EXPLORING PATENT ACTIVITY AND ITS POTENTIAL ASSOCIATION WITH HEALTHCARE OUTCOMES: A CASE STUDY OF OSTOMY PRODUCTS IN SWEDEN.

OBJECTIVES: The aim of this study was to evaluate whether ostomy industry patent activity (PA) is associated with patient outcomes and healthcare costs. METHODS: Two groups of ostomy pouch users based on manufacturer PA (low or high) were compared in terms of ostomy-related wear patterns, adverse events, and healthcare expenditure. Using Swedish registry data, all patients with newly formed stomas were divided between each group and were followed during a 2-year period (2011-12). Propensity score matching and parametric duration analysis were used to compare outcomes between patients of similar characteristics such as sex, age, and ostomy surgery type. RESULTS: In both one- and two-piece systems, the high PA group had significantly lower monthly ostomy-related expenditure than the low PA group (one-piece: 197.47 EUR versus 233.34 EUR; two-piece: 164.00 EUR versus 278.98 EUR). Fewer pouch and skin wafer purchases per month were an important driver of cost differences. Both groups had similar likelihood of purchasing dermatological products for skin complications over time. CONCLUSIONS: PA in the ostomy care industry was associated with reduced healthcare costs, but not necessarily with fewer skin complications. It suggests that there is a health economic benefit from products made by patent intensive companies which may differentiate them from generic comparators, but more research is needed to understand the impact of activities conducive to medical innovation on health outcomes.

From concepts, theory, and evidence of heterogeneity of treatment effects to methodological approaches: a primer.

Implicit in the growing interest in patient-centered outcomes research is a growing need for better evidence regarding how responses to a given intervention or treatment may vary across patients, referred to as heterogeneity of treatment effect (HTE). A variety of methods are available for exploring HTE, each associated with unique strengths and limitations. This paper reviews a selected set of methodological approaches to understanding HTE, focusing largely but not exclusively on their uses with randomized trial data. It is oriented for the "intermediate" outcomes researcher, who may already be familiar with some methods, but would value a systematic overview of both more and less familiar methods with attention to when and why they may be used. Drawing from the biomedical, statistical, epidemiological and econometrics literature, we describe the steps involved in choosing an HTE approach, focusing on whether the intent of the analysis is for exploratory, initial testing, or confirmatory testing purposes. We also map HTE methodological approaches to data considerations as well as the strengths and limitations of each approach. Methods reviewed include formal subgroup analysis, meta-analysis and meta-regression, various types of predictive risk modeling including classification and regression tree analysis, series of n-of-1 trials, latent growth and growth mixture models, quantile regression, and selected non-parametric methods. In addition to an overview of each HTE method, examples and references are provided for further reading.By guiding the selection of the methods and analysis, this review is meant to better enable outcomes researchers to understand and explore aspects of HTE in the context of patient-centered outcomes research.

Acceptance and application of a broad population health perspective when evaluating vaccine.

The traditional health economic analysis is limited to estimating the impact on the treated patient. As vaccines are usually aimed at preventing infectious diseases, they may be associated with additional values for the non-treated wider population. Although there are valid reasons for treating vaccines differently, and a wide support for a broader perspective in the literature (i.e., beyond the net costs and health gain related to the outcome for the vaccinated individual), it remains unclear to what extent the Health Technology Assessment (HTA) agencies accept and apply a broader perspective. The purpose of this study is to examine and discuss what type of consequences are relevant for a health economic analysis of vaccines and which consequences are considered by HTA agencies. The study includes a strategic review of literature and HTA decisions in Sweden and other countries, online round-table discussions with stakeholders in Sweden, and a basic estimation of the value of a COVID-19 vaccination in Sweden. The study shows that, other than herd effect, broader economic consequences for the population are generally not included in the economic evaluation of vaccines. Also, all economic consequences for the treated patient (production loss) and caregiver (health loss) are not always considered. The perspective chosen can have a major impact on the outcome of the analysis. A vaccine for COVID-19 is estimated to provide a value of €744-€956 per dose when using a societal perspective including broader consequences for the population. Providing a complete and appropriate picture of the value of vaccination is of importance to allocate resources efficiently, to provide incentives for vaccine development, and to show the cost of delaying decisions to implement a new vaccine.

Regional Differences in the Prescription of Biologics for Psoriasis in Sweden: A Register-Based Study of 4168 Patients.

BACKGROUND: Observational studies suggest an inequitable prescription of biologics in psoriasis care, which may be attributed to geographical differences in treatment access. Sweden regularly ranks high in international comparisons of equitable healthcare, and is, in connection with established national registries, an ideal country to investigate potential inequitable access. OBJECTIVE: The aim was to determine whether the opportunity for patients to receive biologics depends on where they receive care. METHODS: Biologic-naïve patients enrolled in the Swedish National Register for Systemic Treatment of Psoriasis (PsoReg) from 2008 to 2015 (n = 4168) were included. The association between the likelihood of initiating a biologic and the region where patients received care was analyzed. The strength of the association was adjusted for patient and clinical characteristics, as well as disease severity using logistic regression analysis. The proportion of patients that switched to a biologic (switch rate) and the probability of switch to a biologic was calculated in 2-year periods. RESULTS: The national switch rate increased marginally over time from 9.7 to 11.0%, though the uptake varied across regions. Adjusted odds ratios for at least one region were significantly different from the reference region in every 2-year period. During the latest period (2014-2015), the average patient in the lowest prescribing region was nearly 2.5 times less likely to switch as a similar patient in the highest prescribing region. CONCLUSIONS: Geographical differences in biologics prescription persist after adjusting for patient characteristics and disease severity. The Swedish example calls for further improvements in delivering equitable psoriasis care.

Clinical practice of BOTOX® treatment for overactive bladder syndrome in Sweden: an assessment of resource use and external validity.

OBJECTIVE: The objective of this study was to assess the resource use of treating overactive bladder (OAB) patients in real-world clinical practice and to evaluate whether there is external validity in the treatment of OAB in clinical practice. MATERIALS AND METHODS: The study included 55 patients suffering from OAB and treated with BOTOX® at two Swedish clinics. The study was conducted as an anonymized retrospective chart review study. RESULTS: The estimated yearly direct cost of BOTOX treatment was €902. The mean age of patients in the study was 60 years, and 85% were women. The severity of OAB before BOTOX treatment, given by the mean number of daily leakages, equalled 4.8. The median interval between treatments was 210 days. CONCLUSIONS: Patient characteristics in the real world were similar to those in the clinical trials, showing a high degree of external validity. Treatment intervals were longer in the real world than in clinical trials, indicating that treatment cost could be lower when patients are treated as observed in real-world clinical practice.

Epilepsy in tuberous sclerosis patients in Sweden - Healthcare utilization, treatment, morbidity, and mortality using national register data.

PURPOSE: This study is designed to estimate the prevalence of epilepsy associated with TSC in Sweden and to describe treatment, morbidity, and mortality of TSC patients with epilepsy. METHODS: Register data for 2004-2014 was obtained from the National Board of Health and Welfare in Sweden. Patients with TSC were identified using ICD-10 codes. Epilepsy was identified using ICD-10 codes, interventions aimed to treat epilepsy, or prescriptions for antiepileptic drugs. RESULTS: The prevalence of TSC was 5.38 per 100 000 individuals. We identified 551 unique patients with TSC, of which 386 (70.1%) had epilepsy. The mean study period was 8.82 years. Antiepileptic drugs were dispensed to 97.9% of patients with epilepsy. The most prescribed antiepileptic drug was sodium valproate. Ketogenic diet was used in 6 (1.6%) patients, vagus nerve stimulation in 23 (6.0%) patients, and epilepsy surgery was performed in 25 (6.5%) patients. The mean number of outpatient visits per year was 4.70 (SD 4.17) and the mean number of inpatient days per year was 3.25 (SD 5.61). The mean number of outpatient visits per year with an ICD-10 code for epilepsy was 1.65 (SD 1.95) and the corresponding number of inpatient days was 2.06 (SD 4.50). A total of 30 patients with TSC and epilepsy died during the study period. CONCLUSIONS: The prevalence of epilepsy in this study was in the lower range of previously reported numbers, suggesting that epilepsy may be overestimated in non-population based studies. A substantial part of the healthcare utilization was directly related to epilepsy.

Considerations in developing model-based economic evaluations of glaucoma treatment.

PURPOSE: To facilitate future glaucoma model development and to provide guidance for decision-makers evaluating them, we provide an overview of an innovative glaucoma model and highlight important modeling considerations. CONSIDERATIONS: The considerations that were addressed include: disease outcome that is both relevant and meaningful to current clinical practice; diversity in treatment options and practices; incorporation of therapy discontinuation; and consideration of the variability in patient response to treatment. MODEL SCOPE: A state-transition, Monte Carlo simulation model was developed to simulate the management and treatment of patients with glaucoma and/or ocular hypertension. The model examines strategies involving sequential use of up to 6 pharmacologic interventions. Transitions are based on the monthly probability that a patient is no longer "successfully maintained" on therapy, which can be a consequence of lack of intraocular pressure control, adverse events, lack of compliance, or lack of persistence. Outputs of the model include months on each treatment, frequency of therapy switches, days of intraocular pressure control, frequency of ophthalmologist visits, frequency of surgery, and glaucoma-related costs. The model allows the user to specify country-specific treatment strategies, survival on therapy, surgical rates, practice patterns, and costs. CONCEPT APPLICATION: The model presented offers insights into accommodating patient and clinician variability through the use of persistence distributions. It will facilitate future glaucoma model development and provide insight for decision-makers who must evaluate model-based analyses of the economic value of glaucoma interventions.

Healthcare Provider Type and Switch to Biologics in Psoriasis: Evidence from Real-World Practice.

BACKGROUND: Previous research indicates an uneven uptake of biologics in patients with moderate-to-severe psoriasis in Sweden. Therefore, it is essential to scrutinise variations in treatment patterns. OBJECTIVE: The aim of this study was to evaluate the extent to which the uptake of biologics for psoriasis differs between types of healthcare provider. METHODS: Three types of provider were identified within 52 units participating in the Swedish National Registry for Systemic Psoriasis Treatment (PsoReg): university hospitals, non-university hospitals and individual practices. Biologics-naïve patients (n = 3165) were included in analyses to investigate the probability of switch to biologics. The numbers of patients fulfilling the criteria for moderate-to-severe psoriasis [Psoriasis Area and Severity Index (PASI) ≥10 and Dermatology Life Quality Index (DLQI) ≥10] among patients who switched to biologics and patients who did not switch were reported. A logistic regression model was used to calculate how healthcare provider type influenced the probability of switch to biologics whilst adjusting for patient characteristics and disease severity. RESULTS: During registration, 16% of patients switched to biologics while 84% remained on conventional systemic treatment. In 7% of patients, the criteria PASI ≥10 and DLQI ≥10 was fulfilled at their last visit without switching to biologics, whereas in 10% of patients the criteria was not fulfilled prior to switch. After controlling for patient characteristics and disease severity, small or no difference in the probability of switch was observed between provider types. CONCLUSIONS: Disease severity does not explain the decision to switch or not to switch to biologics for a disproportionate number of patients. There seems to be an uneven uptake of biologics in Swedish clinical practice, but the type of healthcare provider cannot explain this variation. More research is needed on what factors influence the prescription of biologics.

A new index approach to measure lost benefits from progression to blindness.

OBJECTIVES: An index approach for measuring the reduction in daily activities of patients suffering from an eye disease associated with progressive visual loss is proposed. The approach is illustrated using data collected from patients with cataract. METHOD: The approach uses recently developed methods based on index theory together with measurements of daily activities. In a simulation based on observations of visual acuity and daily activities in patients with cataract, indexes of changes in benefits were estimated for varying levels of visual acuity. RESULTS: Results show the development of loss in benefits resulting from a progressive deterioration in visual acuity. The results indicate a 40% loss in benefits at low levels of visual acuity, equivalent to a potential increase in benefits of 75% for these individuals following successful treatment. CONCLUSIONS: The proposed index approach may prove useful for measuring reductions in daily activities resulting from the progressive loss of vision in eye disease. The approach has successfully measured the reduction in daily activities in patients with cataract and may be applicable in patients with other eye diseases, including age-related macular degeneration and open-angle glaucoma.