Patient Reported Outcomes, PRO ---- 解读美国 FDA 的最新文件

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Patient Reported Outcomes, PRO ---- FDA . . 2006. 2 www. fda .gov/cder/ guidance /5460dft.pdf. 2009.12 . What Why How . This guidance describes how the Food and Drug Administration (FDA) reviews and - PowerPoint PPT Presentation

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  • Patient Reported Outcomes, PRO ---- FDA

  • 2006. 2 www.fda.gov/cder/guidance/5460dft.pdf

  • 2009.12

  • What Why How

  • This guidance describes how the Food andDrug 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) .

  • ObjectiveSubjectiveExercise test versus physical functioning, r = 0.40Wiklund I et al.Clin Cardiol 1991;14

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    FEV1HbA1cMRI

  • QOL

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

  • PRO

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  • PRO QOL HRQLPRO QOLHRQLQOL= QOLHRQL= HRQL , HRQL

  • PRO?

  • PROPROPROPROPRO

  • PROM.D. ? 24 010

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  • ________________________________________________________________ 24

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  • PROPROv. ii. iii.iv. PRO

  • I. PRO

  • (labeling claim) ?

    PRO

    (, ) ()

  • ADL\

    PRO PRO

  • PRO

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  • II.

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

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    :

    M.D. Anderson Symptom Inventory (MDASI-CORE)

    M.D. (MDASI-CORE)

    Part 1. How severe are your symptoms?

    People with cancer frequently have symptoms that are caused by their disease or by their treatment.

    We ask you to rate how severe the following symptoms have been in the last 24 hours. Please fill in the circle below from 0 (symptom has not been present) to 10 (the symptom was as bad as you can imagine it could be) for each item.

    24010

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

  • III.

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  • IV.

  • , PRO FDA (SAP): ? ?

  • , , ? PRO.

  • V.

  • PRO?? , PRO ?

  • PROHRQOL, HRQOL PRO PROPROFDAGuidance guidelines2009.12

  • This cartoon concludes what HRQOL stands for..It is actually not the objective exercise test data in an hospital environment that we measure but the everyday activities at home or at work subjectively perceived by the patient himself

    Efficacy is often difficult to measure in disorders for which the patients subjective experience of symptoms and therapy and their impact is critical.

    Assessment of outcome can be achieved in several ways, but most robust involves repeated application of a standardized measurement. Outcome can be defined as a change in this measurement parameterLocates outcome parameters within their primary setting. e.g. clinical or biological markers as tumor size are primarily located within the professional domain. However, parameters such as B/P may be measured by others (e.g. patients).

    Productivity not included as it is secondary to function

    Assessment of outcome can be achieved in several ways, but most robust involves repeated application of a standardized measurement. Outcome can be defined as a change in this measurement parameterLocates outcome parameters within their primary setting. e.g. clinical or biological markers as tumor size are primarily located within the professional domain. However, parameters such as B/P may be measured by others (e.g. patients).

    Productivity not included as it is secondary to function

    Pain intensity and pain relief are known only to the patient.Two treatments may result in same mortality experience but different PRO effectsWhisper effect PROs eliminate 3rd party interpretation of the patients experienceCROs often based on what the patient say

    Good PROs can match results obtained by most expert assessor

    Depends on biologicDepends on disease and manifestationsEvaluating adequacy of Qualitative research does not have rigid criteria for adequacy subjective judgment

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