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Who is involved in making NICE guidance recommendations and what evidence do they look at?
Student Champions Learning about NICE - 27 September 2017 Jane Cowl, Senior Public Involvement Adviser
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Who decides what NICE will recommend?
Specialist staff employed by NICE True or False? The Department of Health True or False? NICE employed administration staff True or False? NHS England True or False? Clinical Commissioning Groups True or False? NHS finance managers True or False? Patients True or False? Independent committees of NICE staff & experts True or False? Independent committees of experts True or False?
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Who decides what NICE will recommend?
Independent committees Chair At least 2 lay members Health and other professionals (specialists and generalists) Sometimes Technical experts e.g. health economist Independent committees of experts – professional and lay All committees and groups developing guidance have a chair, lay members and H&SC professionals – depending on the type of guidance some also have care providers (eg social care) and commissioners (eg social care, public health and service guidance). Some committees include technical experts eg TACs. lay members = patients or people using services, their carers and advocates and other members of the public with relevant experience and expertise. Recruit people who can bring broad perspective not just own personal exp. NICE provides dedicated support and training for lay participants. Open recruitment and selection. Committee members are selected for their knowledge and experience, and do not represent their organisation(s). In most cases, manufacturers of pharmaceutical products or medical devices are not represented on the Committee because of potential conflicts of interest, but they contribute to guidance development as stakeholders. However, when guidance is likely to cover systems and processes relevant to the pharmaceutical or medical devices industries, the Committee may include members of representative bodies to ensure that this perspective is represented 2 types: standing committees and topic specific groups Staff provide technical and administrative support
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Evidence informing committee’s work
Reviews of research evidence (all NICE guidance) Stakeholder consultation (all NICE guidance) Grey literature and unpublished data Economic modelling Manufacturers submissions Expert testimony (patient and professional) Occasional additional consultation with practitioners and patients This slide shows the different sources and range of evidence that inform the work of NICE committees. This will depend on the type of guidance and topic. Only 2 of these are routine to all types of NICE guidance – reviews of the research evidence and consultation with stakeholders. For example expert testimony is always used for TAs (because the standing committee members are not topic experts) but rarely for topic specific GCs. NICE recommendations are based on the best available evidence of what works and what represents value for money. But ‘best available evidence’ will look very different for different types of NICE topics – e.g. evidence for new drugs versus evidence for social care services. NICE recommendations based on best available evidence
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The right evidence type for the question
The question dictates the most appropriate study design What is the cause of this disease? Cohort, case-controlled study What does it feel like? What is important to you? What is your experience of care? Qualitative research What is the most clinically effective therapy? Randomised controlled trial (RCT) What works best in diagnosing the condition Observational study or RCT Move from ‘hierarchy of evidence’ with systematic reviews of RCTs at the top (gold standard) to acknowledgement that the type of question will determine the most appropriate study type – right type of evidence for the right question – And also the value of combining different types of evidence eg RCT for effectiveness of intervention under controlled conditions, plus qualitative evidence on ’barriers and facilitators’ to intervention working in practice, including patients’ views and experiences. Qualitative research is used to explore and understand people’s beliefs, experiences, attitudes or behaviour. It asks questions about how and why as well as some what questions Examples of questions from NICE guidance on patients views and experiences: What information and support should be offered to children with atopic eczema and their families and carers? What elements of care on the general ward are viewed as important by patients following their discharge from critical care areas? Are there cultural differences that need to be considered when delivering information and support on breast or bottle-feeding? What are the perceived risks and benefits of immunisation among parents, carers or young people? Is there a difference in perceived benefits and risks between groups whose children are partially immunised and those who have not been immunised? Includes systematic reviews of studies when available
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HIGH QUALITY PATIENT CARE
The nature of evidence Patient evidence Clinical evidence HIGH QUALITY PATIENT CARE Economic evidence Relevant Effective Acceptable Appropriate Clinical and economic research evidence has a long history of conceptual and methodological development – patient evidence not so and doesn’t have the same recognition – so this is a big challenge for NICE and others because all 3 types of evidence are vital – we need to ensure that committees have appropriately considered what is important from a patient perspective, including how relevant, acceptable and appropriate a treatment or service is. Patient evidence is used in NICE guidance (from existing research and from patient involvement) - lay people on committees help to ensure patient perspectives are considered (also patient stakeholder input). Acknowledgement: Dr Sophie Staniszewska, RCN Research Institute, University of Warwick
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Patient evidence 13/05/2018
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The value of patient evidence
13/05/2018 The value of patient evidence What insights does patient evidence offer us? Personal impact of living with a condition and experience of care People’s preferences and values Outcomes that patients want from treatment or care Impact of treatment or care on outcome, symptoms, physical and social functioning, quality of life Risks, benefits and acceptability of a treatment or service Equality issues and considerations for specific sub-groups Patient evidence may give us new information that doesn’t exist in the research literature, sometimes it can question the assumptions of researchers or a committee, and also bring challenges to the quantitative evidence. And where there is qualitative patient evidence it can contextualise quantitative research.
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Evidence from experience of care
High dose rate brachytherapy for cervical carcinoma People who self-harm Kidney dialysis Example – people who self-harm One finding from focus group discussions was that people in mental distress who self-harmed were not routinely offered anaesthesia for stitching their wounds in emergency department. There was nothing in literature to indicate this was an issue. As a result the guideline included a recommendation that adequate anaesthesia and/or analgesia should be offered to people who have self-harmed throughout the process of suturing or other painful treatments. Other recs include staff training. Example - Psoriasis Clinical research told us the amount of psoriasis was what most affected the quality of life. Patients told us that the location of the flare-up (e.g. face or joints) was more significant. Example - High dose rate brachytherapy for cervical carcinoma Women who had had the procedure told us it was both distressing and painful – nothing in the research evidence to indicate this NICE guidance includes recommendations about pain management and counselling Example – Consultation with patient groups about kidney dialysis Committee assumed patients would prefer dialysis at home Some patients told us they disliked home machines as it meant their illness dominated their lives Psoriasis
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Patient perspectives: impact and challenges
13/05/2018 Patient perspectives: impact and challenges Examples of positive influence of patient evidence on: Scoping and review questions Evidence reviews – how interpreted Guidance recommendations Research recommendations Challenges Ensuring patient voices are heard The weighting of patient evidence Synthesising with clinical and economic evidence PIP has collected examples of impact across NICE work progs - show impact at different stages of guidance development from scoping through to guidance recs and research recs Ensuring patient voices are heard Eg Major gaps eg research not done, or poor quality, or patient perspectives not adequately covered Eg Lay voice on committees – balanced views avoiding single issues; lay members vary in experience – PIP offers support Eg ‘Less heard’ groups eg CYP, people with cognitive impairment, people not represented by a patient organisation, etc Weighting – eg criticism from patient groups re TAs Synthesising different types of evidence – <Health Economist speaker> now say more on HE evidence and synthesising different types…
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Health (Care) Economics
Ross Maconachie Wednesday 27th Sept 2017
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Why Consider Health Economics?
Opportunity Cost If the NHS spends more on one thing, it has to do less of something else (on the margin) Could we do more good by spending money in other ways? The ‘opportunity cost’ is the value of the best alternative use of resources Why we care about cost-effectiveness It would be unethical not to care about cost effectiveness. The National Health Service (NHS) seeks to maximise health of all the people it serves given its limited resources. When it is decided to provide a service for some NHS patients that cost extra money, this will reduce the resources that can be spent on services for other NHS patients. Disregarding this knock on effect of a decision can result in a loss of health across all NHS patients. Therefore we want to assess three issues: What is the evidence that a service intervention is effective (does what it says it does) How much more effective it is than the next best alternative (how much better does it do what it says it does when compared to for example standard care) – be aware that when there are more than two options, comparing these to one another instead of to ‘do nothing’ (also called a common baseline) makes this comparison ‘incremental’. What providing this intervention costs in addition to what the next best alternative (for example standard care) costs (how much must we pay extra for doing new option instead of usual care).
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Which Mobile Phone Contract Would Choose. i) for yourself
Which Mobile Phone Contract Would Choose? i) for yourself ii) if you had to decide for everyone on your course Option A Option B Option C Unlimited minutes Unlimited texts Unlimited data £40 per month 500 minutes 500 texts 1.5 GB of data £20 per month 0 minutes 0 texts 0 GB of data £0 per month Please take 5 minutes to say hello to the people on your table, then consider the two options on the screen. Which option do you recommend? What are the reasons for your choice? * * * Discuss the responses in terms of costs versus benefits; value for money … Would you make a different recommendation for a specific population group? How much would costs have to rise before you decided it was no longer value for money (decision making thresholds)? This is an everyday consideration … let’s go on to see how economic analysis is about setting out a framework for such considerations to help us in our work. (Might this example be analogous to the intensity of a social care intervention – e.g. number of home care hours?) BUT NOTE that the selection of the comparator is crucial – we must ask the right question. If we were writing a guideline on mobile phone contracts we would probably want to address a different question with different comparators. Thinking about cost-effectiveness and opportunity costs – which contract do you pick?
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There is more information that we would need here, ideally:
What is the budget/who is paying? This is our threshold Who are we making the decision for (perspective)? How long for? What benefits will the person/people get? What is the opportunity cost? the value of the best alternative use of that money
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Economic Evaluation What to Consider?
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Economic Evaluation Cost-effectiveness, not cost (or resource) impact
“... the comparative analysis of alternative courses of action in terms of both their costs and consequences.” Drummond, Stoddard & Torrance, 1987 Costs Value of extra resources used (loss to other patients) Current Treatment Consequences/ Outcome Value of health gain for this patient group Based on a CCP slide, the first bullet has been added to emphasise that this is not about resource impact. This definition of economic evaluation (from Drummond, Stoddart & Torrance) has two key aspects: EE should always compare one health care intervention with one or more alternative interventions for the same population group. EE should include both the costs and consequences of interventions - the resources that they consume and the health outcomes that they produce. Note that the choice of comparator is crucial – should include all relevant options for a group of patients (including ‘do nothing’ and ‘current practice’). Analysis should be conducted separately for each subgroup of patients. New Treatment
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What Health Outcomes are Important?
What we want to know…. How much the intervention improves the health of the people involved? What measure to use…..or to develop What outcomes/factors should we be looking at? Types of Outcome….. Cure? ‘Progression-free’? Improved measurement? (e.g. growth; reduced blood pressure Reduction of risk? (e.g. pre-term birth; falls in older people) Improved ‘quality of life’? Additional years (or months) of life? Tools to measure quality of life (QoL) were developed and are available in a wide range of areas of health. At one point or another you will come across one, if not all of these outcomes – there is no one perfect outcome. However…… Outcome measurement is important when establishing whether a new intervention, for example, is more effective than an existing one. There is a great variety of specific outcomes that are used in the different disease areas. One difficulty is that an improvement in one measure for some patients is impossible to compare to an improvement in a different scale of a particular measurement. In clinical guidelines we use quality adjusted life years, that summarise the effects on a person’s health in one measure, thus making comparisons possible across different diseases. ‘Surrogate outcomes’ alone, such as blood pressure, do not provide enough information to decide whether or not something is worthwhile doing given the fixed budget the NHS has available. However, surrogate outcomes, such as ‘return to work’, hospital length of stay in days or number of cases detected, can be important to inform economic work where published evidence is absent.
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Examples of Measures Generic (disease non-specific) – how we get a QALY EQ-5D – EuroQol 5 Dimension SF-6D and SF-36 – Short-Form HUI – Health Utilities Index Disease Specific AQLQ- Asthma Quality of Life Questionnaire MSQOL - Migraine Specific Quality of Life EORTC QLQ-C30 -European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Add-On Tools EQ-5D+C – add on for cognitive factors
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Describing Health State Using EQ-5D
Mobility I have no problems in walking about I have some problems in walking about I am confined to bed Self-care I have no problems with self care I have some problems washing or dressing myself I am unable to wash or dress myself Usual activities (e.g. work, study, housework, family or leisure activities) I have no problems with performing my usual activities I have some problems with performing my usual activities I am unable to perform my usual activities Pain/discomfort I have no pain or discomfort I have moderate pain or discomfort I have extreme pain or discomfort Anxiety/depression I am not anxious or depressed I am moderately anxious or depressed I am extremely anxious or depressed
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Valuing Health States Using EQ-5D
EQ-5D defines 243 possible states Values of states elicited from UK general population (n=3,395) Has been developed in different countries for that specific population Mean values can be used in economic evaluations Values elicited using ‘time trade-off’ – so 1year in perfect health = 2 years in 0.5 health
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EQ-5D Exercise Read the Vignette describing a condition
Fill in the EQ-5D form imagining you are that patient Use the formula to calculate your EQ-5D score Discuss (NB: There is no ‘correct’ answer to this exercise)
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Quality Adjusted Life Years -QALY
What is a QALY? QALYs combine both quantity and health-related quality of life (QoL) into a single measure of health gain Use a self reported preference-based questionnaire score (like EQ-5D or SF-36) QoL scores (utilities) should reflect people’s preferences over health Utilities usually scored with ‘perfect health’=1 and death=0
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Quality Adjusted Life Years
Why use QALYs? Can weigh up net effect of treatment for patients Survival vs QoL (e.g. for cancer chemotherapy) Long-term QoL for chronic & recurrent conditions (e.g. arthritis) Benefits versus harms (e.g. COX II inhibitors) Allows broader comparisons between patient groups The QALY is the preferred measure of NICE when conducting cost- effectiveness analysis (mostly in clinical and technology appraisals). However, not always possible for social care and public health so look to other methods. Cox 2 – NSAID but has adverse vascular events later down the line
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"A QALY is a QALY is a QALY”
Usual value judgements used to calculate QALYs: Allows the effectiveness of different technologies for different people to be compared 1 QALY: One year of ‘perfectly’ health life for one person OR two years of life with QoL of 0.5 for one person OR one year of life with QoL of 0.5 each for two people BUT – they do not incorporate an age weigh function – so 1 QALY at age 86 = 1 QALY at age 3 = =
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Quality Adjusted Life Year
1 Initial QALY loss due to side effects New treatment QALYs gained Health-related quality of life Current treatment We may illustrate the concept of QALYs using this diagram of length of life (in years) against quality of life (or ‘utility’ on a 0-1 scale). We compare the expected profile for some group of patients with some given treatment and without. The curves may cross - for example, if the treatment has initial side effects. The area between the two curves is the number of QALYs gained (large purple area minus small blue). Length of life (years)
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Economic + Cost-Effectiveness Analysis
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Types of Economic Evaluation
Type of analysis Value of resources Value of health gain Cost / Cost impact None Cost-effectiveness Single indicator: Weight loss (kg), blood glucose control (HbA1c) deaths averted, life years saved… Cost-utility Combined index: Quality Adjusted Life Years (QALY) Cost-benefit Monetary value: Willingness to pay (£) These are the main types (also CCA etc) Different types of economic evaluation are defined by the choice of outcome unit. CUA is the preferred option for NICE – but sometimes QALY estimation is difficult, so CEA may be used. Could mention CMA as well (but this is not a ‘true’ economic evaluation). Note that these terms are often misused in the literature - authors call a CMA a CBA, or confuse CBA and CEA. Warning – ‘cost-effectiveness’ often used as general term for any analysis with non-monetary measure of health gain
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Assessing Cost-Effectiveness
High extra cost Low QALY gain £/QALY Cost (£) Treatment cost-effective in shaded region CE threshold ~£20-30K New treatment dominated Low extra cost High QALY gain Using the concept of incremental costs and incremental effects we define the cost-effectiveness plane. We use this to illustrate the relative cost and effect of an intervention compared to some control. If the intervention lies in the south-east quadrant, it is both less expensive and more effective than the control, and so will be preferred. The ICER is illustrated by the slope of a line through the origin and the IE/IC point for an intervention. Compare this with the ‘threshold’ cost-effectiveness ratio – assume around £20,000 to £30,000 per QALY for NICE decisions. In the north-west quadrant, an intervention is more expensive and less effective, and so the control will be preferred. In the other two quadrants there is a trade-off between cost and effectiveness. South-west quadrant: CE interventions are cheaper, but less effective. These are rare examples and difficult decisions to make-taking something away is somehow ‘different’. Perhaps the original decision to implement an intervention was made in error/was too generous etc? The north-east quadrant is where most of NICE decision-making lies. Effect (QALYs) New treatment dominates
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Not set in stone – other considerations:-
The Threshold If one intervention does not ‘dominate’ then we use a threshold of £ k QALY to decide Not set in stone – other considerations:- Health gain not captured in the analysis End of life criteria Significant innovation Certainty around the estimate Theoretical basis - £20k/Q represents maximum WTP in a perfectly allocatively efficient NHS
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A single trial may not capture all the relevant information
Modelling – Why Do It? Trials are of limited duration and so may not capture all costs and benefits A single trial may not capture all the relevant information Not all the relevant outcomes (e.g. adverse effects, quality of life, costs) Not all the relevant comparators [there may be several relevant options that are not feasible to examine concurrently in one single trial] More than one study may addresses the clinical decision Trials often report intermediate outcomes e.g. Blood pressure reduction An intervention may have both positive and negative effects There may not be a trial – social care and public health in particular Trials can be very expensive It may be unethical to carry out a trial in a particular area. In a model we could capture these trade offs. When there is no RCT evidence Technology is very new RCT is not practical or is (perceived to be) unethical Diagnostic and screening technologies
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Cost Effectiveness £/QALY
What is a model A mathematical structure used to combines different sources of data Test accuracy Sensitivity/specificity Treatment effects Survival, health status Resource use GP visits, inpatient stays… Preferences QoL weights Unit costs e.g £ per GP visit Epidemiology Baseline risks, sub-groups MODEL So, for example, we may get data: estimates of unit costs from accounting data; data on resource use by reviewing a sample of patients notes; data on test accuracy from a cohort study; on clinical effectiveness from a trial or meta-analysis of trials; estimates of quality of life (health state valuations) by a special study of a sample of patients; and extrapolate findings forward using epidemiological data on survival. We then use a mathematical or computer model to bring together the data and estimate our summary statistic (the ICER). Ideally data inputs will be based on best available evidence from guideline reviews. But in many cases data is not available – so GDG may be asked to use judgement to make assumptions. Cost Effectiveness £/QALY
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Cost-Effectiveness in NICE Guidelines
Principles set out in NICE’s Social Value Judgements: Principle 2 - “Those developing clinical guidelines, technology appraisals or public health guidance must take into account the relative costs and benefits of interventions (their ‘cost effectiveness’) when deciding whether or not to recommend them.” BUT Principle 3 - “Decisions about whether to recommend interventions should not be based on evidence of their relative costs and benefits alone. NICE must consider other factors when developing its guidance, including the need to distribute health resources in the fairest way within society as a whole.” SVJs are: The judgements that NICE and its advisory bodies should apply when making decisions about the effectiveness and cost effectiveness of interventions, especially where such decisions affect the allocation of resources. Second (current) edition published 2008. Now in the process of updating the document, aiming for third edition that also takes account of: NICE’s growing remit, e.g. in public health and social care Legislative developments, e.g. Equality Act 2010
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Any Questions?
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Making decisions in practice
Student Champions Learning about NICE - 27 September 2017 Jane Cowl, Senior Public Involvement Adviser
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What is shared decision making?
“[shared decision making] is a collaborative process that allows patients and their providers to make health care decisions together, taking into account the best scientific evidence available, as well as the patient’s values and preferences” * Quote from a King’s fund report by Angela Coulter and Alf Collins – ‘Making shared decision-making a reality - No decision about me, without me’ - The quote emphasises the key aspects underpinning shared decision making – collaboration, patients and healthcare professionals making a decision together, using evidence and the patient’s values and preferences. Background info about the paper: It clarifies what is meant by the term shared decision-making and what skills and resources are required to implement it and it also outlines what action is needed to make this vision a reality. The principle of shared decision-making in the context of a clinical consultation is that it should: support patients to articulate their understanding of their condition and of what they hope treatment (or self-management support) will achieve inform patients about their condition, about the treatment or support options available, and about the benefits and risks of each ensure that patients and clinicians arrive at a decision based on mutual understanding of this information record and implement the decision reached. The paper outlines the importance of communication skills and sets out how clinicians might approach consultations to arrive at shared decisions. It also suggests that tools that help patients in making decisions are just as important as guidelines for clinicians. Preference-sensitive decision making More than one clinically reasonable and cost-effective treatment or care option (including the possibility of no treatment) exists The choice between options involves the individual person concerned weighing up significant trade-offs according to their preferences and values *Angela Coulter and Alf Collins, The King’s Fund, 2011
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All NICE guidelines and technology appraisals have text on their landing page that states, in effect: When exercising their judgement, professionals are expected to take the guidance fully into account, alongside the individual needs, preferences and values of their patients or service users The application of the recommendations in the guidance is not mandatory and is at the discretion of health professionals and their individual patients The guidance does not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian Example of decisions that are ‘preference-sensitive’ include: Deciding between radical and more conservative treatment for breast cancer Deciding whether to take a statin to reduce the risk of coronary artery disease or stroke Contraceptive options Deciding whether to or not take HRT to relieve the symptoms of menopause
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SDM – Two sources of expertise
Coulter A, Collins A, Kings Fund report 2011 Clinician’s expertise Patient’s expertise Diagnosis Experience of illness Disease aetiology Social circumstances Prognosis Attitude to risk Treatment options Values Outcome probabilities Preferences MAKING SHARED DECISION-MAKING A REALITY: No decision about me, without me (Coulter A, Collins A, Kings Fund 2011) Pages 10 & 11: Shared decision-making may involve negotiation and compromise, but at its heart is the recognition that clinicians and patients bring different but equally important forms of expertise to the decision-making process. The clinician’s expertise is based on knowledge of the diagnosis, likely prognosis, treatment and support options and the range of possible outcomes based on population data; the patient knows about the impact of the condition on their daily life, and their personal attitude to risk, values and preferences. In shared decision-making the patient’s knowledge and preferences are taken into account, alongside the clinician’s expertise, and the decisions they reach in agreement with each other are informed by research evidence on effective treatment, care or support strategies.
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Patient decision aids 105 studies involving 31,043 participants
Stacey D, Legare F, Lewis K, et al (2017) Cochrane database CD001431 105 studies involving 31,043 participants Decision aids increased Participants’ knowledge Accuracy of risk perceptions Congruency between informed values and care choices Decision aids decreased Decisional conflict related to feeling uninformed Indecision about personal values The proportion of people who were passive in decision-making
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NICE patient decision aids
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From the NICE patient decision aid
“What is my choice? You can choose to take a medicine every day for 5 years to reduce your risk of developing breast cancer. But you do not have to take it: there are pros and cons. The medicine NICE recommends for most women who have been through the menopause is called anastrozole. However, this may not be suitable for all women; for example, women who have severe osteoporosis. NICE recommends that, if anastrozole is not suitable or they prefer not to take it, women could think about taking one of two other medicines, called tamoxifen and raloxifene. The key points to think about are as follows: No one can say for certain what will happen to an individual woman. Being at moderately increased risk does not mean that you will definitely develop breast cancer. If you take one of the medicines you will be less likely to develop breast cancer than if you don’t take it. However, some women who take one of the medicines will still develop breast cancer. Each medicine reduces your risk of breast cancer by a different amount. Taking any of the medicines has not been shown to make a difference to your chance of dying from breast cancer compared with women similar to you who don’t take one of the medicines. Taking any of the medicines might give you side effects, although not every woman gets these. Each medicine has a different pattern of possible side effects. If you start taking a medicine to reduce your risk of breast cancer you can stop at any time. …….”
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Key components Information Patient goals or values Meaningful involvement
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What does the option involve?
What difference will it make to my chance of getting a blood clot such as deep vein thrombosis (DVT) or a blood clot in the lungs (pulmonary embolism)? What difference will it make to my chance of developing breast cancer? What difference will taking it make to my chance of getting endometrial cancer (cancer of the womb)? What difference will it make to my chance of a bone fracture? What are the other common side effects?
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“I’m not sure what the right answer is, so why don’t you decide”
“… I believe that finding the sweet spot for shared decision making will require clinicians to work against their natural impulses to tell the patient what to do when they’re certain of what’s best, and to leave the patient to decide when they’re not “I’m not sure what the right answer is, so why don’t you decide” can be replaced with: “This is a really hard decision because we aren’t sure what will happen if you choose option x; let me show you how I think about this, and you can tell me whether it fits with what’s important to you.” And, equally important, “I’m recommending option x because it provides better outcomes than option y” can become “Let me tell you about the pros and cons of options x and y so that you can decide which one matches your priorities.” Perspective: Shared Decision Making - Finding the Sweet Spot. nej.md/1nfA9nE
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Move from: “What's the matter with you?” To: “What matters to you?”
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Next steps…for everyone
Get to know what decision aids are available Try it out! Spread the word!
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Any questions?
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