Multi-criteria decision analysis for the appraisal of medical needs A pilot study Irina Cleemput, Stephan Devriese, Wendy Christaens, Laurence Kohn 21/06/2016
Context 2014: Unmet Medical Needs Programme tegemoetkoming / intervention for drugs that fall under a Compassionate Use or Medical Needs programme (FAGG/AFMPS), AND target an unmet medical need (LIST), AND will become the subject of a marketing authorization request. 2
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Evidence assessment Therapeutic need Societal need Members of the CATT / CAIT Score Score Score Score Score General public Weight Weight Weight Weight Weight Weighted score Weighted score Weighted score Weighted score Weighted score Total weighted score Total weighted score Ranking Therapeutic need Ranking Societal need KBF Criteria KBF Criteria Final ranking Therapeutic need Final ranking Societal need Patient input Discussion 4
Reference: KBF, November 2015, Terugbetalingen in de gezondheidszorg: een agenda voor verandering. 5
Pre-test and Pilot test 8 health conditions Invasive meningococcal disease Major depression Heart failure Refractive errors Amyotrophic Lateral Sclerosis (ALS) Alzheimer s disease Mesothelioma Deep mixed partial thickness burns of the skin in children 6
Findings from pilot Rankings have face validity Discussion on scores led to minor changes in overall score, no change in rankings Some issues with assigning single score when disease has several phases Quality of evidence? Patient involvement has an added value Variations in interpretation of criteria 7
Conclusion Merit of MCDA: Encourages transparency Improves consistency Decision makers accountable for their judgments 8
Tools provided on website Templates for evidence tables (applicant) Template for consultation of patients or patient organisations Template for quality of evidence assessment Scoring scales for each criterion Excell tool for calculating MCDA scores based on individual raters scores for each criterion in each disease 9
Recommendations To the Belgian legislator Supply-driven needs-driven system, by list of UMN independent of pharmaceutical industry s pipeline. expanded list of stakeholders who can submit proposals for the UMN list other types of innovations allowed in the UMN programme 10
To the Belgian representatives in the European institutions Medical need = needs of patients with seriously debilitating or life-threatening conditions for which no effective alternatives exist, including non-medicinal alternatives 11
To the RIZIV / INAMI Unmet needs = societal + therapeutic needs Limitative list for allocation of UMN budget: Step 1: ranking of needs Step 2: selection of highest ranked diseases to which resources of the programme could be spent Independent assessment of quality of evidence Add information collected directly from patients or patient organisations to evidence tables. Patient umbrella organisations can help 12
To the CAIT / CATT Request use of the templates for evidence tables Use MCDA to rank therapeutic and societal needs Use suggested tools in implementation MCDA Repeat regularly criteria definitions to ensure equal interpretation by voting members 13
Colophon Author(s): Irina Cleemput (KCE), Stephan Devriese (KCE), Wendy Christiaens (KCE), Laurence Kohn (KCE) Publication date: 28 June 2016 Domain: Health Services Research (HSR) MeSH: Decision Making, Decision Support Techniques, Needs Assessment NLM Classification: WA 525 Language: English Format: Adobe PDF (A4) Legal depot: D/2012/10.273/69 Copyright: KCE reports are published under a by/nc/nd Creative Commons Licence http://kce.fgov.be/content/about-copyrights-for-kce-reports. This document is available on the website of the Belgian Health Care Knowledge Centre. 14