Marjolijn van Keep1, Christina Stentoft Hoxer2, Matthew Hemstock3, Andreas Velsing Groth2, Christopher Knight4. 1. BresMed Netherlands BV, Arthur van Schendelstraat 650, 3511MJ Utrecht, The Netherlands. 2. Novo Nordisk A/S, Vandtårnsvej 114, DK-2860 Søborg, Denmark. 3. BresMed Health Solutions, Ltd, North Church House, 84 Queen Street, Sheffield S1 2DW, UK. 4. RTI Health Solutions (Formerly BresMed Health Solutions, Ltd), The Pavilion, Towers Business Park, Wilmslow Road, Didsbury, Manchester M20 2LS, UK.
Abstract
AIM: To develop a modeling approach to compare clinical outcomes of nonacog beta pegol to a standard-acting factor IX (FIX) product. METHODS: Regression analysis linked FIX activity to bleed rates. Pharmacokinetic parameters were used to estimate FIX activity over time. The probability of bleeds was estimated for both treatment arms. A Markov model estimated the presence of target joints and annualized bleed rates (ABRs). RESULTS: Higher FIX activity showed reduced ABRs (p < 0.001). Target joints resulted in higher bleed rates (p < 0.001). When FIX activity levels and bleed risks were applied to the Markov model, ABRs for nonacog beta pegol and its comparator were 2.40 and 6.36, respectively. CONCLUSION: This model provides a starting point for assessing the added value of new FIX products.
AIM: To develop a modeling approach to compare clinical outcomes of nonacog beta pegol to a standard-acting factor IX (FIX) product. METHODS: Regression analysis linked FIX activity to bleed rates. Pharmacokinetic parameters were used to estimate FIX activity over time. The probability of bleeds was estimated for both treatment arms. A Markov model estimated the presence of target joints and annualized bleed rates (ABRs). RESULTS: Higher FIX activity showed reduced ABRs (p < 0.001). Target joints resulted in higher bleed rates (p < 0.001). When FIX activity levels and bleed risks were applied to the Markov model, ABRs for nonacog beta pegol and its comparator were 2.40 and 6.36, respectively. CONCLUSION: This model provides a starting point for assessing the added value of new FIX products.