Economic analysis of cerliponase alfa for treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2)

ABSTRACT Background Cerliponase alfa is an orphan drug approved for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Aim Our goal was to assess the cost-effectiveness of cerliponase alfa in patients with CLN2 in the socioeconomic context of the Republic of Serbia in contrast to symptomatic therapy. Method For this study, a forty-year horizon and the perspective of the Serbian Republic Health Insurance Fund were used. Quality-adjusted life years gained with cerliponase alfa and comparator, as well as direct treatment costs, were the study’s key outcomes. The creation and simulation of a discrete-event simulation model served as the basis for the investigation. Monte Carlo microsimulation was performed on a sample of 1000 virtual patients. Results When compared to symptomatic therapy, cerliponase alfa treatment was not cost-effective and was linked to negative net monetary benefit regardless of when the illness signs started. Conclusion Cerliponase alfa is not more economical than symptomatic therapy for the treatment of CLN2 when using typical pharmacoeconomic analysis. Cerliponase alfa has been shown to be effective but more has to be done to make it accessible to all CLN2 patients.

[1]  M. Milosavljević,et al.  Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C , 2022, International Journal of Clinical Pharmacy.

[2]  S. Resnikoff,et al.  The economics of vision impairment and its leading causes: A systematic review , 2022, EClinicalMedicine.

[3]  Eva M. Raebel,et al.  Impact of the COVID-19 pandemic on access to the cerliponase alfa managed access agreement in England for CLN2 treatment , 2022, Orphanet Journal of Rare Diseases.

[4]  S. Janković,et al.  Rare Neurological Diseases: an Overreview of Pathophysiology, Epidemiology, Clinical Features and Pharmacoeconomic Considerations in the Treating , 2021, Serbian Journal of Experimental and Clinical Research.

[5]  S. Janković,et al.  Cost-Effectiveness of Zonisamide Versus Levetiracetam in Newly Diagnosed Focal Onset Epilepsy in Serbia. , 2021, Value in health regional issues.

[6]  Zhizhen Liu,et al.  Cost-effectiveness of speech and language therapy plus scalp acupuncture versus speech and language therapy alone for community-based patients with Broca’s aphasia after stroke: a post hoc analysis of data from a randomised controlled trial , 2021, BMJ Open.

[7]  A. Olaye,et al.  Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2) , 2021, Orphanet Journal of Rare Diseases.

[8]  M. Nebot-Villacampa,et al.  [Clinical experience with orphan drugs for rare metabolic diseases]. , 2020, Anales de pediatria.

[9]  M. Nebot-Villacampa,et al.  Experiencia clínica con medicamentos huérfanos para enfermedades raras metabólicas , 2020 .

[10]  E. De Los Reyes,et al.  Cerliponase alfa for CLN2 disease, a promising therapy , 2020 .

[11]  B. Erstad,et al.  Pricing methods in outcome-based contracting: δ2: willingness-to-pay-based pricing , 2020, Journal of medical economics.

[12]  J. Wheless,et al.  Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease: Clinical Practice Considerations From US Clinics. , 2020, Pediatric neurology.

[13]  S. Sungkarat,et al.  Multi-System Physical Exercise Intervention for Fall Prevention and Quality of Life in Pre-Frail Older Adults: A Randomized Controlled Trial , 2020, International journal of environmental research and public health.

[14]  I. Scheffer,et al.  Neuronal ceroid lipofuscinosis type 2: an Australian case series , 2020, Journal of paediatrics and child health.

[15]  Amanda M. Morrill,et al.  Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 , 2019, Journal of child neurology.

[16]  M. Neary,et al.  Epilepsy, impaired functioning, and quality of life in patients with tuberous sclerosis complex , 2019, Epilepsia open.

[17]  J. Varni,et al.  Speech difficulties and patient health communication mediating effects on worry and health‐related quality of life in children, adolescents, and young adults with Neurofibromatosis Type 1 , 2019, American journal of medical genetics. Part A.

[18]  S. Berkovic,et al.  Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis , 2019, The Lancet Neurology.

[19]  E. A. Oliveira,et al.  Behavioral disorders and impairment of quality of life in children and adolescents with lower urinary tract dysfunction. , 2018, Journal of pediatric urology.

[20]  V. Lorenzoni,et al.  Rare diseases under different levels of economic analysis: current activities, challenges and perspectives , 2018, RMD Open.

[21]  B. Kosofsky,et al.  Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study. , 2018, The Lancet. Child & adolescent health.

[22]  D. Ballon,et al.  Study of Intraventricular Cerliponase Alfa for CLN2 Disease , 2018, The New England journal of medicine.

[23]  D. Rizopoulos,et al.  Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease , 2017, Orphanet Journal of Rare Diseases.

[24]  G. Shaw.  Practice Matters: Sky-High Costs of Breakthrough Drugs Challenge Neurology , 2017 .

[25]  J. Mink,et al.  Management Strategies for CLN2 Disease. , 2017, Pediatric neurology.

[26]  P. Lefebvre,et al.  Perspectives on the Common Drug Review Process at the Canadian Agency for Drugs and Technologies in Health , 2017 .

[27]  J. Hort,et al.  Costs of dementia in the Czech Republic , 2017, The European Journal of Health Economics.

[28]  J. Cooper,et al.  Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): Expert recommendations for early detection and laboratory diagnosis. , 2016, Molecular genetics and metabolism.

[29]  T. Kielian,et al.  Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis , 2016, Orphanet Journal of Rare Diseases.

[30]  G. Iskrov,et al.  Criteria for Drug Reimbursement Decision-Making: An Emerging Public Health Challenge in Bulgaria. , 2016, Balkan medical journal.

[31]  S. Bryan,et al.  Cognitive status is a determinant of health resource utilization among individuals with a history of falls: a 12-month prospective cohort study , 2016, Osteoporosis International.

[32]  J. Brazier,et al.  Comparison of General Population, Patient, and Carer Utility Values for Dementia Health States , 2015, Medical decision making : an international journal of the Society for Medical Decision Making.

[33]  Jonathan Karnon,et al.  When to Use Discrete Event Simulation (DES) for the Economic Evaluation of Health Technologies? A Review and Critique of the Costs and Benefits of DES , 2014, PharmacoEconomics.

[34]  M. Milá,et al.  Late Infantile Neuronal Ceroid Lipofuscinosis , 2013, Journal of child neurology.

[35]  V. Sikirica,et al.  Cost Effectiveness of Pharmacotherapies for Attention-Deficit Hyperactivity Disorder , 2012, CNS Drugs.

[36]  R. Griggs,et al.  Unintended effects of orphan product designation for rare neurological diseases , 2012, Annals of neurology.

[37]  D McDaid,et al.  The economic impact of dementia in Europe in 2008—cost estimates from the Eurocode project , 2011, International journal of geriatric psychiatry.

[38]  Youwen Zhou,et al.  The US Orphan Drug Act: rare disease research stimulator or commercial opportunity? , 2010, Health policy.

[39]  P. Picon,et al.  [High cost drugs for rare diseases in Brazil: the case of lysosomal storage disorders]. , 2010, Ciencia & saude coletiva.

[40]  K. Boye,et al.  Health utility values associated with diabetic retinopathy , 2008, Diabetic medicine : a journal of the British Diabetic Association.

[41]  H. Goebel,et al.  Late infantile neuronal ceroid lipofuscinosis: quantitative description of the clinical course in patients with CLN2 mutations. , 2002, American journal of medical genetics.