Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)
María Reyes Abad; Carmen Alerany; Luis Ignacio Gonzalez; Olaf Neth; Concepción Payares-Herrera; Carlos Rodríguez-Gallego; Jose Luis Trillo; Kirsten H. Herrmann; Raquel Figueiredo; Alicia Gil
- Published
- 2025-01-02
- Language
- en
Abstract
Background: Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS) is an ultra-rare, potentially life-threatening disease that lacks approved treatments in Spain. This study aimed to apply Multi-Criteria Decision Analysis (MCDA) to assess the value of the first pharmacological treatment for APDS in Spain. Methods: A multidisciplinary group of 8 experts evaluated the selective PI3Kδ inhibitor leniolisib against Standard of Care (SoC). An MCDA framework tailored for Orphan Drugs (ODs), consisting of 5 comparative and 2 quantitative non-comparative criteria, was used. Re-scoring followed a group discussion. Results: Leniolisib scored higher than SoC in all criteria, including efficacy and safety. It was deemed highly valuable as the first disease-modifying treatment, with a positive therapeutic impact and potential to improve patients’ quality of life. Additionally, leniolisib may lead to cost savings. The supporting data was considered of high quality. Conclusion: Based on MCDA methodology and stakeholder experience in APDS management, leniolisib is seen as a value-added treatment option compared to SoC in Spain.
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Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al Legal Disclaimer The information contained in this document is CONFIDENTIAL. MCDA EVIDENCE MATRIX
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 2 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al INTRODUCTION
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 3 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al INTERVENTION: DISEASE: SCOPE: COMPARATOR: INTERVENTION DESCRIPTION COMPARATOR DESCRIPTION
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 4 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al TREATMENT-RELATED CRITERIA CRITERIA RELEVANT SUMMARISED DATA SCORE EFFICACY/EFFECTIVENESS Sub-criteria: Magnitude of clinical benefit or health outcomes compared to the comparator Duration of health benefit ☐ 5 Much safer/more tolerable than comparator ☐ 4 ☐ 3 ☐ 2 ☐ 1 ☐ 0 No difference ☐ -1 ☐ -2 ☐ -3 ☐ -4 ☐ -5 Much less safe/tolerable than the comparator REFLECTIONS BEHIND YOUR SCORE (MANDATORY FIELD):
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 5 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al TREATMENT-RELATED CRITERIA CRITERIA RELEVANT SUMMARISED DATA SCORE SAFETY/TOLERABILITY Sub-criteria: Percentage of adverse events Type (severity) of grade 3 and 4 adverse events Frequency grade 3 and 4 adverse events Percentage of patients who stop treatment due to adverse events ☐ 5 Much safer/more tolerable than comparator ☐ 4 ☐ 3 ☐ 2 ☐ 1 ☐ 0 No difference ☐ -1 ☐ -2 ☐ -3 ☐ -4 ☐ -5 Much less safe/tolerable than the comparator REFLECTIONS BEHIND YOUR SCORE (MANDATORY FIELD):
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 6 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al TREATMENT-RELATED CRITERIA CRITERIA RELEVANT SUMMARISED DATA SCORE PATIENT REPORTED OUTCOMES (PROs) Sub-criteria: Improvement in the health-related quality of life of patients Patient preference and satisfaction with available therapeutic alternatives ☐ 5 The intervention highly improve PROs ☐ 4 ☐ 3 ☐ 2 ☐ 1 ☐ 0 No difference ☐ -1 ☐ -2 ☐ -3 ☐ -4 ☐ -5 The intervention worsen PROs REFLECTIONS BEHIND YOUR SCORE (MANDATORY FIELD):
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 7 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al TREATMENT-RELATED CRITERIA CRITERIA RELEVANT SUMMARISED DATA SCORE TYPE OF THERAPEUTIC BENEFIT Sub-criteria: Symptom reduction Modification of clinical course (interruption or slowing of progression) Cure or impairment of mortality ☐ 5 Curative treatment/lifesaving benefit ☐ 4 ☐ 3 ☐ 2 ☐ 1 ☐ 0 No therapeutic benefit REFLECTIONS BEHIND YOUR SCORE (MANDATORY FIELD):
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 8 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al TREATMENT-RELATED CRITERIA CRITERIA RELEVANT SUMMARISED DATA SCORE OTHER DIRECT MEDICAL COSTS Sub-criteria: Impact on hospitalizations Impact on hospital specialists’ follow-up Impact on tests and interventions Impact on symptom treatment Impact on adverse events treatment Impact on long-term care expenses ☐ 5 Substantial savings ☐ 4 ☐ 3 ☐ 2 ☐ 1 ☐ 0 No difference in expenditure ☐ -1 ☐ -2 ☐ -3 ☐ -4 ☐ -5 Substantial additional expenses REFLECTIONS BEHIND YOUR SCORE (MANDATORY FIELD):
MCDA EVIDENCE MATRIX CONFIDENTIAL Page 9 Glob Reg Health Technol Assess 2025 | DOI: 10.33393/grhta.2025.3199 | Abad et al TREATMENT-RELATED CRITERIA CRITERIA RELEVANT SUMMARISED DATA SCORE NON-MEDICAL (INDIRECT) COSTS Sub-criteria: Impact on patients and caregivers’ productivity Financial impact on patients and caregivers Costs to the social care system ☐ 5 Substantial savings ☐ 4 ☐ 3 ☐ 2 ☐ 1 ☐ 0 No difference in expenditure ☐ -1 ☐ -2 ☐ -3 ☐ -4 ☐ -5 Substantial additional expenses REFLECTIONS BEHIND YOUR SCORE (MANDATORY FIELD):
