Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy

Neurology
Georgina M ChambersMichelle A Farrar

Abstract

To quantify the economic and health-related quality of life (HRQoL) burden incurred by households with a child affected by spinal muscular atrophy (SMA). Hospital records, insurance claims, and detailed resource use questionnaires completed by caregivers were used to capture the direct and indirect costs to households of 40 children affected by SMA I, II, and III in Australia between 2016 and 2017. Prevalence costing methods were used and reported in 2017 US dollar (USD) purchasing power parity (PPP). The HRQoL for patients and primary caregivers was quantified with the youth version of the EQ-5D and CareQoL multiattribute utility instruments and Australian utility weights. The average total annual cost of SMA per household was $143,705 USD PPP for all SMA types (SMA I $229,346, SMA II $150,909, SMA III $94,948). Direct costs accounted for 56% of total costs. The average total indirect health care costs for all SMA types were $63,145 per annum and were highest in families affected by SMA II. Loss of income and unpaid informal care made up 24.2% and 19.8% respectively, of annual SMA costs. Three of 4 (78%) caregivers stated that they experienced financial problems because of care tasks. The loss in HRQoL of children affected by ...Continue Reading

References

Jun 8, 1991·Health Policy·M JohannessonL Råstam
Jul 30, 2003·Archives of Internal Medicine·Peter A UbelA Mark Fendrick
Aug 11, 2006·Quality of Life Research : an International Journal of Quality of Life Aspects of Treatment, Care and Rehabilitation·W B F BrouwerW K Redekop
Apr 21, 2010·Quality of Life Research : an International Journal of Quality of Life Aspects of Treatment, Care and Rehabilitation·Nora WilleUlrike Ravens-Sieberer
Sep 3, 2010·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Jonathan M GitlinR Rodney Howell
Sep 15, 2011·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Rosalie VineyJulie Ratcliffe
Jul 20, 2012·The Journal of Pediatrics·Michelle A FarrarMatthew C Kiernan
Jun 15, 2013·Health Economics·Rosalie VineyDeborah Street
Nov 26, 2015·Annals of Internal Medicine·Tianhua HeJagpreet Chhatwal
May 6, 2016·Orphanet Journal of Rare Diseases·Constanze KlugKlaus H Nagels
Jun 7, 2016·Journal of Medical Economics·Edward P ArmstrongNicholas Sicignano
Dec 28, 2016·Annals of Neurology·Michelle A FarrarMatthew C Kiernan
Dec 28, 2016·JAMA Pediatrics·Anthony L BuiChristopher J L Murray
Feb 23, 2017·Obstetrics and Gynecology·UNKNOWN Committee on Genetics
Aug 20, 2017·Orphanet Journal of Rare Diseases·Julio López-BastidaJuan Oliva-Moreno
Nov 2, 2017·The New England Journal of Medicine·Richard S FinkelUNKNOWN ENDEAR Study Group
Apr 8, 2019·Neuromuscular Disorders : NMD·C H CremersC D Schröder
Apr 10, 2019·European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society·Erik LandfeldtJanbernd Kirschner
Jul 20, 2019·Journal of Medical Economics·Marcus DroegeOmar Dabbous
Oct 15, 2019·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Didu S T KariyawasamMichelle A Farrar

❮ Previous
Next ❯

Citations

Jun 10, 2020·Neurology·Haluk Topaloglu, Nancy Kuntz
Jul 23, 2020·Muscle & Nerve·Arlene D'Silva, Michelle A Farrar
Nov 5, 2020·Disability and Rehabilitation. Assistive Technology·Sarah-Grace PaguintoMichelle Farrar
Jan 25, 2021·Orphanet Journal of Rare Diseases·Tamara DangouloffMickaël Hiligsmann
Feb 13, 2021·Applied Health Economics and Health Policy·Erik LandfeldtThomas Sejersen
Jan 19, 2021·Journal of Neonatal-perinatal Medicine·J J Volpe
Jul 30, 2021·Journal of Neurology, Neurosurgery, and Psychiatry·Sophy Tf ShihGeorgina Chambers

❮ Previous
Next ❯

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.