Objectives: PPP is a chronic, debilitating, painful inflammatory skin disease characterized by localized, sterile pustules on the palms of the hands and soles of the feet. By understanding the burden of disease in this population, targeted interventions that improve patient quality of life can be developed. To our knowledge, this study is the first of its kind to describe HCRU in patients with PPP. Methods: Patients were identified as having PPP if they had $1 inpatient or 2 outpatient ICD-10 L40.3 diagnosis codes, separated by 30 to 365 days. All analyses were conducted via the Aetion Evidence Platform TM v3.17, using Optum® Clinformatics TM Data Mart, a US administrative claims database. The study period was from October 1, 2015 to March 31, 2019, with the first diagnosis code marking the index date. A general population matched cohort (MC) of 4:1, based on age and sex, was generated for context. No formal comparisons were conducted. Patient characteristics, treatment, and all-cause HCRU calculated for each visit type (inpatient, outpatient, and emergency department [ED]) during the 12-month follow up (FU) were analyzed. Results: 1291 patients with PPP were identified at baseline, and 708 had $12 months' FU. Compared with the MC, patients with PPP were more likely to have a diagnosis of hyperlipidemia (PPP: 28.7% vs MC: 20.0%), anxiety (PPP: 7.6% vs MC: 5.5%), and depression (PPP: 7.0% vs MC: 5.1%) at baseline. During the 12-month FU, 391 patients with PPP (55.2%) were treated with a systemic therapy (biologic or non-biologic) and had a median of 18.5 outpatient visits, 23.6% of patients had ED visits (median: 1.0), and 10% had inpatient visits (median: 1.0). Conclusions: Patients with PPP have more comorbidities than those in the MC, as well as high HCRU, highlighting an unmet need among these patients.

Conclusions: Most medicines receiving an initial Orphan Designation in Europe maintain this at market authorization. For the ones that do not, this is due to withdrawal of the orphan designation by the sponsor, caused by the disease prevalence and the significant benefit criteria no longer being met. Although orphan drug legislation has incentivized drug development for rare diseases in Europe, most of these drugs received an earlier FDA approval and overcoming the frequently greater reimbursement/payer hurdle in Europe can further add to this access discrepancy.

Adults reported QALY scores 20% higher than caregivers of adults (P=0.01) after ageadjustment. The mean VAS score was 79 (range: 30, 99) among adults, 55 (15, 90) among caregivers of adults, and 68 (30, 100) among caregivers of minors. Adults reported average VAS scores 24 points above caregiver groups after age adjustment (P,0.01). All groups met or exceeded the 75% instrument acceptability agreementrate (P,0.05). Conclusions: EQ-5D was responsive and acceptable for this multinational rare disease sample. While adults consistently rated their health higher than caregivers in preliminary data, the difference between groups was attenuated using the population-derived QALY estimates as compared to self-evaluation using the VAS.

El presente estudio de tipo cuantitativo, descriptivo, correlacional de corte transversal, se realizó con el objetivo de determinar la relación entre el nivel de conocimiento sobre manejo de aislamiento hospitalario y la aplicación de precauciones de aislamiento hospitalario por la (el) Enfermera(o). El universo muestral fue de 63 enfermeras (os) que laboran en los servicios de emergencia y hospitalización. Se aplicaron dos instrumentos: Test Nivel de Conocimientosobre manejo de aislamiento hospitalario y lista de cotejo para la aplicación de precauciones de aislamiento hospitalario. Los resultados obtenidos demuestran que el 73% del personal posee regular conocimiento, seguido del 14,3%, que tiene buen conocimiento. El 38,1% corresponde al personal que a veces aplica estas precauciones, seguido de lo que no aplican, en un 36,5%; finalmente se ubica un 25,4% que aplica las precauciones de aislamiento hospitalario. Se encontró relación estadísticamente significativa entre las variables de estudio al aplicar el Chi cuadrado para independencia de factores con el 95% de significación.