Ethical questions linked to rare diseases and orphan drugs - A systematic review

Background: Rare or orphan diseases have become an important target of healthcare activities all over the world. The study aims to identify ethical questions linked to rare diseases and orphan drugs and ethical principles or approaches applied to solve them. Methods: Relevant peer-reviewed articles were identified by means of a systematic review. The literature was searched from 20 May 2020 to 20 June 2020. The search included the databases PubMed, Scopus and Web of Science (2010 – April 2020). A total of 4,139 papers related to rare diseases were identified; with 1,205 papers obtained from Scopus; 2,476 papers from PubMed; and 458 from Web of Science with keyword search “ethics” AND “rare” AND “disease”, “ethical” AND “orphan”, “ethical” AND “orphan” AND “drug”, and “ethical” AND “rare“ AND “disease”. Finally, XX studies were chosen for further analysis. Results: The main findings reveal five main ethical issues. The most essential one shows that funding research and development in the field of orphan drugs poses an almost impossible dilemma. Other issues include the significance of non-economic values like compassion and beneficence in decision-making related to orphan drugs and rare diseases; the identification of limits to labelling diseases as rare; barriers to global, supranational and international cooperation; and last but not least, determining and establishing panels of decision-makers. Conclusions: A strictly global approach would be the most appropriate way to deal with rare diseases. Nonetheless, international, let alone global, cooperation seems to be comple-tely beyond the reach of the current international community, although the EU, for instance, has a centralized procedure for labelling orphan drugs. This deficit in international cooperation can be partly explained by the fact that the current technologically globalized world still lacks globally accepted ethical values and rules. This is further aggravated by unresolved international and intercultural conflicts. In addition, the sub-interests of various parties as well as the lack of desire to deal with other people’s problems need to be taken into account. The aforementioned problems are difficult to avoid. Nevertheless, let us be cautiously optimistic. At least, there are people who raise ethical questions about rare diseases and orphan drugs

Estudo comparativo de soluções em alvenaria estrutural e betão armado

No setor da construção, a principal prioridade é a redução de custos relacionados com os processos construtivos e a sua execução. Devido à crise económica global e ao crescimento da competitividade do setor da construção, é necessário analisar outras opções que confiram boas práticas de construção, cumprindo os requisitos definidos na regulamentação atual. Este estudo aborda a temática de algumas das soluções construtivas normalmente utilizadas, focando aspetos de normalização, custos, vantagens e desvantagens. É também apresentada uma avaliação comparativa entre dois sistemas construtivos, alvenaria estrutural vs. betão armado, de uma moradia construída ao abrigo de um projeto de investigação. Nessa avaliação é feita a apresentação do processo de conceção modular e conceção estrutural para ambas as soluções em fase de projeto. Este estudo também apresenta uma comparação de custos entre dois sistemas construtivos, nomeadamente a construção em alvenaria estrutura vs. construção em betão armado. Com o estudo realizado constatou-se que no projeto é notória a complexidade da alvenaria estrutural em detrimento do betão armado. A alvenaria estrutural também apresenta limitações a eventuais alterações executadas durante a vida útil do edifício. Relativamente à comparação de custos efetuada, os resultados mostram que a construção em alvenaria estrutural é um processo tecnológico apropriado e a redução de custos é significativa

Remote monitoring of patients with implantable cardioverter-defibrillators: Can results from large clinical trials be transposed to clinical practice?

SummaryBackgroundRemote monitoring (RM) is increasingly used to follow up patients with implantable cardioverter-defibrillators (ICDs). Randomized control trials provide evidence for the benefit of this intervention, but data for RM in daily clinical practice with multiple-brands and unselected patients is lacking.AimsTo assess the effect of RM on patient management and clinical outcome for recipients of ICDs in daily practice.MethodsWe reviewed ICD recipients followed up at our institution in 2009 with RM or with traditional hospital only (HO) follow-up. We looked at the effect of RM on the number of scheduled ambulatory follow-ups and urgent unscheduled consultations, the time between onset of asymptomatic events to clinical intervention and the clinical effectiveness of all consultations. We also evaluated the proportion of RM notifications representing clinically relevant situations.ResultsWe included 355 patients retrospectively (RM: n=144, HO: n=211, 76.9% male, 60.3±15.2years old, 50.1% with ICDs for primary prevention and mean left ventricular ejection fraction 35.5±14.5%). Average follow-up was 13.5months. The RM group required less scheduled ambulatory follow-up consultations (1.8 vs. 2.1/patient/year; P<0.0001) and a far lower median time between the onset of asymptomatic events and clinical intervention (7 vs. 76days; P=0.016). Of the 784 scheduled ambulatory follow-up consultations carried out, only 152 (19.4%) resulted in therapeutic intervention or ICD reprogramming. We also found that the vast majority of RM notifications (61.9%) were of no clinical relevance.ConclusionRM allows early management of asymptomatic events and a reduction in scheduled ambulatory follow-up consultations in daily clinical practice, without compromising safety, endorsing RM as the new standard of care for ICD recipients

Reduced costs with bisoprolol treatment for heart failure - An economic analysis of the second Cardiac Insufficiency Bisoprolol Study (CIBIS-II)

Background Beta-blockers, used as an adjunctive to diuretics, digoxin and angiotensin converting enzyme inhibitors, improve survival in chronic heart failure. We report a prospectively planned economic analysis of the cost of adjunctive beta-blocker therapy in the second Cardiac Insufficiency BIsoprolol Study (CIBIS II). Methods Resource utilization data (drug therapy, number of hospital admissions, length of hospital stay, ward type) were collected prospectively in all patients in CIBIS . These data were used to determine the additional direct costs incurred, and savings made, with bisoprolol therapy. As well as the cost of the drug, additional costs related to bisoprolol therapy were added to cover the supervision of treatment initiation and titration (four outpatient clinic/office visits). Per them (hospital bed day) costings were carried out for France, Germany and the U.K. Diagnosis related group costings were performed for France and the U.K. Our analyses took the perspective of a third party payer in France and Germany and the National Health Service in the U.K. Results Overall, fewer patients were hospitalized in the bisoprolol group, there were fewer hospital admissions perpatient hospitalized, fewer hospital admissions overall, fewer days spent in hospital and fewer days spent in the most expensive type of ward. As a consequence the cost of care in the bisoprolol group was 5-10% less in all three countries, in the per them analysis, even taking into account the cost of bisoprolol and the extra initiation/up-titration visits. The cost per patient treated in the placebo and bisoprolol groups was FF35 009 vs FF31 762 in France, DM11 563 vs DM10 784 in Germany and pound 4987 vs pound 4722 in the U.K. The diagnosis related group analysis gave similar results. Interpretation Not only did bisoprolol increase survival and reduce hospital admissions in CIBIS II, it also cut the cost of care in so doing. This `win-win' situation of positive health benefits associated with cost savings is Favourable from the point of view of both the patient and health care systems. These findings add further support for the use of beta-blockers in chronic heart failure

Devenir nutritionnel et neurologique à 2 ans des nouveau-nés ayant présenté une entérocolite ulcéro-nécrosante (étude sur la population des enfants hospitalisés au CHRU de Lille entre 2000 et 2004)

LILLE2-BU Santé-Recherche (593502101) / SudocPARIS-BIUM (751062103) / SudocSudocFranceF

Colonisation et infections à Straphylococcus aureus dans les unités de réanimation et des soins intensifs néonataux du CHRU de Lille (incidence, facteurs de risques et modes d'acquisition)

LILLE2-BU Santé-Recherche (593502101) / SudocPARIS-BIUM (751062103) / SudocSudocFranceF

Evaluation de l'impact du clampage tardif sur l'ictère néonatal et autres comorbidités en dehors de l'anémie néonatale chez les enfants prématurés de 32SA et moins (étude comparative avant/après mise en place du clampage tardif en salle de naissance dans le service de médecine néonatale du CHRU de Lille du 1er janvier 2008 au 30 juin 2011)

LILLE2-BU Santé-Recherche (593502101) / SudocSudocFranceF