Personalised medicine, also known as ‘precision’ or ‘stratified’ medicine, targets treatment by using genetics and/or biomarkers to identify patients or subgroups of patients who have distinct mechanisms of disease or who may respond to treatment in a particular way. The goal is to provide more effective care, which potentially not only benefits the patient but also improves the efficiency of the healthcare system.
Although the science underlying drug development has evolved, there has been little change in how we pay for them. As more and more medicines come to market with multiple indications (or even more importantly the unrealised potentialto treat multiple indications), the way we pay for those medicines becomes critical in making sure we can benefit from them. “Indication-based pricing” (IBP) permits price to vary according to indication and has been proposed to tackle this issue.
The World Health Organisation (WHO) has become quite skilled at promoting what changes are desirable in health care systems, and why, but has largely left how to implement those changes to individual countries. Although certainly no one solution will fit all countries, some evidence-informed guidelines and insights about how to implement change successfully may help ensure progress in large-scale health system transformation.
The ‘opioid epidemic’ in the US is the most recent drug-abuse challenge from the misuse of prescription medicines or the use of illicit drugs. It is a particularly difficult challenge because thousands of patients benefit from the prescription pain killers that originally fuelled the crisis. Ceasing production and use of such medications, then, is not the right answer. But the crisis has recently entered a more lethal phase, one that involves the use of illicit synthetic opioids which are both more addictive and more deadly.
Hernandez-Villafuerte, K., Shah, K., Herdman, M., and Lorgelly, P.
Meningococcal disease is a life-threatening infection and can result in severe sequelae. Recent scientific and technical advances have led to the discovery and implementation of novel meningococcal vaccines which have resulted in a substantial reduction in the burden of disease worldwide, representing a major public health achievement (Crum-Cianflone and Sullivan, 2016).
Cubi-Molla, P., Errea, M., Zhang, K. and Garau, M.
Cost-Effectiveness Thresholds (CETs) are used in a selected number of countries as tool in decision-making on funding and reimbursements for new healthcare technologies. In this white paper, OHE presents an analysis of the relative merits and shortfalls of current approaches to defining, estimating and applying CETs in Health Technology Assessments. The paper also puts forward a number of policy recommendations to help guide decision makers in ensuring CETs are used to achieve improved health outcomes in the future.
Gonzalez-Esuerra, J., Karlsberg, S. and Paling, S.
The NHS Improvement Economics team is part of the Strategy Directorate in NHS Improvement. NHS Improvement aims to implement changes to help improve both quality and efficiency. Recent research by the Economics team intended to support this has included research on A&E performance, NHS staffing, and inpatient falls.
Health System Strengthening (HSS) is an important concept now widely discussed, but too often without sufficient structure or an adequate understanding of what actually is involved. The articles on which this seminar is based (Morton, Thomas and Smith, 2016; Smith and Yip, 2016) attempted to present more clearly just what health system strengthening might entail and whether that might be modelled.
Henderson N., Errea M., Skedgel C. and Jofre-Bonet, M.
In light of concerns that not all medicines for ultra-rare (also known as ultra-orphan) conditions are appraised under the same NICE process, a new OHE Consulting Report discusses the distinct ethical and economic challenges faced by medicines for ultra-rare conditions, with particular reference to the challenges of HTA in the UK. A failure to consistently consider all ultra-rare disease medicines under the HST process could lead to inequalities in access and health outcomes for patients with ultra-rare conditions.
Shah, K.K., Ramos-Goñi, J.M., Kreimeier, S. and Devlin, N.J.
To date there have been no value sets to support the use of the EQ-5D-Y in cost-utility analysis. Discrete choice experiments (DCEs) can be used to obtain values on a latent scale, but these values require anchoring at 0 = dead to meet the conventions of quality-adjusted life year (QALY) estimation. This Research Paper describes a study in which four stated preference methods for anchoring EQ-5D-Y values were compared: visual analogue scale, DCE (with a duration attribute), lag-time TTO and the recently developed ‘location-of-dead’ (LOD) element of the personal utility function approach.