BackgroundThe objective of this study was to assess the potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement (P&R) process with regard to orphan drugs.MethodsA four step approach was designed. Firstly, a systematic literature review was conducted to select the MCDA criteria. Secondly, a database of orphan drugs was established. Thirdly, health technology appraisals (HTA recommendations) were categorized and an MCDA appraisal was conducted. Finally, a comparison of HTA and MCDA outcomes was carried out. An MCDA outcome was considered positive if more than 50 % of the maximum number of points was reached (base case). In the sensitivity analysis, 25 % and 75 % thresholds were tested as well.ResultsOut of 2242 publications, 23 full-text articles were included. The final MCDA tool consisted of ten criteria. In total, 27 distinctive drug-indication pairs regarding 21 drugs were used for the study. Six negative and 21 positive HTA recommendations were issued. In the base case, there were 19 positive MCDA outcomes. Of the 27 cases, there were 12 disagreements between the HTA and MCDA outcomes, the majority of which related to positive HTA guidance for negative MCDA outcomes. All drug-indication pairs with negative HTA recommendations were appraised positively in the MCDA framework. Economic details were available for 12 cases, of which there were 9 positive MCDA outcomes. Amongst the 12 drug-indication pairs, two were negatively appraised in the HTA process, with positive MCDA guidance, and two were appraised in the opposite direction.ConclusionsAn MCDA approach may lead to different P&R outcomes compared to a standard HTA process. On the one hand, enrichment of the list of decision making criteria means further scrutiny of a given health technology and as such increases the odds of a negative P&R outcome. On the other hand, it may uncover additional values and as such increase the odds of positive P&R outcomes.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-016-0388-0) contains supplementary material, which is available to authorized users.

ObjectivesDiabetic foot ulcer (DFU) is a common complication of diabetes and not only an important factor of mortality among patients with diabetes but also decreases the quality of life. The short form of Diabetic Foot Ulcer Scale (DFS-SF) provides comprehensive measurement of the impact of diabetic foot ulcers on patients’ health related quality of life (HRQoL). The purpose of this study was to translate DFS-SF into Polish and evaluate its psychometric performance in patients with diabetic foot ulcers.MethodsThe DFS-SF translation process was performed in line with Principles of Good Practice for the Translation and Cultural Adaptation Process for patient reported outcome measures (PROMs) developed by ISPOR TCA group. Assessment of the reliability and validity of Polish DFS-SF was performed in native Polish patients with current DFU.ResultsThe DFS-SF validation study involved 212 patients diagnosed with DFU, with 4.4 years of DFU duration on average. The average ulcer size was 5.5 sq. cm, and generally only one limb was affected. Men (72%) and type 2 diabetes patients (86%) prevailed, with 17.8 years representing the mean time since diagnosis. The mean population age was 62.5 years. The internal consistency of all scales of the Polish DFS-SF was high (Cronbach’s alpha ranged from 0.82 to 0.93). Item convergent and discriminant validity was satisfactory (median corrected item-scale correlation ranged from 0.61 to 0.81). The Polish DFS-SF demonstrated good construct validity when correlated with the SF-36v2 and showed better psychometric performance than SF-36v2.ConclusionsThe newly translated Polish DFS-SF may be used to assess the impact of DFU on HRQoL in Polish patients.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-017-0587-y) contains supplementary material, which is available to authorized users.

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IntroductIon There are no population norms currently available in Poland for any generic health-related quality of life (HRQoL) questionnaire for adults. objEctIvEs The aim of the study was to evaluate the health status of a representative sample of the general Polish population using the EQ-5D questionnaire. MAtErIAL And MEthods Adult subjects who were visiting patients in 8 medical centers in Warsaw, Skierniewice, and Puławy, were inter viewed during the Polish EQ-5D valuation study. Stratified quota sampling was used. The respondents completed the EQ-5D questionnaire and provided information on age, sex, marital status, education, employment, income, housing conditions, medical history, and smoking habits. The inter views were conducted between February and May 2008. rEsuLts The final sample (n = 317) was representative of the general Polish population with respect to age and sex. Moderate problems in at least 1 dimension of the HRQoL were reported by 57% of the respondents, while extreme problems by 4.7%. Pain or discomfort was reported by 40% of the respondents, anxiety or depression by 38%. Problems with mobility were reported by 16% of the respondents, with usual activities (work, school) by 13%, and with self-care by 3%. The mean state of health recorded on the visual analogue scale (VAS) was 81.6 ±14.4 points. The mean VAS value decreased from 87 and 91 points in the youngest age group to 67 and 72 points in the oldest age group, in men and women, respectively. concLusIons Pain and anxiety are commonly reported problems in the Polish population, especially by young women. EQ-5D is a valuable tool for studying health outcomes and differences in health status within the Polish population.

The indirect costs of lost productivity owing to cancer-related sick leave, disability, and premature death have a substantial effect on the Polish economy. In 2009, they accounted for more than 0.8% of GDP.