Hemophilia Care Survey & Gene Therapy Interest in the United States 2022
SURVEY ON HEMOPHILIA CARE & Gene Therapy Interest in the United States
(May 2022)
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, treatment preferences, pricing, reimbursement trends and more,
The FDA approval of Roche’s Hemlibra in October 2018 for hemophilia A prophylactic treatment has opened a new era in hemophilia care, with rapid uptake in patients with inhibitors and regular prophylaxis. Since then, Hemlibra has steadily increased its market share, though other products retain significant market share. With a number of extended half-life recombinant factor VIII now on the market, the variety of new therapeutic options available to hemophilia patients, as well as for persons with von Willebrand disease has already changed the therapeutic landscape. For the clotting factor manufacturers, 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.
Based on thirteen 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 2022 and how it has evolved over the past decade. In addition, over 200 patients and parents of people with hemophilia were surveyed about their interest, awareness and receptiveness to gene therapy in the coming years. The results of this are shared to gauge potential uptake of these disruptive treatments. The report has over 135 pages of charts and analysis, along with over 55 pages of notes from the HTC interviews for all the details.
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 Genentech’s Hemlibra;
- Market shares of coagulation factor products;
- Trends in prophylaxis;
- Gene therapy awareness, interest and receptiveness for uptake anticipation
- 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 & Gene THerapy Interest in the United States 2022
TABLE OF CONTENTS
EXECUTIVE SUMMARY
1) INTRODUCTION
2) PATIENT POPULATION
2.1) Hemophilia
2.2) von Willebrand Disease
3) Hemophilia A and FACTOR VIII Products
3.1) Recombinant Factor VIII Products
3.2) Hemlibra for Hemophilia A
3.3) Plasma-derived Factor VIII Products
4) Hemophilia B and FACTOR IX Products
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) Hemlibra Prophylaxis Patient Share & Dosing Interval
6.3.2) Percent of patients on Prophylaxis by Product
6.4) Number of Patients on Prophylaxis for Hemophilia B
6.5) Dosing Regimen and Individualized Treatment
6.5.1) Weekly Dosage on Prophylaxis
6.6-6.7) 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) Hemlibra and Inhibitor Formation – Reduction in Inhibitor Patients
7.3) Inhibitor Treatment – Hemlibra & Bypassing Agents
8) PATIENT ISSUES AND PRODUCT PREFERENCES
8.1) Hemlibra Switching Analysis
8.2) EHL Factor IX Product Preference
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) CMS Reimbursement Prices
11) NEW PRODUCT DEVELOPMENTS & PATIENT GENE THERAPY INTEREST SURVEY
11.1) Novel Products and Respondents’ Opinion
11.2.1) Alnylam’s fitusiran
11.2) Gene Therapy
11.3.1) Background on Gene Therapy
11.3.2) Results of 200 patient Survey on Gene Therapy Interest
12) MARKET PROSPECTS FOR THE NEXT TWELVE MONTHS
13) METHODOLOGY
14) ANNEX – PATIENT GENE THERAPY QUESTIONNAIRE RESULTS SUMMARY
15) INTERVIEW NOTES