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

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

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

Orphan drugs revisited : [comment]

Hughes et al.1 recently discussed arguments for and against giving special funding status to orphan drugs in this journal. They concluded that there should be a uniform policy across Europe, that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive. The aims of this paper are to correct some inaccuracies in the original paper, develop some of the key issues, and to draw some conclusions regarding the question ‘Do drugs for exceptionally rare disease deserve special status for funding?’ For ease, our paper adopts the same structure as the original

Private Food Assistance in a Small Metropolitan Area: Urban Resources and Rural Needs

Food banks and other private feeding programs have become an institutionalized component of the social welfare system in over 190 urban areas in the U.S. More recently, private food assistance has gained importance in rural areas as well. The density and capacity of agencies to serve the poor is higher in urban areas than in sparsely populated rural locales where distance and dispersal tend to be barriers to supplying and accessing donated food. Rural food distribution strategies thus must be qualitatively different than those in larger communities, because of the smaller-scale, more informal distributional system. Little is known about how urban-based nonprofit services stimulate and support food assistance in surrounding rural locales. Based on intensive interviews with food bank stafffood pan try directors, and food pantry clientele, we examine obstacles affecting the use of food pantries and the amelioration of food insecurity. We also provide an assessment of how changes in federal welfare provisions may be affecting the need for private food assistance

What type of learning journey do students value most? Understanding enduring factors from the NSS leading to responsible decision-making

Purpose The purpose of this research is to support responsible decision-making in Higher Education (HE) settings by understanding what type of learning journey satisfies students most in their HE experience and what they want from the learning. Design/methodology/approach This paper analyses the key tool used to assess satisfaction factors for UK students, the National Student Survey (NSS). It adopts peculiar regression statistical tests to identify the NSS items that influence “overall student satisfaction” by reviewing responses over 9 years from accountancy students at business schools located in England. Findings The findings of the study provide evidence that students are most satisfied with a learning journey where they are part of a course that is “well organised and running smoothly”, which provides “intellectual stimulation” that helps in developing their ability to “present themselves with confidence” and provides “academic advice and support”. The findings of the paper show that students are not satisfied so much by utilitarian aspects of learning but rather those that relate to who they are and where they are in their learning journey, the level of intellectual stimulation they have experienced, the self-confidence they have developed and the supportive relationship they have developed with academics. A factor that did not relate highly was “assessment and feedback” which has been the focus of much university resource. Results show the factors that impacted overall satisfaction are most related to students wanting to develop personal responsibility. These findings shape the key principles of responsible design and management of HE programmes and influence strategic decision-making. Practical implications Focussing on helping students experience, the type of learning journey that develops the virtue of responsibility emergent from the analysis will not only satisfy the student but will also have a knock-on effect of improving NSS scores, university league table ranking and accreditation under the Teaching Excellence Framework. The improved reputation aspects would then feed back into increased student satisfaction (Dean and Gibbs, 2015). The findings will also help HE managers and leaders to evaluate their decisions through three lenses: responsibility, students’ experience and students overall learning journey. Originality/value Much of the information published on the NSS have been predominantly descriptive and has resulted in decisions being made for students based on uninformed analysis of the survey’s results. This study uses advanced statistical modelling to evidence the relationship between factors of the NSS and overall student satisfaction providing key information regarding students’ importance to the type of learning journey they value and that this relates to a desire in wanting to develop responsibility. This study shows the link between factors of the NSS to provide useful lenses for HE managers and leaders to use to support responsible decision-making processes

Searching for a threshold, not setting one: The role of the National Institute for Health and Clinical Excellence

There has been much speculation about whether the National Institute for Health and Clinical Excellence (NICE) has, or ought to have, a'threshold' figure for the cost of an additional quality-adjusted life-year above which a technology will not be recommended for use. We argue that it is not constitutionally appropriate for NICE to set such a threshold, which is properly the business of parliament. Instead, the task for NICE is as a 'threshold-searcher' - to seek to identify an optimal threshold incremental cost-effectiveness ratio, at the ruling rate of expenditure, that is consistent with the aim of the health service to maximize population health. This will involve the identification of technologies currently made available by the National Health Service that have incremental cost-effectiveness ratios above the threshold, and alternative uses for those resources in the shape of technologies not currently provided that fall below the threshol

Food Security of Low-Income Single Parents in East Alabama: Use of Private and Public Programs in the Age of Welfare Reform

Despite a strong economy, the use of private, nonprofit food assistance is increasing. To determine how single parenthood affects the use of both public and private food assistance, a sample of food bank clients and low-income, food-needy non-clients in East Alabama was interviewed. Overall, single-parent food-pantry clients indicated higher levels of food insecurity than other groups, but the non-clients who were not single parents also indicated high levels of need. Although 42 percent of food bank clients were single parents, results showed that married couples with children were more highly represented among the food bank clients than among food-needy individuals who do not use the pantry. Single parents were more likely than others to receive food stamp and Temporary Assistance to Needy Families (TANF) benefits, a finding that corresponds to this group\u27s lower incomes and larger family sizes

Calculating partial expected value of perfect information via Monte Carlo sampling algorithms

Partial expected value of perfect information (EVPI) calculations can quantify the value of learning about particular subsets of uncertain parameters in decision models. Published case studies have used different computational approaches. This article examines the computation of partial EVPI estimates via Monte Carlo sampling algorithms. The mathematical definition shows 2 nested expectations, which must be evaluated separately because of the need to compute a maximum between them. A generalized Monte Carlo sampling algorithm uses nested simulation with an outer loop to sample parameters of interest and, conditional upon these, an inner loop to sample remaining uncertain parameters. Alternative computation methods and shortcut algorithms are discussed and mathematical conditions for their use considered. Maxima of Monte Carlo estimates of expectations are biased upward, and the authors show that the use of small samples results in biased EVPI estimates. Three case studies illustrate 1) the bias due to maximization and also the inaccuracy of shortcut algorithms 2) when correlated variables are present and 3) when there is nonlinearity in net benefit functions. If relatively small correlation or nonlinearity is present, then the shortcut algorithm can be substantially inaccurate. Empirical investigation of the numbers of Monte Carlo samples suggests that fewer samples on the outer level and more on the inner level could be efficient and that relatively small numbers of samples can sometimes be used. Several remaining areas for methodological development are set out. A wider application of partial EVPI is recommended both for greater understanding of decision uncertainty and for analyzing research priorities