Multiple Sclerosis Therapy With Disease-Modifying Treatments in Germany: The PEARL (ProspEctive phArmacoeconomic cohoRt evaluation) Noninterventional Study Protocol

Vormfelde, Stefan Viktor; Ortler, Sonja; Ziemssen, Tjalf

doi:10.2196/resprot.4473

Cited by 11 publications

(10 citation statements)

References 40 publications

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“…Details on study design have been described previously. 20 , 21 In short, MS patients under first-line therapy with glatiramer acetate, interferon beta preparations (PEARL study) or fingolimod (PANGAEA sub-study) were followed under real-world conditions for 2 years, with observational periods ending 2015 (PANGAEA) and 2013 (PEARL), respectively. Further inclusion criteria were a relapsing–remitting multiple sclerosis (RRMS) diagnosis and an age of 18 or older.…”

Section: Methodsmentioning

confidence: 99%

Gender disparities in health resource utilization in patients with relapsing–remitting multiple sclerosis: a prospective longitudinal real-world study with more than 2000 patients

Schriefer¹,

Ness²,

Haase³

et al. 2020

Ther Adv Neurol Disord

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Background: For the case of multiple sclerosis, research on gender differences from a health economics perspective has not received much attention. However, cost-of-illness analyses can provide valuable information about the diverse impact of the disease and thus help decision-makers to allocate scarce resources. The aim of this study was to describe healthcare resource use and associated societal costs from a gender perspective. In particular, we aimed to identify how resource utilization potentially differs in certain cost components between men and women. Methods: Clinical and economic data were extracted from two prospective, multicentre, non-interventional, observational studies in Germany. Information on health resource use was obtained from all patients on a quarterly basis using a validated questionnaire. Cost analyses were conducted from the societal perspective including all direct (healthcare-related) and indirect (work-related) costs, regardless of who bears them. Gender-related differences were analysed by a multivariable generalized linear model with a negative binomial distribution and log link function due to the right-skewed distribution pattern of cost data. In addition, costs for men and women were descriptively analysed within subgroups of two-year disease activity. Results: In total, 2095 patients (women-to-men ratio of 2.7:1) presented a mean age of 41.85 years and a median Expanded Disability Status Scale of 2 (interquartile range 1–3.5) ( p > 0.30 for gender-related differences). Women and men did not statistically differ in total quarterly costs (€2329 ± €2570 versus €2361 ± €2612). For both, costs were higher with advancing disease severity and indirect costs were the main societal cost driver. Regarding healthcare-related resources, women incurred higher costs for ambulant consultations [incidence rate ratio (IRR) 1.16, confidence interval (CI) 1.04–1.31], complementary medicine (IRR 2.41, CI 1.14–5.06), medical consumables (IRR 2.53, CI 1.69–3.79) and informal care (IRR 2.79, CI 1.56–5.01). Among indirect costs, we found higher costs for men for presenteeism (IRR 0.62; CI 0.53–0.72) and higher costs for women for disability pension (IRR 1.62; CI 1.23–2.13). Conclusions: Multiple sclerosis poses a significant economic burden on patients, families and society. While the total economic burden did not differ between male and female patients, we found gender differences in specific cost items that are similar to those in the wider non-MS population.

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Section: Methodsmentioning

confidence: 99%

Gender disparities in health resource utilization in patients with relapsing–remitting multiple sclerosis: a prospective longitudinal real-world study with more than 2000 patients

Schriefer¹,

Ness²,

Haase³

et al. 2020

Ther Adv Neurol Disord

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“…Patients with RRMS receiving BRACE therapies for at least 30 days were recruited into PEARL between September 2010 and March 2011, with the observational period ending in March 2013 [33]. In both studies, prospective visits were conducted at intervals of 3 months.…”

Section: Methodsmentioning

confidence: 99%

“…Patients with RRMS receiving fingolimod 0.5 mg were recruited into PANGAEA between April 2011 (when market approval was received for fingolimod in the EU) and December 2013, with the planned observational period ending in December 2018 [ 32 ]. Patients with RRMS receiving BRACE therapies for at least 30 days were recruited into PEARL between September 2010 and March 2011, with the observational period ending in March 2013 [ 33 ]. In both studies, prospective visits were conducted at intervals of 3 months.…”

Section: Methodsmentioning

confidence: 99%

“…Fingolimod is being assessed in the 5-year P ost- a uthorization N on-interventional G erman S a fety Study of Gil e ny a ® in MS Patients (PANGAEA) [ 32 ]. BRACE therapies were assessed in the independent, recently completed 2-year P rosp e ctive Ph a rmacoeconomic Coho r t Eva l uation (PEARL) [ 33 ]. PANGAEA and PEARL were conducted in Germany to allow analyses of prospectively collected data for substantial cohorts of patients in a single healthcare system that provides a wide range of treatment options for MS [ 34 ].…”

Section: Introductionmentioning

confidence: 99%

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Two studies in one: A propensity-score-matched comparison of fingolimod versus interferons and glatiramer acetate using real-world data from the independent German studies, PANGAEA and PEARL

et al. 2017

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BackgroundThis study compared outcomes following fingolimod or BRACE treatments (beta-interferons/glatiramer acetate) in patients with active MS (≥ 1 relapse in the previous year) following previous BRACE treatment.Methods and findingsPatients with active MS who previously received BRACE were identified from German prospective, observational studies, PANGAEA and PEARL. A novel methodology was developed to compare outcomes between propensity-score-matched cohorts (3:1 ratio) from the independent single-arm studies. Patients in PANGAEA (n = 1287) experienced 48% fewer relapses per year than those in PEARL (n = 429; annualized relapse rate ratio: 0.52; p < 0.001). The risk of 3-month or 6-month confirmed disability progression (CDP) was reduced in PANGAEA versus PEARL (3-month: 37% reduction; hazard ratio [HR], 0.63; p < 0.001; 6-month: 47% reduction; HR, 0.53; p < 0.001). A higher proportion of patients in PANGAEA (n = 1234) than PEARL (n = 401) were free from relapses and 3-month (65.7% vs 38.7%; p < 0.001) or 6-month (68.2% vs 39.2%; p < 0.001) CDP. The probability of confirmed disability improvement was higher in PANGAEA (n = 1163) than PEARL (n = 372; 3-month: 175% increase; HR, 2.75; p < 0.001; 6-month: 126% increase; HR, 2.26; p < 0.001). Patients in PANGAEA (n = 149) were less likely than those in PEARL (n = 307) to have taken sick leave (proportion with 0 days off work: 62.4% vs 44.6%; p = 0.0005). For change in disease severity from baseline (assessed by clinicians using the Clinical Global Impressions scale; PANGAEA, n = 1207; PEARL, n = 427), a larger proportion of patients had subjective improvement and a smaller proportion had worsening status in PANGAEA than PEARL (improvement: 28.2% vs 15.2%; worsening: 16.4% vs 30.4%; p < 0.0001).ConclusionsFingolimod appears to be more effective than BRACE in improving clinical and physician-/patient-reported outcomes in individuals with active MS.

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“…Some observational RWE studies also assess PROs. PANGAEA and PEARL were designed to capture patient experience on effectiveness, tolerability [ 20 ] and treatment satisfaction, as well as on ease and convenience of taking their DMT as instructed [ 20 , 42 ]. Additional PROs on quality of life, physical disability, cognition and fatigue can be obtained through health surveys [ 43 ].…”

Section: How Has Rwe Helped In Understanding the Disease Course And Pmentioning

confidence: 99%

The importance of collecting structured clinical information on multiple sclerosis

Ziemssen

Hillert

Butzkueven

2016

BMC Med

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BackgroundRandomized controlled trials (RCTs) are the ‘gold standard’ in the generation of drug efficacy and safety evidence. However, enrolment criteria, timelines and atypical comparators of RCTs limit their relevance to standard clinical practice.DiscussionReal-world data (RWD) provide longitudinal information on the comparative effectiveness and tolerability of drugs, as well as their impact on resource use, medical costs, and pharmacoeconomic and patient-reported outcomes. This is particularly important in multiple sclerosis (MS), where economic treatment benefits of long-term disability reduction are a cornerstone of payer drug approvals – these are typically not examined in the RCT itself but modelled using real-world datasets. Importantly, surrogate markers used in RCTs to predict the prevention of long-term disability progression can only truly be assessed through RWD methodologies.SummaryWe discuss the differences between RCTs and RWD studies, describe how RWD complements the evidence base from RCTs in MS, summarize the different methods of RWD collection, and explain the importance of structuring data analysis to avoid bias. Guidance on performing and identifying high-quality real-world evidence studies is also provided.

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Multiple Sclerosis Therapy With Disease-Modifying Treatments in Germany: The PEARL (ProspEctive phArmacoeconomic cohoRt evaluation) Noninterventional Study Protocol

Cited by 11 publications

References 40 publications

Gender disparities in health resource utilization in patients with relapsing–remitting multiple sclerosis: a prospective longitudinal real-world study with more than 2000 patients

Gender disparities in health resource utilization in patients with relapsing–remitting multiple sclerosis: a prospective longitudinal real-world study with more than 2000 patients

Two studies in one: A propensity-score-matched comparison of fingolimod versus interferons and glatiramer acetate using real-world data from the independent German studies, PANGAEA and PEARL

The importance of collecting structured clinical information on multiple sclerosis

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