Comparison of US and EU Prices for Orphan Drugs in the Perspective of the Considered US Orphan Drugs Act Modifications and Discussed Price-Regulation Mechanisms Adjustments in US and European Union.

The 2019 worldwide sales of Orphan Drugs were estimated at $136 billion USD, which constituted 16% of the global pharmaceutical prescription market and is expected to grow by 12% in the next 5 years. A better understanding of Orphan Drug pricing may contribute to on-going discussions on Orphan Drug Act (ODA) corrections in US or modifications of price setting mechanisms in EU. The objective of the study was comparison and analysis of the prices of Orphan Drugs in US and EU. All drugs with Orphan Drug status were compared in the US and EU. For the US prices, the US Department of Veterans Affairs (VA) was sourced. The EU List Prices came from six EU countries: Denmark, France, Germany, Greece, Poland, Spain. We found US prices to be higher than the six selected EU countries. The average Price Ratio was 1.64. The prices across EU countries were more homogeneous, while the number of the reimbursed and therefore available to patient medicines varied and was correlated with GDP per capita r = 0.87. Considered implementation of the External Reference Price system in US may generate significant savings in the US but may result in upward pressure on pricing of Orphan Drugs in EU. Centralization of the Orphan Drugs pricing negotiations in EU may prevent such development and offer a win-win opportunity for all involved parties.

Costs of diabetes and its complications in Poland.

OBJECTIVE: Diabetes mellitus (DM) is a major health problem with severe complications and a significant impact on quality of life. It constitutes an enormous burden of disease due to high prevalence, severe co-morbidities and high costs for society. This study is the first comprehensive study on the direct and indirect costs of DM (type 1 and type 2) and associated complications in Poland. METHODS: In order to estimate the direct medical costs of DM and its complications, including the costs of medical consultation, hospitalisation, rehabilitation, drugs and medical equipment, data from the National Health Fund were used. Indirect costs on loss of productivity due to diabetes and its complications were based on data obtained from the ZUS (Social Insurance Institution) and from GUS (Poland's Central Statistical Office). Attributable risk methodology was used to assess the burden of DM complications. RESULTS: A continuous increase of the direct costs of diabetes has been observed since the year 2005. In the analysed time period (2005-2009) the direct costs of medical services for both types of DM doubled. DM is a cause of significant sickness absence and incapacity for work and therefore is associated with a growing productivity decline in Poland. The highest direct costs and indirect costs are associated with treatment of diabetes-related complications. Direct costs of hospital complication treatment were EUR 332 million, which exceeded by more than five times the direct costs of hospital treatment of diabetes per se, which in the same year amounted to EUR 58.5 million. The indirect costs of diabetes-related complications were higher by 41% compared with indirect costs related to DM itself. Total costs of health care services for DM and its complications amounted to EUR 654 million, which constitutes a 2.8% of total health care costs in Poland. Total DM cost in Poland in 2009 amounted EURO 1.5 billion. CONCLUSIONS: DM is causing a growing economic burden on the health care system and on Polish society in terms of health care and productivity losses. Most of the total cost of diabetes are indirect costs caused by productivity losses. Both direct and indirect costs are driven by the cost of diabetes complications.