Modeling the multi‐state natural history of rare diseases with heterogeneous individual patient data: A simulation study

• Published in: Statistics in medicine Vol. 43; no. 1; pp. 184 - 200
• Main Authors: Broomfield, Jonathan, Abrams, Keith R., Freeman, Suzanne, Latimer, Nicholas, Rutherford, Mark J., Crowther, Michael J.
• Format: Journal Article
• Language: English
• Published: Hoboken, USA John Wiley & Sons, Inc 15.01.2024; Wiley Subscription Services, Inc
• Subjects: multi‐state model; Natural history; Rare diseases; simulation; survival analysis; rare diseases; multi-state model; survival analysis; natural history; simulation

Multi‐state survival models are used to represent the natural history of a disease, forming the basis of a health technology assessment comparing a novel treatment to current practice. Constructing such models for rare diseases is problematic, since evidence sources are typically much sparser and mo...