Our aim was to assess the value of nintedanib for non-idiopathic progressive fibrosing interstitial lung disease (non-IPF PF-ILD) and systemic sclerosis-associated ILD (SSc-ILD) in the Spanish context, using a multi-criteria decision analysis (MCDA).

Following an adaptation of the Evidence and Value: Impact on DEcision Making (EVIDEM) MCDA methodology, the estimated value of nintedanib was obtained by means of an additive linear model that combined individual weights (100-points distribution) of criteria with the individual scoring of nintedanib in each criterion for every indication, assigned by a multidisciplinary committee of twelve clinicians, patients, pharmacists, and decision-makers. To assess the reproducibility, an alternative weighting method was applied, as well as a re-test of weights and scores at a different moment of time.

The experts committee recognized nintedanib as an intervention with a positive value contribution in comparison to placebo for the treatment of non-IPF PF-ILD (0.50 ± 0.16, on a scale from −1 to 1) and SSc-ILD (0.40 ± 0.12), diseases which were considered as very severe and with high unmet needs. The drug was perceived as a treatment that provides an added therapeutic benefit for patients (0.06–0.07), given its proven clinical efficacy (0.05–0.06), slight improvements in patient-reported outcomes (0.01–0.02), and similar safety profile than placebo (−0.04–0.00), which will likely be positioned as a recommended therapy in the next clinical practice guidelines updates.

Under this increasingly used methodology, nintedanib has shown to provide a positive value estimate for non-IPF PF-ILD and SSc-ILD when compared to placebo in Spain.

Disturbed eating behaviors are a significant health concern among child and adolescents with type 1 diabetes mellitus (DM1) and are generally related to poor glycemic control, ketoacidosis, hospitalization and microvascular complications. Rates of eating problems among youths with DM1 have been reported to be as high as 38%.

To review clinical characteristics, demographic profiles and risk factors for the development of eating disturbances among child and adolescents with DM1.

We performed a literature research of articles from 1980 until present, in which a Disturbed Eating Behavior appeared comorbid with DM1 in children and adolescents, using Medline database.

Almost all studies selected report a high prevalence of eating disturbances of child and adolescents with DM1 when compared with healthy pairs. This population trend to develop body image discontent and lower self-esteem. They are more likely to diet, skip meals, and omit insulin. All these practices have been associated with worsening diabetic medical complications and poorer psychological outcome.

Due to the high prevalence and severe medical and psychological complications associated with disturbed eating behaviors among pediatric population with DM1, clinicians and school professionals may benefit from specialized training to identify the range of unhealthy weight control behaviors used by youths with DM1. Preventive programs that address disturbed eating behaviors should be provided for adolescents with DM1 in order to reduce the psychological and medical impact of this comorbid situation.

Fear is a distressing emotion aroused by a risk or a damage, real or imaginary. Fears have a warning function against dangers. Nevertheless, fear can also become in one of the most limiting elements of a person's life.

To characterize the profile of fears presented in a sample of 19 children, aged between 8 and 13 years old, who had previously been diagnosed with one of the following: Generalized Anxiety Disorder, Separation Anxiety Disorder or Social Phobia.

The sample was taken from patients who participated in a cognitive-behavioral group therapy. The Spanish version of the Fear Survey Schedule for Children-Revised (FSSC-R) questionnaire was employed. The FSSC-R asks children to indicate on a 3-point scale (‘none’, ‘some’, ‘a lot’) how much they fear 80 specific stimuli or situations. Five basic categories of fears can be stablished: failure and criticism (17 items); the unknown (17 items); minor injury and small animals (13 items); danger and death (16 items); medical fears (5 items).

The sample includes 19 children: 12 boys (63.2%) and 7 girls (36.8%). The median age is 10.74 years. The sample shows 15 excessive fears on average (those scored as ‘a lot’). Girls show higher rates of excessive fears than boys: 19 versus 13. The most common fears were ‘being hit by a car or truck’, ‘bombing attacks. Being invaded’, ‘a burglar breaking into our house’ and ‘falling from high places’.

Most common excessive fears belong to danger and death category. Females report more fears than males.

The authors have not supplied their declaration of competing interest.

Dalbavancin is a new innovative long-acting antimicrobial treatment that allows clinicians to endorse an early discharge program for patients suffering from acute bacterial skin and skin structure Infections (ABSSSI). The aim of this study was to develop a spending predictor model for evaluating the direct costs associated with the management of ABSSSI from the National Health Service (NHS) perspective of Italy, Spain, and Romania. The main purpose is to compare the hospitalization and drug costs due to the treatment of ABSSSI patients treated with standard antibiotics therapy or innovative long-acting treatment dalbavancin.

A decision-analytic model was performed to evaluate the diagnostic and clinical pathways of ABSSSI patients in the hospital, based on clinicians’ expert opinion. The standard of care scenario was compared with the dalbavancin scenario. The epidemiological and cost parameters were extrapolated from national administrative databases (hospital information system) and from a systematic literature review for each country. Only direct costs in the national payer's perspective were considered. Probabilistic sensitivity analysis (PSA) and one-way sensitivity analysis (OSA) were performed to check the robustness of the model assumptions.

Overall, the model estimated an average annual number of patients with ABSSSI equal to around 50,000 in Italy, Spain, and Romania. The introduction of dalbavancin reduced the length of stay of, on average, 3.3 days per ABSSSI patient. From the economic point of view, dalbavancin did not incur any additional cost from the NHS perspective with homogenous results between countries. The PSA and OSA demonstrated the robustness of the results.

The preliminary results highlight that the introduction of dalbavancin could generate a significant reduction in term of length of stay with no incremental cost from the NHS perspective. This model could represent a good tool for policymakers to provide information on the early discharge approach in the ABSSSI management.

Re-purposing of established products (EPs) – defined as marketed for 8 years or more – may represent a high value for patients and society. It has been recognized by the European Commission as an important factor contributing to greater access to new therapies. Due to a lower development cost, it could also represent a cost-effective alternative and help to reduce pressure on healthcare budgets. However, it is perceived that no financial incentives exist for the pharmaceutical industry to invest in new indications for EPs. The objective of this research was to review current European regulations and propose strategies stimulating development in this field.

We performed a targeted literature review and held two international expert panel workshops to discuss current policies and their implications, and issue recommendations for changes.

Within the current regulatory framework EPs face price cuts due to generic competition, reference pricing (RP), price re-negotiations or systematic price cuts, after a period of marketing presence. Extension of indications does not permit to increase or maintain the price. Generic substitution regardless of indication poses another challenge. Limited incentives in the form of an additional year of market protection exists only for new indication(s) registered within the first 8 years following initial approval. The expert panel proposed several strategies to stimulate development in this field, including: (i) extending the period in which registering a new indication results in additional market protection beyond 8 years and extending the duration of additional market protection; (ii) delaying inclusion in RP for EPs with a new value adding indication; (iii) establishing a differential pricing by indication; (iv) preventing temporarily generic substitution when an EP is prescribed for a new indication.

Current regulations represent a serious disincentive to develop new indications for EPs. Regulatory and pricing policy changes are needed to stimulate development in this important field.

We test in practice a Multiple Criteria Decision Analysis (MCDA) framework for the value assessment of a set of therapeutic options for the indication of hormone relapsed metastatic prostate cancer (mPC) through a series of simulation exercises with the participation of decision makers from different Health Technology Assessment (HTA)/insurance agencies across Europe, including TLV (Sweden), AETSA (Andalusia-Spain), INAMI-RIZIV (Belgium) and AOTMiT (Poland). The drugs evaluated were abiraterone, cabazitaxel and enzalutamide.

Using a multi-attribute value theory framework, past research outcomes and literature findings, an mPC-specific value tree was constructed incorporating relevant concerns as criteria. By adopting the MACBETH approach the different drugs were scored against the criteria through the development of value functions, relative weights were assigned to the criteria using a swing weighting technique, scores and weights were combined using an additive aggregation technique, and sensitivity analysis of results was conducted. All stages were informed through the participation of a small group of experts from each HTA/insurance agency at a series of decision conferences taking place in each country.

Value parameters considered spanned the dimensions of therapeutic impact, safety profile, innovation level and socioeconomic impact. Overall weighted preference value scores were produced reflecting the performance of the treatments against the criteria while considering their relative importance. Order of treatments’ rankings was identical across all agencies, with enzalutamide scoring highest and cabazitaxel lowest. Therapeutic impact criteria always produced the greatest relative weight. Hypothetical priority setting decisions were made based on “value-for-money” grounds through the use of “cost per unit of value” metrics by incorporating purchasing costs.

The MCDA framework tested possesses a number of characteristics that could facilitate decision making, including the systematic and explicit incorporation of value trade-offs as part of model assessment and the transparency throughout all its stages. Therefore, it has the prospects to act as a practical evaluation tool for value assessment and communication during the HTA process.