Objectives To study the impact of tendering for off‐patent outpatient medicines in terms of savings for payers and availability of medicines, to explore stakeholder perceptions and to elaborate prerequisites for a successful implementation of the policy. Methods We selected three case studies (Belgium, Denmark and the Netherlands). Information was collected through literature review and stakeholder interviews. Key findings The three case study countries used tendering in different designs. While Dutch health insurers have been tendering for off‐patent medicines for more than a decade, Belgium applied this policy for only two substances in 2007/2008 and then stopped. Denmark has a kind of tendering practice: pharmaceutical companies have been submitting biweekly price bids for reimbursable outpatient medicines to the Medicines Agency since the mid‐1990s. Stakeholder perception varies between the countries: generic industry, pharmacists and partially patients tend to oppose the tendering practice; in Denmark, however, the system is highly appreciated by all stakeholders. All three countries reported savings for public payers. Experiences related to availability limitations were mixed (Belgium – the winner of the second tender had no capacity to procure; Denmark – no indication of availability limitations; and the Netherlands – frequent medicine shortages, both for tendered and nontendered medicines). Conclusions The findings suggest that tendering for off‐patent medicines is able to contribute to cost‐containment. However, as the policy possibly risks leading to availability limitations, it has to be strategically designed to avoid or at least deal with shortages through backup mechanisms. Further prerequisites for a successful introduction of tendering include a robust legal and organisational framework, an appropriate stakeholder management and demand‐side policies to promote generic uptake.

Background and objective Little is known about how much public payers spend on orphan medicines. This study aimed at identifying information on orphan medicine expenditure incurred by public payers that was published in literature globally and at possibly synthesising their shares as portion of the total pharmaceutical expenditure. Methods A literature review was undertaken using Medline, the Orphanet Journal of Rare Diseases and Google Scholar. Titles and abstracts were screened, and full texts of potentially qualifying studies were reviewed for inclusion. Included articles were analysed, and bibliometric parameters as well as public expenditure data on orphan medicines were retrieved. Results Six hundred forty three articles excluding duplicates were identified. After screening of the abstracts and a review of the full texts, 13 articles qualified for in-depth analysis. The 13 selected articles on orphan pharmaceutical expenditure were published between 2010 and 2018. Survey periods varied between 1 year and 12 years. One publication included 22 countries but the majority of the studies were related to a single country. Expenditure data was available in five of the 13 articles, and eight articles used ‘expenditure proxies’ such as sales data. Spending data had been sourced from public institutions (4 studies), private providers (5 studies) and a combination of both (3 studies, no information on data source in 1 study). In all included studies, secondary data were analysed. Reported expenditure shares for orphan medicines in relation to total pharmaceutical spend was frequently below 3%. Countries with higher shares included the USA, Canada, the Netherlands and Bulgaria—the latter reporting spending on orphan medicines as high as 9%. Conclusions A low number of studies that informed about pharmaceutical spending on orphan medicines was published, thereof only a few explicitly analysed expenditure data of public payers. A conclusive synthesis of public spending on orphan medicines is a challenge given to the diversity in methodologies to measure expenditure. There is a need for further research to survey primary data of public spending for orphan medicines, based on a sound methodology to measure these data and to compare them internationally.

of the simulation, the mean number of QALYs accumulated by patients was 3.05 in the treatment arm compared to 2.56 in the natural history arm over the five year time horizon. ConClusions: Real world data may be used to monitor the cost-effectiveness of on-market drugs and to regularly refine their prices based on the value delivered in clinical practice. Performance-based pricing could be used to negotiate earlier market access for pharmaceuticals in advance of mature data on cost-effectiveness.

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for these developments included the global economic downturn (Canada), market consolidation processes (Canada, Spain) and the strategic decision taken by national representatives of generic companies to close their sales or market access departments (Norway, Switzerland). When they announced cost-containment measures, governments were frequently confronted with opposition of pharmaceutical industry (e.g. media campaigns, legal challenges) who expressed their concern of negative consequences for the performance of the pharmaceutical sector. In two countries (Hungary, Spain) industry proposed alternative solutions to prevent the implementation of planned cost-containment measures. ConClusions: While cost-containment measures contributed to impact negatively the performance of pharmaceutical industry in some cases, their consequences appeared to be less severe than previously expected. This suggests that cost-containment is less relevant than other performance-impacting factors. PHP331Are Drug Procurement ProceDure AnD AwArD criteriA in eu HosPitAl setting stAnDArDizeD AnD Do tHey tAke into Accounts effective suPPly QuAlity?