Presentation on theme: "Everything You Ever Wanted to Know About PROs (and Perhaps Even More)"— Presentation transcript:
1 Everything You Ever Wanted to Know About PROs (and Perhaps Even More) Amylou C. Dueck, PhDMayo Clinic Arizona &NCCTGCRA Workshop for Cancer Control StudiesNovember 14, 2009
2 Outline What are PROs? What are the different types of PROs? Why do we measure PROs?How do we measure PROs?Why do we measure PROs the way that we do?Why are there different instruments measuring the same thing?How are these measures created?What can PROs tell us?Why is it so important that they be administered as specified in protocols?Why is the CRA role so important?Are there respondent burden issues?
3 What are PROs? PRO = Patient-reported outcome Definition from FDA Draft Guidance:“a measurement of any aspect of a patient’s health status that comes directly from the patient (i.e., without the interpretation of the patient’s responses by a physician or anyone else).”Or Google “FDA draft guidance patient reported-outcomes”Definition from Wikipedia:“PRO is an umbrella term that covers a whole range of potential types of measurement but is used specifically to refer to questionnaires completed by the patient.”
4 What are the different types of PROs? Health-related quality of life (HRQOL)Quality of life (QOL)SymptomsFunctioningSatisfactionDecision-making / preferencesTreatment complianceHealth utilitiesOthers???
5 Why do we measure PROs?PROs are key to providing a better understanding of treatment outcomes, beyond the data obtained from clinical assessmentsPROs have become to gold standard for assessing subjective experiences of patientsTo get the patient’s perspective!
6 How do we measure PROs? INSTRUMENTS European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)Disease modules such as the Breast (BR23)Brief Pain Inventory (BPI)Brief Fatigue Inventory (BFI)MD Anderson Symptom Inventory (MDASI)Functional Assessment of Cancer Therapy General (FACT-G)Disease specific (FACT-B), disease/symptom specific (FBSI), symptom specific (FACT-An), treatment specific (FACT-Taxane)
7 How do we measure PROs? INSTRUMENT REPOSITORIES Databases of instrumentsQOLIDOLGAAustralian Centre on Quality of LifeAmerican Thoracic Society Quality of Life Resource
13 How do we measure PROs? MODES OF ADMINISTRATION PaperIn clinicTake-homeInterview (in person)Interview (telephone)ePROs (electronic data capture of PROs)Digital penWireless tabletSmart phoneTelephone = Interactive Voice Response System (IVRS)Web
18 Why do we measure PROs the way that we do? Much work goes into instrument developmentNeed to make sure that the questionnaire is measuring what you think it is measuringNeed to make sure that patients are interpreting questions as intendedNeed to make sure that the instrument is sensitive to change (but also produces similar results if nothing has changed)Need to know the size of a clinically meaningful change or difference
19 Why are there different instruments measuring the same thing? Independent groups developed instruments to fit their own needsInstruments vary in level of detail and intended population
20 How are these measures created? May involve some or all of these steps:Conceptual frameworkItem generation (literature, expert opinion, patient focus groups, online chat rooms/blogs, etc.)Cognitive interviewsFeasibility testingValidation study
21 Median Survival (Months) Median (95% CI) What can PROs tell us?Single-item overall QOL at baseline is prognostic of survival in cancer patients! Even after adjusting for performance status!Median Survival (Months) Median (95% CI)Log-rank P-valueQOL CD9.3 (8.1, 10.6)0.0001QOL nCD16.8 (16.1, 17.4)nCDCDTan AD, Novotny PJ, et al. A patient-level meta-analytic investigation of the prognostic significance of baseline quality of life (QOL) for overall survival (OS) among 3,704 patients participating in 24 North Central Cancer Treatment Group (NCCTG) and Mayo Clinic Cancer Center (MC) oncology clinical trials. J Clin Oncol 26: 2008 (May 20 suppl; abstr 9515), ASCO 2008.
22 So is single-item fatigue So is single-item fatigue! Even after adjusting for overall QOL and performance status!Sloan JA, Liu H, et al. A patient-level pooled analysis of the prognostic significance of baseline fatigue for overall survival (OS) among 3,915 patients participating in 43 North Central Cancer Treatment Group (NCCTG) and Mayo Clinic Cancer Center (MC) oncology clinical trials. J Clin Oncol 27:15s, 2009 (suppl; abstr 9599), ASCO 2009.
23 Some things are just better measured by the patient! Hot flash studies (women and men)“Aren’t hot flashes better measured by a device which measures skin conductance? How do you know that the patient is really having a hot flash?”And in response, the famous words of one NCCTG investigator: “I wouldn’t want to be the one to tell a woman that she’s not having a hot flash.”
24 Black Cohosh (n=58)Placebo (n=420)Soy (n=78)Vitamin E (n=53)Clonidine (n=75)Fluoxetine (n=36)Ven (vs MPA) (n=94)Venlafaxine (n=48)MPA 400 mg (n=94)Megestrol (n=74)MPA 500 mg X 3(n=7)Loprinzi CL, Barton DL, et al. Mayo Clinic and North Central Cancer Treatment Group hot flash studies: a 20-year experience. Menopause 2008; 15(4):
25 Why is it so important that they be administered as specified in protocols? May see things like:Administer baseline patient assessment prior to notifying patient of his/her randomization assignmentAdminister onstudy patient assessment prior to discussing outcome of disease assessmentAdminister in a private room or a quite area in a waiting room with adequate privacyWhy?Avoid biasIncrease compliance / patient willingnessConsistency across sites, patients, visits
26 Why is the CRA role so important? I wouldn’t have any data without CRAs!Data quality and quantity is directly impacted by the CRAFollow protocolAdministration guidelines and test scheduleCheck duplication quality of questionnairesMissing pages?Professional-looking copies => better patient complianceAnswer patient questionsEven with ePROsAdministrationPatient questions
27 Are there respondent burden issues? YES!!!We’ve all filled out questionnaires from time to time – how long does the survey have to be for you to say “FORGET IT!”???For cancer patients: ≤50 items