A total of 2.8 million people are living with multiple sclerosis and due to disparities in access to medicines, the ability to treat this condition varies widely. Off-label disease-modifying therapies are sometimes more available or affordable in different health systems. Appropriate methodology is integral in creating high-quality and trustworthy guidelines. In this article, we outline Multiple Sclerosis International Federation's (MSIF) approach to creating guidelines for off-label treatments for multiple sclerosis.We use the Guidelines International Network (GIN)-McMaster Guideline Development Checklist and the Grading of Recommendations, Assessment, Development and Evaluations (GRADE) Evidence-to-Decision (EtD) framework. We developed detailed health descriptors for health outcomes and the panel drafted PICO (Population, Intervention, Comparator, Outcome) questions and prioritised outcomes. We collaborate with independent organisations, which systematically review and collate the information. We are actively engaging stakeholders and consulting with relevant organisations, boards, working groups and individuals.The draft guideline recommendations will be published for open comment and stakeholders will be encouraged to endorse and disseminate the guidelines. Our methodology ensures integrity and transparency in the criteria, evidence and judgement used to make recommendations.This approach will facilitate transparent creation of high-quality and trustworthy guidelines, and allow the global guidelines to be adopted or adapted into national settings.
This is a protocol for a Cochrane Review (Intervention). The objectives are as follows: The primary question is whether interferon beta is more effective than placebo in decreasing the number of SPMS patients who experience disability progression during follow‐up. We also aim to evaluate the incidence and seriousness of side effects and adverse events. The secondary objectives are to examine the effect of interferon beta on relapses during follow‐up, on patient activities of daily living and quality of life, and reducing the need for other treatments. We will also evaluate change in MRI parameters during follow‐up.
Over the last decade the European Union has been coordinating actions addressing various aspects of rare diseases and has funded several cross-border research projects. Recently has initiated the biggest rare disease international collaborative effort by launching the International Rare Diseases Research Consortium (IRDiRC). RARE-Bestpractices is one of the more than 100 collaborative research projects on rare diseases funded under the Seventh Framework Programme for Research and Technological Development (FP7; 2007-2013) (1). As a wide, open and inclusive network, RARE-Bestpractices will build on the knowledge of the experts in rare disease research area and experts in guideline development and health technology assessment area, brought together, for the first time, from academic institutions, agencies, organizations, patient advocacy groups, governmental bodies. The project aims at building a platform to collect and exchange information on best practices for the management of rare diseases; to identify relevant research needs; to promote the development of high quality guidelines; and to contribute in making patients, health professionals and policy makers “informed guideline users”. Besides, RARE-Bestpractices will intend to define the extent to which conclusions from cost-effectiveness analyses for pharmaceuticals are accounted for and implemented in guidelines across a range of countries.
Compared with other areas of the country, very limited data are available on multiple sclerosis (MS) prevalence in Central Italy. We aimed to estimate MS prevalence in the Lazio region and its geographical distribution using regional health information systems (HIS). To identify MS cases we used data from drug prescription, hospital discharge and ticket exemption registries. Crude, age- and gender-specific prevalence estimates on December 31, 2011 were calculated. To compare MS prevalence between different areas within the region, we calculated age- and gender-adjusted prevalence and prevalence ratios using a multivariate Poisson regression model. Crude prevalence rate was 130.5/100,000 (95 % CI 127.5-133.5): 89.7/100,000 for males and 167.9/100,000 for females. The overall prevalence rate standardized to the European Standard Population was 119.6/100,000 (95 % CI 116.8-122.4). We observed significant differences in MS prevalence within the region, with estimates ranging from 96.3 (95 % CI 86.4-107.3) for Latina to 169.6 (95 % CI 147.6-194.9) for Rieti. Most districts close to the coast showed lower prevalence estimates compared to those situated in the eastern mountainous area of the region. In conclusion, this study produced a MS prevalence estimate at regional level using population-based health administrative databases. Our results showed the Lazio region is a high-risk area for MS, although with an uneven geographical distribution. While some limitations must be considered including possible prevalence underestimation, HIS represent a valuable source of information to measure the burden of SM, useful for epidemiological surveillance and healthcare planning.
Objective To determine the accuracy of magnetic resonance imaging criteria for the early diagnosis of multiple sclerosis in patients with suspected disease.Design Systematic review.Data sources 12 electronic databases, citation searches, and reference lists of included studies.Review methods Studies on accuracy of diagnosis that compared magnetic resonance imaging, or diagnostic criteria incorporating such imaging, to a reference standard for the diagnosis of multiple sclerosis.Results 29 studies (18 cohort studies, 11 other designs) were included.On average, studies of other designs (mainly diagnostic case-control studies) produced higher estimated diagnostic odds ratios than did cohort studies.Among 15 studies of higher methodological quality (cohort design, clinical follow-up as reference standard), those with longer follow-up produced higher estimates of specificity and lower estimates of sensitivity.Only two such studies followed patients for more than10 years.Even in the presence of many lesions ( > 10 or > 8), magnetic resonance imaging could not accurately rule multiple sclerosis in (likelihood ratio of a positive test result 3.0 and 2.0, respectively).Similarly, the absence of lesions was of limited utility in ruling out a diagnosis of multiple sclerosis (likelihood ratio of a negative test result 0.1 and 0.5).Conclusions Many evaluations of the accuracy of magnetic resonance imaging for the early detection of multiple sclerosis have produced inflated estimates of test performance owing to methodological weaknesses.Use of magnetic resonance imaging to confirm multiple sclerosis on the basis of a single attack of neurological dysfunction may lead to over-diagnosis and over-treatment. MethodsWe identified studies, published and unpublished, by searching 12 databases from inception until September or November 2004.Search terms were "multiple sclerosis" combined with "magnetic resonance imaging" or "MRI".No language restrictions were applied.We undertook a citation search on the article reporting the McDonald 2001 criteria, 4 screened reference lists of included studies, and assessed studies included in the NICE multiple sclerosis guidelines. 5tudies were eligible that compared magnetic resonance imaging (or diagnostic criteria incorporating such imaging) to a reference standard for the diagnosis of multiple sclerosis and reported sufficient data to enable a 2×2 table of test Additional information and references w1-w43 are on bmj.
OBJECTIVE: To develop a potential amyotrophic lateral sclerosis (ALS) staging system (ALS Milano Torino Staging, ALS-MITOS) and apply it to evaluation of patients9 clinical, quality of life, and cost outcomes. BACKGROUND: Clinical trials track treatment effect on function in ALS patients using functional rating scales, e.g. the ALS Functional Rating Scale – Revised (ALSFRS-R). It may be appropriate to evaluate ALS patients9 natural disease course using defined clinical stages. DESIGN/METHODS: Critical milestones in ALS progression were defined by ALSFRS item score changes in 4 key domains: swallowing, walking/self-care, communicating, and breathing. Stages were defined as: 0 - functional involvement but no loss of independence on any domain; 1-4 -number of domains in which independence was lost; 5 - death. A prospective analysis of consecutively enrolled patients (≥18 to ≤80 yrs) from 11 Italian ALS centers was used to validate these defined stages. Patients within the catchment area with definite, probable or probable laboratory supported ALS, without serious comorbidities were included. At study initiation and at 4, 8, and 12 months, patients9 function (10 item ALSFRS), quality of life (QoL; Short Form-36[SF-36]) and direct costs were evaluated. RESULTS: Of 130 patients included, at baseline 48.7% patients were at Stage 0, 27.7% Stage 1, 11.8% Stage 2, 3.4% Stage 3, and 8.4% Stage 4. At 12 months, 15.7% patients were at Stage 0, 21.7% Stage 1, 15.7% Stage 2, 10.4% Stage 3, 13.9% Stage 4, and 22.6% Stage 5. Mean (±SE) overall ALSFRS score was 31.0 (±0.4), 22.7 (±0.4), 15.8 (±0.5), 9.5 (±0.7), 3.2 (±0.5) in Stages 0-4, respectively. SF-36 physical and mental health composite scores decreased progressively with each stage. Direct costs increased with increasing stages. CONCLUSIONS: The proposed staging system correlated well with functional, QoL and direct cost assessments. Supported by: Italian Ministry of Health. Disclosure: Dr. Chio has received personal compensation for activities with Biogen Idec and Cytokinetics. Dr. Hammond has nothing to disclose. Dr. Mora has nothing to disclose. Dr. Bonito has nothing to disclose. Dr. Filippini has nothing to disclose.
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