# Presentation: 869897

## FDA FVIII vCJD Risk Assessment:A global perspective

**FDA FVIII vCJD Risk Assessment:****A global perspective**

- 15 December 2006 – FDA TSEAC Meeting – Washington, DC

- Mark W. SkinnerWFH President

## 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

- 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

- WFH Guidance on Assessing Product Risk

## 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

- 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

- Risk Communication Core Values

## Potential Global Implications

- Source: WFH Global Survey 2005; Random sample of 45 countries

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

## 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 <30 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, 1992–2001: a prospective cohort study. Journal of Thrombosis and Haemostasis 4 (3), 510-516. doi: 10.1111/j.1538-7836.2006.01808.x

## 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 1:4,225 to 1.8:1,000,000
- FDA 2006:  U.S. plasma-derived concentrates between 1:105,000 & 1:9,400,000
- FDA assessment is significantly lower than the U.K. assessment and thus provides further reassurance for patients & clinicians

## 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

- 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

- Selection of Treatment Product