FDA FVIII vCJD Risk Assessment: A global perspective

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FDA FVIII vCJD Risk AssessmentA global
perspective
Mark W. SkinnerWFH President

• 15 December 2006 FDA TSEAC Meeting
  Washington, DC

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WFH Guidance on Assessing Product Risk

• It is important to avoid the complacency which
  characterized previous blood borne epidemics in
  people with hemophilia, while retaining a sense
  of proportion to this particular issue, and
  maintain a constant watch on the balance between
  safety and supply
• Structured risk-management processes require a
  strategy for continuous learning and communication

A. Farrugia vCJD and Hemophilia Further
Guidance on Assessing the Risk of Plasma-Derived
Products for Treating Hemophilia WFH 2004
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Risk Communication Core Values

• Follow a precautionary approach when scientific
  knowledge is incomplete
• Patients have a right to be consulted and
  informed
• Timely communication with openness and
  transparency
• Clear and comprehensible explanations essential
• Appropriate to acknowledge areas of uncertainty
• Avoid unintended health consequences
• e.g.) patient stigmatization, denial of access to
  care

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Potential Global Implications

• 75 of the patients in the world receive little
  or no care
• Outside of the highly developed nations,
  treatment products are predominantly
  plasma-derived

Source WFH Global Survey 2005 Random sample of
45 countries
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Placing Risk in Context

• Mortality and Causes of Death in PWH
• Before the introduction of clotting factor
  preparations, the mean life expectancy of
  patients with hemophilia was lt30 years, and
  patients mostly died of intracranial or other
  hemorrhages
• In patients with severe hemophilia not infected
  with viruses, mortality is still 40 higher when
  compared with the general population
• 27 (13/49) of the deaths in the cohort of
  patients not infected with HCV or HIV were
  because of hemorrhage

PLUG, I., et. al., Mortality and causes of death
in patients with hemophilia, 19922001 a
prospective cohort study. Journal of Thrombosis
and Haemostasis 4 (3), 510-516. doi
10.1111/j.1538-7836.2006.01808.x
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Quantifying the Risk in Global Context

• FDA risk assessment builds on global knowledge
  and understanding
• U.K. 2004 U.K. plasma-derived concentrates 1
  additional risk beyond general population risk of
  14,225 to 1.81,000,000
• FDA 2006 U.S. plasma-derived concentrates
  between 1105,000 19,400,000
• FDA assessment is significantly lower than the
  U.K. assessment and thus provides further
  reassurance for patients clinicians

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Selection of Treatment Product

• The FDA assessment adds to our understanding and
  provides reassurance for both clinicians and
  patients when making treatment product selection
  decisions
• The WFH continues to view both recombinant and
  plasma-derived products as important treatment
  options for the global bleeding disorders
  community
• Potential remains for adverse events including
  inhibitor development or unknown pathogen risk,
  thus continuous learning and communication are
  required