SURVEY ON HEMOPHILIA CARE & PRICE MONITORING
UNITED STATES — WAVE 25

 

In the past four 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. Some of these new products have an extended half-life (EHL), allowing for less frequent infusion. In October 2018, the first monoclonal antibody was approved. 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.

The “Wave 25” survey is based on some twenty-five in-depth telephone interviews conducted in the first quarter of 2018 with 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 #25

 

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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) Which Factor VIII products are considered EHL products?

6.3) Number of Patients on Prophylaxis for Hemophilia A

6.3.1) Patient Prophylaxis Share

6.3.2) Percent of patients on Prophylaxis by Product

6.3.3) Hemlibra for Prophylaxis

6.4) Number of Patients on Prophylaxis for Hemophilia B

6.5) Dosing Regimen and Individualized Treatment

6.5.1) Weekly Dosage on Prophylaxis

6.5.2) Minimum Trough Level

6.6) Quantity of factor VIII and IX used for prophylaxis

7) INHIBITOR MANAGEMENT

7.1) Background on Inhibitor Management

7.1.1 SIPPET Study Results

7.2) Inhibitor Survey Results

7.2.1) Rate of Inhibitor Formation and Total Patients with Inhibitors 59

7.2.2) Treatment for Patients with Inhibitors

7.2.2.1) Hemlibra

7.2.2.2) Immune Tolerance Induction (ITI)

7.2.2.3) Bypassing Agents

7.2.3) Factor VIII Used to Treat ITI Patients

8) PATIENT ISSUES AND PRODUCT PREFERENCES

8.1) Hemlibra Switching Analysis

8.1.1) Patient Survey on Interest in Hemlibra

8.1.2) Concerns about Hemlibra and Suggestions

8.2) EHL Switching Analysis

8.2.1) Patients not switching to EHL products

8.2.2) Idelvion vs. Alprolix and Rebinyn

8.3) Hospitals Formularies

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

9) SPECIALTY PHARMACIES & 340 B PROGRAM

9.1) Background on the 340 B Program

9.2) 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 and Respondents’ Opinion

11.2.1) Alnylam’s fitusiran

11.2.2) Roche/Genentech’s Hemlibra (emicizumab)

11.3) Gene Therapy

11.3.1) Background on Gene Therapy

11.3.2) Current Gene Therapy Trials

12) MARKET PROSPECTS FOR NEXT YEAR

13) METHODOLOGY

14) SUMMARY OF THE SURVEY RESULTS

15) INTERVIEW NOTES