The Experts below are selected from a list of 20100 Experts worldwide ranked by ideXlab platform
Peter J. Neumann - One of the best experts on this subject based on the ideXlab platform.
-
Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs
Journal of General Internal Medicine, 2020Co-Authors: James D. Chambers, Madison C. Silver, Flora C. Berklein, Joshua T. Cohen, Peter J. NeumannAbstract:Background Orphan Drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan Drugs, leading to questions about their cost-effectiveness. Objective To compare the value of orphan and non-orphan Drugs approved by the FDA from 1999 through 2015. Design We searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan Drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a Drug was approved for multiple Indications, we considered each Drug-Indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan Drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions). Results We identified estimates for 49 orphan Drug-Indication pairs, and for 169 non-orphan Drug-Indication pairs. We found that orphan Drug-Indication pairs offered larger median incremental health gains than non-orphan Drug-Indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p
-
Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs.
Journal of general internal medicine, 2020Co-Authors: James D. Chambers, Madison C. Silver, Flora C. Berklein, Joshua T. Cohen, Peter J. NeumannAbstract:Background Orphan Drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan Drugs, leading to questions about their cost-effectiveness. Objective To compare the value of orphan and non-orphan Drugs approved by the FDA from 1999 through 2015. Design We searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan Drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a Drug was approved for multiple Indications, we considered each Drug-Indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan Drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions). Results We identified estimates for 49 orphan Drug-Indication pairs, and for 169 non-orphan Drug-Indication pairs. We found that orphan Drug-Indication pairs offered larger median incremental health gains than non-orphan Drug-Indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p Conclusions Our study suggests that orphan Drugs often offer larger health gains than non-orphan Drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan Drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan Drugs while achieving value from Drug spending.
Peter L Elkin - One of the best experts on this subject based on the ideXlab platform.
-
coverage of oncology Drug Indication concepts and compositional semantics by snomed ct
American Medical Informatics Association Annual Symposium, 2003Co-Authors: Steven H Brown, Brent A Bauer, Dietlind L Wahnerroedler, Peter L ElkinAbstract:Objective To evaluate SNOMED-CT ‘s ability to represent simple and compositional concepts in FDA approved oncology Drug Indications.
-
AMIA - Coverage of oncology Drug Indication concepts and compositional semantics by SNOMED-CT.
AMIA ... Annual Symposium proceedings. AMIA Symposium, 2003Co-Authors: Steven H Brown, Brent A Bauer, Dietlind L. Wahner-roedler, Peter L ElkinAbstract:Objective To evaluate SNOMED-CT ‘s ability to represent simple and compositional concepts in FDA approved oncology Drug Indications.
James D. Chambers - One of the best experts on this subject based on the ideXlab platform.
-
Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs
Journal of General Internal Medicine, 2020Co-Authors: James D. Chambers, Madison C. Silver, Flora C. Berklein, Joshua T. Cohen, Peter J. NeumannAbstract:Background Orphan Drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan Drugs, leading to questions about their cost-effectiveness. Objective To compare the value of orphan and non-orphan Drugs approved by the FDA from 1999 through 2015. Design We searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan Drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a Drug was approved for multiple Indications, we considered each Drug-Indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan Drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions). Results We identified estimates for 49 orphan Drug-Indication pairs, and for 169 non-orphan Drug-Indication pairs. We found that orphan Drug-Indication pairs offered larger median incremental health gains than non-orphan Drug-Indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p
-
Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs.
Journal of general internal medicine, 2020Co-Authors: James D. Chambers, Madison C. Silver, Flora C. Berklein, Joshua T. Cohen, Peter J. NeumannAbstract:Background Orphan Drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan Drugs, leading to questions about their cost-effectiveness. Objective To compare the value of orphan and non-orphan Drugs approved by the FDA from 1999 through 2015. Design We searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan Drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a Drug was approved for multiple Indications, we considered each Drug-Indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan Drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions). Results We identified estimates for 49 orphan Drug-Indication pairs, and for 169 non-orphan Drug-Indication pairs. We found that orphan Drug-Indication pairs offered larger median incremental health gains than non-orphan Drug-Indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p Conclusions Our study suggests that orphan Drugs often offer larger health gains than non-orphan Drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan Drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan Drugs while achieving value from Drug spending.
Tomasz Hermanowski - One of the best experts on this subject based on the ideXlab platform.
-
Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan Drugs
Orphanet Journal of Rare Diseases, 2016Co-Authors: Katarzyna Kolasa, Krzysztof Miroslaw Zwolinski, Zoltán Kaló, Tomasz HermanowskiAbstract:Background The 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. Methods A 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. Results Out 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. Conclusions An 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.
-
Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan Drugs
Orphanet journal of rare diseases, 2016Co-Authors: Katarzyna Kolasa, Krzysztof Miroslaw Zwolinski, Zoltán Kaló, Tomasz HermanowskiAbstract:The 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. A 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. Out 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. An 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.
Katarzyna Kolasa - One of the best experts on this subject based on the ideXlab platform.
-
Revealed preferences towards the appraisal of orphan Drugs in Poland - multi criteria decision analysis
Orphanet Journal of Rare Diseases, 2018Co-Authors: Katarzyna Kolasa, Krzysztof Miroslaw Zwolinski, Zoltán Kaló, Tadeusz LewandowskiAbstract:Background A Multi Criteria Decision Analysis (MCDA) technique was adopted to reveal the preferences of the Appraisal Body of the Polish HTA agency towards orphan Drugs (OMPs). Results There were 34 positive and 23 negative HTA recommendations out of 54 distinctive Drug-Indication pairs. The MCDA matrix consisted of 13 criteria, seven of which made the most impact on the HTA process. Appraisal of clinical evidence, cost of therapy, and safety considerations were the main contributors to the HTA guidance, whilst advancement of technology and manufacturing costs made the least impact. Conclusions MCDA can be regarded as a valuable tool for revealing decision makers’ preferences in the healthcare sector. Given that only roughly half of all criteria included in the MCDA matrix were deemed to make an impact on the HTA process, there is certainly some room for improvement with respect to the adaptation of a new approach towards the value assessment of OMPs in Poland.
-
Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan Drugs
Orphanet Journal of Rare Diseases, 2016Co-Authors: Katarzyna Kolasa, Krzysztof Miroslaw Zwolinski, Zoltán Kaló, Tomasz HermanowskiAbstract:Background The 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. Methods A 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. Results Out 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. Conclusions An 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.
-
Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan Drugs
Orphanet journal of rare diseases, 2016Co-Authors: Katarzyna Kolasa, Krzysztof Miroslaw Zwolinski, Zoltán Kaló, Tomasz HermanowskiAbstract:The 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. A 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. Out 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. An 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.
