Rare Hemophilia Factors Market Size, Share & Trends Analysis Report By Type, By Treatment (Fresh Frozen Plasma, Factor Concentrates, Cryoprecipitate), By Region, Competitive Landscape, And Segment Forecasts, 2018 - 2025

Industry Outlook

The global rare hemophilia factors market size was valued at USD 213.3 million in 2016. The market is expected to expand at a CAGR of 5.1% during the forecast period. Several new factor concentrates securing approvals from regulatory bodies such as the U.S. FDA and government initiatives aimed at increasing diagnosis and treatment rates are among the key trends stoking market growth.

With increasing R&D activities by key market players such as Novo Nordisk and CSL Behring, the market is witnessing a spike in the number of new factor concentrates. In the recent past, several concentrates have received marketing approvals from regulatory bodies. For instance, in October 2015, FDA approved Coagadex for treatment of hereditary factor X deficiency. Similarly, CSL Behring also received FDA approval for its product Corifact for treatment of congenital factor XIII (FXIII) deficiency in 2011. Advent of new products is likely to augment the market. Wider availability of novel concentrates can help in increasing patient awareness and also providing patients with treatment alternatives.

Government initiatives and mandates have also increased patient and practitioner awareness about the rare hemophilia coagulation disorders, thereby revving up diagnosis and treatment rates of this condition. This, in turn, is likely to escalate the growth of the market over the forecast period. For instance, the Orphan Drug Act (ODA) was enacted by the U.S. Congress in 1983. From 1983 to 2015, around 3,647 drugs were designated the status of orphan drugs and 554 drugs were approved by the U.S. FDA. These orphan drug approvals have resulted in improved treatment alternatives for meeting the unmet needs of patients suffering from unusual diseases such as rare hemophilia disorders.

In addition, initiatives by the International Rare Diseases Research Consortium (IRDiRC) in Europe to develop 200 new therapies for such diseases by 2020 and providing funding for studies and R&D activities are expected to bolster the growth of these hemophilia factors market. Moreover, regional markets such as China are also driven by initiatives taken by patient advocacy groups, academic institutions, other nonprofit organizations, and pharmaceutical companies. For instance, The Chinese Organization for Rare Disorders (CORD) and societies such as Haemophilia Home of China are actively involved in initiating pilot projects to increase awareness, diagnosis rate, and treatment options for rare disorders in China.

Rising adoption of prophylactic treatments by patients is also contributing to the growth of the market. Prophylaxis has several advantages such as reduced incidence of joint pain and reduced joint damage. Patients with severe rare hemophilia disorders are recommended prophylactic treatment to avoid bleeding episodes and improve quality of life. For instance, long-term prophylactic treatment with NovoThirteen (rFXIII-A) is recommended for both adult and pediatric patients with congenital factor XIII A-subunit deficiency. Efficacy and performance of prophylaxis are enhanced by continuous treatment, thereby triggering the adoption rate. This, in turn, is likely to boost market growth over the forecast period.

Type Insights

On the basis of type, the market is segmented into I, II, V, VII, X, XI, and XIII deficiencies. Awareness about rare hemophilia deficiencies is anticipated to increase over the forecast period, due to efforts taken by organizations such as the World Federation of Hemophilia (WFH) for patient advocacy and improving diagnosis and treatment of rare hemophilia disorders. Factor VII deficiency has the highest prevalence of 1 per 300,000-500,000 as per the National Hemophilia Foundation.

The F-VII deficiency segment commanded the largest share in the market in 2016. The segment is estimated to maintain its position, rising at the highest CAGR during the forecast period. The growth of the segment can be attributed to the availability of treatment options such as recombinant factor VIIa (rFVIIa), NovoSeven (manufactured by NovoNordisk, Denmark). Moreover, as per a publication by “Hemophilia of Georgia” in 2013, several new rFVIIa products, counterparts of NovoSeven, were being developed by market players such as CSL Behring, Pfizer/Catalyst Biosciences, and Baxter. Such competitive rivalry among key players is poised to provide a fillip to the market.

Treatment Insights

On the basis of treatment, the rare hemophilia market is segmented into factor concentrates, fresh frozen plasma, cryoprecipitate, and others. The others segment comprises of prothrombin complex concentrate (PCC), desmopressin, and antifibrinolytic drugs.

The fresh frozen plasma segment held the largest share in 2016. Fresh frozen plasma contains all clotting factors and blood proteins. Due to limited availability and affordability of factor concentrates, fresh frozen plasma is used routinely to manage bleeding episodes in rare hemophilia deficiencies.

The factor concentrates segment is projected to expand at the highest CAGR during the forecast period. Factor concentrates are considered to be the safest and most effective treatment option. They are gaining preference for other treatment options such as cryoprecipitate and fresh frozen plasma, as they allow replacement of specific defective or deficient protein without adverse effects created by transfusion reactions or volume overloads.

Regional Insights

North America was at the forefront of the global arena in 2016. Government initiatives to increase the rate of diagnosis & reduce treatment costs, research activities to improve management of rare hemophilia conditions, and the strong presence of key players some of the factors supplementing the growth of the region. For instance, the National Organization for Rare Disorders (NORD), headquartered in the U.S., provides several research grants for the study of rare diseases. In addition, U.S. FDA approvals for certain coagulation factor concentrates in recent years have helped the regional market to gain traction.

Asia Pacific is expected to witness the highest growth over the forecast period owing to the growing population and rising incidence of autosomal recessive disorders due to consanguineous marriages in countries such as India, Pakistan, and China. As per an article titled “Epidemiological study of consanguineous marriage and its effects,” published in the International Journal of Community Medicine and Public Health, the prevalence of consanguineous marriages is high in the Middle East, Africa, and South Asia, estimated at around 20.0% to 50.0% of all marriages.

Rare Hemophilia Factors Market Share Insights

Some of the companies operating in the market are Novo Nordisk; Biogen; Bayer healthcare; Pfizer, Inc.; Baxalta; CSL Behring; and Bio Products Laboratory Ltd. Various Key players are relying on strategies such as collaborations, agreements & joint ventures, and promotions to increase the visibility of their products and strengthen their product portfolios. For instance, in January 2015, Novo Nordisk donated about USD 3 million to its Hemophilia Foundation as a means to provide financial assistance and enhance access to hemophilia treatment. Similarly, in May 2014, in an attempt to help the World Federation of Hemophilia (WFH) in improving the standard of care in developing countries, Biogen and Swedish Orphan Biovitrum AB (Sobi) committed to donating about 500 million units of coagulant therapy.

Report Scope

Segments Covered in the Report

This report forecasts revenue growth and provides an analysis of the industry trends in each of the sub-segments from 2014 to 2025. For the purpose of this study, Grand View Research, Inc. has segmented the global rare hemophilia factors market report on the basis of type, treatment, and region:

• Type Outlook (Revenue, USD Million, 2014 - 2025)

  • Factor I

  • Factor II

  • Factor V

  • Factor VII

  • Factor X

  • Factor XI

  • Factor XIII

• Treatment Outlook (Revenue, USD Million, 2014 - 2025)

  • Factor Concentrates

  • Fresh Frozen Plasma

  • Cryoprecipitate

  • Others

• Regional Outlook (Revenue, USD Million, 2014 - 2025)

  • North America

    • The U.S.

    • Canada

  • Europe

    • Germany

    • The U.K.

  • Asia Pacific

    • Japan

    • China

  • Latin America

    • Brazil

    • Mexico

  • The Middle East and Africa (MEA)

    • South Africa

    • Saudi Arabia