US TRENDS IN HEMOPHILIA CARE & PRICE MONITORING WAVE #27

(UNITED STATES)
May 2020

 

Since 1993, the Marketing Research Bureau has tracked the US hemophilia care market, focusing on the most important clinical as well as socio-economic issues including product’s switching and adoption of new therapies, product’s differentiation, prophylaxis and immune tolerance, treatment preferences, pricing, reimbursement trends and more,

More than a year after the FDA approval of Roche’s Hemlibra in October 2018 for the treatment of hemophilia A patients on prophylaxis, the product continues to gain market share at the expense of other therapies. With increasing numbers of factor products (including extended half-life options) and non-factor therapies for hemophilia A now on the market, the variety of new therapeutic options for patients with bleeding disorders, has made choosing a therapy ever more complicated. Furthermore, the first gene therapy treatment likely be approved by mid-2020. Are hemophilia treatment centers (HTC) ready for gene therapy? In addition to wider product choice, the hemophilia community is gradually experiencing significant changes in patient care, lifestyle expectations, and reimbursement which has become more complex.

For the producers of replacement factor products, the wide choice offered to patients and treating personnel represents a new challenge as it becomes increasingly difficult to maintain differentiation in a crowded market, with pressure on prices, and Hemlibra gaining market share. Additionally, the plasmas-derived therapies face challenges in immune tolerance.

Based on around twenty in-depth telephone interviews with hemophilia treatment centers and other stakeholders, this report provides clients with a detailed description of the US hemophilia care market in early 2020 and how it has evolved over the past decade. Some of the report’s focuses include:

  • Analysis of coagulation factor brands, including the adoption and perception of new recombinant factors VIII and IX, as well as of Hemlibra;
  • Market shares of coagulation factor products;
  • Trends in prophylaxis and immune tolerance;
  • Awareness of new products and technologies in development, including gene therapy;
  • Impact of the 340 B program and patients’ issues and
  • Coagulation factor prices.


 

Survey on Hemophilia Care & Price Monitoring
United States
Wave #27

 

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) Hemlibra & Non-Factor Products

3.3) Plasma-derived Factor VIII Products

4) FACTOR IX

4.1) Recombinant Factor IX Products

4.2) Plasma-derived Factor IX Products

5)VWD 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

7.2) Inhibitor Survey Results

7.2.1) Rate of Inhibitor Formation and Hemlibra’s impact

7.2.2) Treatment for Patients with Inhibitors

7.2.2.1) Hemlibra

7.2.2.2) Decline of 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) Uptake by Center

8.1.2) Hemlibra Adoption Attitudes

8.1.3) Who isn’t switching to Hemlibra and staying on FVIII?

8.2) Idelvion vs. Alprolix vs. Rebiny – Which EHL is preffered?

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) Sanofi’s novel EHL FVIII

11.3) Gene Therapy

11.3.1) Background on Gene Therapy

11.3.2) Current Gene Therapy Trials

11.3.3) Center Expectations for Offering GT in next 12 months?

11.3.4) Concerns about Gene Therapy today

11.3.5) Anticipated uptake of GT in first 12 months?

12) MARKET PROSPECTS FOR NEXT YEAR

13) METHODOLOGY

14) SUMMARY OF THE SURVEY RESULTS

15) INTERVIEW NOTES