EVALUATING THE HOLISTIC VALUE OF INNOVATIVE THERAPIES VIA THE TREATMENT IMPACT MODEL: A CASE STUDY ON EMICIZUMAB FOR HAEMOPHILIA A IN SINGAPORE
Author(s)
Chloe Lee, MSc1, Melina Arnold, MSc, PhD2, Mariana Al-Adwan, MSc, PharmD3, Eng Soo Yap, MBBS (NUS), M.Med (Int Med, S'pore), MRCP (UK), FR4, Pei Lin Koh, MBBS (S’pore), FRCPCH (UK), MMed (Paeds) (S'pore)5, Joyce Ching Mei Lam, MBBS, MMed (Paeds), MRCPCH (UK), FRCPA, FAMS6, Sim Leng Tien, MBBS, MMed (Int Med), FRCPA, FAMS7.
1Roche Singapore Pte. Ltd., Singapore, Singapore, 2Hoffmann-La Roche, Basel, Switzerland, 3F. Hoffmann-La Roche AG, Amman, Jordan, 4Department of Laboratory Medicine, National University Hospital, Singapore, Singapore, 5Department of Paediatrics, Khoo Teck Puat - National University Children's Medical Institute, National University Hospital, Singapore, Singapore, 6Paediatric Haematology / Oncology Service, KK Women's and Children's Hospital, Singapore, Singapore, 7Department of Haematology, Singapore General Hospital, Singapore, Singapore.
1Roche Singapore Pte. Ltd., Singapore, Singapore, 2Hoffmann-La Roche, Basel, Switzerland, 3F. Hoffmann-La Roche AG, Amman, Jordan, 4Department of Laboratory Medicine, National University Hospital, Singapore, Singapore, 5Department of Paediatrics, Khoo Teck Puat - National University Children's Medical Institute, National University Hospital, Singapore, Singapore, 6Paediatric Haematology / Oncology Service, KK Women's and Children's Hospital, Singapore, Singapore, 7Department of Haematology, Singapore General Hospital, Singapore, Singapore.
OBJECTIVES: While cost-effectiveness assessments provide per-patient metrics, the Treatment Impact Model (TIM) offers a broader population-level perspective. The TIM complements these frameworks by quantifying the multi-dimensional value medical innovations bring to patients, healthcare systems, and economies.
To evaluate the population-level impact of introducing emicizumab as a prophylactic treatment for people with haemophilia A (PwHA) in Singapore, focusing on averted clinical events, increased quality-adjusted life years (QALYs), and reduced direct medical and indirect productivity costs over a 25-year period.
METHODS: A dynamic population-based TIM (starting N=137), projected outcomes over a 25-year time horizon by comparing an "absence of emicizumab" scenario (treatment with only on-demand or prophylactic factor VIII replacement and bypassing agents) against an "introduction of emicizumab" scenario (simulating gradual market uptake). The impact of introducing emicizumab as a treatment was established by comparing the number of clinical events (e.g., bleeds, arthroplasties), QALYs, direct medical costs associated with bleeds, indirect costs (e.g. productivity losses) between the two scenarios. The model incorporated Singapore-specific epidemiological and economic data. Clinical assumptions on annualised bleeding rates, hospitalisations and joint damage progression (simulated using Pettersson Score-based algorithms) were based on published literature. This projection assumes a constant treatment environment, excluding potential impacts from newer emerging therapies.
RESULTS: Over a projected decade (2027-2036) in Singapore, with emicizumab uptake peaking at 94% and 65% of the eligible paediatric and adult PwHA, the model estimated a 25.3% reduction in total bleeds. This would result in over 3,900 bleeds averted and 690 hospitalisation days avoided, leading to total savings exceeding S$88 million in direct and indirect costs.
CONCLUSIONS: The study shows that the TIM is adaptable to various disease areas for quantifying socioeconomic value beyond clinical efficacy. We recommend the adoption of the model by payers and policymakers to fully evaluate the comprehensive societal benefits of innovative treatments.
To evaluate the population-level impact of introducing emicizumab as a prophylactic treatment for people with haemophilia A (PwHA) in Singapore, focusing on averted clinical events, increased quality-adjusted life years (QALYs), and reduced direct medical and indirect productivity costs over a 25-year period.
METHODS: A dynamic population-based TIM (starting N=137), projected outcomes over a 25-year time horizon by comparing an "absence of emicizumab" scenario (treatment with only on-demand or prophylactic factor VIII replacement and bypassing agents) against an "introduction of emicizumab" scenario (simulating gradual market uptake). The impact of introducing emicizumab as a treatment was established by comparing the number of clinical events (e.g., bleeds, arthroplasties), QALYs, direct medical costs associated with bleeds, indirect costs (e.g. productivity losses) between the two scenarios. The model incorporated Singapore-specific epidemiological and economic data. Clinical assumptions on annualised bleeding rates, hospitalisations and joint damage progression (simulated using Pettersson Score-based algorithms) were based on published literature. This projection assumes a constant treatment environment, excluding potential impacts from newer emerging therapies.
RESULTS: Over a projected decade (2027-2036) in Singapore, with emicizumab uptake peaking at 94% and 65% of the eligible paediatric and adult PwHA, the model estimated a 25.3% reduction in total bleeds. This would result in over 3,900 bleeds averted and 690 hospitalisation days avoided, leading to total savings exceeding S$88 million in direct and indirect costs.
CONCLUSIONS: The study shows that the TIM is adaptable to various disease areas for quantifying socioeconomic value beyond clinical efficacy. We recommend the adoption of the model by payers and policymakers to fully evaluate the comprehensive societal benefits of innovative treatments.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
EE107
Topic
Economic Evaluation
Topic Subcategory
Novel & Social Elements of Value
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Pediatrics, SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)