SURVEY ON HEMOPHILIA CARE & PRICE MONITORING
UNITED STATES — WAVE 24

 

In the past two years, several new coagulation factor products have been approved in the United States to treat hemophilia and von Willebrand disease. This included four new factor VIII products, three new factors IX products and the first recombinant von Willebrand factor, as well as a product to treat acquired hemophilia. Some of these new products have an extended half-life (EHL), allowing for less frequent infusion. In this new environment, the hemophilia community has begun experiencing significant changes in patient care, lifestyle, treatment options, and cost while reimbursement and access have become more complex.

Since 1993, the Marketing Research Bureau has surveyed and analyzed the hemophilia care market, annually, focusing on the most important issues including the conversion rate and rationale to new products, in particular recombinant factors, the adoption of prophylaxis and immune tolerance, the evolving use of plasma-derived products, the emergence of new companies and therapies, treatment preferences and pricing and reimbursement trends.

This new “Wave 24” survey is based on some thirty in-depth telephone interviews conducted in the first quarter of 2017with hemophilia treatment centers and other hemophilia stakeholders. The report updates the hemophilia care market situation in the United States, with particular focus on:

  • Adoption rate of extended half-life recombinant factors VIII and IX;
  • Market shares of coagulation factor products;
  • Rationale and Decision Process in brand switching;
  • Acquisition and reimbursement prices;
  • Adoption and Barriers relating to immune tolerance and prophylaxis;
  • Awareness of new products and technologies in development;
  • Role of Specialty Pharmacies/home care companies, impact of the 340B program; and
  • Patients’ issues.

The report is delivered electronically (Excel and pdf files) and as a hard copy. For more information or to order this report, please contact The Marketing Research Bureau at mrb_ibpn@marketingresearchbureau.com, or +1 203 799-0298.


 

Survey on Hemophilia Care & Price Monitoring
United States
Wave #24

 

TABLE OF CONTENTS

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EXECUTIVE SUMMARY

 

1) INTRODUCTION

2) PATIENT POPULATION

2.1) Hemophilia

2.2) von Willebrand Disease

3) FACTOR VIII

3.1) Recombinant Factor VIII Products

3.2) Plasma-derived Factor VIII Products

4) FACTOR IX

4.1) Recombinant Factor IX Products

4.2) Plasma-derived Factor IX Products

5) VON WILLEBRAND DISEASE AND RARE BLEEDING DISORDERS

5.1) von Willebrand Disease

5.2) Rare Bleeding Disorders

6) PROPHYLAXIS

7) INHIBITOR MANAGEMENT

8) PATIENT ISSUES AND PRODUCT PREFERENCES

8.1) EHL Product Switching Analysis

8.2) Hospitals Formularies

8.3) Patients Needs and Issues in Hemophilia Care

8.4) Insurance Coverage and its Impact on Brand Choice

8.5) Treatment Log

9) SPECIALTY PHARMACIES & 340 B PROGRAM

9.1) Background on Specialty Pharmacies and the 340 B Program

9.2) Specialty Pharmacies and 340 B Survey Results

10) PRICES

10.1) Government Hospital/340B Program Acquisition Prices

10.2) Other Hospital/Home Care Acquisition Prices

10.3) CMS Reimbursement Prices

11) NEW PRODUCT DEVELOPMENTS

11.1) Number of Patient in Clinical Trials with Investigative Products

11.2) Novel Products Awaited by Respondents

11.3) Gene Therapy

12) MARKET PROSPECTS FOR NEXT YEAR

13) METHODOLOGY

14) SUMMARY OF THE SURVEY RESULTS

15) INTERVIEW NOTES

 

LIST OF TABLES AND CHARTS

Table 1 Distribution of Patients by Severity (Hemophilia/Type (vWD) – Q 1/16

Table 2 Percentage of Patients using recombinant FVIII and IX

Table 3 CDC-UDC Program, ATHN and MRB Survey Hemophilia Comparison

Table 4 Summary of ATHN dataset December 2015 – Hemophilia

Table 5 CDC-UDC Program, ATHN and MRB Survey vWD Comparison

Table 6 Summary of ATHN dataset December 2015 – vWD

Table 7 Patient Market Share of Factor VIII Concentrates

Table 8 Market Penetration of Factor VIII Products by Patient Share

Chart 1 Market Penetration of Recombinant Factors VIII

Chart 2 Market Share of Factor VIII Concentrates

Chart 3 Factor VIII Market Share Changes – Q4/14-Q1/16

Table 9 Patient Market Share of Factor IX Concentrates

Table 10 Market Penetration of Factor IX Products by Patient Share

Chart 4 Market Penetration of Recombinant Factors IX

Chart 5 Market Share of Factor IX Concentrates

Chart 6 Factor IX Market Share Changes – Q4/14-Q1/16

Table 11 Patient Share of VWD Products

Table 12 Patients with Rare Coagulation Deficiencies

Table 13 Prevalence and Rate of Inhibitor Development

Table 14 Market Share for ITI treated Inhibitor Patients

Table 15 Mode of treatment for Hemophilia A Patients with Inhibitors

Table 16 Distribution of Hemophilia Patients using 340B Programs

Table 17 Government/340B Program Acquisition Prices of Factor Concentrates

Table 18 Hospital/Home Care Company Acquisition Prices of Factor Concentrates

Table 19 CMS Reimbursement Prices of Factor Concentrates – Q2/07 – Q2/16

Chart 7 Hospital/Home Care Acquisition Prices of Factor VIII Concentrates

Chart 8 Hospital/Home Care Acquisition Prices of Factor IX and Other Concentrates

Chart 9 CMS Reimbursement Prices of Factor VIII Concentrates – Q2/12 – Q2/16

Chart 10 CMS Reimbursement Prices of Factor IX & Other Concentrates – Q2/12 – Q2/16

Table 20 CMS Reimbursement vs. Hospital/Home Care Acquisition Prices  – March 2016