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Are we failing patients in our assessment of treatment failure?

Are we failing patients in our assessment of treatment failure? Treat-to-target principles of managing rheumatic and musculoskeletal diseases (RMDs) such as RA [1] are now widely accepted as effective strategies for achieving optimal disease outcomes. Despite this, a proportion of patients continue to have poor disease outcomes, regardless of the use of highly effective treatments that are currently available. The term ‘treatment failure’ is becoming increasingly replaced by ‘treatment success’, the latter capturing headlines in the news and declaring the power of newer treatments to achieve disease control and states of disease remission. Yet, the meaning of treatment failure or treatment success can be vastly different from the patient’s perspective, compared with that of their treating rheumatologist. It is therefore perhaps not surprising to see discordance in the perception of disease severity between patients and physicians [2]. Rheumatologists traditionally use the term treatment failure to indicate the inability of a specific treatment to achieve treat-to-target goals, that is, remission or at least low disease activity in RA, based on composite measures of disease activity, all of which in fact integrate the perspectives of the patient and the physician by being a composition of subjective and objective measures. The most prominent representatives are the DAS-28 [3], the Simplified Disease Activity Index and the clinical Disease Activity Index [4]. Whereas some patients will appreciate this technical aspect of their disease, embracing the DAS-28 as a tool to self-manage their disease and participating in know your DAS (https://www.nras.org.uk/publications/know-your-das) or know your clinical Disease Activity Index initiatives, others find this too technical and not sufficiently representative of their disease burden. To many, other parameters of their illness will be as or even more important than the mere technical disease activity. These could include levels of fatigue, quality of sleep, impact on sexual life, and ability to socially engage and to work; the lack of these measures in the DAS-28 questions its value in accurately determining treatment success from the perspective of the patient. Aspects of disease such as sleep and fatigue remain generally poorly addressed in routine clinical care, despite their significant impact on other disease outcomes including disease activity [5]. Other examples of patient-reported outcomes (PROs) that are more traditionally used in routine clinical care include the HAQ [6], which since the 1980s has offered a feasible and reliable tool to measure disability across the spectrum of RMDs. Regulatory bodies in the UK such as the National Institute for Health and Care Excellence have used the HAQ to derive utility and cost-effectiveness measures for specific treatments. Still, the HAQ faces similar challenges to the composite disease activity measures, including the inability to capture other important elements of disease as outlined above. That said, we witness an abundance of PROs for RMDs [7] being developed over time, but which of these really matter to the patient? Many of these tools seem to lack significant measures of other important parameters of disease such the impact on social participation, work ability and sexual life. It could be argued that PROs to address these parameters are not really necessary and all that a rheumatologist needs to do, is to simply ask the relevant question(s), for example, ‘Are you able to go out, socialize with others? Are you able to work? How does your disease affect your ability to work?’ In reality though, the significant pressures that many national health care systems face such as capacity, time pressures and staff issues, can make the possibility of having even a simple, one-to-one heartfelt and unrushed chat with the patient rather challenging. We read with interest of new PROs being developed to measure other, less commonly talked about factors relating to disease such as fear [8]. High fear scores are reported in an important proportion of patients with these conditions and are in turn also associated with psychological distress [8]; we therefore consider the development of such measures of great importance, probably even greater than some of the more traditional measures of disease. Fear, among other emotions experienced by patients such as anxiety and low mood, could influence the way patients perceive treatment and potential side effects, with consequences on compliance and treatment outcomes. We consider it necessary that the future assessment of RMDs is regularly revisited to reflect both objective but also subjective measures of disease and with emphasis on biopsychosocial elements that influence disease outcomes. In the current era of multiple therapeutic options on the market, all with an important side effect profile, it is imperative that not only are patient activation measures encouraged (stimulating patients to use their knowledge, skills and confidence to manage their own health/care), making patients central to treatment decisions, but also that rheumatologists use effective leadership skills and highly adaptive language and approach, tailored to the individual patient needs. With this reconciliation of perspectives of these two important stakeholders, the future will move to an even better implementation of the mentioned treat-to-target strategy with probably the most important of its overarching principles, namely to manage RA (and other diseases), based on shared decision making. Novel technologies, self-management applications and other gadgets, along with an impressive (and at times concerning!) surge of artificial intelligence and social media into our lives, herald a new epoch for people with RMDs, which spares no room for anything less than excellence. Failed consultations (Table 1) should be firmly locked in the past and the future should be characterized by more effective bidirectional interaction with our patients. Empowering our patients to take control of their disease and not ignoring potential barriers to treatment success that go beyond drugs, such as language barriers, cultural beliefs, social isolation and the stigma that comes along with having a diagnosis of long-term and potentially disabling condition, is crucial. This calls for better alignment of our interpretation of treatment failure with that of the person with the RMD. Table 1 Perceived treatment success—a dialogue example that ‘fails’ patients Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ Table 1 Perceived treatment success—a dialogue example that ‘fails’ patients Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ In the end, aligned objectives and aligned strategies will help to overcome one of the great challenges in the management of chronic rheumatic disease, which is to keep patients adherent to and persistent with their therapies. Poor adherence is a great unknown player in most rheumatologists’ offices and clinics, but is a major harbinger of treatment failure. We initially thought that treatment failure was a clinically defined tight term, but maybe redefining it would eventually yield to treatment success. Funding: No specific funding was received from any bodies in the public, commercial or not-for-profit sectors to carry out the work described in this manuscript. Disclosure statement: M.B. has been sponsored to attend regional, national and international meetings by UCB Celltech, Roche/Chugai, Pfizer, AbbVie, Merck, Mennarini, Janssen, Bristol-Myers Squib, Novartis and Eli-Lilly, and has received honoraria for speaking and attended advisory boards with Bristol-Myers Squib, UCB Celltech, Roche/Chugai, Pfizer, Abbvie, Merck, Mennarini, Sanofi-aventis, Eli-Lilly, Janssen and Novartis. The other authors have declared no conflict of interest. References 1 Smolen JS. Treat-to-target as an approach in inflammatory arthritis . Curr Opin Rheumatol 2016 ; 28 : 297 – 302 . Google Scholar Crossref Search ADS PubMed 2 Cho S-K , Sung Y-K , Choi C-B et al. What factors affect discordance between physicians and patients in the global assessment of disease activity in rheumatoid arthritis? Mod Rheumatol 2017 ; 27 : 35 – 41 . Google Scholar Crossref Search ADS PubMed 3 Prevoo ML , van’t Hof MA , Kuper HH et al. Modified disease activity scores that include twenty-eight-joint counts. Development and validation in a prospective longitudinal study of patients with rheumatoid arthritis . Arthritis Rheum 1995 ; 38 : 44 – 8 . Google Scholar Crossref Search ADS PubMed 4 Aletaha D , Smolen J. The Simplified Disease Activity Index (SDAI) and the Clinical Disease Activity Index (CDAI): A review of their usefulness and validity in rheumatoid arthritis . Clin Exp Rheumatol 2005 ; 23 : 100 – 8 . Google Scholar PubMed 5 Austad C , Kvien TK , Olsen IC. Sleep disturbance in patients with rheumatoid arthritis is related to fatigue, disease activity, and other patient-reported outcomes . Scand J Rheumatol 2017 ; 46 : 95 – 103 . Google Scholar Crossref Search ADS PubMed 6 Kirwan JR. Stanford Health Assessment Questionnaire modified to assess disability in British patients with rheumatoid arthritis . Br J Rheumatol 1986 ; 25 : 206 – 9 . Google Scholar Crossref Search ADS PubMed 7 van Tuyl LHD , Boers M. Patient-reported outcomes in core domain sets for rheumatic diseases . Nat Rev Rheumatol 2015 ; 11 : 705 . Google Scholar Crossref Search ADS PubMed 8 Gossec L , Chauvin P , Saraux A et al. Development and psychometric validation of a patient-reported outcome measure to assess fears in rheumatoid arthritis and axial spondyloarthritis: the Fear Assessment in Inflammatory Rheumatic diseases (FAIR) questionnaire . Ann Rheum Dis 2017 ; 77 : 258 – 63 . Google Scholar Crossref Search ADS PubMed © The Author(s) 2018. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: journals.permissions@oup.com This article is published and distributed under the terms of the Oxford University Press, Standard Journals Publication Model (https://academic.oup.com/journals/pages/open_access/funder_policies/chorus/standard_publication_model) http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Rheumatology Oxford University Press

Are we failing patients in our assessment of treatment failure?

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Publisher
Oxford University Press
Copyright
© The Author(s) 2018. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: journals.permissions@oup.com
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1462-0324
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10.1093/rheumatology/key107
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Abstract

Treat-to-target principles of managing rheumatic and musculoskeletal diseases (RMDs) such as RA [1] are now widely accepted as effective strategies for achieving optimal disease outcomes. Despite this, a proportion of patients continue to have poor disease outcomes, regardless of the use of highly effective treatments that are currently available. The term ‘treatment failure’ is becoming increasingly replaced by ‘treatment success’, the latter capturing headlines in the news and declaring the power of newer treatments to achieve disease control and states of disease remission. Yet, the meaning of treatment failure or treatment success can be vastly different from the patient’s perspective, compared with that of their treating rheumatologist. It is therefore perhaps not surprising to see discordance in the perception of disease severity between patients and physicians [2]. Rheumatologists traditionally use the term treatment failure to indicate the inability of a specific treatment to achieve treat-to-target goals, that is, remission or at least low disease activity in RA, based on composite measures of disease activity, all of which in fact integrate the perspectives of the patient and the physician by being a composition of subjective and objective measures. The most prominent representatives are the DAS-28 [3], the Simplified Disease Activity Index and the clinical Disease Activity Index [4]. Whereas some patients will appreciate this technical aspect of their disease, embracing the DAS-28 as a tool to self-manage their disease and participating in know your DAS (https://www.nras.org.uk/publications/know-your-das) or know your clinical Disease Activity Index initiatives, others find this too technical and not sufficiently representative of their disease burden. To many, other parameters of their illness will be as or even more important than the mere technical disease activity. These could include levels of fatigue, quality of sleep, impact on sexual life, and ability to socially engage and to work; the lack of these measures in the DAS-28 questions its value in accurately determining treatment success from the perspective of the patient. Aspects of disease such as sleep and fatigue remain generally poorly addressed in routine clinical care, despite their significant impact on other disease outcomes including disease activity [5]. Other examples of patient-reported outcomes (PROs) that are more traditionally used in routine clinical care include the HAQ [6], which since the 1980s has offered a feasible and reliable tool to measure disability across the spectrum of RMDs. Regulatory bodies in the UK such as the National Institute for Health and Care Excellence have used the HAQ to derive utility and cost-effectiveness measures for specific treatments. Still, the HAQ faces similar challenges to the composite disease activity measures, including the inability to capture other important elements of disease as outlined above. That said, we witness an abundance of PROs for RMDs [7] being developed over time, but which of these really matter to the patient? Many of these tools seem to lack significant measures of other important parameters of disease such the impact on social participation, work ability and sexual life. It could be argued that PROs to address these parameters are not really necessary and all that a rheumatologist needs to do, is to simply ask the relevant question(s), for example, ‘Are you able to go out, socialize with others? Are you able to work? How does your disease affect your ability to work?’ In reality though, the significant pressures that many national health care systems face such as capacity, time pressures and staff issues, can make the possibility of having even a simple, one-to-one heartfelt and unrushed chat with the patient rather challenging. We read with interest of new PROs being developed to measure other, less commonly talked about factors relating to disease such as fear [8]. High fear scores are reported in an important proportion of patients with these conditions and are in turn also associated with psychological distress [8]; we therefore consider the development of such measures of great importance, probably even greater than some of the more traditional measures of disease. Fear, among other emotions experienced by patients such as anxiety and low mood, could influence the way patients perceive treatment and potential side effects, with consequences on compliance and treatment outcomes. We consider it necessary that the future assessment of RMDs is regularly revisited to reflect both objective but also subjective measures of disease and with emphasis on biopsychosocial elements that influence disease outcomes. In the current era of multiple therapeutic options on the market, all with an important side effect profile, it is imperative that not only are patient activation measures encouraged (stimulating patients to use their knowledge, skills and confidence to manage their own health/care), making patients central to treatment decisions, but also that rheumatologists use effective leadership skills and highly adaptive language and approach, tailored to the individual patient needs. With this reconciliation of perspectives of these two important stakeholders, the future will move to an even better implementation of the mentioned treat-to-target strategy with probably the most important of its overarching principles, namely to manage RA (and other diseases), based on shared decision making. Novel technologies, self-management applications and other gadgets, along with an impressive (and at times concerning!) surge of artificial intelligence and social media into our lives, herald a new epoch for people with RMDs, which spares no room for anything less than excellence. Failed consultations (Table 1) should be firmly locked in the past and the future should be characterized by more effective bidirectional interaction with our patients. Empowering our patients to take control of their disease and not ignoring potential barriers to treatment success that go beyond drugs, such as language barriers, cultural beliefs, social isolation and the stigma that comes along with having a diagnosis of long-term and potentially disabling condition, is crucial. This calls for better alignment of our interpretation of treatment failure with that of the person with the RMD. Table 1 Perceived treatment success—a dialogue example that ‘fails’ patients Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ Table 1 Perceived treatment success—a dialogue example that ‘fails’ patients Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ Patient: ‘These drugs have been making me feel sick…’ Doctor: ‘You are on great treatment! Glad we managed to get funding for the biologic drug …!’ Patient: ‘… I’m feeling so tired… can’t be bothered to get out of the house … .’ Doctor: ‘Are these joints tender?’ Patient: ‘… driving is not easy anymore… had to skip my best friend’s birthday party …’ Doctor: ‘One a scale of 1–10…’? Patient: ‘Doctor … I have been feeling rather low too… sometimes all I want to do is just sleep …’ Doctor: ‘Your disease activity score is 2.3! This is great news!’ Patient: ‘… work hasn’t been easy … missed 3 days last week … I wasn’t feeling well … everything hurts … . The drugs make me feel sick … .’ Doctor: ‘You are officially in remission! Keep your drugs going, you are doing great! See you in a year’s time!’ In the end, aligned objectives and aligned strategies will help to overcome one of the great challenges in the management of chronic rheumatic disease, which is to keep patients adherent to and persistent with their therapies. Poor adherence is a great unknown player in most rheumatologists’ offices and clinics, but is a major harbinger of treatment failure. We initially thought that treatment failure was a clinically defined tight term, but maybe redefining it would eventually yield to treatment success. Funding: No specific funding was received from any bodies in the public, commercial or not-for-profit sectors to carry out the work described in this manuscript. Disclosure statement: M.B. has been sponsored to attend regional, national and international meetings by UCB Celltech, Roche/Chugai, Pfizer, AbbVie, Merck, Mennarini, Janssen, Bristol-Myers Squib, Novartis and Eli-Lilly, and has received honoraria for speaking and attended advisory boards with Bristol-Myers Squib, UCB Celltech, Roche/Chugai, Pfizer, Abbvie, Merck, Mennarini, Sanofi-aventis, Eli-Lilly, Janssen and Novartis. The other authors have declared no conflict of interest. References 1 Smolen JS. Treat-to-target as an approach in inflammatory arthritis . Curr Opin Rheumatol 2016 ; 28 : 297 – 302 . Google Scholar Crossref Search ADS PubMed 2 Cho S-K , Sung Y-K , Choi C-B et al. What factors affect discordance between physicians and patients in the global assessment of disease activity in rheumatoid arthritis? Mod Rheumatol 2017 ; 27 : 35 – 41 . Google Scholar Crossref Search ADS PubMed 3 Prevoo ML , van’t Hof MA , Kuper HH et al. Modified disease activity scores that include twenty-eight-joint counts. Development and validation in a prospective longitudinal study of patients with rheumatoid arthritis . Arthritis Rheum 1995 ; 38 : 44 – 8 . Google Scholar Crossref Search ADS PubMed 4 Aletaha D , Smolen J. The Simplified Disease Activity Index (SDAI) and the Clinical Disease Activity Index (CDAI): A review of their usefulness and validity in rheumatoid arthritis . Clin Exp Rheumatol 2005 ; 23 : 100 – 8 . Google Scholar PubMed 5 Austad C , Kvien TK , Olsen IC. Sleep disturbance in patients with rheumatoid arthritis is related to fatigue, disease activity, and other patient-reported outcomes . Scand J Rheumatol 2017 ; 46 : 95 – 103 . Google Scholar Crossref Search ADS PubMed 6 Kirwan JR. Stanford Health Assessment Questionnaire modified to assess disability in British patients with rheumatoid arthritis . Br J Rheumatol 1986 ; 25 : 206 – 9 . Google Scholar Crossref Search ADS PubMed 7 van Tuyl LHD , Boers M. Patient-reported outcomes in core domain sets for rheumatic diseases . Nat Rev Rheumatol 2015 ; 11 : 705 . Google Scholar Crossref Search ADS PubMed 8 Gossec L , Chauvin P , Saraux A et al. Development and psychometric validation of a patient-reported outcome measure to assess fears in rheumatoid arthritis and axial spondyloarthritis: the Fear Assessment in Inflammatory Rheumatic diseases (FAIR) questionnaire . Ann Rheum Dis 2017 ; 77 : 258 – 63 . Google Scholar Crossref Search ADS PubMed © The Author(s) 2018. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: journals.permissions@oup.com This article is published and distributed under the terms of the Oxford University Press, Standard Journals Publication Model (https://academic.oup.com/journals/pages/open_access/funder_policies/chorus/standard_publication_model)

Journal

Rheumatology – Oxford University Press

Published: Apr 1, 2019

There are no references for this article.