Hemophilia Market Size, Share & Trends Analysis Report By Type (Hemophilia A, B, C), By Treatment (On-demand, Prophylaxis), By Therapy, By Region, And Segment Forecasts, 2018 - 2024

Industry Insights

The global hemophilia market size was valued at USD 9.86 billion in 2016 and is expected to grow at a CAGR of 5.6% during the forecast period. Hemophilia is also known as the royal blood disease, it is a rare genetic bleeding disorder. About 400,000 people have been estimated to be affected by hemophilia globally as of 2013.

Increasing need for diagnosis of the target population in order to initiate prophylaxis treatment and supportive government programs are anticipated to be the vital impact rendering drivers. Rising neonatal population has been driving the diagnostic rate. Active efforts are being taken by organizations such as the WHO, CDC, Healthy Newborn Network, etc., to educate parents for the possible disorders and their diagnostic procedures.

Furthermore, as of 2013, according to the World Federation of Hemophilia, only 30-40% individuals are diagnosed. By mandating the screening procedures on birth, is expected to help in boosting the diagnosis and early detection of the disease.

With mandating of neonatal screening for hemophilia and other associated diseases, the government is also working towards identification of moderate and mild cases in adults. Furthermore, to curb the financial pressure on patients over the treatment cost the hemophilia federations across the globe are providing co-pay assistance programs in collaboration with industry players.

Advantages such as supportive financial assistance, reduced contamination and incidences of joint damage and prevention of bleeding episodes are anticipated to boost the market growth. Therefore, it may be concluded that the efficacy and performance of treatment is enhanced by continuous treatment, thereby propelling adoption rate.

Presence of limited therapies and rising burden over the regulatory authorities towards treatment of these disorders is expected to drive R&D focused on identifying curative agents. This, in turn, is expected to boost growth during the forecast period. Furthermore, increasing campaigns and supportive government initiatives plan to introduce early screening of neonates, which is further expected to propel the industry’s growth.

Initiatives aimed at promoting the adoption of prophylaxis treatment by patients are expected to boost the market growth over the forecast period. Supportive financial assistance, reduced contamination, joint damage and prevention of bleeding episodes are the vital impact rendering factors. According to a study published in the New England Journal of Medicine, on opting for prophylaxis treatment, about 93.0% of the patients were observed to be free from any joint damages.

Type Insights

On the basis of type, the hemophilia market has been segmented into type A, B, C and others. The classification has been done based on the deficiency of coagulation factor in a certain patient. The most common type is A and has been identified to be four times more prevailing than other types of hemophilia.

In addition, in the field of pharmaceuticals, the drugs that are used for the treatment of hemophilia type A is enormous, thereby limiting its growth over the forecast period. Recombinate and Advate are the popular drugs manufactured by Baxalta that are used for the prophylaxis treatment of type A patients across the U.S., the UK, Japan, Germany, South Africa, and China

Some other diseases associated with the deficiency of clotting factors are Parahemophilia and von Willebrand disease. Von Willebrand disease arises from a quantitative or qualitative deficiency of von Willebrand factor (vWF). Parahemophilia is a very rare bleeding disease that occurs due to the deficiency of clotting factor V.

Treatment Insights

Based on treatment, the hemophilia market has been segmented into prophylaxis, and on-demand. The on-demand segment has been estimated to dominate the treatment segment with about 60.0% of the market share in 2015.

In patient with mild to moderate hemophilia and some patient with severe hemophilia, treatment can be given at the time when bleeding occurs, is termed as on-demand therapy. The advantage of on-demand therapy is that one does not need to infuse the coagulating factors regularly and the replacement factor is injected into the vein immediately after an injury has occurred.

The principle behind attributing prophylaxis is to efficiently convert a patient suffering with severe hemophilia to moderate hemophilia by giving clotting factors on a regular basis. There are generally two types of prophylaxis regimes that can be used, viz. primary prophylaxis and secondary prophylaxis. Primary prophylaxis is when continuous treatment is started before the age of two years or after the first joint bleed has occurred. Secondary prophylaxis is when continuous treatment is started after two or more joint bleeds or after the age of two years.

The rising treatment cost, and regular visits to healthcare facilities prevents the patient from opting for prophylaxis treatment. However, prophylaxis treatment is anticipated to witness lucrative growth during the forecast period owing to the efforts by healthcare providers to maintain a continuous replacement regime of the deficient coagulant factors in order to prevent rise in severity of the disease. For instance, the co-pay programs by the regional federations such European Hematology Association. Also, improving healthcare infrastructure and rising disposable income are factors promoting growth.

Therapy Insights

On the basis of therapy, the segments are replacement therapy, gene therapy, and Immune Tolerance Induction (ITI) therapy. Gene therapy is a developing field that is focusing on identifying the defective DNA bases and restoring with functional ones. Therefore, the segment is expected to present the key player of the industry with the most lucrative opportunities over the forecast period. Moreover, replacement therapy is expected to witness lucrative growth due to the launch of extended half-life versions of currently available drugs.

In addition, about 60.0% cases of type A fall into the severe category within the U.S, as per the statistics by the NHF stated in their annual report, driving the demand for a more permanent solution rather than replacement through chemically synthesized factors.

BioMarin announced the first clinical results for their gene therapy molecule BMRN-270 indicated to be used for curing type A in April 2016. According to the study, six patients from a total of eight illustrated production of the deficient factor VIII.

Regional Insights

The hemophilia market was dominated by North America and is expected to contribute for about 45.0% of the market share. Rising prevalence of the disease along with rising adoption towards prophylaxis treatment are anticipated to be the key drivers of this market. About 63.0% of the U.S patients have opted for prophylaxis treatment, as per the annual global survey, 2013 of WFH, thereby propelling the growth.

However, Asia-Pacific and Latin American regions are expected to witness substantial growth over the forecast, increasing awareness about technologically advanced products and primarily rising number of hemophilic patients in these regions. Additionally, owing to better diagnostic technique, growing medical research and increasing prophylactic treatments in Asia-Pacific and Latin America, the demand for recombinant and plasma derived products are further increasing the growth in these regions.

Hemophilia Market Share Insights

Key players in this industry include CSL Behring, Baxalta, Pfizer Inc, BioMarin, Bayer Healthcare, Biogen, Novo Nordisk, Roche, Shire Plc., and others. Shire Plc. is the most progressive company currently due to its acquisition of Baxalta in June 2016. About 40.0% of the annual revenue were gained by Baxalta through the sale of their drugs such as Advate, Recombinate, Hemofil, Obizur, And Rixubis

The market players are predicted to adopt strategies such as product pipeline development and geographic expansion in under developed and developing regions such as Middle Eastern and West African countries. Competitive pricing strategy is a vital factor promoting market players’ growth over the forecast period. For instance, Biogen launched Eloctate in 2014 in the U.S. at a lower price than the existing and vastly used drug Advate.

Increasing R&D expenditure and exploration of new geographic regions in order to develop longer acting alternatives are the key drivers observed. For instance, for marketing authorization of Adynovi (Adynovate in the U.S.), Baxalta filed an application with the EMA. The drug is currently under regulatory evaluation in Switzerland.

Mergers, acquisitions, and collaborations help the market players to maintain their market positions. For instance, Novo Nordisk announced the acquisition of a new production plant in New Hampshire. The acquisition was done as an initiative to ensure the production capacity for the company’s hemophilia products was sustained over the forecast period.

Similarly, Biogen in collaboration with bodies such as the American Thrombosis and Hemostasis Network (ATHN), the Puget Sound Blood Center (PSBC), and the National Hemophilia Foundation (NHF) announced the launch of a nation-wide program for free genetic testing of individuals suffering from hemophilia and their family members. By this initiative, the company and healthcare institutes aimed are increasing awareness and raising the diagnostic rate for the industry.

Report Scope

Segments Covered in the Report

This report forecasts revenue growth and provides an analysis on the latest trends in each of the sub-segments from 2013 to 2024. For the purpose of this study, Grand View Research, Inc. has segmented the global hemophilia market report on the basis of type, therapy, treatment and region:

• Type Outlook (Revenue, USD Million, 2013 - 2024)

  • Hemophilia A

  • Hemophilia B

  • Hemophilia C

  • Others

• Therapy Outlook (Revenue, USD Million, 2013 - 2024)

  • On-demand

  • prophylaxis

• Treatment (Revenue, USD Million, 2013 - 2024)

  • Replacement therapy

  • ITI therapy

  • Gene therapy

• Regional Outlook (Revenue, USD Million, 2013 - 2024)

  • North America

    • U.S.

    • Canada

  • Europe

    • Germany

    • UK

  • Asia Pacific

    • Japan

    • China

  • Latin America

    • Brazil

    • Mexico

  • Middle East and Africa (MEA)

    • South Africa

    • Saudi Arabia