Priorisation of rare diseases for public health surveillance

1
Priorisation of rare diseases for public health
surveillance

• What means public health surveillance ?
• WHY do we need to establish priority
• What priorisation of rare diseases for health
  surveillance is not
• What is the best approach to define priority in
  Rare diseases

2
Priorisation of rare diseases for public health
surveillance

• What means public health surveillance ?
• To produce indicators

3
Priorisation of rare diseases for public health
surveillance

• What means public health surveillance ?
• To produce reliable indicators

4
Priorisation of rare diseases for public health
surveillance

• What means public health surveillance ?
• To produce reliable indicators
• Various epidemiological tools
• Cross-sectional studies
• Cohort / retrospective prospective
• Register

5
Priorisation of rare diseases for public health
surveillance

• WHY do we need to establish priority among RD ?
• A major reason
• numerous RD
• reliable indicators are defined for precise
  disease
• limited resources
• epidemiological techniques are quite costly
  register
• Additional reasons
• A rationale approach of the choices is warranted
• The only way to be sure to monitor each disease
  which really need to be monitored

6
Priorisation of rare diseases for public health
surveillance

• What priorisation of rare diseases for public
  health surveillance is not !
• Priority for care
• Priority for basic research
• A moral ranking of the diseases

7
Priorisation of rare diseases for public health
surveillance

• HOW ?
• Need to have tools criteria of priorisation
• Need to document each diseases
• Need to rank the rare diseases for public health
  surveillance

8
Priorisation of rare diseases for public health
surveillance

• Need to have tools criteria of priorisation
• A Delphi method in France
• A working group 10 including 3 representatives
  of patients association
• A panel of 110 experts 90 medical experts or
  responsible of network in RD, 15 insurance or
  institution, 5 industrials

9
Priorisation of rare diseases for public health
surveillance

• A Delphi method in France
• round 1 July sept
• round 2 mailing in feb see questionnaire
• Expected results
• multiple criteria approach
• to define relative weight between criteria

10
Priorisation

• Perspectives A review of a limited number of
  diseases (300 ?) since the end of 2006
• Next Do the criteria and the priority could be
  endorsed by other country ? Do we need a global
  EU approach ?