Highlights • Cost–benefit analysis to compare liraglutide vs. sitagliptin for the control of T2DM in Italy. • The cost of control is lower for liraglutide at 26 weeks. • The cost of control is ...similar for liraglutide and sitagliptin at 52 weeks. • The cost of control is lower for sitagliptin-to-liraglutide switched patients at 78 weeks.
To explore the preferences of Spanish healthcare professionals (haematologists and hospital pharmacists) for the treatment selection of active Chronic Lymphocytic Leukaemia (CLL) patients at first ...relapse, condition that mainly afflicts older adults.
A discrete choice experiment (DCE) was conducted among haematologists and hospital pharmacists. A literature review and a focus group informed the DCE design. CLL treatment settings were defined by seven attributes: four patient/disease-related attributes (age, functional status, comorbidities, and risk of the disease) and three treatment-related attributes (efficacy hazard ratio of progression-free survival, HR-PFS, rate of discontinuations due to adverse events and cost). A mixed-logit model was used to determine choice-based preferences. Relative importance (RI) of attributes was calculated and compared between stakeholders. Willingness-to-pay (WTP) was estimated through the DCE. Besides, nine ad-hoc questions were posed, to explore more in depth CLL treatment decision making.
A total of 130 participants (72 haematologists and 58 hospital pharmacists) answered the DCE. All attributes were significant predictors of preferences (p < 0.05) in the multinomial model. Higher RI was obtained for treatment-related attributes: the highest rated being ‘cost’ (23.8%) followed by ‘efficacy’ (20.9%). Regarding patient-related attributes, the highest RI was obtained for ‘age’ (18.1%). No significant differences (p > 0.05) in RI between haematologists and pharmacists were found. WTP for the treatment was higher for younger CLL patients. Ad-hoc questions showed that patient age and functional status influence treatment decisions.
For healthcare professionals, ‘cost’ and ‘efficacy’ (treatment-related attributes) and age (patient-related attribute) are the main factors that determine CLL treatment selection at first relapse. WTP decreases as patient's age increases.
Introduction
NutriQoL
®
(Nestlé Health Science, Vevay, Switzerland) is a questionnaire developed to assess the health-related quality-of-life (HRQoL) of patients with home enteral nutrition (HEN) ...irrespective of their underlying condition and route of administration. The aim of this work is assessing the questionnaire’s reliability and responsiveness to change.
Methods
Two cohorts of patients with HEN and their primary caregivers were enrolled to assess reliability and responsiveness, respectively. All participants had to be 18 years of age or older, without mental deterioration (≤3 or 4 errors in the Pfeiffer’s test) and with sufficient functional status (>40 points on Karnovsky’s performance status scale). When the patients’ ability to respond to the questionnaire was impaired due to underlying disease, their caregivers answered on their behalf. NutriQoL was administered in two and three visits to reliability and responsiveness cohorts, respectively. Test–retest reliability and internal consistency were assessed by the intra-class correlation coefficient (ICC) and the Cronbach’s
α
, respectively. Responsiveness was evaluated by standardized effect size and standardized response mean between basal visit and third visit. Finally, the minimal clinically important difference (MCID) was estimated.
Results
A total of 54 and 86 participants were recruited to the reliability and responsiveness cohort, respectively. Thirty-five caregivers were selected to assess the inter-observer reliability. ICC values confirmed the good reproducibility level (ICC >0.75) of the questionnaire in both “physical functioning and activities of daily living” and “social life” domains and total score. The assessment of internal consistency in both domains of the questionnaire showed good internal consistency in visit 2. ICC showed the excellent agreement level between caregiver and patient in the global NutriQoL score. Finally, patients classified as having a minimal change in their health reported a mean (standard deviation) MCID in NutriQoL score of 0.63 (11.51).
Conclusion
NutriQoL is a reliable and unique instrument to measure the HRQoL in HEN patients. NutriQoL detects changes in the health status of the patient. Nevertheless, further research is needed to determine the full extent of the questionnaire responsiveness.
Introduction
The management of postmenopausal osteoporosis (PMO) in routine clinical practice differs considerably from guideline recommendations. The objective of our study was to reach a consensus ...on the management of PMO, considering prevention, diagnosis, treatment and follow-up, according to expert opinion in Spain.
Methods
A two-round Delphi technique was conducted, including 38 experts. The questionnaire contained 35 sections, each one including 1–10 questions (
n
= 308) based on a literature review and contributions from the scientific steering committee. Each question was scored by experts from the current (1 = no occurrence, 9 = occurrence in all cases), wish (1 = total rejection; 9 = wish) and prediction (1 = no occurrence at all; 9 = occurs with maximum probability) perspectives. Consensus (wish and prediction perspectives) was considered when ≥75% of experts scored 7–9 (agreement) or 1–3 (disagreement).
Results
Overall, consensus was achieved on 75% of questions. While protocols of clinical management and consultation/referral should be followed, their implementation is unlikely. Furthermore, the medical specialties currently involved in PMO management are poorly defined. PMO patients without fracture should be managed (prevention, diagnosis, treatment and follow-up) in both primary care and rheumatology settings; however, experts predicted that only treatment and follow-up will be assumed by these specialties. A multidisciplinary team should be involved in patients with fracture. No assessment tools are usually applied, and prediction indicated that they will not be used.
Conclusion
Efforts should be focused on questions with high divergence between wishes and predictions, defining actions that will improve PMO management. Collaboration between scientific societies and health authorities to address the identified opportunities of improvement is proposed.
Funding
Amgen S.A.
Introduction
An Excel
®
(Microsoft Corporation) model was adapted to estimate the short-term (1-year) cost effectiveness of insulin detemir (IDet) versus neutral protamine Hagedorn (NPH) insulin in ...patients initiating insulin treatment with type 1 diabetes mellitus (T1DM) and type 2 diabetes mellitus (T2DM) in Spain.
Methods
Clinical benefits included the non-severe hypoglycemia rate for T1DM and T2DM, and weight change for T2DM. Three scenarios were included with different hypoglycemia rates estimated on the basis of clinical trials and observational studies. Costs, estimated from perspective of the Spanish Public Healthcare System (Euros 2014), included insulin treatment and non-severe hypoglycemia management costs. Non-severe hypoglycemia, defined as a self-managed event, implied the use of extra glucose testing strips and a general practitioner visit during the week following the event for 25% of patients. An average disutility value was associated to non-severe hypoglycemia events and, for T2DM, to one body mass index unit gain to calculate quality-adjusted life years (QALYs).
Results
For the three scenarios a range of 0.025–0.076 QALYs for T1DM and 0.014–0.051 QALYs for T2DM were gained for IDet versus NPH due to non-severe hypoglycemia and weight gain avoidance, in return of an incremental cost of €145–192 for T1DM and €128–206 for T2DM. This resulted in the IDet versus NPH incremental cost-effectiveness ratio (ICER) ranging between €1910/QALY and €7682/QALY for T1DM and €2522/QALY and €15,009/QALY for T2DM.
Conclusion
IDet was a cost-effective alternative to NPH insulin in the first year of treatment of patients with T1DM and patients with T2DM in Spain, with ICERs under the threshold value commonly accepted in Spain (€30,000/QALY).
Funding
Novo Nordisk.
Understanding the epidemiology of moderate-to-severe psoriasis is essential for its management.
To assess the epidemiological characteristics of patients with moderate-to-severe psoriasis.
...Cross-sectional, observational epidemiological study conducted in Spain and Portugal. Data were collected by 332 dermatologists for >or=10 consecutive presenting patients.
Based on body surface area (BSA) and Psoriasis Area and Severity Index (PASI) criteria, moderate-to-severe psoriasis was confirmed in >or=79.3% of patients (n = 3,320). Preexisting comorbid conditions included psoriatic arthropathy (13%), dyslipidemia (14.1%) and hypertension (20.2%). The mean BSA involvement was 23% (95% confidence interval, CI: 22.2-23.3%), and the mean PASI score was 14.3 (95% CI: 13.9-14.6%). During the 2 years prior to assessment, 97.0% of patients had received topical treatments, whereas 31.3% had not received systemic treatment or phototherapy. The median annual cost of treatment was 825 EUR.
Moderate-to-severe psoriasis is accurately diagnosed, but inadequately treated in many patients in Spain and Portugal.
To assess whether the use of median overall survival to define clinically meaningful outcomes in the area of oncology could yield different decisions compared with those obtained with a more ...realistic measure such as individual survival.
Two scenarios that offered equivalent health gains/money spent were presented: 'median overall survival' scenario (new treatment provided small clinical benefits for the average population) and 'individual survival'scenario (new treatment provided substantial clinical benefits for a small percentage of the patients and no benefits for the rest). Responses from both scenarios were compared.
Responses between the two scenarios were different for oncologists, healthcare policy makers and patients (p < 0.05). 'Individual survival' scenario obtained higher percentage of positive answers compared with 'median overall survival'.
Expressing the benefits of new oncologic treatments in terms of 'individual survival' may yield to different healthcare decisions compared with the widely used median overall survival.
Abstract Psoriasis is a chronic skin disease that negatively impacts on patient’s life. A holistic approach integrating well-being assessment could improve disease management. Since a consensus ...definition of well-being in psoriasis is not available, we aim to achieve a multidisciplinary consensus on well-being definition and its components. A literature review and consultation with psoriasis patients facilitated the design of a two-round Delphi questionnaire targeting healthcare professionals and psoriasis patients. A total of 261 panellists (65.1% patients with psoriasis, 34.9% healthcare professionals) agreed on the dimensions and components that should integrate the concept of well-being: emotional dimension (78.9%) stress (83.9%), mood disturbance (85.1%), body image (83.9%), stigma/shame (75.1%), self-esteem (77.4%) and coping/resilience (81.2%), physical dimension (82.0%) sleep quality (81.6%), pain/discomfort (80.8%), itching (83.5%), extracutaneous manifestations (82.8%), lesions in visible areas (84.3%), lesions in functional areas (85.8%), and sex life (78.2%), social dimension (79.5%) social relationships (80.8%), leisure/recreational activities (80.3%), support from family/friends (76.6%) and work/academic life (76.5%), and satisfaction with disease management (78.5%) treatment (78.2%), information received (75.6%) and medical care provided by the dermatologist (80.1%). This well-being definition reflects patients’ needs and concerns. Therefore, addressing them in psoriasis will optimise management, contributing to better outcomes and restoring normalcy to the patient’s life.
To synthesize information available in the literature on patients' preferences and satisfaction with osteoporosis treatment and their unmet needs on the treatment decision-making process.
Systematic ...literature review consulting international database and grey literature of articles published between January 1, 2009 and January 1, 2019.
Nineteen publications were reviewed, 79% of them focused on evaluating the importance that patients attached to the mode and frequency of administration, adverse events and treatment efficacy. 21% of them provided information about treatment satisfaction and 26% regarding unmet needs on treatment-decision making process.
Aligning treatment with patients' preferences, promoting physician-patient communication and identifying patients' concerns with treatment may contribute to improve treatment satisfaction and adherence and ultimately achieve the treatment goal.
The purpose of this work is to estimate the costs associated with managing patients with MS in Panama and evaluating the impact of the disease on their health-related quality of life (HRQoL). ...Multicentric observational, retrospective, cross-sectional study. The costs were estimated from societal and patient perspectives and expressed in USD, 2015. The focus of the study is based on prevalence and on a "bottom-up" approach. To estimate the total cost per patient, annual reported use for each resource was multiplied by its unit cost. To evaluate HRQoL, patients completed the EQ-5D-3L questionnaire. 108 patients took part in the study. 82.41% were women with 44.78 (SD: 12.27) years. 61.11% presented mild (EDSS = 0-3.5), 25.93% moderate (EDSS = 3.5-6) and 12.96%, severe disability (EDSS≥6.5). The mean annual cost from the patient's perspective was estimated at 777.99 USD (SD: 1,741.45) per patient. The mean cost from a societal perspective was estimated at 23,803.21 USD (SD: 13,331.83) per patient. Disease-modifying therapies (DMT) accounted for the main component of the cost. A deterioration in HRQoL was observed as the disease advances and as disability increases, with mobility and usual activities being the areas most affected by its progression. From both perspective, the cost per MS patient in Panama is high. In addition to the high economic impact, MS also exerts a negative impact on patient HRQoL, which increases as the disease advances.
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