SURVEY ON HEMOPHILIA CARE & PRICE MONITORING
UNITED STATES — WAVE 23

 

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 23” survey is based on some thirty in-depth telephone interviews conducted in the first quarter of 2016with 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@earthlink.net, or +1 203 799-0298.


 

Survey on Hemophilia Care & Price Monitoring
United States
Wave #23

 

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

6.2.1) Influence of the Extended Half-life Products on Prophylaxis Treatment4

6.3.1 Pharmacokinetic studies (PK) before switching to EHL products

6.3.2 Weekly Dosage on Prophylaxis

6.3.3 Minimum Trough Level

7) INHIBITOR MANAGEMENT

7.1.1 RODIN and SIPPET Study Results

7.2) Inhibitor Survey Results

8) PATIENT ISSUES AND PRODUCT PREFERENCES

8.1) EHL Product Switching Analysis

8.1.1) Patients Mostly likely to switch to EHL products

8.1.2) Product Switching Drivers (Patients vs. HTCs)

8.1.3) Eloctate vs. Adynovate

8.1.4) Current satisfaction with EHL products

8.2) Hospitals Formularies

8.3) Patients Needs and Issues in Hemophilia Care

8.4) Insurance Coverage and its Impact on Brand Choice

8.4.1) Type of Insurance and Patient Coverage

8.4.2) Private Insurance influence on brand

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

11.2.1) CSL Behring’s Idelvion

11.2.2) Baxalta’s Vonvendi

11.2.3) Novel mAB Therapies

11.3) Gene Therapy

11.3.1) Background on Gene Therapy

11.3.2) Current Gene Therapy Trials

12) MARKET PROSPECTS FOR THE NEXT YEAR

13) METHODOLOGY

14) SUMMARY OF THE SURVEY RESULTS

15) INTERVIEW NOTES