Value Assessment of Orphan Drugs and Treatments for Rare Diseases

Thesis (Master's)--University of Washington, 2016-06

Does landscape context affect habitat value? The importance of seascape ecology in back-reef systems

Seascape ecology provides a useful framework from which to understand the processes governing spatial variability in ecological patterns. Seascape context, or the composition and pattern of habitat surrounding a focal patch, has the potential to impact resource availability, predator-prey interactions, and connectivity with other habitats. For my dissertation research, I combined a variety of approaches to examine how habitat quality for fishes is influenced by a diverse range of seascape factors in sub-tropical, back-reef ecosystems. In the first part of my dissertation, I examined how seascape context can affect reef fish communities on an experimental array of artificial reefs created in various seascape contexts in Abaco, Bahamas. I found that the amount of seagrass at large spatial scales was an important predictor of community assembly on these reefs. Additionally, seascape context had differing effects on various aspects of habitat quality for the most common reef species, White grunt Haemulon plumierii. The amount of seagrass at large spatial scales had positive effects on fish abundance and secondary production, but not on metrics of condition and growth. The second part of my dissertation focused on how foraging conditions for fish varied across a linear seascape gradient in the Loxahatchee River estuary in Florida, USA. Gray snapper, Lutjanus griseus, traded food quality for quantity along this estuarine gradient, maintaining similar growth rates and condition among sites. Additional work focused on identifying major energy flow pathways to two consumers in oyster-reef food webs in the Loxahatchee. Algal and microphytobenthos resource pools supported most of the production to these consumers, and body size for one of the consumers mediated food web linkages with surrounding mangrove habitats. All of these studies examined a different facet of the importance of seascape context in governing ecological processes occurring in focal habitats and underscore the role of connectivity among habitats in back-reef systems. The results suggest that management approaches consider the surrounding seascape when prioritizing areas for conservation or attempting to understand the impacts of seascape change on focal habitat patches. For this reason, spatially-based management approaches are recommended to most effectively manage back-reef systems.

Does Landscape Context Affect Habitat Value? The Importance of Seascape Ecology in Back-reef Systems

Seascape ecology provides a useful framework from which to understand the processes governing spatial variability in ecological patterns. Seascape context, or the composition and pattern of habitat surrounding a focal patch, has the potential to impact resource availability, predator-prey interactions, and connectivity with other habitats. For my dissertation research, I combined a variety of approaches to examine how habitat quality for fishes is influenced by a diverse range of seascape factors in sub-tropical, back-reef ecosystems. In the first part of my dissertation, I examined how seascape context can affect reef fish communities on an experimental array of artificial reefs created in various seascape contexts in Abaco, Bahamas. I found that the amount of seagrass at large spatial scales was an important predictor of community assembly on these reefs. Additionally, seascape context had differing effects on various aspects of habitat quality for the most common reef species, White grunt Haemulon plumierii. The amount of seagrass at large spatial scales had positive effects on fish abundance and secondary production, but not on metrics of condition and growth. The second part of my dissertation focused on how foraging conditions for fish varied across a linear seascape gradient in the Loxahatchee River estuary in Florida, USA. Gray snapper, Lutjanus griseus, traded food quality for quantity along this estuarine gradient, maintaining similar growth rates and condition among sites. Additional work focused on identifying major energy flow pathways to two consumers in oyster-reef food webs in the Loxahatchee. Algal and microphytobenthos resource pools supported most of the production to these consumers, and body size for one of the consumers mediated food web linkages with surrounding mangrove habitats. All of these studies examined a different facet of the importance of seascape context in governing ecological processes occurring in focal habitats and underscore the role of connectivity among habitats in back-reef systems. The results suggest that management approaches consider the surrounding seascape when prioritizing areas for conservation or attempting to understand the impacts of seascape change on focal habitat patches. For this reason, spatially-based management approaches are recommended to most effectively manage back-reef systems.

INTEGRATED MANAGEMENT SYSTEMS: CRITICAL SUCCESS FACTORS

The purpose of this paper is to, in a holistic way, identify and explore the critical success factors (CSFs) that are considered in the context of the growing discussions, movements, proposed models, and case studies about the integration of management systems (MSs). This work is an investigation focused on the integration of MSs into an integrated management system (IMS) and the proposed approach takes into account the literature review as well as the experience gained by the authors on researches about Portuguese enterprises. This qualitative and empirical research, investigated the integration of MSs from existing scientific publications for the period 1999 to 2014, on-going case studies and one inquiry conducted by the authors. This research contributes to a better understanding of the CSFs regarding the integration of MSs and thus provides an insight on the preventive management. This research shows an evident lack of information regarding case studies on CSFs for integrated management systems (IMSs) and has identified a set of relevant CSFs, for MSs integration and associated guidelines that organizations should take as a priority, in order to be able to manage, on a preventive way, the implementation of IMSs, and consequently to become more competitive with added value for the stakeholders.

Learning to manage in knowledge based organizations: the role of portfolios

Paper presented at the 8th European Conference on Knowledge Management, Barcelona, 6-7 Sep. 2008 URL: http://www.academic-conferences.org/eckm/eckm2007/eckm07-home.htm

Planeamento espacial marinho das áreas marinhas protegidas: o caso de estudo da ára protegida de gestão de recursos: Caloura - ilhéu de Vila Franca do Campo

Tese de Doutoramento, Ciências do Mar (Ecologia Marinha), 26 de Novembro de 2013, Universidade dos Açores.

Avaliação da performance empresarial: o economic value added (EVA®) e a sua relação com o valor da empresa

Mestrado em Contabilidade e Gestão das Instituições Financeiras

Learning to manage in knowledge-based organizations: the role of learning logs or portfolios

Paper presented at the 8th European Conference on Knowledge Management, Barcelona, 6-7 Sep. 2008 URL: http://www.academic-conferences.org/eckm/eckm2007/eckm07-home.htm

Models and tools for value systems analysis in collaborative environments

Dissertation to obtain the degree of Doctor in Electrical and Computer Engineering, specialization of Collaborative Networks

Eficiência Energética no Hospital Pedro Hispano

Os crescentes custos ligados ao consumo elétrico, não apenas de cariz financeiro mas também ambiental, despertam cada vez mais para a importância da definição de estratégias de melhor utilização de recursos e eficiência energética. Esta importância tem sido reforçada pela definição de decretos-lei que vêm colocar metas e limites relativamente às despesas energéticas. Estes diplomas são também acompanhados por programas de incentivo para um setor ligado à eficiência energética. Em Portugal as medidas ligadas ao setor tem vindo a ser redirecionadas para o consumo final de energia, com a definição de metas para as instalações de maior consumo. As instalações hospitalares são grandes centros de consumo energético devido não só ao elevado número de utentes que recebem mas também pelos diversos tipos de equipamentos elétricos usados para a prestação dos serviços médicos. Como consequência disso, os investimentos e os custos operacionais são elevados, o que reforça a necessidade de gerir os gastos e consumos energéticos com a procura constante de melhoria na recolha de informação sobre todo o sistema e na adequação de intervenções com vista a uma maior eficiência energética. O Hospital Pedro Hispano vem desde algum tempo a investir no sentido de conhecer mais e melhor toda a instalação bem como os consumos energéticos a ela associados. Algumas medidas foram tomadas nesse sentido nomeadamente a instalação de analisadores de energia, de modo a obter um retrato mais fiel e fidedigno dos principais vetores de consumo. Neste momento a gestão técnica do hospital tem em análise uma grande parte da instalação recolhendo dados do consumo elétrico real do hospital. Nesta dissertação procurou-se fazer uma análise e enquadramento dos programas e metas ligados ao setor energético com ênfase nos diplomas que visão e abrangem as instalações hospitalares. Dos vários programas de incentivo à adoção de políticas de maior eficiência energética é dado especial destaque ao programa ECO.AP que visa a celebração de contratos para implementação de medidas de poupança energética ao setor público. Em colaboração com o HPH, iniciaram-se os trabalhos pelo estudo e identificação das principais fases e ferramentas utilizadas na gestão energética do edifício tendo como objetivo a reavaliação dos vetores energéticos já identificados no HPH e a criação e contabilização de novos grupos de consumo. Através de várias medições do consumo elétrico, num total superior a 650 horas de funcionamento, foi possível a criação do mapa de desagregação de consumos para o ano de 2013. A desagregação realizada conta com 3 novos vetores energéticos e com a reavaliação do peso relativo de mais 5 grupos de consumo. Das medições efetuadas destaca-se a reavaliação do consumo da central de bombagem onde a parcela considerada até à data estava 3 vezes acima do valor real medido. Com base na desagregação feita foram apontadas e estudadas medidas de implementação com o objetivo de reduzir os consumos energético em todo o hospital, destacando-se a solução apresentada para a central de bombagem. Esta medida traria um grande impacto em toda a fatura energética, não só pela sua viabilidade, mas também porque atuaria num grande centro de consumo onde até ao momento nenhuma ação do género foi implementada.

Cidadão e comunidade : que relevância no processo de contratualizaç

RESUMO - Nas últimas décadas, a especialização dos cuidados médicos tem conduzido a uma fragmentação do sistema de prestação, que, associada a uma deficiente coordenação entre serviços, cuidados e prestadores, torna a navegabilidade dos utentes nos sistemas de saúde uma tarefa complexa. Um novo modelo de organização, assente na procura de valor para os cidadãos, deve adoptar uma abordagem sistémica, que tenha subjacente uma coordenação integrada de serviços, numa perspectiva de ciclo de cuidados. Reorientar a prestação de cuidados para a obtenção de resultados e valor em saúde, exige uma reengenharia em torno da estrutura, organização e avaliação1 dos cuidados, requerendo, nomeadamente: i) instrumentos e ferramentas que auxiliem e estruturem este novo modelo; ii) assumpção dos papéis definidos para cada um dos actores do sistema, nomeadamente ao nível da coordenação; iii) encorajamento à adopção de modelos de contratualização, pagamento e competição, que responsabilizem os actores envolvidos não só pela prática que desenvolvem, mas pelos resultados em saúde. Estes mecanismos constituem uma oportunidade para expandir e sustentar abordagens, programas e intervenções integradas. Investir num sistema de pagamento por valor em saúde — P4V — payment for value, traduz uma aposta na relação entre diagnóstico, tratamento, resultados clínicos e custos, enquanto estratégia para assegurar ganhos em qualidade dos cuidados, eficiência dos processos e valor em saúde para o cidadão. Neste contexto, a gestão da doença enquanto modelo direccionado para o reforço da perspectiva e participação activa do cidadão, e avaliação compreensiva de novas formas de organização e gestão do sistema de prestação, constitui um instrumento para informar e sustentar esse processo de reengenharia do sistema. Um modelo que procura assegurar o encontro entre o estado da arte na prestação de cuidados e um nível óptimo, garantindo a qualidade de vida expectável para a pessoa com doença crónica. ----------------- ---------ABSTRACT – In the last decades advanced medical sciences trend to specialized care and fragmented health systems, leaving patients with a challenge on navigating services and care, requiring them to see a sequence of specialists each delivering discrete interventions. To overcome these challenges, every health system must redefine health care delivery to use its resources more efficiently and improve quality of care through an organization of the system as a whole. A system currently organized around value for patients, entails a framework that comprises the entire set of activities needed to address a patient´s medical condition, over the full cycle of care. Value- based care delivery therefore requires an integrated practice, both across services and time, and implies a movement through new structures, organization models, evaluation efforts and payment systems that enables, catalyze and reinforces the extension and sustainability of the steps needed to the change required. A shift from a payment for performance to a payment for value focuses attention on maximizing the overall value of care, and encourages coordination and integration between components of care that extends from screening, diagnoses, all the way through treatment, outcomes and costs, and ensuring an incentive for potentially high value types of care as well as innovation. These leave the actors of the system with the task of best allocating and valuing components of care. Disease management as a model designed to structure patient engagement and involvement in their care, and assure a comprehensive evaluation and monitoring of new organization and care delivery strategies align an opportunity as a source of information and sustainability for the progress of a growing number of likeminded efforts now underway across care delivery for chronic diseases. This framework will allow the fulfillment of the gap between sta

Indoor location-based analytics systems: Portuguese market compettive assessment and entry procing strategy of BIPs' technology in Sonae Sierra Shopping malls stores

This project attempts to provide an in-depth competitive assessment of the Portuguese indoor location-based analytics market, and to elaborate an entry-pricing strategy for Business Intelligence Positioning System (BIPS) implementation in Portuguese shopping centre stores. The role of industry forces and company’s organizational resources platform to sustain company’s competitive advantage was explored. A customer value-based pricing approach was adopted to assess BIPS value to retailers and maximize Sonae Sierra profitability. The exploratory quantitative research found that there is a market opportunity to explore every store area types with tailored proposals, and to set higher-than-tested membership fees to allow a rapid ROI, concluding there are propitious conditions for Sierra to succeed in BIPS store’s business model in Portugal.

Prognostic value of serial blood S100B determinations in stage IIB-III melanoma patients: a corollary study to EORTC trial 18952.

S100B is a prognostic factor for melanoma as elevated levels correlate with disease progression and poor outcome. We determined its prognostic value based on updated information using serial determinations in stage IIb/III melanoma patients. 211 Patients who participated in the EORTC 18952 trial, evaluating efficacy of adjuvant intermediate doses of interferon α2b (IFN) versus observation, entered a corollary study. Over a period of 36 months, 918 serum samples were collected. The Cox time-dependent model was used to assess prognostic value of the latest (most recent) S100B determination. At first measurement, 178 patients had S100B values <0.2 μg/l and 33 ≥ 0.2 μg/l. Within the first group, 61 patients had, later on, an increased value of S100B (≥ 0.2 μg/l). An initial increased value of S100B, or during follow-up, was associated with worse distant metastasis-free survival (DMFS); hazard ratio (HR) of S100B ≥ 0.2 versus S100B < 0.2 was 5.57 (95% confidence interval (CI) 3.81-8.16), P < 0.0001, after adjustment for stage, number of lymph nodes and sex. In stage IIb patients, the HR adjusted for sex was 2.14 (95% CI 0.71, 6.42), whereas in stage III, the HR adjusted for stage, number of lymph nodes and sex was 6.76 (95% CI 4.50-10.16). Similar results were observed regarding overall survival (OS). Serial determination of S100B in stage IIb-III melanoma is a strong independent prognostic marker, even stronger compared to stage and number of positive lymph nodes. The prognostic impact of S100B ≥ 0.2 μg/l is more pronounced in stage III disease compared with stage IIb.

Argemone mexicana decoction versus artesunate-amodiaquine for the management of malaria in Mali: policy and public-health implications.

A classic way of delaying drug resistance is to use an alternative when possible. We tested the malaria treatment Argemone mexicana decoction (AM), a validated self-prepared traditional medicine made with one widely available plant and safe across wide dose variations. In an attempt to reflect the real situation in the home-based management of malaria in a remote Malian village, 301 patients with presumed uncomplicated malaria (median age 5 years) were randomly assigned to receive AM or artesunate-amodiaquine [artemisinin combination therapy (ACT)] as first-line treatment. Both treatments were well tolerated. Over 28 days, second-line treatment was not required for 89% (95% CI 84.1-93.2) of patients on AM, versus 95% (95% CI 88.8-98.3) on ACT. Deterioration to severe malaria was 1.9% in both groups in children aged </=5 years (there were no cases in patients aged >5 years) and 0% had coma/convulsions. AM, now government-approved in Mali, could be tested as a first-line complement to standard modern drugs in high-transmission areas, in order to reduce the drug pressure for development of resistance to ACT, in the management of malaria. In view of the low rate of severe malaria and good tolerability, AM may also constitute a first-aid treatment when access to other antimalarials is delayed.

Arvojen integrointi johtamiseen - case Engel

Tutkimuksen tavoitteena on selvittää kuinka Engel-Yhtymässä arvot on liitetty johtamiseen. Keskeinen kysymys on: kuinka arvot ovat integroitu yrityksen nykyiseen johtamisjärjestelmään. Arvoja ei käsitellä irrallisena asiana, vaan liitettyinä yhteen organisaatiokulttuuriin ja johtamiseen. Tutkimus tukee arvojen merkityksen tärkeyttä osana Engelin hyvää liiketaloudellista menestystä viime vuosina. Tutkimuksen empiirinen osuus koostuu pääasiassa vuoden 2003 Arvomittari-henkilöstökyselystä ja kyselyn tulosten havainnoista. Tutkimuksessa haetaan vastauksia henkilöstön näkökulmasta strategian sisäistämiseen engeliläisten päivittäisessä työskentelyssä. Kuinka arvot on sisäistetty ja kuinka ne näkyvät arkipäivässä? Mitkä asiat estävät arvojen toteutumista ja ovatko arvot omistajien, johdon vai koko henkilöstön arvoja? Tutkimuksen tuloksia analysoidaan valitun viitekehyksen pohjalta, metodina kvalitatiivinen analyysi. Sen muodostaa ensisijaisesti Daniel Denisonin 2003 esittämä malli. Denisonin mallin mukaan tavoitteena on tarjota arvojen integroinnille eri näkökulmia. Johtopäätöksenä on kuitenkin selkeä liiketaloudellisen tuloksen parantuminen arvojohtaminen merkityksen myötä.