The availability and Expenditure of Orphan Medicines in Poland

Sujkowska, G.; Jagodzinska-Kalinowska, K.; Matusewicz, W

doi:10.1016/j.jval.2015.03.1770

Cited by 2 publications

(2 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Th e budget impact of orphan drugs represented 2.5% of total drug expenditure in Sweden and 3.1% in France in 2012 [62]. Th e share of orphan drugs in total pharmaceutical market in 2013 reached 3.6% in Czech Republic [26] and 3.2% in Poland [27]. [23].…”

Section: Volume 3 • Number 3 • October 2016 • Hophmentioning

confidence: 99%

“…Studies conducted in various countries reported that share of orphan drug expenditure had increased since introduction of orphan drug legislation [10,18,[23][24][25][26][27][28]. However, this share represented mainly low percentage (less than 8%) of overall drug expenditure [10,18,[23][24][25][26][27][28]. Picavet et al observed that principal determinants in the market uptake of orphan drugs across 23 countries in Europe were the gross domestic product and the availability of a formal technology assessment organization [29].…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia

Pejčić¹,

Искров²

2016

Hosp Pharm Int Multi J

View full text Add to dashboard Cite

Background/Objective: Orphan drugs are medicinal products which treat life-threatening or chronically debilitating diseases which aff ect small patient populations. So far there is a substantial knowledge gap on actual expenditure on orphan drugs in Serbia. Therefore, the aim of this study was to provide insight into the expenditure trends of orphan drugs in Serbia. Material and methods: Annual reports on turnover and the consumption of pharmaceuticals in Serbia published by Medicines and Medical Devices Agency of Serbia (ALIMS) for the period 2006-2013 were used to extract data on expenditure on orphan drugs. Drugs were eligible for inclusion in the analysis if they had active European orphan designation and European marketing authorization during the period covered by the study (2006)(2007)(2008)(2009)(2010)(2011)(2012)(2013). Data were analysed across time series. Simple descriptive analysis and observation of chronological trends were applied. Results: Data for annual expenditure on 12 diff erent orphan drugs were available in the analysed reports. Orphan drug expenditure and share of orphan drug expenditure in total drug expenditure increased constantly from 2006 to 2010. Both began to slightly decrease over the next two years (2011)(2012), and then suddenly declined nearly threefold in 2013. Share of orphan drug expenditure didn't exceed 1% of total drug expenditure over a period of eight years. The highest share of expenditure on orphan drugs was attributed to imatinib (87.36%-97.13%), so abrupt decline in 2013 could be explained by its withdrawal from European Community Register of Orphan Medicinal Products in 2012. Conclusions: Expenditure on orphan drugs in Serbia from 2006 to 2013 was considerably low compared to total expenditure on drugs. In the last available year we noted substantial decline of orphan drug expenditure, mainly because of imatinib losing its orphan designation. A broader time horizon would be needed to investigate long term trends.

show abstract

Section: Volume 3 • Number 3 • October 2016 • Hophmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia

Pejčić¹,

Искров²

2016

Hosp Pharm Int Multi J

View full text Add to dashboard Cite

show abstract

Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review

et al. 2021

View full text Add to dashboard Cite

Objectives: This research aims to evaluate the methodological quality of budget impact analyses for orphan drugs and to provide suggestions for future analyses.Methods: Conference abstracts and peer-reviewed literature on budget impact analyses were collected through searches of Pubmed and Embase. ISPOR good practice guidelines were used as a methodological standard for budget impact analyses. Examined parameters encompassed: perspective, target population, data sources, intervention and comparator(s), time horizon, scope of costs, discounting, validation, assumptions and sensitivity analysis.Results: Seventy studies on individual orphan drugs and 21 studies on a combination of orphan drugs analyzing budget impact were identified. Overall, analyses considered a third-party payer perspective, reported periodic budget impacts over a one-to-five-year time horizon, and did not apply discounting. A dynamically fluctuating population and costs beyond drug costs were accounted for in 18.7% and 51.7% of studies, respectively. Input data were retrieved from published literature, clinical trials, registries, claims databases, expert opinions, historical data and market research. Assumptions were mostly made about population size and intervention/comparator(s) market uptake, but these assumptions were rarely justified and their impact was insufficiently explored through sensitivity analyses. Budget impact results were rarely validated.Conclusion: Existing budget impact analyses for orphan drugs are concise, vary greatly and are of substandard methodological quality. To eliminate possible bias in future budget impact analyses, future studies should adhere to national or ISPOR good practice guidelines on budget impact analysis.

show abstract

The availability and Expenditure of Orphan Medicines in Poland

Cited by 2 publications

References 0 publications

Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia

Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia

Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review

Contact Info

Product

Resources

About