Patient reported outcome measures in clinical practice

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1 Patient reported outcome measures in clinical practice Brought to you by Pfizer Limited Date of preparation: August 2016; Job code: PP-GEP-GBR

2 Objective To provide healthcare professionals (HCPs) with an introduction to patient reported outcomes measures (PROMs), and how they are important in developing a patientcentred approach to care 2

3 Learning outcomes Upon completion of this module, the participant will be able to: 1. Demonstrate an increase in, or affirmation of, their knowledge of patient reported outcome (PRO) research in terms of: Understanding the meaning and purpose of PRO research Recognising the different types of measures used in PRO research 2. Identify and utilise PROMs for the needs of the patient and for their clinical setting 3. Understand how to synthesise findings from PRO research studies 3

4 Content overview The principles of PRO research Self-assessment quiz PROs The applications of PROMs in clinical practice Conclusions PRO clinical case study 4

5 The principles of PRO research 5

6 What is PRO research? PRO research can be defined as: Any report on the status of a patient s health condition that comes directly from the patient, without interpretation of the response by a clinician or anybody else 1,2 PRO research takes into consideration, and stresses the importance of, research that is informed by the perspectives, interests and values of the patient, 2 and are used to evaluate the effect of treatment on how a patient feels or functions 3 PROs are used when measuring a concept that is best known by the patient or measured from the perspective of the patient 4 Other than reporting of outcomes by the patients themselves, a proxy-reported outcome is a measurement based on a report by someone other than the patient, reporting as if he or she is the patient 5 An observer (e.g. caregiver) may give a measure based on their opinion, referred to as an observerreported outcome 5 PROMs are typically based on questionnaires, referred to as PRO instruments, that are based on health and quality of life 6 PROs are often used to evaluate change in health status over time with PRO instruments administered at baseline and during/or after treatment, or when a certain milestone has been reached 7 1. U.S. Department of Health and Human Services FDA Center for Drug Evaluation and Research. Health Qual Life Outcomes 2006;4:79; 2. Snyder CF, et al. Med Care 2013;51:S73 9; 3. Rothman M, et al. Value Health 2009;12: ; 4. Coons SJ, et al. Value Health 2009;12:419 29; 5. Matza LS, et al. Value Health 2013;16:461 79; 6. Spiegal BM. 6 J Neurogastroenterol Motil 2013;19:137 48; 7. Nixon A, et al. Health Qual Life Outcomes 2016;14:25.

What instruments are used to assess PROs?

and social functioning, and are generally classed as either generic or disease-specific 1 Generic

angina Overall feelings about a condition or treatment e.g. worry about the condition deteriorating General disease concepts e.

7 What instruments are used to assess PROs? PRO instruments collect data across several areas of health, including physical, psychological and social functioning, and are generally classed as either generic or disease-specific 1 Generic PRO instruments 2 Disease-specific PRO instruments 2 Overall state of the condition e.g. angina Overall feelings about a condition or treatment e.g. worry about the condition deteriorating General disease concepts e.g. physical functioning Specific disease concepts e.g. frequency/severity of symptoms Treatment side effects e.g. severity of adverse events 1. Spiegal BM. J Neurogastroenterol Motil 2013;19:137 48; 2. U.S. Department of Health and Human Services FDA Center for Drug Evaluation and Research. Health Qual Life Outcomes 2006;4:79. 7

8 Advantages and disadvantages of PRO instruments Generic PRO instruments 1 Advantages Suitable for the general public and for comparisons across populations Suitable for comparisons with disparate conditions/disease groups Captures commonly experienced health domains Disadvantages Content may be redundant for certain conditions/illnesses Not sensitive to detecting disease-specific illnesses Disease-specific PRO instruments 1 Advantages Specific for a given group (disease/population) Sensitive enough to detect clinically significant changes Captures clinically-relevant information that is applicable to a specific target group Disadvantages Cannot compare with the general population 1. Snyder CF, et al. Qual Life Res 2012;21:

9 Examples of PRO instruments Physical function Role physical Bodily pain General health Generic PRO instruments measure health across several areas, examples described below are the short-form health questionnaire (SF-36) and the EuroQOL five dimensions questionnaire (EQ-5D) 1,2 A large number of disease-specific PRO instruments have been developed, for example over 110 PRO instruments have been developed in gastroenterology alone 1 SF-36 Includes 36 items that are organised into eight discrete domains 1,3 Physical Mental Mental health Role emotional Social function Vitality EQ-5D Measures health-related quality of life (HRQoL) in five distinct domains 2 Anxiety /depression Pain/discomfort Mobility EQ-5D domains Self-care Usual activities 1. Spiegal BM. J Neurogastroenterol Motil 2013;19:137 48; 2. Brettschneider C, et al. Health and Quality of Life Outcomes 2013;11:3; 3. Chiu Y-W, et al. Clin J Am 9 Soc Nephrol 2009;4:

Administering PRO instruments Responses to PRO instruments can be gathered through a number of different channels 1,2,3 Telephone Paper Personal

Impersonal if using automated systems Efficient Interview Resource intensive May encourage patients to report sensitive information Personal Can

real-time data interpretation and allows for inperson explanation and timely interventions Mail Larger recruitment catchment area, less Expensive

intensive Requires trained personnel and software High technical costs and initially expensive 1. Liu H, et al.

10 Administering PRO instruments Responses to PRO instruments can be gathered through a number of different channels 1,2,3 Telephone Paper Personal Low technology and Convenient cost Only requires Not for the illiterate auditory Can lose data proficiency Resource Computer/Internet intensive Impersonal if using automated systems Efficient Interview Resource intensive May encourage patients to report sensitive information Personal Can be linked to electronic health records Doesn t require Immediate scoring and presentation is literacy possible Can be more in-depth May allow for real-time data interpretation and allows for inperson explanation and timely interventions Mail Larger recruitment catchment area, less Expensive restricted by geographies and boundaries Invasive Low tech and cost Smaller investment in staff time and cost Can lose data savings Resource intensive Requires trained personnel and software High technical costs and initially expensive 1. Liu H, et al. J Clin Epidemiol 2010;63: ; 2. Snyder CF, et al. Qual Life Res 2012;21: ; 3. Coons SJ, et al. May require training of patients Value Health 2009;12: Data privacy is an issue 10

order to reach a common ground for integrated management 1 PROMs can be used to gauge patient opinion on their care and improve clinical practice 2 PROMs can be used to quantify

in measuring the effects of a medical intervention 4 PROs are also important from an pharmacoeconomic perspective and are used when calculating quality-adjusted life years 5 Impro ve

11 Why do we need PRO studies? Patient-centred care is an emerging concept in healthcare and can be defined as: Understanding of the patient s thoughts, feelings, and expectations in the context of their illnesses in order to reach a common ground for integrated management 1 PROMs can be used to gauge patient opinion on their care and improve clinical practice 2 PROMs can be used to quantify consequences of interventions and quality of life following an intervention 3 Multiple stakeholders including, patients, payors, government bodies and the general public are interested in measuring the effects of a medical intervention 4 PROs are also important from an pharmacoeconomic perspective and are used when calculating quality-adjusted life years 5 Impro ve care Gather data from PRO instru ments Feedb ack results Analys is of PRO data 1. Lam CLK, et al. Front Med (Lausanne) 2014;1:29; 2. Kotronoulas G, et al. J Clin Oncol 2014;32: ; 3. Weldring T and Smith SM. Health Services Insights 2013;6:61 8; 4. Banerjee AK, et al. Drug Safety 2013;36: ; 5. Appleby J, et al. J R Soc Med 2013;106:

12 The applications of PROMs in clinical practice 12

13 How are PROMs used in clinical practice? PRO research generates measures, referred to as PROMS, that can be used to inform clinical practice 1 A one-time assessment may help to identify problems that would otherwise have gone unnoticed 1 Multiple assessments can track patient outcomes over time and may help to modify treatments, as required 1 As a screening and treatment monitoring tool Linking to disease registries Part of a patient-centred approach to care As a treatment decision aid Linking PROMs to disease registries offers a cost-effective way to gather real-world data on disease progression and treatment outcomes 2 Helps to facilitate discussions between patients and clinicians regarding patient issues, including concerns and priorities for care 1 Helps patients understand the treatment options that are available and assists in the selection of a treatment that is most suitable to the individual patient 1 1. International Society for Quality of Life Research. User s Guide to Implementing Patient-Reported Outcomes Assessment in Clinical Practice. Can be accessed from: 2. Ashley L, et al. J Med Internet Res 2013;15:e

14 Using PROMs as a treatment monitoring tool Linking PROMs to treatment outcomes provides an opportunity to monitor quality of life while undergoing treatment 1 Example: Monitoring treatment and HRQoL using PRO instruments in patients with metastatic breast, genitourinary, gynaecologic, or lung cancer Objectives Patients Assessments Use PRO instruments to monitor symptoms and HRQoL during cancer treatment Patients were randomised to report symptoms via an electronic device or means at the discretion of clinicians HRQoL was evaluated using the EQ-5D PRO instrument at 6 months Outcomes Patients who reported symptoms via an electronic device improved HRQoL compared with means at the clinicians discretion 1. Basch E, et al. J Clin Oncol 2015; Dec 7. pii: JCO [Epub ahead of print]. 14

15 Linking PROMs to disease registries Linking PROMs to disease registries allows the practitioner to gather real-world data in real time 1 Example The electronic Patient-reported Outcomes from Cancer Survivors (epocs) system Objectives Link PROMs to disease registries for recently diagnosed patients with breast, colorectal, and prostate cancer Assessments Six different PRO instruments were administered at 6, 9 and 15 months, post diagnosis Participation Patient participation was 55.21% (636/1152) overall, although this varied by mode of approach Participation was considerably higher among patients approached face-to-face (61.4%, 490/798) compared with telephone (48.8%, 21/43) or by letter (41.0%, 125/305) Outcomes The PROMs were successfully linked with cancer registry data for 100% of patients (N=636) 1. Ashley L, et al. J Med Internet Res 2013;15:e

16 Implementing PROMs into clinical practice Implementing PROMs into clinical practice typically involves the consideration of a number of factors 1 1. Identifying the goals for collecting PROMs 2. Selecting patients, settings and timings of assessments 3. Determining which PRO instrument to use 4. Mode for administration and scoring PROMs 5. Reporting results 6. Interpreting scores 7. Responding to issues identified by the PROMs 8. Evaluating the impact that PROMs may have on clinical practice 1. Snyder CF, et al. Qual Life Res 2012;21:

Challenges associated with PRO research There are some difficulties associated with developing PRO research 1,2 Poor literacy and understanding of PRO instruments 1 Challenge:

Additional costs 2 Challenge: Additional costs required to implement PRO research, such as training, administration and data interpretation Validation: It would be expected that

17 Challenges associated with PRO research There are some difficulties associated with developing PRO research 1,2 Poor literacy and understanding of PRO instruments 1 Challenge: Patients with low literacy may fail to understand some questions Solution: Format the PRO instrument so that it is easy to follow and use illustrations where appropriate Additional costs 2 Challenge: Additional costs required to implement PRO research, such as training, administration and data interpretation Validation: It would be expected that the additional costs are offset by the benefits to the patient and the practitioner 1. Jahagirdar D, et al. BMC Health Serv Res 2012;12:431; 2. Antunes B, et al. Palliative Med 2014;28:

18 PRO clinical case study: Northumbria NHS Healthcare Foundation Trust: The use of PROMs to assess the impact of knee replacement surgery 18

19 Case study background The programme An NHS programme that began in April 2009 in the United Kingdom (UK) that used PRO instruments to evaluate health before and after elective knee replacement, hip replacement, varicose vein and groin hernia surgeries 1 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: PROMs-benefits-case-study/pdf/promscasestudy.pdf. Copyright 2016, Re-used with the permission of the Health and Social Care Information Centre. All rights reserved 19

20 Implementing PROMs in clinical research The objectives of the study To use PROMs to inform service changes and enable providers and other stakeholders to improve quality of care To empower patients and clinicians to make an informed choice on the course of treatment Selecting patients The programme selected patients that were to undergo elective knee replacement, hip replacement, varicose vein and groin hernia surgery Timing of assessment STEP 1 Identifying goals STEP 2 Identifying patients Pre-operative and post-operative PRO instruments were used to assess whether patient health improved following surgery; post-operative questionnaires STEP 2 were sent out 3 and 6 months after treatment Timing assessments 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: PROMs-benefits-case-study/pdf/promscasestudy.pdf. Copyright 2016, Re-used with the permission of the Health and Social Care Information Centre. All rights reserved 20

21 Selecting the PRO instrument The generic PRO instruments that were used for all procedures were: EQ-5D Index Patients provided responses to five questions, each covering a distinct health domain 1. Mobility 2. Self-care 3. Performing usual activities 4. Pain/discomfort 5. Anxiety/depression Patients rated their health on one of three levels, ranging from no problems to severe problems EQ Visual Analogue Scale (EQ VAS) Patients rated their general health on an analogue linear scale ranging between 0 and 100, with 100 representing the best state of health 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: PROMs-benefits-case-study/pdf/promscasestudy.pdf. Copyright 2016, Re-used with the permission of the Health and Social Care Information Centre. All rights reserved STEP 3 & 4 Selecting PRO instruments 21

Disease-specific PRO instruments The disease-specific PRO instruments selected were: STEP 3 & 4 Oxford Hip Score Oxford Knee Score Aberdeen

it s condition affects quality of life Responses combined to give an overall score, ranging from 0 (worst possible score) to 48 (best possible

(worst possible score) 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: http://www.hscic.gov.

22 Disease-specific PRO instruments The disease-specific PRO instruments selected were: STEP 3 & 4 Oxford Hip Score Oxford Knee Score Aberdeen Varicose Questionnaire Selecting PRO instruments 12 questions relating to the patient s hip (Oxford Hip Score) or knee (Oxford knee score) and how it s condition affects quality of life Responses combined to give an overall score, ranging from 0 (worst possible score) to 48 (best possible score) 13 questions relating to the patient s varicose veins The responses are combined into an index, ranging from 0 (best possible score) to 100 (worst possible score) 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: PROMs-benefits-case-study/pdf/promscasestudy.pdf. Copyright 2016, Re-used with the permission of the Health and Social Care Information Centre. All rights reserved 22

Linking PROMS to clinical outcomes Identifying an unmet need PROMs demonstrated that the Northumbria NHS Healthcare Foundation Trust was below the UK average for

Registry data for 22,691 primary total knee replacements to detect a correlation between PROMs scores and various surgical factors, including STEP 7 surgical

with two other brands (brands A and B). 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: http://www.hscic.gov.

23 Linking PROMS to clinical outcomes Identifying an unmet need PROMs demonstrated that the Northumbria NHS Healthcare Foundation Trust was below the UK average for adjusted health gain following replacement knee surgery STEP 5 & 6 Reporting results Interpreting PROMs Analysis of PROMs PROMs were linked to the National Joint Registry data for 22,691 primary total knee replacements to detect a correlation between PROMs scores and various surgical factors, including STEP 7 surgical implants Responding Linking PROMs to clinical outcomes to issues One implant brand (brand C) returned the highest health gain for the Oxford Knee Score, compared with two other brands (brands A and B). 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: PROMs-benefits-case-study/pdf/promscasestudy.pdf. Copyright 2016, Re-used with the permission of the Health and Social Care Information Centre. All rights reserved 23

24 Evaluating clinical practice informed by PROMs The Northumbria NHS Foundation Trust moved to solely using the knee replacement implant brand C that demonstrated the superior Oxford Knee Score To assess the effectiveness of the implant brand change, Northumbria Healthcare Foundation Trust analysed 1623 completed PRO instruments for the period April 2009 to March 2014 STEP 8 Evaluating the impact on clinical practice Brand Number of primary knee replacement surgeries Adjusted health gain for Oxford Knee Score Implant brand A Implant brand B Implant brand C Total 1623 N/A The analysis showed that changing to implant brand C significantly improved adjusted health gain scores for the Oxford Knee Score 1. Basser MR. Health and Social Care Information Centre. Can be accessed from: PROMs-benefits-case-study/pdf/promscasestudy.pdf. Copyright 2016, Re-used with the permission of the Health and Social Care Information Centre. All rights reserved 24

25 Conclusions 25

26 Summary PRO instruments help to encourage a patient-centric approach to care 1 PRO instruments are typically questionnaires that collect data across several areas of health, including physical, psychological and social functioning 2 o PRO instruments can be either generic or disease-specific PROMs can be used to influence clinical practice in a number of ways, these include: o As a screening tool to identify conditions/symptoms that would otherwise have been overlooked 3 o To monitor treatment and respond to patient outcomes over time 3 o To aid treatment choices and assist in selecting treatments that are most suited to the patient 3 Limitations of PROMs include comprehension of PRO instruments and associated costs of implementing and evaluating results 4,5 o However, PRO instruments can be improved for comprehension and the clinical benefits of implementing changes as a result of PROMs can offset initial economic costs 4,5 1. Lam CLK, et al. Front Med (Lausanne) 2014;1:29; 2. Spiegal BM. J Neurogastroenterol Motil 2013;19:137 48; 3. International Society for Quality of Life Research. Us Guide to Implementing Patient-Reported Outcomes Assessment in Clinical Practice. Can be accessed from: 4. Jahagirda 26 al.

27 Further reading 1. Health and Social Care Information Centre. Can be accessed from: Patient-reported outcomes: Measurements, implementation and interpretation (Chapman & Hall/CRC Biostatistics Series). Cappelleri JC, Zou KH, Bushmakin AG, Alvir JJ, Alemayehu D, Symonds T. CRC Press Taylor & Francis Group. 3. Nelson EC, et al. Patient-reported outcome measures in practice. BMJ 2015; Feb 10;350:g7818. doi: /bmj.g Santan Mj, et al. Training clinicians in how to use patient-reported outcome measures in routine clinical practice. Qual Life Res 2015;24:

28 Self-assessment quiz 28

29 Question 1 How many health domains does the SF-36 PRO instrument evaluate? 1. Eight 2. Six 3. Four 4. Two 29

30 Question 2 How many health domains does the EQ-5D PRO instrument evaluate? 1. Two 2. Four 3. Five 4. Eight 30

31 Question 3 Which of the following is not a type of PRO instrument? 1. Disease-specific 2. Individual patient-specific 3. Generic 31

32 Question 4 Which of the following is a disadvantage of disease-specific PRO instruments? 1. Specific for a given group (disease/population) 2. Sensitive enough to detect clinically significant changes 3. Captures clinically-relevant information that is applicable to a specific target group 4. Cannot compare with the general population 32

33 Question 5 Which of the following is a disadvantage of generic PRO instruments? 1. Suitable for the general public and for comparisons across populations 2. Suitable for comparisons with disparate conditions/disease groups 3. Captures commonly experienced health domains 4. Not sensitive to detecting disease-specific illnesses 33

34 Question 6 Which of the following is a potential drawback to PRO research 1. PROs are a poor reflection of the patient experience 2. Exclusion of patients due to poor literacy 3. An inability to link PRO outcomes to clinical registries 34

35 Question 7 Linking PROMs with clinical registries allows the clinician to establish: 1. Real world, retrospective data as it is processed 2. Prospective, randomised data 35

36 Question 8 Which of the following uses for PROMs is not true? PROMs can be used in clinical practice: 1. As a screening and monitoring tool 2. To replace randomised clinical trials to monitor the efficacy and safety of a treatment 3. As part of a patient-centred approach to care 4. As a treatment decision aid 36

37 Question 9 The SF-36 and EQ-5D PRO instruments are examples of a: 1. Generic PRO instrument 2. Disease-specific PRO instrument 37

38 Question 10 To compare the quality of life of a patient with a specific disease with that of healthy individuals, which of the two types of PRO instrument should be used? 1. Generic 2. Disease-specific 38

Patient Reported Outcomes: are they appropriate for clinical practice? Jose M Valderas Clinical Lecturer

Patient Reported Outcomes: are they appropriate for clinical practice? Jose M Valderas Clinical Lecturer Key issues What are PROMs? Why PROMs? Assessing PROMs Interpreting PROMs Evidence for use in clinical