Modeling long-term health and economic implications of new treatment strategies for Parkinson’s disease: an individual patient simulation study

References

• Rogers G, Davies D, Pink J, et al. Parkinson’s disease: summary of updated NICE guidance. BMJ. 2017;358:j1951.
   PubMed Web of Science ®Google Scholar
• Dawson VL, Dawson TM. Promising disease-modifying therapies for Parkinson’s disease. Sci Transl Med. 2019;11:520.
   Web of Science ®Google Scholar
• Hoehn MM, Yahr MD. Parkinsonism: onset, progression and mortality. Neurology. 1967;17(5):427–11.
   PubMed Web of Science ®Google Scholar
• Goetz CG, Poewe W, Rascol O, et al. Movement disorder society task force report on the Hoehn and Yahr staging scale: status and recommendations. Mov Disord. 2004;19(9):1020–1028.
   PubMed Web of Science ®Google Scholar
• Movement Disorder Society Task Force on Rating Scales for Parkinson’s D. The Unified Parkinson’s disease rating scale (UPDRS): status and recommendations. Mov Disord. 2003;18(7):738–750.
   PubMed Web of Science ®Google Scholar
• Goetz CG, Tilley BC, Shaftman SR, et al. Movement Disorder Society-sponsored revision of the Unified Parkinson’s Disease Rating Scale (MDS-UPDRS): scale presentation and clinimetric testing results. Mov Disord. 2008;23(15):2129–2170.
   PubMed Web of Science ®Google Scholar
• Venuto CS, Potter NB, Dorsey ER, et al. A review of disease progression models of Parkinson’s disease and applications in clinical trials. Mov Disord. 2016;31(7):947–956.
   Web of Science ®Google Scholar
• Fundament T, Eldridge PR, Green AL, et al. Deep brain stimulation for Parkinson’s disease with early motor complications: a UK cost-effectiveness analysis. PLoS One. 2016;11(7):e0159340.
   PubMed Web of Science ®Google Scholar
• Dams J, Bornschein B, Reese JP, et al. Modelling the cost effectiveness of treatments for Parkinson’s disease: a methodological review. Pharmacoeconomics. 2011;29(12):1025–1049.
   PubMed Web of Science ®Google Scholar
• Folse H, Chandler C, Alvarez P, et al. Model-based economic evaluations of treatments for Parkinson’s disease: a systematic literature review. Paper presented at: International Society for Pharmacoeconomics and Outcomes Europe; Barcelona, Spain; 2018.
   Google Scholar
• Jorgensen J, Servos S, Kefalas P. The potential price and access implications of the cost-utility and budget impact methodologies applied by NICE in England and ICER in the US for a novel gene therapy in Parkinson’s disease. J Mark Access Health Policy. 2018;6(1):1500419.
   PubMedGoogle Scholar
• Hoerger TJ, Bala MV, Rowland C, et al. Cost effectiveness of pramipexole in Parkinson’s disease in the US. Pharmacoeconomics. 1998;14(5):541–557.
   PubMed Web of Science ®Google Scholar
• ClinicalTrials.gov. Exenatide once weekly over 2 years as a potential disease modifying treatment for Parkinson’s disease (exenatide-PD3). [ cited 2021 Apr 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT04232969
   Google Scholar
• ClinicalTrials.gov. A study to evaluate the efficacy of prasinezumab (RO7046015/PRX002) in participants with early Parkinson’s disease (PASADENA). [ cited 2021 Apr 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT03100149
   Google Scholar
• Carroll CB, Webb D, Stevens KN, et al. Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study. BMJ Open. 2019;9(10):e029740.
   Google Scholar
• ClinicalTrials.gov. Evaluating the safety, pharmacokinetics, and pharmacodynamics of BIIB054 in participants with Parkinson’s disease (SPARK). [ cited 2021 Apr 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT03318523
   Google Scholar
• Smedinga M, Darweesh SKL, Bloem BR, et al. Towards early disease modification of Parkinson’s disease: a review of lessons learned in the Alzheimer field. J Neurol. 2021;268(2):724–733.
   Web of Science ®Google Scholar
• Caro JJ. Discretely integrated condition event (DICE) simulation for pharmacoeconomics. Pharmacoeconomics. 2016;34(7):665–672.
   PubMed Web of Science ®Google Scholar
• Kaltenboeck A, Johnson SJ, Davis MR, et al. Direct costs and survival of medicare beneficiaries with early and advanced Parkinson’s disease. Parkinsonism Relat Disord. 2012;18(4):321–326.
   PubMed Web of Science ®Google Scholar
• Parkinson’s Progression Markers Initiative. Home page. [ cited 2018 Jan]. Available from: www.ppmi-info.org
   Google Scholar
• Writing Group for the NINDS Exploratory Trials in Parkinson Disease Investigators, Kieburtz K, Tilley BC, et al. Effect of creatine monohydrate on clinical progression in patients with Parkinson's disease: a randomized clinical trial. JAMA. 2015;313(6):584–593.
   Web of Science ®Google Scholar
• National Institute of Neurological Disorders and Stroke. Archived clinical research data (NET-PD LS-1 creatine in Parkinson’s disease; Karl Kieburtz, MD, MPH). [ cited 2021 Jan]. Available from: https://www.ninds.nih.gov/Current-Research/Research-Funded-NINDS/Clinical-Research/Archived-Clinical-Research-Datasets
   Google Scholar
• Goetz CG, Stebbins GT, Tilley BC. Calibration of unified Parkinson’s disease rating scale scores to movement disorder society-unified Parkinson’s disease rating scale scores. Mov Disord. 2012;27(10):1239–1242.
   Web of Science ®Google Scholar
• Parkinson Study Group PI. Mixed lineage kinase inhibitor CEP-1347 fails to delay disability in early Parkinson disease. Neurology. 2007;69(15):1480–1490.
   PubMed Web of Science ®Google Scholar
• Ravina B, Tanner C, Dieuliis D, et al. A longitudinal program for biomarker development in Parkinson’s disease: a feasibility study. Mov Disord. 2009;24(14):2081–2090.
   Web of Science ®Google Scholar
• Johnson SJ, Diener MD, Kaltenboeck A, et al. An economic model of Parkinson’s disease: implications for slowing progression in the USA. Mov Disord. 2013;28(3):319–326.
   PubMed Web of Science ®Google Scholar
• Eggington S, Valldeoriola F, Chaudhuri KR, et al. The cost-effectiveness of deep brain stimulation in combination with best medical therapy, versus best medical therapy alone, in advanced Parkinson’s disease. J Neurol. 2014;261(1):106–116.
   PubMed Web of Science ®Google Scholar
• Lowin J, Sail K, Baj R, et al. The cost-effectiveness of levodopa/carbidopa intestinal gel compared to standard care in advanced Parkinson’s disease. J Med Econ. 2017;20(11):1207–1215.
   PubMed Web of Science ®Google Scholar
• Walter E, Odin P. Cost-effectiveness of continuous subcutaneous apomorphine in the treatment of Parkinson’s disease in the UK and Germany. J Med Econ. 2015;18(2):155–165.
   PubMed Web of Science ®Google Scholar
• Office for National Statistics. National life tables, UK: 2016 to 2018. Fit parametric distributions to reported survival data.
   Google Scholar
• Kalabina S, Belsey J, Pivonka D, et al. Cost-utility analysis of levodopa carbidopa intestinal gel (Duodopa) in the treatment of advanced Parkinson’s disease in patients in Scotland and Wales. J Med Econ. 2019;22(3):215–225.
   PubMed Web of Science ®Google Scholar
• Findley LJ, Wood E, Lowin J, et al. The economic burden of advanced Parkinson’s disease: an analysis of a UK patient dataset. J Med Econ. 2011;14(1):130–139.
   PubMedGoogle Scholar
• Kalilani L, Friesen D, Boudiaf N, et al. The characteristics and treatment patterns of patients with Parkinson’s disease in the USA and UK: a retrospective cohort study. PLoS One. 2019;14(11):e0225723.
   Web of Science ®Google Scholar
• Fasano A, Fung VSC, Lopiano L, et al. Characterizing advanced Parkinson’s disease: OBSERVE-PD observational study results of 2615 patients. BMC Neurol. 2019;19(1):50.
   PubMedGoogle Scholar
• National Institute for Health and Care Excellence (NICE). Guide to the methods of technology appraisal 2013. Published 2013 [cited 2021 Jan]. Available from: https://www.nice.org.uk/process/pmg9/chapter/foreword
   Google Scholar
• National Institute for Health and Care Excellence (NICE). Reviewing our methods for health technology evaluation: consultation. [ cited 2021 Feb 1]. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/chte-methods-consultation
   Google Scholar
• Caro JJ, Briggs AH, Siebert U, et al. Force I-SMGRPT. modeling good research practices–overview: a report of the ISPOR-SMDM modeling good research practices task force-1. Med Decis Making. 2012;32(5):667–677.
   PubMed Web of Science ®Google Scholar
• Roberts M, Russell LB, Paltiel AD, et al. Conceptualizing a model: a report of the ISPOR-SMDM modeling good research practices task force-2. Med Decis Making. 2012;32(5):678–689.
   PubMed Web of Science ®Google Scholar