APDS Treatment in Germany: Decision Criteria, Costs, and Contribution of Leniolisib to Outcomes
Author(s)
Ulrich Baumann, MD1, Marie-Celine Deau, PhD2, Karsten Franke, MD3, Leif G. Hanitsch, MD4, Georgios Sogkas, MD PhD5, Kirsten H Herrmann, PhD6, Ulrich Bosch dos Santos, PhD6, Christof Minartz, PhD7, Aljoscha S Neubauer, MD MBA7, Fabian Hauck, MD PhD8.
1Immunology Unit, Paediatric Pulmonology, Allergy and Neonatology, Hannover Medical School (MHH), Hannover, Germany, 2Institute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Medical Center – University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany, 3Institute of Clinical Immunology, Siegen, Germany, 4Charité – Universitätsmedizin Berlin, Berlin, Germany, 5Department of Rheumatology and Immunology, Hannover Medical School (MHH), Hannover, Germany, 6Pharming, Leiden, Netherlands, 7Institute for Health- and Pharmacoeconomics (IfGPh), Muenchen, Germany, 8Division of Pediatric Immunology and Rheumatology, Department of Pediatrics, Dr. von Hauner Children’s Hospital, University Hospital, LMU Munich, Muenchen, Germany.
1Immunology Unit, Paediatric Pulmonology, Allergy and Neonatology, Hannover Medical School (MHH), Hannover, Germany, 2Institute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Medical Center – University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany, 3Institute of Clinical Immunology, Siegen, Germany, 4Charité – Universitätsmedizin Berlin, Berlin, Germany, 5Department of Rheumatology and Immunology, Hannover Medical School (MHH), Hannover, Germany, 6Pharming, Leiden, Netherlands, 7Institute for Health- and Pharmacoeconomics (IfGPh), Muenchen, Germany, 8Division of Pediatric Immunology and Rheumatology, Department of Pediatrics, Dr. von Hauner Children’s Hospital, University Hospital, LMU Munich, Muenchen, Germany.
OBJECTIVES: Activated PI3 Kinase Delta Syndrome (APDS) is a rare, inborn error of immunity. To better classify the contribution of a potential new therapeutic precision agent, leniolisib, medically relevant decision criteria in Germany were assessed upon which a model was built.
METHODS: Two online surveys and two roundtable discussions with 6 German medical experts were conducted to obtain and discuss clinical and cost components, based on an existing full UK cost-effectiveness model of leniolisib. Based on the discussion results, key decision outcomes were modelled in a Markov-Model (MS-Excel).
RESULTS: Key decision therapeutic criteria of physicians in Germany were found reliably in two rounds of MaxDiff rating, based on importance: efficacy > safety > further attributes like disease severity and quality of life impact. Relevant clinical outcomes in APDS, which were found well quantifiable were infection numbers, lymphoproliferation, bronchiectasis, lymphoma and mortality. For these outcomes, relevant improvements versus current standard of care were expected by the new therapeutic precision agent leniolisib. Expert assessments in the online surveys, which were carried out by the 6 participants independently, showed low variance across participants. Results supported an expected mortality reduction by use of leniolisib of ~15% by an age of 40 years. This age is currently reached only by 65% of APDS patients. Regarding costs, immunoglobulin replacement, treatment of lymphoma and other malignancies, bronchiectasis and advanced lung disease, as well as allogeneic hematopoietic stem cell transplantation (alloHSCT) were important considerations.
CONCLUSIONS: Decision criteria of German physicians for treating APDS are well in line with other therapeutic areas, with efficacy considered to be the most relevant issue. Based on data on leniolisib available today, mortality and several patient-relevant morbidity endpoints are expected to improve in future clinical practice in Germany. Leniolisib may also significantly modify the course of disease in APDS patients.
METHODS: Two online surveys and two roundtable discussions with 6 German medical experts were conducted to obtain and discuss clinical and cost components, based on an existing full UK cost-effectiveness model of leniolisib. Based on the discussion results, key decision outcomes were modelled in a Markov-Model (MS-Excel).
RESULTS: Key decision therapeutic criteria of physicians in Germany were found reliably in two rounds of MaxDiff rating, based on importance: efficacy > safety > further attributes like disease severity and quality of life impact. Relevant clinical outcomes in APDS, which were found well quantifiable were infection numbers, lymphoproliferation, bronchiectasis, lymphoma and mortality. For these outcomes, relevant improvements versus current standard of care were expected by the new therapeutic precision agent leniolisib. Expert assessments in the online surveys, which were carried out by the 6 participants independently, showed low variance across participants. Results supported an expected mortality reduction by use of leniolisib of ~15% by an age of 40 years. This age is currently reached only by 65% of APDS patients. Regarding costs, immunoglobulin replacement, treatment of lymphoma and other malignancies, bronchiectasis and advanced lung disease, as well as allogeneic hematopoietic stem cell transplantation (alloHSCT) were important considerations.
CONCLUSIONS: Decision criteria of German physicians for treating APDS are well in line with other therapeutic areas, with efficacy considered to be the most relevant issue. Based on data on leniolisib available today, mortality and several patient-relevant morbidity endpoints are expected to improve in future clinical practice in Germany. Leniolisib may also significantly modify the course of disease in APDS patients.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO12
Topic
Clinical Outcomes, Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Clinician Reported Outcomes, Comparative Effectiveness or Efficacy
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)