Efficiency, equity, and NICE clinical guidelines

The stated purpose of clinical guidelines from the United Kingdom's National Institute for Clinical Excellence (NICE) is to "help healthcare professionals and patients make the right decisions about healthcare in specific clinical circumstances." However, what constitutes "the right decisions" depends on your point of view. For individual patients the right decision is that which maximises their wellbeing, and this is properly the concern of the clinician. Yet in resource constrained healthcare systems this will not always coincide with the right decisions for patients in general or society as a whole, thereby leading to some understandable tensions. NICE is a national policy making body whose responsibility is clearly broader than the individual patient. This wider viewpoint is reflected in NICE's technology appraisals by the central role afforded to cost effectiveness. We argue that the methods currently used by the NICE clinical guideline programme confuse these two viewpoints

Disk Evolution in Young Binaries: from Observations to Theory

The formation of a binary system surrounded by disks is the most common outcome of stellar formation. Hence studying and understanding the formation and the evolution of binary systems and associated disks is a cornerstone of star formation science. Moreover, since the components within binary systems are coeval and the sizes of their disks are fixed by the tidal truncation of their companion, binary systems provide an ideal "laboratory" in which to study disk evolution under well defined boundary conditions. In this paper, we review observations of several inner disk diagnostics in multiple systems, including hydrogen emission lines (indicative of ongoing accretion), $K-L$ and $K-N$ color excesses (evidence of warm inner disks), and polarization (indicative of the relative orientations of the disks around each component). We examine to what degree these properties are correlated within binary systems and how this degree of correlation depends on parameters such as separation and binary mass ratio. These findings will be interpreted both in terms of models that treat each disk as an isolated reservoir and those in which the disks are subject to re-supply from some form of circumbinary reservoir, the observational evidence for which we will also critically review. The planet forming potential of multiple star systems is discussed in terms of the relative lifetimes of disks around single stars, binary primaries and binary secondaries. Finally, we summarize several potentially revealing observational problems and future projects that could provide further insight into disk evolution in the coming decadeComment: 16 pages, 7 figures, chapter in Protostars and Planets

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

Health State Values for the HUI 2 descriptive system: results from a UK survey

This paper reports the results of a study to estimate a statistical health state valuation model for a revised version of the Health Utilities Index Mark 2, using Standard Gamble health state preference data. A sample of 51 health states were valued by a sample of the 198 members of the UK general population. Models are estimated for predicting health state valuations for all 8,000 states defined by the revised HUI2. The recommended model produces logical and significant coefficients for all levels of all dimensions in the HUI2. These coefficients appear to be robust across model specifications. This model performs well in predicting the observed health state values within the valuation sample and for a separate validation sample of health states. However, there are concerns over large prediction errors for two health states in the valuation sample. These problems must be balanced against concerns over the validity of using the VAS based health state valuation data of the original HUI2 valuation model

Health state values for the HUI 2 descriptive system: results from a UK survey

This paper reports the results of a study to estimate a statistical health state valuation model for a revised version of the Health Utilities Index Mark 2, using Standard Gamble health state preference data. A sample of 51 health states were valued by a sample of the 198 members of the UK general population. Models are estimated for predicting health state valuations for all 8,000 states defined by the revised HUI2. The recommended model produces logical and significant coefficients for all levels of all dimensions in the HUI2. These coefficients appear to be robust across model specifications. This model performs well in predicting the observed health state values within the valuation sample and for a separate validation sample of health states. However, there are concerns over large prediction errors for two health states in the valuation sample. These problems must be balanced against concerns over the validity of using the VAS based health state valuation data of the original HUI2 valuation model.HUI2

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

Estimating population cardinal health state valuation models from individual ordinal (rank) health state preference data

Ranking exercises have routinely been used as warm-up exercises within health state valuation surveys. Very little use has been made of the information obtained in this process. Instead, research has focussed upon the analysis of health state valuation data obtained using the visual analogue scale, standard gamble and time trade off methods. Thurstone’s law of comparative judgement postulates a stable relationship between ordinal and cardinal preferences, based upon the information provided by pairwise choices. McFadden proposed that this relationship could be modelled by estimating conditional logistic regression models where alternatives had been ranked. In this paper we report the estimation of such models for the Health Utilities Index Mark 2 and the SF-6D. The results are compared to the conventional regression models estimated from standard gamble data, and to the observed mean standard gamble health state valuations. For both the HUI2 and the SF-6D, the models estimated using rank data are broadly comparable to the models estimated on standard gamble data and the predictive performance of these models is close to that of the standard gamble models. Our research indicates that rank data has the potential to provide useful insights into community health state preferences. However, important questions remain

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

Estimating population cardinal health state valuation models from individual ordinal (rank) health state preference data

Ranking exercises have routinely been used as warm-up exercises within health state valuation surveys. Very little use has been made of the information obtained in this process. Instead, research has focussed upon the analysis of health state valuation data obtained using the visual analogue scale, standard gamble and time trade off methods. Thurstone’s law of comparative judgement postulates a stable relationship between ordinal and cardinal preferences, based upon the information provided by pairwise choices. McFadden proposed that this relationship could be modelled by estimating conditional logistic regression models where alternatives had been ranked. In this paper we report the estimation of such models for the Health Utilities Index Mark 2 and the SF-6D. The results are compared to the conventional regression models estimated from standard gamble data, and to the observed mean standard gamble health state valuations. For both the HUI2 and the SF-6D, the models estimated using rank data are broadly comparable to the models estimated on standard gamble data and the predictive performance of these models is close to that of the standard gamble models. Our research indicates that rank data has the potential to provide useful insights into community health state preferences. However, important questions remain.health state valuation; HUI-2; SF-6D