Economic Modeling Considerations for Rare Diseases

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Isobel PearsonChristopher Knight

Abstract

To identify challenges that affect the feasibility and rigor of economic models in rare diseases and strategies that manufacturers have employed in health technology assessment submissions to demonstrate the value of new orphan products that have limited study data. Targeted reviews of PubMed, the National Institute for Health and Care Excellence's (NICE's) Highly Specialised Technologies (HST), and the Scottish Medicines Consortium's (SMC's) ultra-orphan submissions were performed. A total of 19 PubMed studies, 3 published NICE HSTs, and 11 ultra-orphan SMC submissions were eligible for inclusion. In rare diseases, a number of different factors may affect the model's ability to comply with good practice recommendations. Many products for the treatment of rare diseases have an incomplete efficacy and safety profile at product launch. In addition, there is often limited available natural history and epidemiology data. Information on the direct and indirect cost burden of an orphan disease also may be limited, making it difficult to estimate the potential economic benefit of treatment. These challenges can prevent accurate estimation of a new product's benefits in relation to costs. Approaches that can address such challenges inc...Continue Reading

Citations

Sep 10, 2019·Expert Review of Pharmacoeconomics & Outcomes Research·Kentaro KogushiShunya Ikeda
Jan 15, 2020·Orphanet Journal of Rare Diseases·Gonzalo EncinaGabriela M Repetto
Mar 11, 2020·Frontiers in Pharmacology·Miquéias Lopes-Pacheco
Jul 21, 2020·Journal of Health Economics and Outcomes Research·Jonas HjelmgrenGunnar Birgegård
Sep 29, 2020·International Journal of Technology Assessment in Health Care·Michela MeregagliaMichael Drummond
Jun 25, 2020·Journal of Medical Ethics·Sophie Rhys-Evans
May 29, 2021·International Journal of Technology Assessment in Health Care·Amanda WhittalKaren Facey
Jun 10, 2021·Journal of Medical Ethics·Andreas Albertsen
Jun 1, 2021·Frontiers in Pharmacology·Alessandra BlondaSteven Simoens
Aug 24, 2021·Frontiers in Medicine·Richard Huan XuShuyang Zhang
Oct 30, 2021·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·Elena NicodMichael Drummond

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