Orphan drugs and the NHS: Should we value rarity

Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients. Several countries have put measures in place to safeguard research and development of orphan drugs, but few get close to meeting the cost effectiveness criteria for funding by healthcare providers. We examine the justifications for special status for rare diseases and ask whether the cost effectiveness of drugs for rare or very rare diseases should be treated differently from that of other drugs and interventions

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

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.

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

The NICE Cost-Effectiveness Threshold: What it is and What that Means

The National Institute for Health and Clinical Excellence (NICE) has been using a cost-effectiveness threshold range between £20 000 and £30 000 for over 7 years. What the cost-effectiveness threshold represents, what the appropriate level is for NICE to use, and what the other factors are that NICE should consider have all been the subject of much discussion. In this article, we briefly review hese questions, provide a critical assessment of NICE’s utilization of the incremental cost-effectiveness ratio (ICER) threshold to inform its guidance, and suggest ways in which NICE’s utilization of the ICER threshold could be developed to promote the efficient use of health service resources. We conclude that it is feasible and probably desirable to operate an explicit single threshold rather than the current range; the threshold should be seen as a threshold at which ‘other’ criteria beyond the ICER itself are taken into account; interventions with a large budgetary impact may need to be subject to a lower threshold as they are likely to displace more than the marginal activities; reimbursement at the threshold transfers the full value of an innovation to the manufacturer. Positive decisions above the threshold on the grounds of innovation reduce population health; the value of the threshold should be reconsidered regularly to ensure that it captures the impact of changes in efficiency and budget over time; the use of equity weights to sustain a positive recommendation when the ICER is above the threshold requires knowledge of the equity characteristics of those patients who bear the opportunity cost. Given the barriers to obtaining this knowledge and knowledge about the characteristics of typical beneficiaries of UK NHS care, caution is warranted before accepting claims from special pleaders; uncertainty in the evidence base should not be used to justify a positive recommendation when the ICER is above the threshold. The development of a programme of disinvestment guidance would enable NICE and the NHS to be more confident that the net health benefit of the Technology Appraisal Programme is positive

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

Opportunities and challenges of new product development and testing for longevity in clothing

Many types of clothing are now seen as disposable by consumers in the UK even though durability is among the top criteria that consumers claim to use when buying garments (WRAP, 2012). Routine tests for clothing performance carried out by retailers are generally designed to ensure garments are 'fit for purpose', not to establish durability or longevity. Designing clothing that lasts longer is, however, key to reducing waste and has become a government policy objective (Defra, 2011). This paper discusses the findings from a recent research project, carried out for WRAP (Waste and Resources Action Programme), that investigated the opportunities for measuring, specifying and communicating aspects of clothing longevity within a Longevity Protocol. The Protocol is intended to enable retailers to obtain a reliable indication of garment life expectancy and was piloted in conjunction with clothing industry practitioners. It incorporates recommendations for best practice in product development and a testing regime that provides an indication of garment life expectancy (WRAP, 2014). Overall, the findings from the pilot suggest that it is possible to test for garment longevity, however, this process can be drawn-out and may not fit easily into the normal product development process. Furthermore, variations in consumer wearing patterns and laundering make it difficult for retailers to guarantee and communicate product lifetimes in absolute terms. The research adds to a growing body of evidence that supports the concept of design for clothing longevity. The findings will help to inform strategies for the implementation of government policy on sustainable clothing, but point to the need for refined testing processes to support this agenda