4/1/2015
Biogen Global Medical Grants Office
Hemophilia: Areas of Interest (Cycle B)
April 1, 2015
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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
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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
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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:
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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.
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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.
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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.
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Educational Gap Data continued…
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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.
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Cycle B: Areas of Interest Details
The Biogen Grants Office is seeking proposals for Cycle B targeting the below 2
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.
Medical Education: Physicians and other HCPs
Medical Education: Nurses
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Cycle B: Please reference the following cycle code on your grant application/proposal: HEMCYCLEB-2015
Activity Start Date: Cycle B-2015 is for grants with start dates on or after Aug. 31, 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.
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The importance of joint pain as a sign of progressive degenerative joint changes.
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The latest trial data on emerging therapies.
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Cycle B: Please reference the following cycle code on your grant application/proposal: HEMCYCLEB-2015
Activity Start Date: Cycle B-2015 is for grants with start dates on or after Aug. 31, 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.
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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:
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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.
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Contact Information
Questions? please contact the Biogen Grants Office:
Phone:
Email:
617-914-1299
[email protected]
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