Global Barriers to the Diagnosis of Multiple Sclerosis: Data From the Multiple Sclerosis International Federation Atlas of MS, Third Edition.

BACKGROUND AND OBJECTIVES: Recent data suggest increasing global prevalence of multiple sclerosis (MS). Early diagnosis of MS reduces the burden of disability-adjusted life years and associated health care costs. Yet diagnostic delays persist in MS care and even within national health care systems with robust resources, comprehensive registries, and MS subspecialist referral networks. The global prevalence and characteristics of barriers to expedited MS diagnosis, particularly in resource-restricted regions, have not been extensively studied. Recent revisions to MS diagnostic criteria demonstrate potential to facilitate earlier diagnosis, but global implementation remains largely unknown. METHODS: The Multiple Sclerosis International Federation third edition of the Atlas of MS was a survey that assessed the current global state of diagnosis including adoption of MS diagnostic criteria; barriers to diagnosis with respect to the patient, health care provider, and health system; and existence of national guidelines or national standards for speed of MS diagnosis. RESULTS: Coordinators from 107 countries (representing approximately 82% of the world population), participated. Eighty-three percent reported at least 1 "major barrier" to early MS diagnosis. The most frequently reported barriers included the following: "lack of awareness of MS symptoms among general public" (68%), "lack of awareness of MS symptoms among health care professionals" (59%), and "lack of availability of health care professionals with knowledge to diagnose MS" (44%). One-third reported lack of "specialist medical equipment or diagnostic tests." Thirty-four percent reported the use of only 2017 McDonald criteria (McD-C) for diagnosis, and 79% reported 2017 McD-C as the "most commonly used criteria." Sixty-six percent reported at least 1 barrier to the adoption of 2017 McD-C, including "neurologists lack awareness or training" by 45%. There was no significant association between national guidelines pertaining to MS diagnosis or practice standards addressing the speed of diagnosis and presence of barriers to early MS diagnosis and implementation of 2017 McD-C. DISCUSSION: This study finds pervasive consistent global barriers to early diagnosis of MS. While these barriers reflected a lack of resources in many countries, data also suggest that interventions designed to develop and implement accessible education and training can provide cost-effective opportunities to improve access to early MS diagnosis.

Barriers to access and utilization of multiple sclerosis care services in a large cohort of Latin American patients.

BACKGROUND: Multiple sclerosis (MS), is an emergent disease in Latin America (LATAM), which raises substantial socioeconomic challenges to a region where most countries remain as economies in development. OBJECTIVE: To assess barriers to access and utilization of MS care services in a regional cohort survey. METHODS: We conducted a cross-sectional study based on a self-reported survey. Patients with MS (PwMS) completed this regional survey in 12 Latin American (LATAM) countries. PwMS were also divided into those with healthcare insurance (including certain local national social security programs) and those without healthcare insurance (treated at public institutions). RESULTS: We surveyed 1469 PwMS and identified significant regional differences in relation to access to complementary tests, rehabilitation services, and prescription of disease-modifying therapies (DMTs). Between 44.4% and 73.5% of PwMS were unemployed and nearly 50% had completed higher education. PwMS receiving care from the private sector reported greater access to imaging, DMTs, and fewer problems obtaining DMTs compared to those treated at public institutions. Multivariate analysis showed that lack of private insurance (OR = 2.21, p < 0.001), longer MS duration (OR = 1.02, p = 0.001), lower level of education (OR = 0.66, p = 0.009), and unemployment (OR = 0.73, p = 0.03) were independently associated with inappropriate delivery of DMTs. CONCLUSION: These findings suggest barriers to access and utilization of MS care services across LATAM are prevalent. We identified several factors predicting unmet healthcare needs in PwMS.

Adaptation and validation of a Spanish version of the treatment burden questionnaire in patients with multiple sclerosis.

BACKGROUND: The Treatment Burden Questionnaire (TBQ) is a self-reported measure of the effect of treatment workload on patient wellbeing. We sought to validate the TBQ in Spanish and use it to estimate the burden of treatment in Argentinian patients with multiple sclerosis (MS). METHODS: The TBQ was forward-backward translated into Spanish. Two focus groups and 25 semi-structured interviews focused on wording and possible item exclusion. Validation was performed in 2 steps. First, 162 patients across a range of MS severity completed the questionnaire. Confirmatory factor analysis assessed the dimensional structure of the TBQ. Construct validity was assessed by studying correlations with fatigue and quality of life (QoL). Then, in a second cohort of 171 patients, we evaluated the association between TBQ scores and patients' sex, age, education level, employment status, type of MS, disease duration, comorbidities, EDSS, pharmacological treatment and medication adherence. RESULTS: The questionnaire presented a 3-factor structure in which burden was related to pharmacological treatment; comprehensive health assistance; and psycho-social-economic context. Composite reliability was > 0.8 for all factors. TBQ showed positive correlation with fatigue (rs = 0.467, p = 0.006), negative correlation with QoL (rs - 0.446, p = 0.009). For the second cohort, total TBQ score was 43 (SD 29). Lowest scores were observed on self-monitoring (0.53, SD 1.3) and highest for administrative load (4.2, SD 3.4). Inverse association was found between the TBQ score and medication adherence (r 0.243 p = 0.001). TBQ scores also correlated with daily patient pill/injection requirements (r 0.175 p = 0.020). Individuals receiving injectable treatment scored higher than patients on oral drugs (total TBQ 51 (SD 32) vs 39 (SD 27) p = 0.002). CONCLUSIONS: The TBQ in Spanish is a reliable instrument and showed adequate correlation with QoL and adherence scales in MS patients. TBQ may benefit health resources allocation and provide tailor therapeutic interventions to construct a minimally disruptive care.

Follow-on products for treatment of multiple sclerosis in Latin America: An update.

Both proprietary and non-proprietary medicines are expected to undergo rigorous pre-approval testing and both should meet stringent health authority regulatory requirements related to quality to obtain approval. Non-proprietary (also known as copy or generic) medicines, which base their authorization and use on the proprietary documentation and label, are often viewed as a means to help lower cost and thus increase patient access. If these medicines fail to meet quality standards, such as good manufacturing practice and bioequivalence (in humans), they are then defined as substandard copies and can pose serious risks to patients in terms of safety and efficacy. Availability of this type of compounds is more prevalent in regions where health authorities do not enforce registration regulations as stringent as those of the Food and Drug Administration, European Medicines Agency, or World Health Organization, including preestablished quality standard requirements. This article focuses on non-proprietary medicines for multiple sclerosis, that are not identical to proprietary versions and could thus fail to meet efficacy or have different impact on the safety of patients with multiple sclerosis.

Quality of Life Assessment in Multiple Sclerosis: Different Perception between Patients and Neurologists.

BACKGROUND: In recent years, neurologists are noticing that evaluation of multiple sclerosis (MS) patients based on combining relapses, disability progression, and magnetic resonance imaging activity may be insufficient to adequately assess suboptimal responses to available therapy. Inclusion of quality of life (QoL) parameters may contribute to breach this gap. OBJECTIVE: To evaluate agreement levels between doctor and patient perception of QoL in MS. METHODS: A total of 700 MS patients and 300 neurologists were invited to participate in a cross-sectional study by answering an e-mail questionnaire. The survey collected information on demographical data and included the Short Form questionnaire (SF-36). After completing the questionnaire, patients were given a standard written description of each of the subdomains assessed by SF-36 and asked to identify which three were the most important determinants of their overall health-related QoL. RESULTS: A total of 135 neurologists and 380 MS patients responded the survey. Study population mean age was 42.1 ± 10.5 years, with 61% presenting relapsing-remitting MS. SF-36 results were physical function 68.4 ± 30, physical role limitation 56.8 ± 41.7, vitality 47.6 ± 21.4, pain 71.2 ± 26.1, social function 72.6 ± 28.6, emotional role limitation 63.2 ± 39.8, mental health 60 ± 14.1, and general health 55.8 ± 22. Doctors considered physical function (75%) and physical role limitation (70%) as the most important QoL determinants in MS, followed by emotional role limitation (52%). Patients however, assigned significantly different levels of importance to physical function (58%), and physical role limitation (46%) and considered vitality (52%) more important than their physicians (p < 0.001). Important to note, the results of SF-36 questionnaire were highly correlated with the perception gap between patients and neurologists (r = 0.89; p = 0.0004). CONCLUSION: Concerns on QoL in MS are different for patients and physicians. It is essential to enhance communication in order to better understand actual patient needs.

Use of Fingolimod in the Management of Relapsing-Remitting Multiple Sclerosis: Experience from Latin America.

UNLABELLED: Once-daily fingolimod 0.5 mg (FTY720; Gilenya(®), Novartis Pharma AG, Basel, Switzerland) is a sphingosine 1-phosphate receptor modulator that is approved for the treatment of relapsing multiple sclerosis (MS); currently, this includes approval in 13 Latin American countries. However, despite a well-characterized efficacy and safety profile in a large clinical development program, thus far there has been limited representation of patients from across the Latin American region. Differences in MS disease characteristics have been reported for the Latin American population compared with Caucasians, which may be additional to recent improvements in MS diagnosis. Furthermore, healthcare provision and regional socioeconomic factors exist that are unique to Latin America compared with other regions. Therefore, to optimize MS treatment pathways and improve patient clinical outcomes, it is important to investigate the efficacy and safety profile of fingolimod using ethnically relevant data. Here, we review key data from Hispanic patients enrolled in the fingolimod clinical trial program, summarize recent findings from the FIRST LATAM study, and appraise fingolimod data from real-world patient populations. FUNDING: Novartis Pharma AG, Basel, Switzerland.

There is much to be learnt about the costs of multiple sclerosis in Latin America.

METHOD: A systematic review of the literature from 1990 to 2011 was conducted. Outcome measures included: mean cost of disease modifying therapies (DMTs), mean cost of treatment of relapses and mean cost of disease by stage stratification measured by the expanded disability status scale (EDSS). RESULTS: Seven studies from three countries (Brazil, Argentina and Colombia) were included. In 2004, in Argentina, the mean cost of DMT treatment was reported to be USD 35,000 per patient treated. In Brazil, the total MS expenditure of DMTs rose from USD 14,011,700 in 2006 to USD 122,575,000 in 2009. Patient costs ranged between USD 10,543 (EDSS 8-9.5) and USD 25,713 (EDSS 3-5.5). Indirect costs markedly increased for the EDSS 8-9.5 patients. CONCLUSION: Further research assessing the economic burden of MS in LA is warranted.

There is much to be learnt about the costs of multiple sclerosis in Latin America / Hay mucho a ser aprendido sobre los costos de la esclerosis multiple en America Latina

METHOD: A systematic review of the literature from 1990 to 2011 was conducted. Outcome measures included: mean cost of disease modifying therapies (DMTs), mean cost of treatment of relapses and mean cost of disease by stage stratification measured by the expanded disability status scale (EDSS). RESULTS: Seven studies from three countries (Brazil, Argentina and Colombia) were included. In 2004, in Argentina, the mean cost of DMT treatment was reported to be USD 35,000 per patient treated. In Brazil, the total MS expenditure of DMTs rose from USD 14,011,700 in 2006 to USD 122,575,000 in 2009. Patient costs ranged between USD 10,543 (EDSS 8-9.5) and USD 25,713 (EDSS 3-5.5). Indirect costs markedly increased for the EDSS 8-9.5 patients. CONCLUSION: Further research assessing the economic burden of MS in LA is warranted. .

MÉTODOS: Revisión sistemática de la literatura desde 1990 hasta 2011. Los resultados evaluados fueron: coste medio de los tratamientos modificadores de la enfermedad (DMTs), coste medio del tratamiento de las recaídas y la media de coste de la enfermedad estratificado por la Expanded Disability Status Scale (EDSS). RESULTADOS: Siete estudios de tres países (Brasil, Argentina y Colombia) fueron incluidos. El costo promedio del tratamiento de DMTs fue de USD 35.000 por paciente para el año 2004 en Argentina y el total del costo de los DMTs aumentó de USD 14.011,700 en 2006 a USD 122.575,000 en Brasil en 2009. Los costos de pacientes oscilaron entre USD 10.543 (EDSS 8-9.5) y USD 25.713 (EDSS 3.5 a 5). Los costes indirectos aumentaron para la EDSS mayor discapacidad (EDSS 8-9.5). CONCLUSIÓN: Estudios adicionales del costo de la EM en AL son necesarios.. .