Patient Reported Outcomes, PRO ---- ????FDA?????

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Patient Reported Outcomes, PRO ----
????FDA?????

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• ??????????

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2006. 2 ??www.fda.gov/cder/guidance/5460dft.pdf
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2009.12 ??
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• What?
• Why?
• How?

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• This guidance describes how the Food and
• Drug Administration (FDA) reviews and
• evaluates existing, modified, or newly created
• patient-reported outcome (PRO) instruments
• used to support claims in approved medical
• product labeling.
• ???????????????????
• ?????????????????????
• ????????????????(PRO) ??
• ??.

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Objective
Subjective
Exercise test versus physical functioning, r
0.40
Wiklund I et al. Clin Cardiol 199114
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??? PRO?

• PRO ??????? ????????????????????????????????????
  (???? ??????)
• PRO ?? ???????
• PRO ?? ????????????????????? (? ???????????)
• PRO ?? ???????????????PRO ????

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• PRO ????????, ????????,??????????????? ?
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PRO ? QOL ? HRQL

• PRO ???QOL?HRQL??????
• QOL ???????????,??????????
• QOL?????????????
• HRQL ???????????????????
• HRQL????????????, ??????????????????
• ?????????????, HRQL ???????
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????? PRO?

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PRO?????

• PRO??????,??????
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PRO??????M.D.??????? ???? ?????????
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• ??????
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• ?PRO???????
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PRO???? ??????

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PRO ????

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I. ??????

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??????? (labeling claim) ?

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• PRO?????????????
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PRO ????????
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PRO ??
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??ADL
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PRO ????????
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II. ???????????

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????

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????

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• 1. ??????????????
• 2. ?????????????
• 3. ???????????
• 4. ?????? (cognitive interviewing)
  ???????????????????

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?????
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????????
Long List of Items Item Source Final Decision
How much difficulty do you have swallowing? Qualitative interviews\ Focus groups Dropped high frequency, low severity, too vague in meaning, didnt discriminate between severity levels
How much difficulty do you have swallowing liquids? Qualitative interviews\ Focus groups Retained high frequency of report, high importance to patients, worked well in cognitive debriefing and discriminated between severity levels
How much difficulty do you have swallowing soft foods? Qualitative interviews\ Focus groups Dropped highly correlated with swallowing solid foods and will be covered by this item
How much difficulty do you have swallowing solid foods? Existing swallowing diary (SWALLOW) Retained worked well in cognitive debriefing and discriminated between severity levels
How often do you need to spit? Qualitative interviews\ Focus groups Dropped highly correlated with swallowing salivarelated to other conditions.
How much difficulty do you have swallowing your saliva? Qualitative interviews\ Focus groups Retained worked well in cognitive debriefing high importance to patients
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III. ???????????????

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IV. ??????????

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?????

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????

• ?????, PRO???
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• FDA ??????????(SAP)???
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?????

• ?????, ???????????, ??????
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• ?PRO?????????????????????????.

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V. ????

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???PRO????

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• ??????????, ????????????????PRO ???????

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• ???????,PRO??????,???HRQOL, ????HRQOL
• ?? PRO ????????????????,????????
• PRO??????????,???????
• ??PRO?????????????
• FDA?Guidance ??,??? guidelines
• 2009.12????,?????????

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