SURVEY ON HEMOPHILIA CARE & PRICE MONITORING
UNITED STATES — WAVE 22

 

February 2015

Introduction

After almost a decade with no new clottting factor product introduced in the U.S., the hemophilia community has now begun to experience major changes in their care, lifestyle, safety, and possibly economic situation due to the commercialization of several new recombinant factors, in particular long acting products. In the coming months new products are expected to come to the market, not only the traditional manufacturers of clotting factors (Baxter, Bayer, CSL Behring, Novo Nordisk, Octapharma, etc) but also new ones (Biogen IDEC, Emergent Biosolutions, Green Cross, and rEVO Biologics)

New coagulation factor products will appear in all facets of treatment, including von Willebrand disease and inhibitor treatments These developments will make the market more competitive, creating a challenge to differentiate brands. At the same time, the Affordable Care Act (ACA) will continue to impact healthcare delivery and financing.

Since the early 1990’s, the Marketing Research Bureau has surveyed and analyzed the hemophilia care market, focusing on important trends, such as the conversion to novel products, the adoption of prophylaxis and immune tolerance, the plasma industry consolidation, the emergence of new companies, patient’s aging and treatment preferences, and new developments in pricing and reimbursement.

The first survey, titled Hemophilia Care and price Monitoring Wave #1  was conducted in 1993. In subsequent years, it has been conducted once a year on average, and made available to clients as a syndicated report.


 

Survey on Hemophilia Care & Price Monitoring
United States
Wave #22

 

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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

6.1) Background on Prophylaxis use

6.2) Number of Patients on Prophylaxis

6.2.1) Influence of the Longer-Acting Product on Prophylaxis Treatment

6.3) Dosing Regimen and Individualized Treatment

7) INHIBITOR MANAGEMENT

7.1) Background on Inhibitor Management

7.2) Inhibitor Survey Results

7.2.1) Obizur (Porcine rFVIII)

8) PATIENT ISSUES AND PRODUCT PREFERENCES

8.1) Longer-Acting Product Switching Analysis

8.1.1) Profile of the Patients on longer-acting products and Rationale for

Switching

8.1.2) Product Switching Drivers (Patients vs. HTCs)

8.1.3) Correlation Between Centers Requiring PK testing and Percentage of

Patients on Longer-Acting Products

8.1.4) Timeline Expectations on  Switching to Longer-Acting Products

8.2) Hospitals Formularies

8.3) Patients Needs and Issues in Hemophilia Care

8.4) Insurance Coverage and its Impact on Brand Choice

8.5) Treatment Log

9) HOME CARE/340 B Program

9.1) Background on Home Care Companies and the 340 B Program

9.2) Home Care 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) Novel Products Awaited by Respondents

11.2) Gene Therapy

12) MARKET PROSPECTS FOR 2015

13) METHODOLOGY

14) SUMMARY OF SURVEY RESULTS

15) INTERVIEW NOTES