Background and Objective
Patient support programs aim to provide solutions beyond the medication itself, by enhancing treatment adherence, improving clinical outcomes, elevating patient experience, ...and/or increasing quality of life. As patient support programs increasingly play an important role in assisting patients, numerous observational studies and pragmatic trials designed to evaluate their impact on healthcare have been conducted in recent years. This review aims to characterize these studies.
Methods
A systematic literature review, supplemented by a broad search of gray literature, was conducted following PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) and Cochrane recommendations. Observational studies and pragmatic trials conducted in Europe to evaluate the impact of patient support programs, published in English or Spanish between 17/03/2010 and 17/03/2020, were reviewed. Two patient support program definitions were applied starting with Ganguli et al.’s broad approach, followed by the European Medicines Agency definition, narrowed to Marketing Authorization Holders organized systems and their medicines. The quality of publications was assessed using the STROBE (Strengthening the Reporting of Observational Studies in Epidemiology) statement 22-item checklist.
Results
Of the 49 identified studies following the Ganguli et al. definition, 20 studies met the European Medicines Agency definition and were reviewed. Patient support program impact was evaluated based on a wide range of methodologies: 70% assessed patient support program-related patient-reported outcomes, 55% reported clinical outcomes, and 25% reported economic impacts on health resources. Only 45% conducted a comparative analysis. Overall, 75% of the studies achieved their proposed objectives.
Conclusions
The heterogeneity of the observational studies reviewed reflects the complexity of patient support programs that are built ad hoc for specific diseases, treatments, and patients. Results suggest that patient support programs play a key role in promoting treatment effectiveness, clinical outcomes, and satisfaction. However, there is a need for standardizing the definition of patient support programs and the methods to evaluate their impact.
This paper presents the possibility of using Gallium Nitride (GaN) high-electron-mobility transistors (HEMTs) instead of the conventional silicon metal oxide semiconductor field effect transistor ...(MOSFET) to implement a high-frequency intermediate bus converter (IBC) as part of a typical distributed power architecture used in a space power application. The results show that processing the power at greater frequencies is possible with a reduction in mass and without impacting the system efficiency. The proposed solution was experimentally validated by the implementation of a 1 MHz zero-voltage and zero-current switching (ZVZCS) current-fed half-bridge converter with synchronous rectification compared with the same converter using silicon as the standard technology on power switches and working at 100 kHz. In conclusion, the replacement of silicon (Si) transistors by GaN HEMTs is feasible, and GaN HEMTs are promising next-generation devices in the power electronics field and can coexist with silicon semiconductors, mainly in some radiation-intensive environments, such as power space converters. The best physical properties of GaN HEMTs, such as inherent radiation hardness, low on resistance and parasitic capacitances, allow them to switch at higher frequencies with high efficiency achieving higher power density. We present an optimized design procedure to guaranty the zero-voltage switching condition that enables the power density to be increased without a penalization of the efficiency.
Patient compliance with oral nutritional supplements (ONS) is not optimal for meeting energy and nutritional requirements in a high proportion of patients with disease-related malnutrition (DRM). ...Energy density or prescribed volume of ONS may impact compliance.
A randomized, open-label crossover trial was conducted in outpatients with DRM to compare compliance with a high energy-dense ONS (edONS, 2.4 kcal/mL) and a reference ONS (heONS, 2.0 kcal/mL; NCT05609006). Patients were randomly assigned to two 8-week treatment sequences of four-weeks periods: edONS + heONS (sequence A) or heONS + edONS (sequence B). Patients daily reported the amount of product left over gastrointestinal tolerance and satisfaction with ONS. A non-inferiority analysis was performed to compare the compliance rate (percentage of consumed energy over the prescribed) for each period and sequence.
Fifty-three patients were assigned to sequence A and 50 to sequence B (55.7 ± 13.9 years, 37.0% female, 67.1% oncology patients). In sequence A, the compliance rates were 88.6% ± 14.3% vs. 84.1 ± 21.8% (
= 0.183), while in sequence B, they were 78.9% ± 23.8% vs. 84.4% ± 21.4% (
< 0.01). In both sequences, the lower range of the confidence interval for compliance with edONS was greater than the non-inferiority threshold (for sequence A Δ
was 4.5% 95% CI, -2.0% to 10.0%, and for sequence, B Δ
was 5.6% 95% CI, -3.0% to 14.0%). The total discarded cost for each ONS was higher for heONS than edONS, being the difference statistically significant in sequence B. BMI increased slightly and not significantly in both sequences, and the percentage of patients with severe malnutrition was reduced. The frequency of gastrointestinal symptoms was low for both sequences, and satisfaction with ONS was slightly higher for edONS.
Our findings highlight that edONS was non-inferior to heONS in terms of consumed energy over the prescribed, with a lower amount of edONS discarded, which suggests a higher efficiency of edONS.
Multiple myeloma is a hematologic malignancy affecting bone marrow derived plasma cells. Current therapies are not able to eradicate the disease and most patients become refractory to the treatment. ...Lenalidomide and bortezomib have proved effective in the second-line treatment of these patients.
To evaluate the cost-effectiveness of lenalidomide in combination with dexamethasone compared to bortezomib in combination with dexamethasone in patients with multiple myeloma previously treated with bortezomib, from the perspective of the Chilean National Health Service.
A four-state Markov model (preprogression on treatment; preprogression off treatment, progression and death) was used to simulate the evolution of a cohort of multiple myeloma patients over a 25-year time horizon. Efficacy data, resource use and frequency of adverse events were extracted from MM009/010 studies and a retrospective analysis of retreatment with bortezomib. All inputs were validated by experts. A 3% annual discount rate was used for costs and health outcomes. The robustness of the results was evaluated through univariate and probabilistic sensitivity analyses.
Lenalidomide in combination with dexamethasone treatment provided 1.41 incremental life years and 0.83 incremental quality-adjusted life years in comparison with bortezomib in combination with dexamethasone, with an incremental cost of 11 864 597.86 CLP (19 589.86 US$). The incremental cost-effectiveness and cost-utility ratio were estimated at 8 410 266.92 CLP (13 886,35 US$) / incremental life year and 14 271 896.16 CLP (23 564,59 US$)/incremental quality-adjusted life years, respectively.
Lenalidomide in combination with dexamethasone represents a potentially cost-effective alternative for the second-line treatment of patients with multiple myeloma who are not eligible for transplantation, from the perspective of the Chilean National Health Service.
Parkinson's disease (PD) is a progressive, neurodegenerative disorder whose symptoms and manifestations greatly deteriorate the health, functional status and quality of life of patients, has severe ...consequences on their families and caregivers and supposes a challenge for the healthcare system and society. The aim of this paper is to comprehensively and descriptively review studies on the economic impact of the disease and interventions, analyzing major contributing factors to direct and indirect costs in PD. Cost-of-illness studies have shown that costs of PD are high, mainly due to drug, hospitalization and productivity loss, and tend to increase as the disease progresses. Studies on PD treatment have suggested that therapies for advanced PD (levodopa/carbidopa intestinal gel and apomorphine) and surgical procedures are cost-effective and cost saving, despite their high expenditures; however, further research such as on the economic impact of non-motor manifestations or on the cost-effectiveness of non-medical interventions is still needed.
Introduction
To determine patient and rheumatologist preferences for rheumatoid arthritis (RA) treatment attributes in Spain and to evaluate their attitude towards shared decision-making (SDM).
...Methods
Observational, descriptive, exploratory and cross-sectional study based on a discrete choice experiment (DCE). To identify the attributes and their levels, a literature review and two focus groups (patients P = 5; rheumatologists R = 4) were undertaken. Seven attributes with 2–4 levels were presented in eight scenarios. Attribute utility and relative importance (RI) were assessed using a conditional logit model. Patient preferences for SDM were assessed using an ad hoc questionnaire.
Results
Ninety rheumatologists 52.2% women; mean years of experience 18.1 (SD: 9.0); seeing an average of 24.4 RA patients/week (SD: 15.3) and 137 RA patients mean age: 47.5 years (SD: 10.7); 84.0% women; mean time since diagnosis of RA: 14.2 years (SD: 11.8) and time in treatment: 13.2 years (SD: 11.2), mean HAQ score 1.2 (SD: 0.7) participated in the study. In terms of RI, rheumatologists and RA patients viewed: time with optimal QoL: R: 23.41%/P: 35.05%; substantial symptom improvement: R: 13.15%/P: 3.62%; time to onset of treatment action: R: 16.24%/P: 13.56%; severe adverse events: R: 10.89%/P: 11.20%; mild adverse events: R: 4.16%/P: 0.91%; mode of administration: R: 25.23%/P: 25.00%; and added cost: R: 6.93%/P: 10.66%. Nearly 73% of RA patients were involved in treatment decision-making to a greater or lesser extent; however, 27.4% did not participate at all.
Conclusion
Both for rheumatologists and patients, the top three decision-making drivers are time with optimal quality, treatment mode of administration and time to onset of action, although in different ranking order. Patients were willing to be more involved in the treatment decision-making process.
Resumen: Introducción: La fibrosis pulmonar idiopática (FPI) impacta en la vida del paciente y de sus familiares, por ello, resulta necesario conocer su percepción en el abordaje de su enfermedad. ...Métodos: Estudio observacional mediante un cuestionario específico que recoge: variables sociodemográficas y clínicas, percepción sobre el impacto de la enfermedad (escala Likert 5 puntos), preferencias acerca de las características de la medicación (grado importancia/preocupación 1-10) y satisfacción con el tratamiento (Treatment Satisfaction with Medicines Questionnaire SATMED-Q® escala 0-100). Resultados: Participaron 69 individuos (edad: 66,5±7,6 años; tiempo hasta diagnóstico: 16,5±17,4 meses; tiempo de diagnóstico: 49,6±42,3 meses). La mayoría indicó que la FPI les limita física (90%) y emocionalmente (75%). Las características más valoradas del tratamiento fueron: condición de enlentecer la progresión (7,4±2,8), estabilizar la capacidad pulmonar (6,9±2,8) y mejorar la calidad de vida (6,9±2,8), por encima de mejorar/estabilizar los síntomas (6,1±2,8/6,3±2,8) o evitar la hospitalización (6,6±2,7). El riesgo de sufrir molestias gástricas (7,1±2,9), fotosensibilidad (6,6±3,0) e interacciones con otros fármacos (6,0±3,0) fueron los principales factores de preocupación. La satisfacción global con el tratamiento actual fue de 61,1 puntos, otorgando las puntuaciones más altas al seguimiento médico (79,5) y la opinión general sobre la medicación (74,3). Conclusiones: Estudio realizado en España que describe la perspectiva del paciente con FPI con respecto a la enfermedad y su tratamiento. Los resultados muestran un elevado nivel de concienciación acerca de la gravedad de la enfermedad por parte de los pacientes, cuyo principal interés es enlentecer su progresión. La información proporcionada puede contribuir a optimizar el manejo del paciente con FPI. Abstract: Introduction: Idiopathic pulmonary fibrosis (IPF) impacts the life of patients and their families, so it is necessary to ascertain their perception in the approach to their disease. Methods: Observational study by means of a specific questionnaire that includes socio-demographic and clinical variables, perception of the impact of the disease (5-point Likert scale), preferences regarding the characteristics of the medication (degree of importance/concern 1-10) and satisfaction with treatment (SATMED-Q® scale 0-100). Results: 69 individuals participated (age: 66.5±7.6 years; time until diagnosis: 16.5±17.4 months; diagnosis time: 49.6±42.3 months). The majority state that IPF limits them physically (90%) and emotionally (75%). The most highly valued features of the treatment were: slowing down progression of the disease (7.4±2.8), stabilising lung capacity (6.9±2.8) and improving quality of life (6.9±2.8), above stabilising/improving symptoms (6.1±2.8/6.3±2.8) or avoiding hospitalisation (6.6±2.7). The principal factors of concern were suffering gastric disorders (7.1±2.9), photosensitivity (6.6±3.0) or interaction with other drugs (6.0±3.0). Overall satisfaction with the current treatment scored 61.1 points, with the highest scores being for medical follow-up (79.5) and overall opinion of the medicine (74.3). Conclusions: A study conducted in Spain on the perspective of IPF patients regarding the disease and its treatment. The results show a high level of awareness in terms of the seriousness of the disease on the part of patients, whose main concern is to slow down its progression. The information provided may help to optimise the management of IPF patients.
Objective: To compare the cost consequence of biologic drugs for moderate-to-severe psoriasis from the perspective of the Spanish National Health System.
Methods: We built a decision tree with a ...two-year time horizon. Efficacy data for biologics (etanercept, infliximab, adalimumab, ustekinumab and secukinumab) were drawn from published meta-analyses: PASI75 for the induction phase and PASI90 for the rest of follow-up. Patients with PASI < 75 at week 10-16 were switched to another biologic agent. Efficacy at week 24 was considered the highest possible efficacy for each drug and assumed to remain constant throughout the two-year period. Only drug treatment costs were used. The number needed to treat (NNT), annual cost per patient, annual cost per patient with PASI90 (cost per responder) and cost of primary failure (PASI < 75 at first efficacy evaluation) were calculated.
Results: Secukinumab monotherapy was associated with the lowest cost per responder, followed by infliximab and ustekinumab. Treatment sequences starting with secukinumab were the most efficient, having the lowest NNT and cost per responder. Although the annual cost per treatment is similar for all drugs, there are huge differences in the cost per responder.
Conclusions: Secukinumab as first-line biologic treatment is the most efficient treatment for moderate-to-severe plaque psoriasis in the short-to-medium term.
We aimed to validate the Spanish version of the Decisional Conflict Scale (DCS) and analyze its psychometric properties in people with migraine.
The DCS validation comprised two phases. First, a ...translation and cross-cultural adaptation following the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Good Practices: 1-preparation, 2-independent forward translation, 3-reconciliation, 4-back-translation, 5-harmonization, 6-clinical review, and 7-content validation in a group of migraine patients. Second, the analysis of the psychometric properties. The reliability or internal consistency of the DCS scale and subscales was assessed using Cronbach's α value. The item-subscale correlation was also evaluated. A floor and ceiling effects for DCS score was considered when at least 15% of respondents obtained DCS >90 (ceiling) or <10 (floor). The construct validity was studied through the correlation between the DCS subscales and by the correlation between the DCS and other questionnaires (Decision Self-Efficacy Scale DSES and 9-item Shared Decision-Making SDM-Q-9). Spearman's coefficients were estimated for the correlations.
The cross-cultural adaptation was conducted on 17 patients who completed the questionnaire in a mean of 2.4 ±1.1 minutes. Generally, more than 75% of them considered that DCS items were adequate, easy to understand, and relevant. The psychometric properties were evaluated in a sample of n=128 patients. Accordingly, the internal consistency of DCS was high, with a Cronbach's α of 0.97 for the scale and between 0.87 to 0.96 for subscales. Also, a slight floor effect was observed, with 24.2% of patients having DCS scores <10. The correlation between subscales exceeded Spearman's coefficient of 0.7. Whereas the correlation between the DCS and the other questionnaires was generally moderate (Spearman's coefficient >0.4).
The Spanish version (Spain) of the DCS has very acceptable psychometric properties (reliability and construct validity) and good potential for assessing decisional conflicts among migraine patients.
Background: To describe the benefit of patient-reported symptom monitoring on clinical, other patient-reported, and economic outcomes. Methods: We conducted a systematic literature review using ...Medline/PubMed, limited to original articles published between 2011 and 2021 in English and Spanish, and focused on the benefit of patient-reported symptom monitoring on cancer patients. Results: We identified 16 reports that deal with the benefit of patient-reported symptom monitoring (collected mostly electronically) on different outcomes. Five studies showed that patient-reported symptom surveillance led to significantly improved survival compared with usual care—mainly through better symptom control, early detection of tumor recurrence, and extended chemotherapy use. Additionally, three evaluations demonstrated an improvement in Health-Related Quality of Life (HRQoL) associated with this monitoring strategy, specifically by reducing symptom severity. Additionally, six studies observed that this monitoring approach prevented unplanned emergency room visits and hospital readmissions, leading to a substantial decrease in healthcare usage. Conclusions: There is consistent evidence across the studies that patient-reported symptom monitoring might entail a substantial survival benefit for cancer patients, better HRQoL, and a considerable decrease in healthcare usage. Nonetheless, more studies should be conducted to demonstrate their effectiveness in addition to their cost-effectiveness in clinical practice.
PDF
