Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain)
Early access to medicines allows the prescription of a medicine before it is available in the public formulary to patients with severe or rare diseases with high unmet needs who have no authorised therapeutic alternatives available. In this context, consistent decision making is difficult, and a sys...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-03-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | https://www.mdpi.com/2077-0383/11/5/1353 |
| _version_ | 1797474689221656576 |
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| author | Montse Gasol Noelia Paco Laura Guarga Josep Àngel Bosch Caridad Pontes Mercè Obach |
| author_facet | Montse Gasol Noelia Paco Laura Guarga Josep Àngel Bosch Caridad Pontes Mercè Obach |
| author_sort | Montse Gasol |
| collection | DOAJ |
| description | Early access to medicines allows the prescription of a medicine before it is available in the public formulary to patients with severe or rare diseases with high unmet needs who have no authorised therapeutic alternatives available. In this context, consistent decision making is difficult, and a systematic assessment procedure could be useful to tackle complex situations and guarantee the equity of medicines’ access. A multidisciplinary panel (MP) conducted four workshops to develop an early access framework based on a reflective multiple criteria decision analysis (MCDA). A set of 12 criteria was agreed: eight quantitative (severity of disease, urgency, efficacy, safety, internal and external validity, therapeutic benefit and plausibility) and four qualitative (therapeutic alternative, existence of precedents, management impact and costs). Quantitative criteria were weighted using a five-point scale. The relative importance of quantitative criteria had mean weights from 4.7 to 3.6, showing its relevance in the decisions. The framework was tested using two case studies, and reliability was assessed by re-test. The re-test revealed no statistical differences, indicating the consistency and replicability of the framework developed. MCDA may help to structure discussions for heterogeneous treatment requests, providing predictability and robustness in decision making involving sensitive and complex situations. |
| first_indexed | 2024-03-09T20:34:42Z |
| format | Article |
| id | doaj.art-212b3a04cd18438b87a9380f86a336f9 |
| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-09T20:34:42Z |
| publishDate | 2022-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-212b3a04cd18438b87a9380f86a336f92023-11-23T23:14:35ZengMDPI AGJournal of Clinical Medicine2077-03832022-03-01115135310.3390/jcm11051353Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain)Montse Gasol0Noelia Paco1Laura Guarga2Josep Àngel Bosch3Caridad Pontes4Mercè Obach5Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, 08193 Bellaterra, SpainCatalan Health Service (CatSalut), 08007 Barcelona, SpainCatalan Health Service (CatSalut), 08007 Barcelona, SpainDepartment of Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, SpainDepartment of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, 08193 Bellaterra, SpainCatalan Health Service (CatSalut), 08007 Barcelona, SpainEarly access to medicines allows the prescription of a medicine before it is available in the public formulary to patients with severe or rare diseases with high unmet needs who have no authorised therapeutic alternatives available. In this context, consistent decision making is difficult, and a systematic assessment procedure could be useful to tackle complex situations and guarantee the equity of medicines’ access. A multidisciplinary panel (MP) conducted four workshops to develop an early access framework based on a reflective multiple criteria decision analysis (MCDA). A set of 12 criteria was agreed: eight quantitative (severity of disease, urgency, efficacy, safety, internal and external validity, therapeutic benefit and plausibility) and four qualitative (therapeutic alternative, existence of precedents, management impact and costs). Quantitative criteria were weighted using a five-point scale. The relative importance of quantitative criteria had mean weights from 4.7 to 3.6, showing its relevance in the decisions. The framework was tested using two case studies, and reliability was assessed by re-test. The re-test revealed no statistical differences, indicating the consistency and replicability of the framework developed. MCDA may help to structure discussions for heterogeneous treatment requests, providing predictability and robustness in decision making involving sensitive and complex situations.https://www.mdpi.com/2077-0383/11/5/1353early accessmulticriteria decision analysisMCDAdrug policyassessmentpharmaceuticals |
| spellingShingle | Montse Gasol Noelia Paco Laura Guarga Josep Àngel Bosch Caridad Pontes Mercè Obach Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain) Journal of Clinical Medicine early access multicriteria decision analysis MCDA drug policy assessment pharmaceuticals |
| title | Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain) |
| title_full | Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain) |
| title_fullStr | Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain) |
| title_full_unstemmed | Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain) |
| title_short | Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain) |
| title_sort | early access to medicines use of multicriteria decision analysis mcda as a decision tool in catalonia spain |
| topic | early access multicriteria decision analysis MCDA drug policy assessment pharmaceuticals |
| url | https://www.mdpi.com/2077-0383/11/5/1353 |
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