Realising the broader value of vaccines in the UK.

Many health technology assessment (HTA) agencies limit their assessments of vaccines to the health benefits for the vaccinated individual, the costs associated with vaccine administration and the disease avoided. However, because the value of vaccines tends to accrue to a large extent beyond the vaccinated individual, they are systematically undervalued in many current HTA processes. This is also the case in the UK, where the Joint Committee on Vaccination and Immunisation (JCVI) is in charge of assessing preventative vaccines, while therapeutic vaccines fall in the realm of the National Institute for Clinical Excellence (NICE). To contribute to a forward-looking perspective, we designed a framework to capture the broader value of vaccination. We reviewed the current state of the global vaccines pipeline and selected seven preventative and three therapeutic vaccines that are likely to enter the UK market within five years. We assessed on which value elements the selected vaccines would potentially generate value, and compared those against the novel broader value framework. A review of the current value elements considered by the JCVI and NICE allowed identifying the critical gaps between potential value generation and value recognition. To our knowledge, this is the first time that the broader value of vaccination has been pro-actively assessed for pipeline vaccinations. Our findings show that the existing narrow evaluation frameworks are likely to systematically undervalue the value of potential future vaccines coming to the UK market. This is particularly relevant, where their impact on AMR and other health interventions, and on the productivity of the workforce is of concern. Recommendations to overcome this include an explicit and more consistent inclusion of, and data collection on, the impact of vaccines on AMR and other health interventions by JCVI and NICE; the consideration of a societal perspective and the fiscal impact of vaccines to societies.

Does NICE influence the adoption and uptake of generics in the UK?

The aim of this paper is to examine generic competition in the UK, with a special focus on the role of Health Technology Assessment (HTA) on generic market entry and diffusion. In the UK, where no direct price regulation on pharmaceuticals exists, HTA has a leading role for recommending the use of medicines providing a non-regulatory aspect that may influence the dynamics in the generic market. The paper focuses on the role of Technology Appraisals issued by the National Institute for Health and Care Excellence (NICE). We follow a two-step approach. First, we examine the probability of generic entry. Second, conditional on generic entry, we examine the determinants of generic market share. We use data from IQVIA British Pharmaceutical Index (BPI) for the primary care market for 60 products that lost patent between 2003 and 2012. Our results suggest that market size remains one of the main drivers of generic entry. After controlling for market size, intermolecular substitution and difficulty of manufacturing increase the likelihood of generic entry. After generic entry, our estimates suggest that generic market share is highly state dependent. Our findings also suggest that while NICE recommendations do influence generic uptake, there is only marginal evidence they affect generic entry.

Comparing access to orphan medicinal products in Europe.

OBJECTIVES: The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service. METHODS: Data were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared. RESULTS: We found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation - but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average. CONCLUSIONS: Marketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain.

The Influence of Cost-Effectiveness and Other Factors on Nice Decisions.

The National Institute for Health and Care Excellence (NICE) emphasises that cost-effectiveness is not the only consideration in health technology appraisal and is increasingly explicit about other factors considered relevant but not the weight attached to each. The objective of this study is to investigate the influence of cost-effectiveness and other factors on NICE decisions and whether NICE's decision-making has changed over time. We model NICE's decisions as binary choices for or against a health care technology in a specific patient group. Independent variables comprised of the following: clinical and economic evidence; characteristics of patients, disease or treatment; and contextual factors potentially affecting decision-making. Data on all NICE decisions published by December 2011 were obtained from HTAinSite [www.htainsite.com]. Cost-effectiveness alone correctly predicted 82% of decisions; few other variables were significant and alternative model specifications had similar performance. There was no evidence that the threshold has changed significantly over time. The model with highest prediction accuracy suggested that technologies costing £40 000 per quality-adjusted life-year (QALY) have a 50% chance of NICE rejection (75% at £52 000/QALY; 25% at £27 000/QALY). Past NICE decisions appear to have been based on a higher threshold than £20 000-£30 000/QALY. However, this may reflect consideration of other factors that cannot be easily quantified. © 2014 The Authors. Health Economics published by John Wiley & Sons Ltd.

Projecting expenditure on medicines in the UK NHS.

BACKGROUND: Expenditure on medicines is a readily identifiable element of health service costs. It is the focus of much attention by payers, not least in the UK even though the cost of medicines represents less than 10 % of total UK National Health Service (NHS) expenditure. Projecting future medicines spending enables the likely cost pressure to be allowed for in planning the scale and allocation of NHS resources. Simple extrapolations of past trends in expenditure fail to account for changes in the rate and mix of new medicines becoming available and in the scope for windfall savings when some medicines lose their patent protection. The objective of this study is to develop and test an improved method to project NHS pharmaceutical expenditure in the UK for the period 2012-2015. METHODS: We have adopted a product-by-product, bottom-up approach, which means that our projections are built up from individual products to the total market. Our projections of the impact of generic and biosimilars entry on prices and quantities of medicines sold, and of the rate of uptake of newly launched medicines, have been obtained from regression analysis of UK data. To address uncertainty, we have created a baseline and two other illustrative scenarios. We have compared our projections with actual expenditure for 2012. RESULTS: Our projections estimate that, between 2011 and 2015, with no change in policy or price regulation, the UK total medicines bill would increase at an average compound annual growth rate (CAGR) of between 3.1 and 4.1 %. Total NHS spending on branded medicines and total NHS spending on generics are projected to increase at average CAGRs of 0.5-1.8 and 10.0-11.0 %, respectively, over the same time period. For the total market, the actual growth rate for 2012 lay within our projected range. CONCLUSIONS: Our methodology provides a useful framework for projecting UK NHS medicines expenditure over the medium term and captures the impacts of existing medicines losing exclusivity and of new medicines being launched onto the market.

An analysis of NICE's 'restricted' (or 'optimized') decisions.

BACKGROUND: A common way of describing UK National Institute for Health and Clinical Excellence (NICE) decisions is to distinguish between cases where NICE recommended use of a healthcare technology by all relevant patients ('yes'); those where it did not recommend use ('no'); and those where its decisions are a mixture of 'yes' to some patient subgroups, and 'no' to others. Over half of NICE's decisions are of this mixed type, which involve restricting (or 'optimizing') patient use in some way. OBJECTIVE: To report an attempt to develop a robust and defensible means of measuring and describing the degree of patient access in mixed NICE decisions. METHODS: A list of mixed decisions made from 2006 to the end of 2009 was identified using HTAinSite™. The following calculation was used: M = (p/P) × 100, where M is a measure of the level of patient access (0 = no access, 100 = full access), P is the set of patients considered in the guidance as Potential candidates for treatment (given the licensed use and the scope of NICE's appraisal), and p is a subset of those patients, for whom NICE did recommend treatment. M can be estimated either for a specific product or for a group of technologies (Multiple Technology Appraisals). Both product-specific and overall M were estimated, using estimates of p obtained from NICE costing templates. These data are subject to some important limitations, so the results should be regarded as illustrative. RESULTS: Of the 69 medicines that have received a mixed decision since January 2006, 34 included details that allowed the estimation of M. Of these 34 decisions, 24 (71%) had a product-specific M ≤50, 16 (47%) M ≤25 and 11 (32%) M ≤10. That is, in just under three-quarters of the mixed decisions for which P and p were available, NICE recommended use for less than half of patients for whom the medicine is licensed, and in nearly one-third of these sorts of decisions, NICE recommended use in ≤10% of potential patients. The estimates of M for groups of technologies provide a slightly different picture: for example, grouped M was ≤10 in <20% of decisions. CONCLUSIONS: The measure of patient access, M, proposed here has the potential to provide a more informative way of reporting all NICE decisions, particularly 'restricted' (or 'optimized') decisions.