Forecasting pharmaceutical expenditure in Europe : adjusting for the impact of rebates and discounts

European healthcare systems are under constant pressure to contain healthcare expenditure. Understanding future drug expenditure is an important consideration for payers when formulating policies. QuintileIMS publishes European forecasts that are underpinned by its audited volume data and publicly available list prices. With increasing price pressures, list to net price divergence is growing, although some of this information is commercially sensitive and thus not publicly available. The objective of this study was to further develop an established forecast to account for this divergence and explore its impact

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

Does the use of health technology assessment have an impact on the utilisation of health care resources? Evidence from two European countries

Objectives: A centralised approach to health technology assessment (HTA) may facilitate optimal use of HTA resources. A regional approach may increase the chances of local implementation of recommendations. This study aimed to compare assessment procedures in England (centralised HTA approach) with Spain (regional HTA approach) discussing key challenges and opportunities from both approaches. Methods: We compared technology assessments of anticancer medicines in the two jurisdictions from 2008 to 2015. In order to assess the implementation of HTA recommendations, we assessed trends in medicine usage using regression methods. We used IQVIA data, from 2011 to 2016, for a sample of 11 medicines. We used CatSalut data from Catalonia to assess the implementation of local recommendations. Results: In England 66 assessments were undertaken by the National Institute for Health and Care Excellence (NICE), using a standardised methodology. In Spain there were 79 reports undertaken by a range of bodies using a shared process and coordinated through the GENESIS collaboration; the assessment methods used varied substantially. Overall, the recommendations in the two jurisdictions were similar. Regression analyses indicate that where there is a positive recommendation by HTA bodies, the usage of the medicine responds most strongly (p<0.001) in Catalonia (=4.892), followed by England ( =3.120) and Spain ( =1.693). Conclusions: This study suggests that medicine utilisation does respond to the positive recommendations of HTA bodies. However, if HTA capacity is organised primarily regionally, considerable effort may be required in coordination, in order to ensure consistent and rigorous assessments and adequate implementation of HTA findings

Policy instruments (‎non-price)‎ for medical innovation

Innovation policy instruments are policy interventions with a specific mechanism of action that influences the innovation process. This Oslo Medicines Initiative technical report presents a broad range of such instruments available to national policy-makers in support of innovation for new medicines (excluding those focused on price, which are covered elsewhere in the report series). This report explores various types of policy instruments, based on reviews of the literature on policies for innovation in the medical and other sectors. For each type identified, the report explores the mechanisms of action, the effects these have and where they occur, and the extent to which these instruments have been implemented globally. It also sets out considerations for their effective implementation. The report demonstrates that the long-established push/pull (supply/ demand) framing that dominates discourse around medical innovation can be broadened, providing policy-makers with instruments to supplement push/pull approaches, by emphasizing the role of communication, collaboration and coordination in supporting the emergence of medicines to address societal needs

Appraising research policy instrument mixes: a multicriteria mapping study in six European countries of diagnostic innovation to manage antimicrobial resistance

This article provides prospective appraisal of key policy instruments intended to stimulate innovation to combat antimicrobial resistance (AMR). AMR refers to the ability of microbes to evolve resistance to those treatments designed to kill them, and is associated with the overuse or misuse of medicines such as antibiotics. AMR is an emerging global challenge with major implications for healthcare and society as a whole. Diagnostic tests for infectious diseases can guide decision making when prescribing medicines, so reducing inappropriate drug use. In the context of growing international interest in policies to stimulate innovation in AMR diagnostics, this study uses multicriteria mapping (MCM) to appraise a range of policy instruments in order to understand their potential performance while also highlighting the uncertainties that stakeholders hold about such interventions in complex contexts. A contribution of the article is the demonstration of a novel method to analyse and visualise MCM data in order to reveal stakeholder inclinations towards particular options while exploring interviewees’ uncertainties about the effectiveness of each instrument's design or implementation. The article reports results from six European countries (Germany, Greece, Italy, the Netherlands, Spain and the UK). The findings reveal which policy instruments are deemed most likely to perform well, and why, across stakeholder groups and national settings, with areas of common ground and difference being identified. Importantly, the conclusions presented here differ from prominent policy discourse, with international implications for the design of mixes of policy instruments to combat AMR. Strategic and practical methodological implications also emerge for general appraisal of innovation policy instrument mixes

Projecting Pharmaceutical Expenditure in EU5 to 2021: Adjusting for the Impact of Discounts and Rebates

Within (European) healthcare systems, the main goal for pharmaceutical expenditure is cost containment. This is due to a general belief among healthcare policy makers that pharmaceutical expenditure—driven by high prices—will be unsustainable unless further reforms are enacted.The aim of the research published in this paper is to provide more realistic expectations of pharmaceutical expenditure for all key stakeholder groups by estimating pharmaceutical expenditure at ‘net’ prices. We also aim to estimate any gaps developing between list and net pharmaceutical expenditure for the EU5 countries (i.e. France, Germany, Italy, Spain, and the UK). We adjusted an established forecast of pharmaceutical expenditure for the EU5 countries, from 2017 to 2021, by reflecting discounts and rebates not previously considered, i.e. we moved from ‘list’ to ‘net’ prices, as far as data were available.We found an increasing divergence between expenditure measured at list and net prices. When the forecasts for the five countries were aggregated, the EU5 (unweighted) average historical growth (2010–2016) rate fell from 3.4% compound annual growth rate at list to 2.5% at net. For the forecast, the net growth rate was estimated at 1.5 versus 2.9% at list.Our results suggest that future growth in pharmaceutical expenditure in Europe is likely to be (1) lower than previously understood from forecasts based on list prices and (2) below predicted healthcare expenditure growth in Europe and in line with long-term economic growth rates. For policy makers concerned about the sustainability of pharmaceutical expenditure, this study may provide some comfort, in that the perceived problem is not as large as expected

Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

International audienceAbstractRare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP’s is realised