Rights, responsibilities and NICE: a rejoinder to Harris

Harris' reply to our defence of the National Institute for Clinical Excellence's (NICE) current cost-effectiveness procedures contains two further errors. First, he wrongly draws a conclusion from the fact that NICE does not and cannot evaluate all possible uses of healthcare resources at any one time and generally cannot know which National Health Service (NHS) activities would be displaced or which groups of patients would have to forgo health benefits: the inference is that no estimate is or can be made by NICE of the benefits to be forgone. This is a non-sequitur. Second, he asserts that it is a flaw at the heart of the use of quality-adjusted life years (QALYs) as an outcome measure that comparisons between people need to be made. Such comparisons do indeed have to be made, but this is not a consequence of the choice of any particular outcome measure, be it the QALY or anything else

Drugs for exceptionally rare diseases: a commentary on Hughes et al

Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive on orphan drugs. We critically assess these arguments, demonstrating that they failed to justify special status for treatments for rare diseases

Pre-operative optimisation employing dopexamine or adrenaline for patients undergoing major elective surgery: a cost-effectiveness analysis

<b>Objective</b>: To compare the cost and cost-effectiveness of a policy of pre-operative optimisation of oxygen delivery (using either adrenaline or dopexamine) to reduce the risk associated with major elective surgery, in high-risk patients. <b>Methods</b>: A cost-effectiveness analysis using data from a randomised controlled trial (RCT). In the RCT 138 patients undergoing major elective surgery were allocated to receive pre-operative optimisation employing either adrenaline or dopexamine (assigned randomly), or to receive routine peri-operative care. Differential health service costs were based on trial data on the number and cause of hospital in-patient days and the utilisation of health care resources. These were costed using unit costs from a UK hospital. The cost-effectiveness analysis related differential costs to differential life-years during a 2 year trial follow-up. <b>Results</b>: The mean number of in-patient days was 16 in the pre-optimised groups (19 adrenaline; 13 dopexamine) and 22 in the standard care group. The number (%) of deaths, over a 2 year follow-up, was 24 (26%) in the pre-optimised groups and 15 (33%) in the standard care group. The mean total costs were EUR 11,310 in the pre-optimised groups and EUR 16,965 in the standard care group. Life-years were 1.68 in the pre-optimised groups and 1.46 in the standard care group. The probability that pre-operative optimisation is less costly than standard care is 98%. The probability that it dominates standard care is 93%. Conclusions: Based on resource use and effectiveness data collected in the trial, pre-operative optimisation of high-risk surgical patients undergoing major elective surgery is cost-effective compared with standard treatment

Drugs for exceptionally rare diseases: a commentary on Hughes et al

Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive on orphan drugs. We critically assess these arguments, demonstrating that they failed to justify special status for treatments for rare diseases

Bayesian Value-of-Information Analysis: An Application to a Policy Model of Alzheimer's Disease

A framework is presented which distinguishes the conceptually separate decisions of which treatment strategy is optimal from the question of whether more information is required to inform this choice in the future. The authors argue that the choice of treatment strategy should be based on expected utility and the only valid reason to characterise the uncertainty surrounding outcomes of interest is to establish the value of acquiring additional information. A Bayesian decision theoretic approach is demonstrated though a probabilistic analysis of a published policy model of Alzheimer’s disease. The expected value of perfect information is estimated for the decision to adopt a new pharmaceutical for the population of US Alzheimer’s disease patients. This provides an upper bound on the value of additional research. The value of information is also estimated for each of the model inputs. This analysis can focus future research by identifying those parameters where more precise estimates would be most valuable, and indicating whether an experimental design would be required. We also discuss how this type of analysis can also be used to design experimental research efficiently (identifying optimal sample size and optimal sample allocation) based on the marginal cost and marginal benefit of sample information. Value-of-information analysis can provide a measure of the expected payoff from proposed research, which can be used to set priorities in research and development. It can also inform an efficient regulatory framework for new health care technologies: an analysis of the value of information would define when a claim for a new technology should be deemed “substantiated” and when evidence should be considered “competent and reliable” when it is not cost-effective to gather anymore information.stochastic CEA; Bayesian decision theory; value of information.

Do Actions Speak Louder than Knowledge? Action Manipulation, Parental Discourse, and Children’s Mental State Understanding in Pretense

Studies on pretense mental state understanding in young children have produced inconsistent findings. These findings could potentially emerge from the confounding influences of action manipulation or the failure to examine possible influences on individual children’s performances. To address these issues, we created a task in which 68 3- and 4-year-olds viewed two actors, side by side, on a monitor. Children were told that one actor was knowledgeable about a specific animal, whereas the other actor was not. The actors performed identical movements that were either related or unrelated to the animal they were mimicking or engaged in different behaviors contradictory to their knowledge. Saliency of action was also manipulated by presenting either dynamic images or a paused frame of the actors’ behavior (i.e., the static condition). Children performed similarly on the dynamic and static conditions. Children selected the knowledgeable actor more often in the unrelated and related trials but were not as successful at selecting the knowledgeable actor when the actor’s knowledge contradicted the actor’s behavior. Therefore, by 3 years of age, some children may understand that pretend play involves mental representations and appreciate that the mind influences a pretender’s behavior. To investigate the observed individual differences, we also examined children and parents as they engaged in reading and pretense activities prior to data collection. The frequency of parents’ cognitive mental state utterances strongly predicted performance on the mental state task. Individual differences in performance as a result of parental language and executive functioning abilities are discussed

Modelling the cost effectiveness of interferon beta and glatiramer acetate in the management of multiple sclerosis

OBJECTIVE: To evaluate the cost effectiveness of four disease modifying treatments (interferon betas and glatiramer acetate) for relapsing remitting and secondary progressive multiple sclerosis in the United Kingdom. DESIGN: Modelling cost effectiveness. SETTING: UK NHS. PARTICIPANTS: Patients with relapsing remitting multiple sclerosis and secondary progressive multiple sclerosis. MAIN OUTCOME MEASURES: Cost per quality adjusted life year gained. RESULTS: The base case cost per quality adjusted life year gained by using any of the four treatments ranged from £42 000 ($66 469; 61 630) to £98 000 based on efficacy information in the public domain. Uncertainty analysis suggests that the probability of any of these treatments having a cost effectiveness better than £20 000 at 20 years is below 20%. The key determinants of cost effectiveness were the time horizon, the progression of patients after stopping treatment, differential discount rates, and the price of the treatments. CONCLUSIONS: Cost effectiveness varied markedly between the interventions. Uncertainty around point estimates was substantial. This uncertainty could be reduced by conducting research on the true magnitude of the effect of these drugs, the progression of patients after stopping treatment, the costs of care, and the quality of life of the patients. Price was the key modifiable determinant of the cost effectiveness of these treatments

Eperythrozoonosis In Swine

Eperythrozoonosis is an acute febrile, icteroanemic disease of shoats caused by the rickettsial organism Eperythrozoon spp. There are two species which are commonly associated with swine, E. suis, which produces a febrile disease, and E. parvum, which is innoxious

Challenges for Novice School Leaders: Facing Today’s Issues in School Administration

Challenges for novice school leaders evolve as information is managed differently and as societal and regulatory expectations change. This study addresses unique challenges faced by practicing school administrators (n=159) during their first three years in a school leadership position. It focuses on their perceptions, how perceptions of present novices compare to those of experienced school leaders, and how pre-service programs can better prepare them for these challenges. Findings showed that perceptions of present novice school leaders vary somewhat from those of experienced school leaders. Two themes shared relatively the same prominence among experienced leaders as they did with novices: navigating politics and gaining a sense of credibility. Experienced leaders expounded more on specific political hurdles with school boards, other teachers, and parents, and—regarding these same populations—experienced leaders voiced that they wished they had been more successful in their earlier years in developing credibility more quickly with stakeholders. A theme not occurring at all among present novices but noted by five experienced leaders was that of adjusting to the culture of a new school as its leader. This phenomenon may be explained by the value more seasoned leaders have developed for factors inherent in a campus culture