- Hemophilia is the result of improperly functioning or deficient clotting factor, caused by a mutation in the gene responsible for producing the clotting factor protein. It is a life-long and life-threatening disease which confers significant burden not only on the patient, but also onto the patient’s family, and the health care system.
- Hemophilia is treated by replacing the patient’s missing clotting factor. Patient access to the broad range of factor replacement products is essential as the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation notes, “(t)he lack of therapeutic interchangeability of products, in terms of efficacy, tolerance, and adverse drug reactions, could result in life threatening bleeding”1.
- The World Federation of Hemophilia guidelines state that prophylaxis should be the goal of therapy to preserve musculoskeletal function, especially for pediatric patients, while acknowledging resource constraints in developing economies to achieve that goal2.
- Clinical studies have shown that, compared to on-demand treatment, prophylaxis can substantially reduce the number of bleeds per year3, prevent joint damage4, and reduce healthcare costs5.
- An estimated 400,000 people worldwide are living with hemophilia6, but only 25% receive adequate treatment7.
- Lack of treatment and inadequate treatment of hemophilia can lead to:
- Severe bruising
- Prolonged bleeding from an injury, or following an invasive medical or dental procedure
- Joint damage caused by repeated and/or spontaneous bleeds:
- Can potentially lead to reduced physical functioning, increased pain8, and subsequent need for synovectomy or joint replacement surgery9.
- As joint damage can begin after one serious bleed, the use and adherence to prophylactic treatment can potentially prevent arthropathy10.
- With prophylaxis being the goal of therapy, treatment should be optimized to account for the uniqueness of each patient and pharmacokinetic profile, including disparate genetics, joint status, bleeding phenotype and lifestyle of patients.
Shire’s Legacy in Hemophilia Treatment
Shire is a global leader in the development and manufacturing of therapies for bleeding conditions such as Hemophilia. Shire’s heritage spans decades of innovation in the Hemophilia community. For over 60 years, we’ve listened to, learned from and championed the needs of patients with rare hematologic conditions. As an estimated 75% of people with Hemophilia remain untreated or undertreated, we focus on building public and private partnerships in communities around the world to expand awareness, diagnosis, and access to treatments (Shire Annual report 2016, p.37). Shire’s commitment to rare hematologic conditions continues to strengthen.
Shire’s Position on Hemophilia Treatment
We continue to develop a deep understanding of patients' needs with the goal of delivering transformative and personalized therapies intended to help improve the lives of patients with Hemophilia and other bleeding conditions to support them in living their life to the fullest.
At Shire, we believe:
- Every person with a rare hematologic condition deserves early diagnosis
- Access to hemophilia treatment centers, which provide comprehensive care to people with bleeding disorders, is essential for patients to receive optimal care
- Early prophylaxis can effectively prevent bleeds and help reduce the burden on patients
- A personalized approach supports optimization of each individual’s outcomes
Based on these core beliefs, we help transform lives of people with bleeding disorders by improving global Standards of Care. We support localized policies that ensure prescribers and patients are empowered to allow Healthcare Practitioners to make individualized treatment decisions that take into account patient’s clinical needs, caregiver burden, and local health care infrastructure capabilities.
- MASAC Document #166 “MASAC Resolution Regarding Preferred Drug Lists”, 30 October 2005
- World Federation of Hemophilia, “Guidelines for Management of Hemophilia, 2nd Edition”, 6 July 2012
- Valentino, LA et al. “Multicentre, randomized, open-label study of on-demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects.” Haemophilia. 2014 May;20(3):398-406.
- Manco-Johnson, MJ et al. “Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia” N Engl J Med. 2007 Aug 9;357(6):535-44.
- Miners, AH et al. “Cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia.” Pharmacoeconomics. 2002;20(11):759-74.
- Hemophilia Federation of America website http://www.hemophiliafed.org/bleeding-disorders/hemophilia/ accessed on 20 July 2017
- World Federation of Hemophilia website https://www.wfh.org/en/about-us accessed on 20 July 2017
- Miners, A “Assessing health-related quality-of-life in individuals with haemophilia” Haemophilia, (5) 378 – 385, 1999
- Luck, JV et al. “Hemophilic arthropathy” J Am Acad Orthop Surg. 2004 Jul-Aug;12(4):234-45.
- Gringeri, A et al. “The burden of bleeding in haemophilia: is one bleed too many?” Haemophilia. 2014 Jul;20(4):459-63.