Potential Undiagnosed VWD Or Other Mucocutaneous Bleeding Disorder Cases Estimated From Private Medical Insurance Claims

Abstract

Introduction

Von Willebrand disease (VWD) is a common inherited bleeding disorder, but awareness among health care professionals is low. We estimated the number of cases of undiagnosed VWD or other mucocutaneous bleeding disorders among commercially insured patients in the United States with a recent history of bleeding events.

Methods

Patients with a VWD diagnosis who were users of or candidates for von Willebrand factor replacement were identified from the IMS PharMetrics Plus Database (2006–2015). We constructed a unary patient-finding model based on 12 prediagnosis variables that best defined this population, and applied this to undiagnosed patients with recent bleeding events from the same database. Cases of symptomatic undiagnosed VWD or other mucocutaneous bleeding disorders in the commercially insured population were estimated from the “best fit” (positive predictive value [PPV] 83%) and “good fit” (PPV 75%) patients thus identified.

Results

Overall, 507,668 undiagnosed patients with recent bleeding events were identified (86% female, 14% male). Application of the VWD model identified 3318 best-fit and 37,163 good-fit patients; 91% of best-fit patients were females aged <46 years, with heavy menstrual bleeding as the most common claim. Projection to the full commercially insured US population suggested that 35,000–387,000 patients may have symptomatic, undiagnosed VWD or other mucocutaneous bleeding disorders.

Discussion

Computer modeling suggests there may be a significant number of patients with symptomatic, undiagnosed VWD or other mucocutaneous bleeding disorder in the commercially insured population. Enhanced awareness of VWD symptoms and their impact, and of screening and testing procedures, may improve the diagnosis of VWD and reduce disease burden.

Keywords:

• medical insurance claims
• delayed diagnosis
• von Willebrand disease

Acknowledgments

Medical writing support was provided by Mary Berrington, PhD, and Iain Patefield, MS, of Parexel, Hackensack, NJ, USA, and was funded by Baxalta US Inc., a member of the Takeda group of companies, Lexington, MA, USA. Sarah A. Hale, Baxalta US Inc., a member of the Takeda group of companies, Cambridge, MA, USA, and Emily Brouwer, Shire US Inc., a member of the Takeda group of companies, Cambridge, MA, USA, also provided input and reviewed the manuscript for scientific accuracy. Imrran Halari, Charles River Associates, Boston, MA, provided additional statistical analyses for the work, in addition to providing input and reviewing the manuscript drafts.

Data Sharing Statement

Data are the proprietary property of Baxalta US Inc., a member of the Takeda group of companies, Lexington, MA, USA and access can be requested by contacting their legal representative.

Author Contributions

All authors contributed to data analysis, drafting and revising the article, gave final approval of the version to be published, and agree to be accountable for all aspects of the work. Although employees of the sponsor were involved in the design, collection, analysis, interpretation, fact checking of information, and coordination and collation of comments, the content of this manuscript, the interpretation of the data, and the decision to submit the manuscript for publication in the Journal of Blood Medicine was made by the authors.

Disclosure

AZ has received an investigator-initiated grant from Shire (a member of the Takeda group of companies). DF was an employee of Charles River Associates, which received consulting fees from Shire, at the time of the study and is currently an employee of UCB, Atlanta, GA, USA. RFS has acted as a paid consultant to Bayer, Shire, Novo Nordisk, Aptevo, Bioverativ, LFB, HEMA Biologics, and CSL Behring, and has received investigator-initiated grants from Bioverativ and Shire. He reports grants, personal fees from Genentech, Grifols, Kedrion, Takeda, and Octapharma, during the conduct of the study; also an investigator initiated grant (as well as personal consulting) from Octapharma, Genentech, Grifols, Kedrion, and Takeda. The authors report no other conflicts of interest in this work.

Additional information

Funding

Research was funded by the sponsor, Baxalta US Inc., a member of the Takeda group of companies, Lexington, MA, USA. Data were obtained from the IMS PharMetrics Plus Health Plan Claims Database, IMS Health Incorporated, all rights reserved. Analyses undertaken by Charles River Associates, Boston, MA, including Dana Fallaize, Mike Roy, Michael Agne and Imrran Halari, and was funded by Baxalta US Inc., a member of the Takeda group of companies, Lexington, MA, USA.