PurposeThe purpose of this study was to assess the overall burden of pancreatic cancer in Europe, with a focus on survival time in a real-world setting, and the overall healthy life lost to the disease.MethodsReal-world data were retrieved from peer-reviewed, observational studies identified by an electronic search. We performed two de novo analyses: a proportional shortfall analysis to quantify the proportion of healthy life lost to pancreatic cancer and an estimation of the aggregate life-years lost annually in Europe.ResultsNinety-one studies were included. The median, age-standardised incidence of pancreatic cancer per 100,000 was 7.6 in men and 4.9 in women. Overall median survival from diagnosis was 4.6 months; median survival was 2.8–5.7 months in patients with metastatic disease. The proportional shortfall analysis showed that pancreatic cancer results in a 98 % loss of healthy life, with a life expectancy at diagnosis of 4.6 months compared to 15.1 years for an age-matched healthy population. Annually, 610,000–915,000 quality-adjusted life-years (QALYs) are lost to pancreatic cancer in Europe. Patients had significantly lower scores on validated health-related quality of life instruments versus population norms.ConclusionsTo the best of our knowledge, this is the first study to systematically review real-world overall survival and patient outcomes of pancreatic cancer patients in Europe outside the context of clinical trials. Our findings confirm the poor prognosis and short survival reported by national studies. Pancreatic cancer is a substantial burden in Europe, with nearly a million aggregate life-years lost annually and almost complete loss of healthy life in affected individuals.Electronic supplementary materialThe online version of this article (doi:10.1007/s12029-015-9724-1) contains supplementary material, which is available to authorized users.

BackgroundOrphan medicinal product (OMP) prices are considered by some to be a challenge to the sustainability of healthcare expenditure. These concerns are compounded by the increasing number of OMPs receiving marketing authorisation (MA) annually. The aim of this study was to explore the sustainability of OMP expenditure within the context of total European pharmaceutical expenditure.MethodsUsing historical IQVIA data, an analysis was conducted on total pharmaceutical and OMP expenditure in eight countries (using values / volumes) in the branded, non-branded and overall pharmaceutical market. Country level and aggregated data was considered for EU5 countries, Austria, Belgium and Ireland.Three key analyses were conducted: The OMP share of total pharmaceutical expenditure was calculated from 2000 to 2017, to assess its evolution over time.The results of this analysis were compared with a 2011 forecast of OMP budget impact.The evolution of the total pharmaceutical market and its different segments (branded OMPs, non-OMP branded and unbranded) were assessed by estimating the compound annual growth rate (CAGR) and percentage of pharmaceutical expenditure for each market segment from 2010 to 2017.ResultsAcross countries, OMP share of total pharmaceutical expenditure has increased each year since 2000, rising to 7.2% of total pharmaceutical expenditure in 2017. OMP expenditure has increased at a CAGR of 16% since 2010. The number of OMPs receiving MA each year showed a CAGR of 11% since 2001, four percentage points greater than the CAGR for all medicines receiving MA over the same period. OMP share of total pharmaceutical expenditure is higher than forecasted in 2011 due to slower than expected growth in the non-OMP market. OMP growth has been offset by reduced expenditure in the general market and increased use of generics and biosimilars.ConclusionsRelative spending on OMPs has increased over the last 20 years, but this has been largely compensated for within the current allocation of total pharmaceutical spending by flat expenditure for non-OMPs and increased volumes of (lower-priced) generics/biosimilars, reflecting a shift towards expenditure in higher cost, lower volume patient populations and a shift in drug development towards more specialised targeting of diseases.

Aims: To compare, using a decision model, performance, treatment pathways and effects of different newborn screening strategies for developmental hip dysplasia with no screening. Methods: Detection rate, radiological absence of subluxation at skeletal maturity and avascular necrosis of the femoral head, as favourable and unfavourable treatment outcomes respectively, were compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; the addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). Results: Universal or selective ultrasound detects more more affected children (76% and 60% respectively) than clinical screening alone (35%), results in a higher proportion of affected children with favourable treatment outcomes (92% and 88% respectively) than clinical screening alone (78%) or no screening (75%), and the highest proportion of these achieved without recourse to surgery (64% and 79% respectively) compared with clinical screening alone (18%). However, ultrasound based strategies are also associated with the highest number of unfavourable treatment outcomes arising in unaffected children treated following a false positive screening result. The detection rate of clinical screening alone becomes similar to that reported for universal ultrasound when based on studies using experienced examiners (80%) rather than junior medical staff (35%). Conclusion: From the largely observational data available, ultrasound based screening strategies appear to be most sensitive and effective but are associated with the greatest risk of potential adverse iatrogenic effects arising in unaffected children.

BackgroundWithin (European) healthcare systems, the predominant 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.ObjectiveThe aim of 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).MethodsWe 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.ResultsWe 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.ConclusionsOur 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.

Aims: To assess, using a decision model, the efficiency of ultrasound based and clinical screening strategies for developmental dysplasia of the hip. Methods: The additional cost per additional favourable outcome was compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). Results: Ultrasound based screening strategies are predicted to be more effective but more costly than clinical screening or no screening. Estimated total costs per 100 000 live births are approximately £4 million for universal ultrasound, £3 million for selective ultrasound, £1 million for clinical screening alone, and £0.4 million for no screening. The relative efficiency of selective ultrasound and clinical screening is poorly differentiated, and depends on how infants are selected for ultrasound as well as the expertise of clinical screening examiners. If training costs less than £20 per child screened, clinical screening alone would be more efficient than selective ultrasound. Relative to no screening, each of the 16 additional favourable outcomes achieved as a result of selective ultrasound costs approximately £0.2 million, while each of the five favourable outcomes achieved through universal ultrasound screening, over and above selective ultrasound, costs approximately £0.3 million. Conclusions: Policy choice depends on values attached to the different outcomes, willingness to pay to achieve these and total budget.