Multiple Sclerosis Outcome Assessments Consortium: Genesis and initial project plan

2013 ◽  
Vol 20 (1) ◽  
pp. 12-17 ◽  
Author(s):  
Richard A Rudick ◽  
Nicholas LaRocca ◽  
Lynn D Hudson ◽  
Keyword(s):  
Multiple Sclerosis ◽  
Unmet Need ◽  
European Medicines Agency ◽  
Disability Progression ◽  
Consensus Approach ◽  
Disease Modifying Drugs ◽  
Outcome Assessments ◽  
Therapeutic Landscape ◽  
Measuring Outcomes ◽  
Disease Modifying

The need for improved clinical outcome measures in multiple sclerosis trials has been recognized for two decades, but only recently has the Food and Drug Administration (FDA) created a pathway for qualification of new clinician-reported outcome (ClinRO) assessments. Additionally, drug development in multiple sclerosis (MS) has been extraordinarily active, with numerous disease-modifying drugs now on the market. This shifting therapeutic landscape, along with the unmet need for drugs to treat the progressive forms of MS and the changing expectations of clinicians, patients, and payers, have led to the call for more sensitive and meaningful disability progression measures. In response to these drivers, the Multiple Sclerosis Outcome Assessments Consortium (MSOAC) was launched. A public-private partnership, MSOAC aims to accelerate the development of new therapies for MS by generating new tools for measuring outcomes in clinical trials. At the first annual MSOAC/FDA meeting, a regulatory path was outlined for qualifying a new tool for assessing efficacy in registration trials of MS. The European Medicines Agency (EMA) and FDA will provide parallel consultation and review. The consensus approach with engagement by all of the stakeholders, prominently including patients with MS, should also increase acceptance of the measure by clinicians and patients.

scholarly journals Optimizing treatment strategies in paediatric, adult and late-onset multiple sclerosis

Brain ◽  
2020 ◽  
Vol 143 (10) ◽  
pp. 2866-2868
Author(s):  
Cristina Gaudioso ◽  
Robert T Naismith
Keyword(s):  
Multiple Sclerosis ◽  
Late Onset ◽  
Treatment Strategies ◽  
Disability Progression ◽  
Disease Modifying Drugs ◽  
Disease Modifying ◽  
Relapsing Multiple Sclerosis

This scientific commentary refers to ‘Disease-modifying drugs can reduce disability progression in paediatric, adult and late-onset relapsing multiple sclerosis’, by Amato etal. (doi:10.1093/brain/awaa251).

Preapproval and postapproval evidence on drugs for multiple sclerosis

Neurology ◽  
2018 ◽  
Vol 90 (21) ◽  
pp. 964-973 ◽  
Author(s):  
Chiara Gerardi ◽  
Vittorio Bertele' ◽  
Silvia Rossi ◽  
Silvio Garattini ◽  
Rita Banzi
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Effectiveness ◽  
Dimethyl Fumarate ◽  
New Drugs ◽  
Cochrane Library ◽  
European Medicines Agency ◽  
The European Union ◽  
Disability Progression ◽  
Disease Modifying Drugs ◽  
Approved Drugs

ObjectiveTo review the evidence supporting the European Union marketing authorization of drugs for multiple sclerosis (MS) and assess how far postmarketing research addresses information gaps at the time of approval.MethodsThrough its database, we identified drugs approved by the European Medicines Agency and gathered data on pivotal trials from the European Public Assessment Reports and corresponding publications. We searched Medline, Embase, Cochrane Library, and trial registries for postmarketing randomized controlled trials testing the drugs identified in any form of the disease.ResultsSince approval of interferon and glatiramer up to 2017, the Agency has examined 10 drugs for the treatment of MS, and 8 were included in this study: alemtuzumab, daclizumab, dimethyl fumarate, fampridine, fingolimod, peginterferon-β-1a, natalizumab, and teriflunomide. We analyzed 16 pivotal trials enrolling almost 16,000 participants. Eleven compared new drugs to placebo, 5 to interferon-β-1a. Annualized relapse rate was the primary outcome in two-thirds and coprimary with disability progression in the 2 studies of alemtuzumab. Of the 52 postmarketing trials, 24 reported final results and 28 were ongoing, terminated, or completed but no results were available. None directly compared the approved drugs, thus leaving their respective therapeutic values unknown. Data on the prevention of disease progression were scarce: none of the disease-modifying drugs showed any effect on disability progression.ConclusionThe lack of comparative evidence and data on clinical effectiveness hamper the assessment of therapeutic value and place in therapy of drugs approved for MS.

Cladribine for multiple sclerosis

2018 ◽  
Vol 56 (2) ◽  
pp. 21-24 ◽  
Keyword(s):  
Multiple Sclerosis ◽  
Oral Treatment ◽  
Oral Formulation ◽  
Simple Approach ◽  
European Medicines Agency ◽  
Disease Modifying Drugs ◽  
Highly Active ◽  
Disease Modifying Therapy ◽  
Disease Modifying ◽  
The Uk

In the UK, there are twelve disease-modifying drugs licensed for various forms of multiple sclerosis (MS), of which three are oral therapies. An oral formulation of cladribine (Mavenclad - Merck Serono Europe Limited) was recently licensed by the European Medicines Agency (EMA) for the treatment of adult patients with highly active relapsing MS.1,2 It is claimed to be “an innovatively simple approach” for treating this form of MS and “the only disease modifying therapy that can deliver and sustain 4 years of disease control with a maximum of 20 days oral treatment in the first 2 years.”3 Here, we consider the evidence for its use in the treatment of highly active relapsing MS.

scholarly journals Long-term effectiveness in patients previously treated with cladribine tablets: a real-world analysis of the Italian multiple sclerosis registry (CLARINET-MS)

2020 ◽  
Vol 13 ◽  
pp. 175628642092268 ◽  
Author(s):  
Francesco Patti ◽  
Andrea Visconti ◽  
Antonio Capacchione ◽  
Sanjeev Roy ◽  
Maria Trojano ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Real World ◽  
Event Analysis ◽  
Treatment Change ◽  
Real World Data ◽  
Indirect Measure ◽  
Disease Modifying Drugs ◽  
Disease Modifying ◽  
Using Data

Background: The CLARINET-MS study assessed the long-term effectiveness of cladribine tablets by following patients with multiple sclerosis (MS) in Italy, using data from the Italian MS Registry. Methods: Real-world data (RWD) from Italian MS patients who participated in cladribine tablets randomised clinical trials (RCTs; CLARITY, CLARITY Extension, ONWARD or ORACLE-MS) across 17 MS centres were obtained from the Italian MS Registry. RWD were collected during a set observation period, spanning from the last dose of cladribine tablets during the RCT (defined as baseline) to the last visit date in the registry, treatment switch to other disease-modifying drugs, date of last Expanded Disability Status Scale recording or date of the last relapse (whichever occurred last). Time-to-event analysis was completed using the Kaplan–Meier (KM) method. Median duration and associated 95% confidence intervals (CI) were estimated from the model. Results: Time span under observation in the Italian MS Registry was 1–137 (median 80.3) months. In the total Italian patient population ( n = 80), the KM estimates for the probability of being relapse-free at 12, 36 and 60 months after the last dose of cladribine tablets were 84.8%, 66.2% and 57.2%, respectively. The corresponding probability of being progression-free at 60 months after the last dose was 63.7%. The KM estimate for the probability of not initiating another disease-modifying treatment at 60 months after the last dose of cladribine tablets was 28.1%, and the median time-to-treatment change was 32.1 (95% CI 15.5–39.5) months. Conclusion: CLARINET-MS provides an indirect measure of the long-term effectiveness of cladribine tablets. Over half of MS patients analysed did not relapse or experience disability progression during 60 months of follow-up from the last dose, suggesting that cladribine tablets remain effective in years 3 and 4 after short courses at the beginning of years 1 and 2.

scholarly journals HIV infection and multiple sclerosis: a case with unexpected “no evidence of disease activity” status

2021 ◽  
Vol 49 (3) ◽  
pp. 030006052199957
Author(s):  
Fernando Labella ◽  
Fernando Acebrón ◽  
María del Carmen Blanco-Valero ◽  
Alba Rodrígez-Martín ◽  
Ángela Monterde Ortega ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Hiv Infection ◽  
Disease Activity ◽  
Demyelinating Disease ◽  
Clinical Course ◽  
Disease Modifying Drugs ◽  
Immunodeficiency Virus ◽  
Relapsing Remitting ◽  
The Central Nervous System ◽  
Disease Modifying

Multiple sclerosis (MS) is an inflammatory demyelinating disease of the central nervous system whose etiology remains unclear. It has been suggested that MS can be triggered by certain viruses; however, human immunodeficiency virus (HIV) infection is associated with reduced incidence of MS. We present the case of a young patient diagnosed with active relapsing-remitting MS whose clinical course substantially improved following HIV infection and treatment. The patient achieved no evidence of disease activity status without any disease-modifying drugs. Both HIV-induced immunosuppression and antiretroviral therapy may have attenuated the clinical course in this patient.

scholarly journals Utilization Patterns of Oral Disease-Modifying Drugs in Commercially Insured Patients with Multiple Sclerosis

2019 ◽  
Vol 25 (1) ◽  
pp. 113-121 ◽  
Author(s):  
Rishi J. Desai ◽  
Mufaddal Mahesri ◽  
Joshua J. Gagne ◽  
Eimir Hurley ◽  
Angela Tong ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Oral Disease ◽  
Disease Modifying Drugs ◽  
Utilization Patterns ◽  
Disease Modifying ◽  
Insured Patients

scholarly journals Pregnancy in multiple sclerosis: from scientific aspects to practical

2021 ◽  
Vol 64 (3) ◽  
pp. 78-84
Author(s):  
Anna Belenciuc ◽  
◽  
Ana-Maria Bubuioc ◽  
Olesea Odainic ◽  
Marina Sangheli ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Family Planning ◽  
Immune Reconstitution ◽  
Interferon Beta ◽  
Reproductive Age ◽  
Childbearing Age ◽  
Disease Modifying Drugs ◽  
Choice Of Treatment ◽  
Key Issues ◽  
Disease Modifying

Background: Multiple sclerosis (MS) is a disease that affects young people of reproductive age (20-40 years old), predominantly women. Therefore, almost every patient has questions about pregnancy and breastfeeding. Family planning is one of the key issues in the choice of treatment tactics. Despite the growing number of therapeutic options for individualized treatment, it is still a question how to manage women with MS who become pregnant while taking disease-modifying drugs or want to become pregnant after starting this treatment. Conclusions: Women with MS should not be discouraged from pregnancy due to their illness. It is necessary to proactively discuss pregnancy planning with all women with MS of childbearing age. Based on available data, interferon beta and glatiramer acetate appear to be most suitable for use up until the time of confirmed pregnancy. A large amount of data (more than 1000 cases) obtained from registries shows that use of interferon beta before conception and during pregnancy suggests no evidence of increase in the rate of congenital anomalies or spontaneous abortions. For women with persistent high disease activity, pulsed immune reconstitution therapy gives additional opportunity for family planning after the last dose. The choice between available options for pulsed immune reconstitution therapy should be based on efficacy balanced against the risks.

Sign in / Sign up

Export Citation Format

Share Document