Treatment burden should be included in clinical practice guidelinesBMJ 2018; 363 doi: https://doi.org/10.1136/bmj.k4065 (Published 12 October 2018) Cite this as: BMJ 2018;363:k4065
- Claudia C Dobler, consultant pulmonologist1 2 3,
- Nathan Harb, medical student3,
- Catherine A Maguire, patient involvement and engagement2,
- Carol L Armour, professor of pharmacology24,
- Courtney Coleman, patient involvement and engagement5,
- M Hassan Murad, professor of medicine1
- 1Evidence Based Practice Center, Robert D and Patricia E Kern Center for the Science of Health Care Delivery, Mayo Clinic, Rochester, MN, USA
- 2Woolcock Institute of Medical Research, University of Sydney, Sydney, NSW, Australia
- 3South Western Sydney Clinical School, University of New South Wales, NSW, Sydney, Australia
- 4Central Sydney Area Health Service, Sydney, NSW, Australia
- 5European Lung Foundation, Sheffield, UK
- Correspondence to: C C Dobler
Implementation of medical treatment regimens demands a lot of time and effort and can result in substantial burden for patients with chronic conditions. Guideline recommendations, however, are traditionally made from a perspective that places the highest value on achieving certain clinical outcomes (such as reducing shortness of breath or increasing survival). More recent guidelines may consider patients’ values and the acceptability and feasibility of the recommended action, but the treatment burden is not made explicit. This makes it hard for patients to make informed decisions about treatments in line with their values and capacity.
What is burden of treatment?
Although the burden of disease is a well known epidemiological concept, the burden of treatment is a less known and fairly recently coined term. Burden of treatment is the workload of healthcare and its effect on patient functioning and wellbeing.1 The cumulative treatment workload for patients with chronic conditions when enacting all recommendations in disease specific guidelines can be overwhelming.2
To cope with the demands of complex self care, patients rely on a range of personal, social, and healthcare resources.3 Disease treatment can occupy a large part of patients’ day and limit the amount of time they can spend on other activities such as work, studying, leisure, childcare, and being with friends and family. A study found that in order to comply with all disease specific guidelines patients with three chronic conditions (any combination of chronic obstructive pulmonary disease (COPD), coronary heart disease, diabetes, osteoarthritis, hypertension, and depression) would take from six to 13 different drugs a day, visit a health professional 1.2 to 5.9 times a month, and spend a mean (SD) of 49.6 (27.3) to 71.0 (34.5) hours each month in health related activities.2 In patients with all six chronic conditions, the workload rose to 18 medications a day, 6.6 healthcare visits a month, and 80.7 (35.8) hours a month in health related activities.2
The work that patients do to manage their health often remains invisible to health professionals, who only see the results (eg, attended exercise programme, achieved weight loss, glucose measurement log).4 This means that they often underappreciate the workload and overestimate patients’ capacity for more interventions.5 Components of the work that patients must do to care for their health include learning about their conditions and care, getting prescriptions, taking medications, attending medical appointments, monitoring their health (eg, measuring lung function or glucose monitoring), making lifestyle or behavioural changes (exercise, diet, smoking cessation), and managing medical equipment or devices (eg, regularly cleaning a nebuliser).6
Consequences of overburden
Patients who do not take prescribed medications or perform the prescribed daily exercises may be viewed as irresponsible, neglectful, or lacking motivation or insight into the importance of these treatments. Many patients who do not adhere to prescribed treatments, however, say that they know that they should do differently but that they lack the information and support to do so.7 Patients will also reject medical advice if the perceived benefit is considered insufficient in relation to the required investment of time, energy, and cost.8 These assessments about return on investment may be done consciously or intuitively, particularly when patients are overburdened by their treatment workload. In this case, they may resort to treatment prioritisation without discussing these choices with a health professional.
Shared decision making is therefore important because it enables patients, in collaboration with their clinician, to make conscious decisions about treatments using evidence based information.
How do guidelines handle burden of treatment?
The modern process of developing clinical practice guidelines starts by forming a panel that includes content and methodological experts and sometimes patient representatives. The panel develops clinical questions, which are examined in a systematic review focused on benefits and adverse effects of the interventions. The panel incorporates this evidence with other factors such as patients’ values and preferences to generate recommendations. Each recommendation is denoted by the grade of evidence (certainty or quality of evidence) and the strength of the recommendation (how compelling is the recommendation).9
Patients’ work to enact specific treatment recommendations is usually ignored in older guidelines, which have typically emphasised the evidence for the effectiveness of an intervention (or lack thereof). Modern guideline frameworks such as GRADE (Grading of Recommendations Assessment, Development, and Evaluation), which is the most used guideline approach, do attempt to capture the patient perspective. The evidence to decision (EtD) framework developed by the GRADE Working Group acknowledges the importance of patients’ values and preferences.10 Following this framework, the 2017 guidelines on prevention of COPD exacerbations published by the European Respiratory Society and the American Thoracic Society added a paragraph on “values and preferences” to every treatment recommendation.11 For example, the statement accompanying the conditional recommendation to use oral mucolytic agents to prevent future COPD exacerbations reads: “This recommendation places a high value on avoiding hospitalisations and a lower value on the cost and burden of taking daily medication.”
The EtD framework also incorporates some parts of the construct of burden of treatment in the domains of acceptability and feasibility.10 A strong recommendation for an intervention is contingent on the intervention being feasible to implement (by patients and clinicians) and acceptable (by patients and clinicians). Yet, the framework does not include the burden of treatment as a discrete construct.
Integration of treatment burden into guidelines
If future guidelines are to help clinicians and patients to make treatment decisions taking into account patients’ values and preferences, they should explicitly state the treatment burden associated with enacting different recommendations (the quantifiable workload as well as the potential effect on a patient’s life).
The information currently available on treatment burden for specific conditions and interventions is sparse and probably insufficient for most guidelines. The major challenges to including information on treatment burden in guidelines are therefore finding robust high quality methods for assessing treatment burden and meaningful ways of adding this information to clinical practice guidelines.
At least two validated measurement tools exist for self reported burden of treatment for patients with chronic conditions.121314 The Treatment Burden Questionnaire (TBQ) consists of seven items (two of which have four subitems), evaluating quantifiable workload as well as the effect of this workload on patients’ lives such as the frequency, time spent, and inconvenience of lab tests and other examinations.12 The Patient Experience with Treatment and Self-management (PETS) is a 48 item tool that focuses on the effect of treatment workload on patients’ lives and wellbeing—for example, “How much of a problem has it been for you to plan your daily activities around your medicine schedule?” (Responses: not at all, a little, somewhat, quite a bit, very much.) The suitability of these tools to collect information for use in clinical practice guidelines has yet to be evaluated, and they might have to be adjusted for this purpose.
Questionnaires that collect patient reported outcome measures have the advantage that they can be used in large scale surveys. They are not the best way to measure time spent on treatments, however, and a time and motion study might be useful to collect this information. Time and motion studies using observers have been used in healthcare mainly for understanding workflow of healthcare professionals and time spent on different activities.1516 Further research would be required to determine the feasibility of using this study design to document patients’ treatment workload around the clock, as using observers in this context does not seem feasible.
Drug companies are likely to have limited interest in measuring treatment burden in sponsored randomised controlled trials. Also, information collected in a real life setting may differ from that in a trial. Patient advocacy groups may have greater interest in information on treatment burden, and research partnerships between patient advocacy groups and investigators might be a good way to develop the research.
Treatment burden is not the same for all patients with the same condition. It depends on numerous factors such as age, disease burden, comorbidities, social support network, and patients’ values and preferences. For example, one patient may perceive the need for regular exercise as a burden whereas another patient may experience it as empowering action that improves their situation.17 Research is required to determine the best way to integrate information on treatment burden into guidelines so that it reflects the variations in experiences and is robust and useful to guideline developers and users. The value of integrating information on the potential burden for carers should also be explored. Box 1 gives some examples of how information on treatment burden could be included in guidelines.
Examples of how treatment burden could be included in guidelines
Chronic obstructive pulmonary disease (COPD)
The 2017 guidelines on prevention of COPD exacerbations make a conditional recommendation for initiation of pulmonary rehabilitation within three weeks after hospital discharge.11 The current “values and preferences” section states: “This recommendation places a high value on improving clinical outcomes and a lower value on the burden and cost of pulmonary rehabilitation.”
If the treatment burden was made explicit, the guideline could include a statement like this: “A typical effective pulmonary rehabilitation programme lasts 8 to12 weeks with two to three sessions a week, lasting one hour each. Many pulmonary rehabilitation programmes are fully or partially conducted at hospitals, therefore travel time to exercise classes may be added to the treatment burden. Although exercise is beneficial, more strenuous exercise is likely to be associated with (temporary) discomfort from shortness of breath.”
Type 2 diabetes
The 2015 NICE guideline on management of type 2 diabetes in adults says: “In adults with type 2 diabetes, if metformin is contraindicated or not tolerated, and if dual therapy with 2 oral drugs …has not continued to control HbA1c to below the person’s individually agreed threshold for intensification, consider insulin-based treatment.”18
Information on treatment burden could include information on how daily routines need to be changed with the initiation of insulin therapy (potential effect on working life, dietary choices, etc), the frequency and average time required for insulin injections and blood sugar controls, discomfort associated with insulin injection, risk of hypoglycaemia, and its potential impact on patients’ quality of life.
Chronic kidney disease
The 2014 NICE guidelines on assessment and management of chronic kidney disease in adults state: “Offer dietary advice about potassium, phosphate, calorie and salt intake appropriate to the severity of CKD. Where dietary intervention is agreed this should occur within the context of education, detailed dietary assessment and supervision to ensure malnutrition is prevented.”19
Information on treatment burden could include information on foods that should be avoided, extra time spent on grocery shopping and cooking, challenges when eating out, the typical number of consultations with a dietitian, transport requirements to see a dietitian, time spent documenting the diet, and required intensity of blood tests to monitor electrolytes.
Clinical practice guidelines should also explicitly recommend that clinicians discuss values and preferences with patients, especially for treatments associated with a high workload. This would help clinicians to understand how individual patients prioritise different outcomes and treatment burden and would help patients to understand what components of their treatment are the essentials, even on a bad day, so that they can prioritise treatments appropriately.
Individualised treatment and shared decision making
The process of weighing pros and cons (including treatment burden) of different treatments happens during shared decision making between patient and clinician. This should ensure that the final choice for or against a medical intervention aligns with the patient’s values and preferences rather than being driven by the clinician’s agenda. Information on treatment burden in guidelines could provide high quality information for developers of decision aids and facilitate shared decision making when a decision aid is not available for the specific clinical scenario.
Clinical decision making, particularly for patients with multimorbidity, has to consider trade-offs between competing outcomes and prioritisation. The Ariadne principles on how to handle multimorbidity in primary care consultations emphasise the careful and critical use of guidelines in multimorbidity and promote sharing of realistic treatment goals by physicians and patients and prioritising health problems in accordance with the patient’s preferences.20 Including information on treatment burden in guidelines would increase their applicability to patients with multimorbidity in line with these principles. Better understanding of the work required to follow treatment recommendations would help to improve treatment decisions and avoid overburdening patients.
Treatment burden, the work that patients need to do to care for their health and its effect on their life, is often substantial for people with chronic conditions
Although modern clinical guidelines may consider acceptability to patients, evaluations of treatment burden are not included
Information on treatment burden would enable patients to make informed decisions about treatments
High quality methods for assessing treatment burden need to be identified and meaningful ways of adding this information to clinical practice guidelines need to be explored
Contributors and sources: CCD is a consultant pulmonologist. Her research focuses on evidence based medicine, knowledge translation, and medical decision making. NH is a medical student who is conducting research on the treatment burden of patients with chronic respiratory diseases. CAM balances a busy life as a tertiary education disability adviser and provisional psychologist studying her masters, mother, and wife while also spending 2-3 hours daily on treatments for her cystic fibrosis. CLA is a professor of respiratory pharmacology and director of a medical research institute and has authored more than 240 scientific publications, with a major focus on patient centred models of care. CC’s role includes working with European Respiratory Society members to facilitate patient input into clinical practice guideline development. MHM has expertise in evidence based medicine, evidence synthesis, and guideline development and has authored more than 500 scientific publications (including more than 50 guidelines). CCD and MHM conceived the initial idea. CCD drafted the manuscript. All authors contributed to critical discussions of the ideas, revised the manuscript, and approved the final submission.
Competing interests: We have read and understood BMJ policy on declaration of interests and have no relevant interests to declare. CCD is supported by a fellowship of the National Health and Medical Research Council (APP1123733). The fellowship sponsor had no role in manuscript design, data interpretation, or writing of the manuscript.
Provenance and peer review: Not commissioned; externally peer reviewed.