Biogen Global Medical Grants Office
Biogen Global Medical Grants Office Hemophilia: Areas of Interest (Cycle C) June 2, 2015 1 Introduction • Hemophilia A and B are rare bleeding disorders caused by a lack of clotting factor VIII (hemophilia A) or a lack of clotting factor IX (hemophilia B) in circulation. Insufficient levels of clotting factor can make it difficult or impossible for blood to clot. People with hemophilia A or B do not bleed harder or faster than a person without hemophilia A or B—they bleed longer. • Hemophilia A and B are genetic conditions; they are lifelong and present from birth. Both forms of hemophilia are inherited disorders that affect mostly males. However, in about 30% of cases, there is no family history of hemophilia and the condition is likely the result of a spontaneous mutation of a gene. 1 The genetic mutation negatively affects the clotting factor, either in quantity or quality. Approximately 1 in 5,000 males are born with hemophilia A, and approximately 1 in 25,000 males are born with hemophilia B.1 • Hemophilia A and B are classified into 3 categories: mild, moderate, and severe. Each category is determined by the specific level of clotting factor VIII/factor IX a person has in circulation. 1, 2 People with mild hemophilia A/B have >5% to <40% of normal clotting factor in circulation People with moderate hemophilia A/B have 1% to 5% of normal clotting factor in circulation People with severe hemophilia A/B have <1% of normal clotting factor in circulation • In addition to bleeding following an injury, people with severe hemophilia A/B may have frequent spontaneous bleeding episodes, often into their joints and muscles, which can cause long‐term damage. 1, 3 2 Educational Gap Data • In a recent independent study 4 of practicing hematologists and nurses in the US who manage patients with Hemophilia it was discovered that: Almost half of hematologists (47%) would not change the clotting factor regimen for a patient with hemophilia B who is experiencing weekly joint bleeding episodes. Almost a quarter (23%) did not select "decreased frequency of clinically apparent soft tissue damage compared with an on demand regimen" as a clinical benefit of primary prophylaxis. For a non‐adherence, physically active teen with severe hemophilia A who has experienced bilateral ankle pain for several years, 39% of hematologists did not select "symptomatic joint pain in ankles, signifying progressive degenerative changes" and 23% did not select “data showing the superiority of prophylaxis for preventing joint damage, compared with an enhanced episodic regimen” when asked “Which of the following factors will most influence your selection of clotting factor concentrate replacement at this juncture?” For a patient with severe hemophilia B experiencing knee bleeds every 2‐3 weeks while on an on‐demand regimen, 47% of hematologists did not select "increasing frequency of bleeding episodes (once every week)" as a prompt to change regimens. 3 Educational Gap Data continued… Most hematologists do not include annualized bleed rates at each visit (61%) or clinical scales i.e. QOL, ADL assessment (59%) for assessing efficacy of on‐demand regimens for patients repeated bleeding episodes in 1 or more joints. Less than half of hematologists were very familiar with albumin‐fused recombinant clotting factors, PEGylated recombinant clotting factors, Fc fusion recombinant clotting factors, tissue factor pathway inhibitor (TFPI) antagonists, single chain recombinant FVIII, and gene therapy. When asked which approaches they would include to facilitate adherence with a 4‐year‐old patient with severe hemophilia, 32% of hematology nurses did not include "nursing education in the hemophilia clinic on infusion technique". Over three‐quarters of hematology nurses consider "patient/parental lack of adherence to prescribed prophylaxis regimens" to be a very significant barrier to the optimal management of patients with hemophilia. REFERENCES 1. Roberts HR, Key NS, Escobar MA. Chapter 124. Hemophilia A and Hemophilia B. In: Prchal JT, Kaushansky K, Lichtman MA, Kipps TJ, Seligsohn U, eds. Williams Hematology. 8th ed. New York: McGraw‐Hill; 2010. http://www.accessmedicine.com/content.aspx?aID=6117504. Accessed May 11, 2012. 2. White GC, Rosendaal F, Aledort LM, et al; on behalf of the Factor VIII and Factor IX Subcommittee. Definitions in hemophilia: Recommendation of the Scientific Subcommittee on factor VIII and factor IX of the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 2001;85:560. 3. Berntorp E. Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens. Haemophilia. 2009;15:1219‐1227. 4. Source: CE Outcomes Needs Assessment Data. Collected February 2013. 4 Cycle C: Areas of Interest Details The Biogen Grants Office is seeking proposals for Cycle C targeting the below 3 categories that are aimed at addressing the independently identified educational needs of the Hemophilia Healthcare Professional (HCP) community, referenced in the previous slides. 1. 2. 3. Medical Education: Physicians and other HCPs Medical Education: Nurses 2016 National/Regional Congresses 5 Cycle C: Please reference the following cycle code on your grant application/proposal: HEMCYCLEC-2015 Activity Start Date: Cycle C-2015 is for grants with start dates on or after Dec. 6, 2015 Medical Education: Physicians and other HCPs • The Biogen Grants Office is seeking proposals that are aimed at addressing the independently identified educational needs in the following areas: • The various treatment regimens (prophylaxis vs. on demand) and their correlation with clinical outcomes such as frequent bleeding episodes or annualized bleed rates and tools for measuring joint damage progression. • The importance of joint pain as a sign of progressive degenerative joint changes. • The latest trial data on emerging therapies. 6 Cycle C: Please reference the following cycle code on your grant application/proposal: HEMCYCLEC-2015 Activity Start Date: Cycle C-2015 is for grants with start dates on or after Dec. 6, 2015 Medical Education: Nurses • The Biogen Grants Office is seeking proposals that are aimed at addressing the independently identified educational needs in the following areas: • Tools and techniques to educate patients on the risks and benefits of hemophilia treatments. • Best practices for achieving or improving patient adherence to prescribed treatment regimens (coaching, peer‐to‐peer, mentoring). • Communication tactics in educating patients, caregivers and/or HCP peers on the basics of treatment; how they work in the body and ways to assess their effectiveness including monitoring options. 7 Cycle C: Please reference the following cycle code on your grant application/proposal: HEMCYCLEC-2015 Activity Start Date: Cycle C-2015 is for grants with start dates on or after Dec. 6, 2015 2016 National and Regional Congresses • We acknowledge the value physicians, nurses, and other healthcare professionals place on professional meetings, as they provide them with the opportunity to stay up to date on advancements in clinical practice, specifically within their specialty. Therefore, we are seeking requests to fund live activities (face‐to‐face meetings) at major national/regional congresses. 8 Special Note The Biogen Grants Office supports independently developed educational programming designed to address knowledge and performance practice gaps (levels 3‐64) of the healthcare professional community, ultimately directed at enhancing patient care and community health. Special consideration will be given to multi‐component initiatives that offer scientifically rigorous formats, unique delivery methods, and incorporates adult learning principles (for example, interactive interventions that reinforce the relevance of the presented content to real‐world practice through case scenarios). Consistent with Biogen policy and our commitment to conduct business ethically, all proposals for continuing medical education programs and initiatives must comply with ACCME criteria and Standards for Commercial Support™ as well as the AMA, PhRMA Code, FDA, and OIG guidance’s. In addition: • • • • • Proposals must be fair, balanced, and scientifically sound; Data must be objectively selected; Content must be independently developed; Biogen will not provide input relating to content, presenters, moderators, or program format; and Biogen will not support proposals that are linked to prescribing, purchasing, formulary status, or reimbursement activities. 9 Contact Information Questions? please contact the Biogen Grants Office: Phone: Email: 617-914-1299 [email protected] 10
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