Using N-of-1 trials to improve patient management and save costs.
BACKGROUND
N-of-1 trials test treatment effectiveness within an individual patient.


OBJECTIVE
To assess (i) the impact of three different N-of-1 trials on both clinical and economic outcomes over 12 months and (ii) whether the use of N-of-1 trials to target patients' access to high-cost drugs might be cost-effective in Australia.


METHODS
Descriptive study of management change, persistence, and costs summarizing three N-of-1 trials.


METHODS
Volunteer patients with osteoarthritis, chronic neuropathic pain or ADHD whose optimal choice of treatment was uncertain.


METHODS
Double-blind cyclical alternative medications for the three conditions.


METHODS
Detailed resource use, treatment and health outcomes (response) data collected by postal and telephone surveys immediately before and after the trial and at 3, 6 and 12 months. Estimated costs to the Australian healthcare system for the pre-trial vs. 12 months post-trial.


RESULTS
Participants persisting with the joint patient-doctor decision 12 months after trial completion were 32% for osteoarthritis, 45% for chronic neuropathic pain and 70% for the ADHD trials. Cost-offsets were obtained from reduced usage of non-optimal drugs, and reduced medical consultations. Drug costs increased for the chronic neuropathic pain and ADHD trials due to many patients being on either low-cost or no pharmaceuticals before the trial.


CONCLUSIONS
N-of-1 trials are an effective method to identify optimal treatment in patients in whom disease management is uncertain. Using this evidence-based approach, patients and doctors tend to persist with optimal treatment resulting in cost-savings. N-of-1 trials are clinically acceptable and may be an effective way of rationally prescribing some expensive long-term medicines.
Scuffham
Paul A
PA
Health Economics, School of Medicine, Griffith University, Brisbane, Queensland, Australia. p.scuffham@griffith.edu.au
Nikles
Jane
J
Mitchell
Geoffrey K
GK
Yelland
Michael J
MJ
Vine
Norma
N
Poulos
Christopher J
CJ
Pillans
Peter I
PI
Bashford
Guy
G
del Mar
Chris
C
Schluter
Philip J
PJ
Glasziou
Paul
P
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001289:Attention Deficit Disorder with Hyperactivity / Q000188:drug therapy; D001315:Australia; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease / Q000188:drug therapy / Q000191:economics; D003362:Cost-Benefit Analysis; D018592:Cross-Over Studies; D004311:Double-Blind Method; D019317:Evidence-Based Medicine / Q000191:economics*; D005260:Female; D006801:Humans; D008297:Male; D055118:Medication Adherence; D008875:Middle Aged; D009437:Neuralgia / Q000188:drug therapy; D010003:Osteoarthritis / Q000188:drug therapy; D057285:Precision Medicine / Q000191:economics*; D012107:Research Design; D016896:Treatment Outcome; D055815:Young Adult
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