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. Epub 2017 May 30.

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.

Keywords: alternative indirect treatment comparison; factor IX; hemophilia B; modeling; nonacog beta pegol; pharmacokinetics; prophylaxis.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Factor IX / therapeutic use*
  • Factor X / therapeutic use*
  • Hemophilia B / complications
  • Hemophilia B / drug therapy*
  • Hemorrhage / etiology
  • Hemorrhage / prevention & control
  • Humans
  • Markov Chains
  • Polyethylene Glycols / therapeutic use*
  • Recombinant Proteins / therapeutic use
  • Time
  • Treatment Outcome

Substances

  • Recombinant Proteins
  • nonacog beta pegol
  • Polyethylene Glycols
  • Factor IX
  • Factor X