Paediatric indications and dosing guidance for advanced targeted treatments in Australia.

As with adults, paediatric patients may benefit from a number of advanced targeted therapies for inflammatory skin disease. This brief report aims to be an accessible reference tool with respect to regulatory approval and reimbursement of these treatments within Australia.

Costs of UK community care for individuals with recessive dystrophic epidermolysis bullosa: Findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study.

Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare inherited skin fragility disorder requiring multidisciplinary management. Information regarding costs of current standard treatment is scant. Objectives: As part of a longitudinal natural history study, we explored the community care costs of UK patients with different forms of RDEB. Methods: Fifty-nine individuals with RDEB provided detailed information on multiple facets of RDEB including disease severity scores (iscorEB, BEBS) and patient reported outcomes (quality of life evaluation in epidermolysis bullosa, iscorEB patient questionnaire). Costs data included time spent doing dressings, frequency of dressing changes, details of materials used, and paid and unpaid care. Results: Overall costs of dressing materials and associated care were high in RDEB. Median annual costs across all subtypes for those using dressings (n = 51) were over £26 000. For severe RDEB (RDEB-S), median costs were almost £90 000 per annum, with a median of 18 h per week spent on dressing changes. Half of working-age adults with RDEB were unemployed and 39% of carers were unable to take on full-time or part-time paid employment, adding to indirect costs and the financial burden from RDEB on families and society. Conclusions: The findings demonstrate the high costs of care of RDEB, particularly for RDEB-S. The current expense supports the drive to develop new therapies which accelerate wound healing and diminish total wound burden, thereby reducing costs of dressings and care. While costly to bring to market, these might ultimately reduce the overall cost of treatment and also the impact on individuals living with this rare disease. The data also highlight the need for adequate reimbursement for EB care which can place significant financial strain on families.

Rubella and congenital rubella syndrome: global update / La rubéola y el síndrome de rubéola congénita: resumen de la situación actual en el mundo

Se calcula que cada año nacen en el mundo más de 100 000 niños con síndrome de rubéola congénita (SRC). La Organización Mundial de la Salud (OMS) estandarizó en 1998 las definiciones de casos para la vigilancia del SRC y de la rubéola. En 2001, 123 países o territorios notificaron 836 356 casos de rubéola y se espera que el número de países se incremente a medida que se desarrolla, bajo la coordinación de la OMS, una red mundial de laboratorios para la detección del sarampión y la rubéola. Se están realizando investigaciones para mejorar la vigilancia de esta última enfermedad, entre ellas algunos proyectos encaminados a echar a andar la vigilancia y a comparan métodos diagnósticos, así como estudios de epidemiología molecular para lograr entender más a fondo los patrones de circulación del virus de la rubéola en el mundo. En 1996 una encuesta efectuada por la OMS reveló que 78 (36 per ciento) de los 214 países o territorios que habían notificado casos de la enfermedad aplicaban la vacuna contra la rubéola como parte de su régimen de vacunación estándar. Para fines de 2002 un total de 124 de esos 214 (58 per ciento) países o territorios aplicaban la vacuna antirrubeólica cuyo uso depende del nivel de desarrollo económico: 100 per ciento en países industrializados, 71 per ciento en países con economías en transición y 48 per ciento en países en desarrollo. Se dispone de una vacuna inocua y eficaz y se ha demostrado la eficacia de algunas estrategias de vacunación para la prevención de la rubeóla y el SRC. En un trabajo de posición de la OMS se ofrece orientación acerca de lo que entraña, desde el punto de vista programático, emprender la vacunación antirrubeólica. Se trata de una medida cuya efectividad y beneficios superan su costo, pero que exige un continuo fortalecimiento de los servicios de vacunación y sistemas de vigilancia habituales.

Worldwide, it is estimated that there are more than 100 000 infants born with congenital rubella syndrome (CRS) each year. In 1998, standard case definitions for surveillance of CRS and rubella were developed by the World Health Organization (WHO). In 2001, 123 countries/territories reported a total of 836 356 rubella cases. In the future more countries are expected to report on rubella as a global measles/rubella laboratory network is further developed under the coordination of the WHO. Operational research is being conducted to improve rubella surveillance. This includes projects on initiating CRS surveillance, comparative studtes on diagnostic laboratory methods, and molecular epidemiology research to expand the global understanding of patterns of rubella virus circulation. In 1996 a WHO survey found that 78 of 214 reporting countries/territories (36%) were using rubella vaccine in their routine immunization services. By the end of 2002 a total of 124 of the 214 counties/territories (58%) were using rubella vaccine. Rubella vaccine use varies by stage of economic development: 100% for industrialized countries, 71% for countries with economies in transition, and 48% for developing countries. A safe and effective rubella vaccine is available, and there are proven vaccination strategies for preventing rubella and CRS. A WHO position paper provides guidance on programmatic aspects of rubella vaccine introduction. The introduction of rubella vaccine is cost-effective and cost-beneficial but requires ongoing strengthening of routine immunization services and surveillance systems