Keep MV, Hoxer CS, Hemstock M, Groth AV, Knight C. A new modeling approach allowing prediction and comparison of the long-term outcomes of treatments for hemophilia B. J Comp Eff Res. 2018 Jan;7(1):39-48. doi: 10.2217/cer-2017-0028.


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.

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