Multi-criteria decision analysis for assessment and appraisal of orphan drugs
Background: Limited resources and expanding expectations push all countries and types of health systems to adopt new approaches in priority setting and resources allocation. Despite best efforts, it is difficult to reconcile all competing interests and trade-offs are inevitable. This is why multi-cr...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2016-09-01
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| Series: | Frontiers in Public Health |
| Subjects: | |
| Online Access: | http://journal.frontiersin.org/Journal/10.3389/fpubh.2016.00214/full |
| _version_ | 1819097197941096448 |
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| author | Georgi Iskrov Georgi Iskrov Tsonka Miteva-Katrandzhieva Tsonka Miteva-Katrandzhieva Rumen Stefanov Rumen Stefanov |
| author_facet | Georgi Iskrov Georgi Iskrov Tsonka Miteva-Katrandzhieva Tsonka Miteva-Katrandzhieva Rumen Stefanov Rumen Stefanov |
| author_sort | Georgi Iskrov |
| collection | DOAJ |
| description | Background: Limited resources and expanding expectations push all countries and types of health systems to adopt new approaches in priority setting and resources allocation. Despite best efforts, it is difficult to reconcile all competing interests and trade-offs are inevitable. This is why multi-criteria decision analysis (MCDA) has played a major role in recent uptake of value-based reimbursement. MCDA framework enables exploration of stakeholders’ preferences, as well as explicit organization of broad range of criteria on which real-world decisions are made.Assessment and appraisal of orphan drugs tend to be one of the most complicated health technology assessment (HTA) tasks. Access to market approved orphan therapies remains an issue. Early constructive dialogue among rare disease stakeholders and elaboration of orphan drug-tailored decision support tools could set the scene for ongoing accumulation of evidence, as well as for proper reimbursement decision-making.Objective: The objective of this study was to create a MCDA value measurement model to assess and appraise orphan drugs. This was achieved by exploring the preferences on decision criteria’s weights and performance scores through a stakeholder-representative survey and a focus group discussion that were both organized in Bulgaria.Results/Conclusions: Decision criteria that describe the health technology’s characteristics were unanimously agreed as the most important group of reimbursement considerations. This outcome, combined with the high individual weight of disease severity and disease burden criteria underlined some of the fundamental principles of healthcare – equity and fairness. Our study proved that strength of evidence may be a key criterion in orphan drug assessment and appraisal. Evidence is not only used to shape reimbursement decision-making, but also to lend legitimacy to policies pursued. The need for real-world data on orphan drugs was largely stressed.Improved knowledge on MCDA feasibility and integration to HTA is of paramount importance, as progress in medicine and innovative health technologies should correspond to patient, healthcare system and societal values. |
| first_indexed | 2024-12-22T00:11:16Z |
| format | Article |
| id | doaj.art-72c5f444fbb44d6eb51612678d0e20f0 |
| institution | Directory Open Access Journal |
| issn | 2296-2565 |
| language | English |
| last_indexed | 2024-12-22T00:11:16Z |
| publishDate | 2016-09-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Public Health |
| spelling | doaj.art-72c5f444fbb44d6eb51612678d0e20f02022-12-21T18:45:26ZengFrontiers Media S.A.Frontiers in Public Health2296-25652016-09-01410.3389/fpubh.2016.00214225569Multi-criteria decision analysis for assessment and appraisal of orphan drugsGeorgi Iskrov0Georgi Iskrov1Tsonka Miteva-Katrandzhieva2Tsonka Miteva-Katrandzhieva3Rumen Stefanov4Rumen Stefanov5Medicial University of PlovdivInstitute for Rare DiseasesMedicial University of PlovdivInstitute for Rare DiseasesMedicial University of PlovdivInstitute for Rare DiseasesBackground: Limited resources and expanding expectations push all countries and types of health systems to adopt new approaches in priority setting and resources allocation. Despite best efforts, it is difficult to reconcile all competing interests and trade-offs are inevitable. This is why multi-criteria decision analysis (MCDA) has played a major role in recent uptake of value-based reimbursement. MCDA framework enables exploration of stakeholders’ preferences, as well as explicit organization of broad range of criteria on which real-world decisions are made.Assessment and appraisal of orphan drugs tend to be one of the most complicated health technology assessment (HTA) tasks. Access to market approved orphan therapies remains an issue. Early constructive dialogue among rare disease stakeholders and elaboration of orphan drug-tailored decision support tools could set the scene for ongoing accumulation of evidence, as well as for proper reimbursement decision-making.Objective: The objective of this study was to create a MCDA value measurement model to assess and appraise orphan drugs. This was achieved by exploring the preferences on decision criteria’s weights and performance scores through a stakeholder-representative survey and a focus group discussion that were both organized in Bulgaria.Results/Conclusions: Decision criteria that describe the health technology’s characteristics were unanimously agreed as the most important group of reimbursement considerations. This outcome, combined with the high individual weight of disease severity and disease burden criteria underlined some of the fundamental principles of healthcare – equity and fairness. Our study proved that strength of evidence may be a key criterion in orphan drug assessment and appraisal. Evidence is not only used to shape reimbursement decision-making, but also to lend legitimacy to policies pursued. The need for real-world data on orphan drugs was largely stressed.Improved knowledge on MCDA feasibility and integration to HTA is of paramount importance, as progress in medicine and innovative health technologies should correspond to patient, healthcare system and societal values.http://journal.frontiersin.org/Journal/10.3389/fpubh.2016.00214/fullRare Diseasesdecision-makingreimbursementHealth Technology Assessmentorphan drugsMulti-criteria Decision Analysis |
| spellingShingle | Georgi Iskrov Georgi Iskrov Tsonka Miteva-Katrandzhieva Tsonka Miteva-Katrandzhieva Rumen Stefanov Rumen Stefanov Multi-criteria decision analysis for assessment and appraisal of orphan drugs Frontiers in Public Health Rare Diseases decision-making reimbursement Health Technology Assessment orphan drugs Multi-criteria Decision Analysis |
| title | Multi-criteria decision analysis for assessment and appraisal of orphan drugs |
| title_full | Multi-criteria decision analysis for assessment and appraisal of orphan drugs |
| title_fullStr | Multi-criteria decision analysis for assessment and appraisal of orphan drugs |
| title_full_unstemmed | Multi-criteria decision analysis for assessment and appraisal of orphan drugs |
| title_short | Multi-criteria decision analysis for assessment and appraisal of orphan drugs |
| title_sort | multi criteria decision analysis for assessment and appraisal of orphan drugs |
| topic | Rare Diseases decision-making reimbursement Health Technology Assessment orphan drugs Multi-criteria Decision Analysis |
| url | http://journal.frontiersin.org/Journal/10.3389/fpubh.2016.00214/full |
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