unlikely to consider stopping MS therapy if their disease was considered “non-active.” As the results of studies concerning DMT discontinuation are obtained, information from providers will be an important part of individuals’ decision-making process.

scholarly journals Disease-modifying therapies modulate retinal atrophy in multiple sclerosis

Neurology ◽  
2017 ◽  
Vol 88 (6) ◽  
pp. 525-532 ◽  
Author(s):  
Julia Button ◽  
Omar Al-Louzi ◽  
Andrew Lang ◽  
Pavan Bhargava ◽  
Scott D. Newsome ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
First Year ◽  
Time Interval ◽  
Inclusion Criteria ◽  
Disease Modifying Therapies ◽  
Relapsing Remitting ◽  
Retinal Atrophy ◽  
Disease Modifying ◽  
Relapsing Remitting Multiple Sclerosis

Objective:To retrospectively investigate whether disease-modifying therapies (DMTs) exert differential effects on rates of retinal atrophy in relapsing-remitting multiple sclerosis (RRMS), as assessed using optical coherence tomography (OCT).Methods:A total of 402 patients with RRMS followed at the Johns Hopkins MS Center who underwent Cirrus-HD OCT were assessed for eligibility. Inclusion criteria included at least 1 year of OCT follow-up and adherence to a single DMT during the period of follow-up. Combined thickness of the ganglion cell + inner plexiform (GCIP) and other retinal layers was computed utilizing automated macular segmentation. Retinal thickness changes were analyzed using mixed-effects linear regression.Results:The effects of glatiramer acetate (GA; n = 48), natalizumab (NAT; n = 46), and interferon-β-1a subcutaneously (IFNSC; n = 35) and intramuscularly (IFNIM; n = 28) were assessed. Baseline analyses revealed no significant differences between groups in terms of age, sex, optic neuritis history, or follow-up duration. During follow-up, relative to NAT-treated patients, IFNSC- and GA-treated patients exhibited 0.37 μm/y (p < 0.001) and 0.14 μm/y (p = 0.035) faster rates of GCIP thinning, respectively, adjusting for the interval between initiation of DMT and OCT monitoring (gap time), age, sex, relapses, and disease duration. In the IFNSC group, GCIP thinning was 1.53 μm/y faster during the first year of therapy vs during the time interval afterwards (p < 0.001).Conclusions:Rates of GCIP atrophy in patients with RRMS vary according to DMT utilization. Our findings support OCT for monitoring neurodegenerative treatment effects in the retina, an easily accessible tissue, and as a practical outcome measure in RRMS clinical trials.

scholarly journals She Doesn’t Even Go Here: The Role of Inflammatory Astrocytes in CNS Disorders

2021 ◽  
Vol 15 ◽  
Author(s):  
Jacqueline Kelsey Reid ◽  
Hedwich Fardau Kuipers
Keyword(s):  
Neurodegenerative Diseases ◽  
Neurological Disorders ◽  
Therapeutic Targeting ◽  
Future Directions ◽  
Cns Disorders ◽  
Disease Modifying Therapies ◽  
Astrocyte Heterogeneity ◽  
Disease Modifying ◽  
Innovative Techniques

Astrocyte heterogeneity is a rapidly evolving field driven by innovative techniques. Inflammatory astrocytes, one of the first described subtypes of reactive astrocytes, are present in a variety of neurodegenerative diseases and may play a role in their pathogenesis. Moreover, genetic and therapeutic targeting of these astrocytes ameliorates disease in several models, providing support for advancing the development of astrocyte-specific disease modifying therapies. This review aims to explore the methods and challenges of identifying inflammatory astrocytes, the role these astrocytes play in neurological disorders, and future directions in the field of astrocyte heterogeneity.

scholarly journals COVID-19 is associated with multiple sclerosis exacerbations that are prevented by disease modifying therapies

2021 ◽  
Author(s):  
Afagh Garjani ◽  
Rodden M Middleton ◽  
Rachael Hunter ◽  
Katherine A Tuite-Dalton ◽  
Alasdair Coles ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Cohort Study ◽  
Expanded Disability Status Scale ◽  
Community Based ◽  
Web Based ◽  
The United Kingdom ◽  
Disease Modifying Therapies ◽  
Disability Status ◽  
Disease Modifying ◽  
The Impact

ABSTRACTBackgroundInfections can trigger exacerbations of multiple sclerosis (MS). The effects of the coronavirus disease 2019 (COVID-19) on MS are not known. The aim of this study was to understand the impact of COVID-19 on new and pre-existing symptoms of MS.MethodsThe COVID-19 and MS study is an ongoing community-based, prospective cohort study conducted as part of the United Kingdom MS Register. People with MS and COVID-19 were invited by email to complete a questionnaire about their MS symptoms during the infection. An MS exacerbation was defined as developing new MS symptoms and/or worsening of pre-existing MS symptoms.ResultsFifty-seven percent (230/404) of participants had an MS exacerbation during their infection; 82 developed new MS symptoms, 207 experienced worsened pre-existing MS symptoms, and 59 reported both. Disease modifying therapies (DMTs) reduced the likelihood of developing new MS symptoms during the infection (OR 0.556, 95%CI 0.316-0.978). Participants with a higher pre-COVID-19 webEDSS (web-based Expanded Disability Status Scale) score (OR 1.251, 95%CI 1.060-1.478) and longer MS duration (OR 1.042, 95%CI 1.009-1.076) were more likely to experience worsening of their pre-existing MS symptoms during the infection.ConclusionCOVID-19 infection was associated with exacerbation of MS. DMTs reduced the chance of developing new MS symptoms during the infection.

Reporting treatment outcomes in observational data: A fine balance

2016 ◽  
Vol 23 (1) ◽  
pp. 21-22 ◽  
Author(s):  
Tomas Kalincik ◽  
Maria Pia Sormani
Keyword(s):  
Multiple Sclerosis ◽  
Cost Effectiveness ◽  
High Risk ◽  
Treatment Outcomes ◽  
Observational Data ◽  
Risk Of Bias ◽  
Statistical Methodology ◽  
Disease Modifying Therapies ◽  
Disease Modifying

Analyses of observational data have been gaining momentum in the evaluation of ever increasing spectrum of disease modifying therapies for multiple sclerosis. While high cost-effectiveness and generalisability represent their main advantages, these studies are also burdened with high risk of bias that may lead to erroneous conclusions. In this viewpoint, we highlight the key role of rigorous and transparent statistical methodology in the studies of observational data and encourage its thorough editorial scrutiny.

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