ARE WE USING THE RIGHT OUTCOME MEASURE TO ASCERTAIN PATIENT BENEFIT FROM DRUG THERAPY?  Professor Sam Salek Centre for Soc...
Background  <ul><li>Traditionally  –  measurement of the benefits of drug therapy using clinical outcomes such as changes ...
Definitions of Quality of Life <ul><li>The difference, at a particular moment in time, between the hopes and expectations ...
Definitions of HRQoL Quality of life in clinical medicine represents the functional effect of an illness and its consequen...
Outcomes <ul><li>Clinical </li></ul><ul><li>Humanistic </li></ul><ul><li>Economic </li></ul>Decision should not be made on...
Patient Outcomes Model (1) <ul><li>During course of disease and/or treatment </li></ul><ul><li>Itching </li></ul><ul><li>S...
Patient Outcomes Model (2) <ul><li>After treatment </li></ul><ul><li>Contend with physical, emotional and social problems ...
Linking Clinical & HRQoL Variables Measures of Patient Outcomes <ul><li>Biological/physiological </li></ul><ul><li>Symptom...
Linking Clinical and HRQoL Variables Source of Data    Overall QoL    General Health     Functional Status   ...
Biological/Physiological <ul><li>Diagnosis </li></ul><ul><li>Lab values </li></ul><ul><li>Physiological function (skin les...
Symptom Status <ul><li>Perception of an abnormal physical, emotional or cognitive state </li></ul>
Functional Status <ul><li>Physical, social, psychological functioning and behaviour </li></ul>
General Health Perceptions <ul><li>Evaluation of overall happiness </li></ul><ul><li>General sense of physical and mental ...
Example Overall QoL General Health Inability to walk stairs Inability to sleep Functional Status Hip pain Itching Symptom ...
Example Depression, anxiety Inability to handle stressful situations “ in general, how would you rate your health?” “ over...
Association of Clinical & HRQoL Variables Cystic Fibrosis 0.75 Patient-Reported MRC Dyspnea Scale Satisfaction HRQoL (QWB,...
Association of Clinical &  HRQoL Variables Acne <ul><li>QoL </li></ul><ul><li>Patient age -0.01 </li></ul><ul><li>Clinicia...
Association of Clinical & HRQoL Variables Atopic Dermatitis 0.65 0.82 0.72 0.70 0.75 0.80 0.70 0.67 Patient-Reported Sleep...
Why Care About Error? <ul><li>Too much of it renders any measurement useless </li></ul><ul><li>Comparing clinical and HRQo...
How to do it? <ul><li>Literature Review </li></ul><ul><ul><li>Reliability statistics to establish high, medium and low </l...
Guidelines for Instrument Reliability/Precision <ul><li>Data Statistic   Reliability </li></ul><ul><li>High    Mod  Low </...
Reliability of Common  Clinical Measures <ul><li>Classification of vital sign measurements </li></ul><ul><li>(Edmonds et a...
Reliability of Common  Clinical Measures <ul><li>Cardiovascular measurements  </li></ul><ul><li>(Gerin et al, J Psychosoma...
Reliability of Common  Clinical Measures <ul><li>Airway inflammation  </li></ul><ul><li>(Faul et al, Am J Resp Crit Care M...
Reliability of Common  Clinical Measures Oncology <ul><li>Time to tumour prog. </li></ul><ul><li>Survival </li></ul><ul><l...
Reliability of HRQoL Data  SF-36 (McHorney & Tarlov, Qual Life Res, 1995) <ul><li>Pain </li></ul><ul><li>Role – emotional ...
Reliability of HRQoL Data  NHP (McHorney & Tarlov, Qual Life Res, 1995) <ul><li>Sleep </li></ul><ul><li>Social isolation <...
Clinical Significance Problems and Solutions <ul><li>There are problems with assessing the clinical significance of QOL as...
Why is it Difficult to Define  “Clinical Significance” for QOL? <ul><li>Blood pressure analogy </li></ul><ul><ul><li>100 y...
Usefulness of QOL Measures for Clinicians <ul><li>Inform practitioners and patients decisions about alternative treatments...
Take Home Message (1) <ul><li>HRQoL data is a relevant outcome measure </li></ul><ul><li>Use of HRQoL in practice is is ju...
Take Home Message (2) <ul><li>Establishment of a dialogue </li></ul><ul><li>Involvement of patient in clinical decision-ma...
What is the Basic Problem with Quality of Life? How are you? I’m a little different and relatively young
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ARE WE USING THE RIGHT OUTCOME MEASURE TO ASCERTAIN PATIENT BENEFIT FROM DRUG THERAPY?

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PharmaCon2007 Conference Dubrovnik Croatia

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ARE WE USING THE RIGHT OUTCOME MEASURE TO ASCERTAIN PATIENT BENEFIT FROM DRUG THERAPY?

  1. 1. ARE WE USING THE RIGHT OUTCOME MEASURE TO ASCERTAIN PATIENT BENEFIT FROM DRUG THERAPY? Professor Sam Salek Centre for Socioeconomic Research Welsh School of Pharmacy Cardiff University
  2. 2. Background <ul><li>Traditionally – measurement of the benefits of drug therapy using clinical outcomes such as changes in disease severity and disease activity </li></ul><ul><li>Recently – a shift from clinician-centred to patient-centred approach to treatment decision making </li></ul><ul><li>growing recognition of the importance of HRQOL as a relevant patient-reported outcome measure </li></ul>
  3. 3. Definitions of Quality of Life <ul><li>The difference, at a particular moment in time, between the hopes and expectations of the individual and that individual’s present experiences. (Calman 1984) </li></ul>
  4. 4. Definitions of HRQoL Quality of life in clinical medicine represents the functional effect of an illness and its consequent therapy upon a patient, as perceived by the patient. (Schipper, et al 1996)
  5. 5. Outcomes <ul><li>Clinical </li></ul><ul><li>Humanistic </li></ul><ul><li>Economic </li></ul>Decision should not be made on two dimensional basis
  6. 6. Patient Outcomes Model (1) <ul><li>During course of disease and/or treatment </li></ul><ul><li>Itching </li></ul><ul><li>Sleep </li></ul><ul><li>Scaling </li></ul><ul><li>Physical activity limitations </li></ul><ul><li>Pain, fatigue, discomfort </li></ul><ul><li>Treatment side effects </li></ul><ul><li>Coping with mode, self-image and emotional changes </li></ul>
  7. 7. Patient Outcomes Model (2) <ul><li>After treatment </li></ul><ul><li>Contend with physical, emotional and social problems </li></ul><ul><li>Physical </li></ul><ul><li>Relapse of skin lesions </li></ul><ul><li>Restricted physical activity/sports </li></ul><ul><li>Return of certain symptoms </li></ul><ul><li>Possibility of skin cancer </li></ul><ul><li>Social </li></ul><ul><li>Employability/career prospect </li></ul><ul><li>Restriction in using public places </li></ul><ul><li>Stigma </li></ul><ul><li>Socialising and travel </li></ul><ul><li>Emotional </li></ul><ul><li>Fears about relapse </li></ul><ul><li>Self-image </li></ul><ul><li>Self-esteem </li></ul><ul><li>Adjustment to physical limitations </li></ul><ul><li>Fear of job discrimination and unemployment </li></ul><ul><li>Stress, mood change, depression, anxiety </li></ul>Consultations should address patient’s needs during and after treatment
  8. 8. Linking Clinical & HRQoL Variables Measures of Patient Outcomes <ul><li>Biological/physiological </li></ul><ul><li>Symptom status </li></ul><ul><li>Functional status </li></ul><ul><li>General health perception </li></ul><ul><li>Overall quality of life </li></ul>
  9. 9. Linking Clinical and HRQoL Variables Source of Data    Overall QoL    General Health     Functional Status     Symptom Status  Biological/Physiological Patient Carer Physiological Clinician
  10. 10. Biological/Physiological <ul><li>Diagnosis </li></ul><ul><li>Lab values </li></ul><ul><li>Physiological function (skin lesions, mast cells, immunological response, growth hormone secretion) (other examples, pulmonary function tests etc) </li></ul><ul><li>Physical examination findings (itching, sleep disturbance etc) </li></ul><ul><li>Radiographic and imaging data </li></ul>
  11. 11. Symptom Status <ul><li>Perception of an abnormal physical, emotional or cognitive state </li></ul>
  12. 12. Functional Status <ul><li>Physical, social, psychological functioning and behaviour </li></ul>
  13. 13. General Health Perceptions <ul><li>Evaluation of overall happiness </li></ul><ul><li>General sense of physical and mental well-being </li></ul><ul><li>Perception of prognosis and comparison to others </li></ul>
  14. 14. Example Overall QoL General Health Inability to walk stairs Inability to sleep Functional Status Hip pain Itching Symptom Status Biological/Physiological
  15. 15. Example Depression, anxiety Inability to handle stressful situations “ in general, how would you rate your health?” “ overall, how would you rate your quality of life?” Overall QoL General Health Functional Status Symptom Status Biological/Physiological
  16. 16. Association of Clinical & HRQoL Variables Cystic Fibrosis 0.75 Patient-Reported MRC Dyspnea Scale Satisfaction HRQoL (QWB, SIP) Carer Proxy family carer 0.57 Clinician-Reported Maximal capacity- Exercise QoL 0.40 0.33 Satisfaction MRC Carer EX FEV 1 0.84 SaO 2 CT Physiological HR CT SaO 2 FEV 1
  17. 17. Association of Clinical & HRQoL Variables Acne <ul><li>QoL </li></ul><ul><li>Patient age -0.01 </li></ul><ul><li>Clinician-Reported </li></ul><ul><li>Acne Clinical Severity -0.06 </li></ul><ul><li>Patient-Reported </li></ul><ul><li>General Health Perception 0.40 </li></ul>
  18. 18. Association of Clinical & HRQoL Variables Atopic Dermatitis 0.65 0.82 0.72 0.70 0.75 0.80 0.70 0.67 Patient-Reported Sleep Itch Tolerability Efficacy EDI 0.38 0.19 0.57 0.67 UK-SIP 0.18 0.01 0.50 0.65 Clinician-Reported Disease activity Extent of disease Tolerability Efficacy
  19. 19. Why Care About Error? <ul><li>Too much of it renders any measurement useless </li></ul><ul><li>Comparing clinical and HRQoL measures on this basis offers a common ground </li></ul><ul><ul><li>Places HRQoL error in a familiar context </li></ul></ul><ul><ul><li>Allows one to evaluate measurement precision by degree of error </li></ul></ul><ul><li>Exposes useful generalisations and analogues </li></ul><ul><ul><li>Same patient providing data over time in comparison to going to the same lab for repeat tests </li></ul></ul><ul><li>May encourage more general use of HRQoL in practice </li></ul>
  20. 20. How to do it? <ul><li>Literature Review </li></ul><ul><ul><li>Reliability statistics to establish high, medium and low </li></ul></ul><ul><ul><li>Reliability reports in clinical measurement studies </li></ul></ul><ul><ul><li>Value of practice-based HRQoL assessment </li></ul></ul><ul><li>Classification of the obtained range of clinical measurements compared side by side with HRQoL measurements </li></ul>
  21. 21. Guidelines for Instrument Reliability/Precision <ul><li>Data Statistic Reliability </li></ul><ul><li>High Mod Low </li></ul><ul><li>Nominal Kappa >.74 .40-.74 <.40 </li></ul><ul><li>Ordinal Weighted K >.74 .40-.74 <.40 </li></ul><ul><li>Interval/Ratio ICC, r ICC </li></ul><ul><li>Pearson r >.90 .70-.90 <.70 </li></ul><ul><li>Alpha r  </li></ul><ul><li>SEM <1 sem </li></ul>
  22. 22. Reliability of Common Clinical Measures <ul><li>Classification of vital sign measurements </li></ul><ul><li>(Edmonds et al, Ann Emerg Med, 2002) </li></ul><ul><li>Dias hypertension </li></ul><ul><li>Sys hypertension </li></ul><ul><li>Bradycardia </li></ul><ul><li>Sys hypotention </li></ul><ul><li>Tachypnea </li></ul><ul><li>Tachycardia </li></ul>Low Moderate High
  23. 23. Reliability of Common Clinical Measures <ul><li>Cardiovascular measurements </li></ul><ul><li>(Gerin et al, J Psychosomatic Res, 1998) </li></ul><ul><li>Heart rate </li></ul><ul><li>Distolic BP </li></ul><ul><li>Systolic BP </li></ul>Low Moderate High
  24. 24. Reliability of Common Clinical Measures <ul><li>Airway inflammation </li></ul><ul><li>(Faul et al, Am J Resp Crit Care Med, 1992) </li></ul><ul><li>Bronchodilator response </li></ul><ul><li>FEV 25-75% </li></ul><ul><li>FIV 1 </li></ul>Low Moderate High
  25. 25. Reliability of Common Clinical Measures Oncology <ul><li>Time to tumour prog. </li></ul><ul><li>Survival </li></ul><ul><li>Tumour measurement over time (esp. inter-observer) </li></ul><ul><li>Tumour CT/MR </li></ul><ul><li>Haemoglobin </li></ul><ul><li>Tumour palpation </li></ul><ul><li>FACT-G Scale </li></ul><ul><li>FACT – Fatigue </li></ul>Low Moderate High
  26. 26. Reliability of HRQoL Data SF-36 (McHorney & Tarlov, Qual Life Res, 1995) <ul><li>Pain </li></ul><ul><li>Role – emotional </li></ul><ul><li>Role - emotional </li></ul><ul><li>Role – physical </li></ul><ul><li>Role – physical </li></ul><ul><li>Physical function </li></ul><ul><li>Physical function </li></ul>Low Moderate High
  27. 27. Reliability of HRQoL Data NHP (McHorney & Tarlov, Qual Life Res, 1995) <ul><li>Sleep </li></ul><ul><li>Social isolation </li></ul><ul><li>Energy </li></ul><ul><li>Emotional reactions </li></ul><ul><li>Physical mobility </li></ul><ul><li>Pain </li></ul>Low Moderate High
  28. 28. Clinical Significance Problems and Solutions <ul><li>There are problems with assessing the clinical significance of QOL as indicators of clinical efficacy in clinical trials </li></ul><ul><li>There are scientifically sound solutions to these problems. The problems have been disseminated widely and consistently. The solutions have not. </li></ul>
  29. 29. Why is it Difficult to Define “Clinical Significance” for QOL? <ul><li>Blood pressure analogy </li></ul><ul><ul><li>100 years ago, clinical significance of BP scores was unknown </li></ul></ul><ul><ul><li>Massage therapy was the gold standard </li></ul></ul><ul><ul><li>Present guidelines for BP clinical significance today? </li></ul></ul><ul><li>Pain analogy </li></ul><ul><ul><li>25 years ago physicians were the sole raters of patient pain </li></ul></ul><ul><ul><li>JCAHO 2000 guideline: every patient’s pain to be assessed upon intake on a 0-10 scale </li></ul></ul><ul><li>Time and experience and shift from “med-centrism” alleviates novelty and scepticism </li></ul>
  30. 30. Usefulness of QOL Measures for Clinicians <ul><li>Inform practitioners and patients decisions about alternative treatments </li></ul><ul><li>Efficient way of gathering data about functioning and well-being </li></ul><ul><li>Alert clinicians to problems that require intervention </li></ul><ul><li>Ultimately, improve patient outcomes </li></ul>
  31. 31. Take Home Message (1) <ul><li>HRQoL data is a relevant outcome measure </li></ul><ul><li>Use of HRQoL in practice is is justifiable based on patient benefit </li></ul><ul><li>More work is needed to: </li></ul><ul><ul><li>Further refine measures </li></ul></ul><ul><ul><li>Provide data on “meaningfulness” of changes in scores </li></ul></ul><ul><ul><li>Provide data on “minimally important difference” </li></ul></ul><ul><ul><li>Educate clinicians on appropriate use of HRQoL Data </li></ul></ul><ul><li>HRQoL is the right outcome measure to ascertain patient benefit for drug therapy. </li></ul>
  32. 32. Take Home Message (2) <ul><li>Establishment of a dialogue </li></ul><ul><li>Involvement of patient in clinical decision-making </li></ul><ul><li>Improvement in concordance, adherence/compliance </li></ul><ul><li>Optimisation of outcomes </li></ul>
  33. 33. What is the Basic Problem with Quality of Life? How are you? I’m a little different and relatively young

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