SURVEY ON HEMOPHILIA CARE & PRICE MONITORING
UNITED STATES — WAVE 21
Over the past few decades, the hemophilia care market has experienced considerable changes, both qualitatively and quantitatively. Hemophilia patients have gradually converted from plasma-derived to recombinant products, first factor VIII (Baxter’s Recombinate, 1992, Bayer’s Kogenate, 1993), and then factor IX (Genetics Institute’s BeneFIX, 1997) and factor VIIa (Novo Nordisk’s NovoSeven, in 1999). Today, the majority of young boys with hemophilia have not been exposed to any plasma-derived product.
At the same time, the safety of plasma-derived clotting factors improved with the introduction of robust virus inactivation techniques, NAT testing of the blood and plasma collected, more stringent donor selection, and other safety measures.
As the benefits of prophylactic treatment gained recognition, and clotting factor products were acceptably safe, hemophilia patients opted increasingly for this mode of treatment from a young age onward, and well into adulthood. In patients developing an inhibitor to factor VIII or IX, immune tolerance induction became the standard approach offered to young patients, as recommended by the National Hemophilia Foundations’ Medical Advisory Committee (MASAC), and advocated in numerous scientific articles. As a result, the life expectancy of hemophilia patients has increased to be comparable to the general population’s, contributing to a growing hemophilia patient population. These clinical and social events led to an impressive growth of the U.S. consumption of coagulation factor concentrates: the U.S. market size almost quadrupled in twenty years, as it went from about 600 million units in 1992 to over 2.2 billion units in 2012. Similarly, the treatment of von Willebrand disease improved with the introduction of several new products (CSL Behring’s Humate P, 1999, Grifols’ Alphanate, 2005 and Octapharma’s Wilate, 2009).
In 2003, the introduction of a third generation recombinant factor VIII (rFVIII) devoid of albumin in the final container and cell growth medium (Baxter’s Advate) brought a new level of product safety. Pfizer’s Xyntha, approved in 2008 was also a third generation recombinant rFVIII. Since 2008, no new coagulation factor VIII or IX has been introduced in the U.S., but a number of new products have been in development by companies, some of which take advantage of the expiration of manufacturing patents, while others have sought to expand the half-life of products by means of a variety of protein engineering technologies. Consequently, several new recombinant factors are expected to reach the U.S. and European markets in the next two to three years. Some of them will significantly enhance the patient’s comfort by extending the interval between infusions in those treating prophylactically (Baxter, Bayer, Biogen IDEC, CSL Behring, and Novo Nordisk) while Octapharma is developing a recombinant factor VIII grown in human cells. Other companies outside the United States are also active in recombinant factor development.
Recombinant products are also in development in the area of von Willebrand disease (Baxter’s recombinant von Willebrand factor) and inhibitor treatment, including recombinant factor VIIa (CSL Behring, LFB), and recombinant porcine factor VIII (Baxter.) All these developments will make the coagulation factors’ markets more competitive, creating a challenge to differentiate brands.
The growth in recombinant factor production of the past few years has contributed to an increase in overall product availability not only in the U.S. and other industrialized countries, but in emerging markets, too. More factor VIII and IX units will become available once the new products in development reach the market, allowing treating more patients worldwide with plasma-derived products, and increasingly, recombinant factors as well.
The Marketing Research Bureau has surveyed hemophilia treatment centers for twenty years to track and analyze these trends. The first survey, titled Hemophilia Care and price Monitoring 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.
The hemophilia care market is constantly shifting: new products are coming to market, prophylaxis and immune tolerance induction continue to gain acceptance, the number of patients reaching advanced ages increases, and new infusion frequencies and dosages are in development, while cost remains a major issue for hemophilia patients. For these reasons, a completely new study was conducted and the report published in April 2013. The objective of this survey was to provide clients with a comprehensive description of the hemophilia care market in early 2013, as outlined in the interview topics listed below.
- The Marketing Research Bureau staff will conduct thirty to forty confidential telephone interviews with hemophilia treatment centers, distributors, manufacturers, home care organizations and other stakeholders. The bulk of the interviews will be conducted with nurse coordinators at selected HTCs, as well as with some hemophilia-treating hematologists, including several opinion leaders.
The sample of surveyed treatment centers will be as representative as possible of the U.S. hemophilia care market. The same centers that were contacted in the previous waves will be interviewed in order to maintain consistency of the data year to year. The treatment centers to be interviewed represent about a third of the total number of centers and patient population.
Survey on Hemophilia Care & Price Monitoring
TABLE OF CONTENTS
EXECUTIVE SUMMARY 6
1) INTRODUCTION 12
2) PATIENTS POPULATION 12
3) FACTOR VIII 19
4) FACTOR IX 28
5) VON WILLEBRAND DISEASE 34
6) PROPHYLAXIS USE IN HEMOPHILIA A & B 37
7) INHIBITOR MANAGEMENT 41
8) PRODUCT PREFERENCE AND PATIENTS’ ISSUES 47
9) HOME CARE/340 B PROGRAM 56
10) PRICES 61
11) NEW PRODUCT DEVELOPMENTS 72
12) MARKET PROSPECTS FOR 2013 76
13) METHODOLOGY 78
14) SUMMARY OF SURVEY RESULTS 80
15) INTERVIEW NOTES 81-150