Patient preferences Flashcards
why do patient preferences matter?
- Perceptions influence behaviour, and behaviour influences health outcomes, lifestyle and compliance to treatment
- Health Belief Model: Beliefs about susceptibility to disease, severity of disease are modifiable: Through education, beliefs can be reshaped to influence health behaviours and ultimately improve outcomes.
- Ethical and efficient drug development: Minimises investment waste by aligning treatments with what patients are willing to accept - patient-focused drug dev
- Core to NHS strategy and patient-focused drug development models
how are patient beliefs important in drug use?
Patient beliefs, expectations, and preferences are modifiable predictors of:
- Treatment uptake / adherence
- Use of health services
- Clinical outcomes
are we good at predicting who will respond to RA treatment? how does this impact patient preferences?
Not good at predicting who will respond to treatment in RA:
- treatment responses aren’t universal - not clear which patients will respond and which won’t
- Treatments for RA have side effects – infection risk
- Change lifestyle
- These are preference sensitive – personal choice to have treatments and change lifestyle
Patient preferences differ from physician preferences
what are common preference-sensitive decisions of patients?
- Side effects and lifestyle impacts (e.g., fertility, career, daily functioning) affect acceptance.
- Patients weigh benefits and harms differently than physicians.
- Decisions are complex and context-dependent, especially in immunotherapy where treatment may have:
o Delayed benefits
o Long-term risk
o Impact on daily life or identity
what is the importance of shared decision-making?
Recognises that patients and clinicians may value outcomes differently.
Requires:
o Transparent communication
o Understanding of individual preferences
o Joint decisions based on clinical evidence and patient values
what are key treatment attributes?
efficacy: inflammation, pain, fatigue, depression
side effects: mild (nausea, rash), negative impact on quality of life e.g. hospital visits, serious but not fatal, fatal
lifestyle changes e.g. avoid pregnancy, alcohol
method of administration
frequency, duration, convenience
cost
These factor into patient preferences:
Different people value different treatment attributes
Many variables – complex decision
what can patient preference studies tell us?
What treatment attributes are most important to patients?
What is the relative importance of treatment attributes?
How do patients trade off risks and benefits?
How much does this vary across patients?
e.g. option A: lowest risk but less benefit vs option B: highest risk but greater benefit
when can patient preference studies inform drug development?
Patient preference data about benefits and risk can inform endpoints of clinical trials and result evaluation
- patient-driven trial
- Patients can be involved in trial design
Regulatory review – whether drug is approved
- Patients are present on the board
should patient preferences be included in clinical trials?
yes, patients should be involved in:
- Trial design (selecting relevant outcomes, acceptable risk thresholds)
- Evaluation and interpretation of results
- Approval processes (e.g., EMA, NICE)
- Improves relevance, regulatory decision-making, and patient adherence.
what are common issues with preference studies?
Are the methods comparable? Should different methods be used in different settings
No standardised guidelines for how to assess preferences in drug development.
Preferences can change depending on context, e.g. by disease stage, personal experience, culture
- Hard to measure
- Can have irrational preferences under uncertainty
Weigh up benefit and risk
- e.g. 5% risk of side effect is still uncertain
- May not be a rational decision – generates unreliable data
Need to present info to people in a way that doesn’t bias their response - question wording can influence answer
Preferences may not be transferable between populations
- They may not be valid in the real word
Preference data collection/ elicitation needs to be incorporated in drug development, but methodological questions can generate bias - skews response
what methods can be used in patient preference studies?
Qualitative methods:
- See what is important to patients when deciding on treatments
- Used to identify key attributes and context (e.g., interviews, focus groups).
- Nominal group technique
- Inform design of quantitative study, but can’t give estimate
Quantifiable/quantitative methods
- many options
- translate insights from qualitative methods into measurable data
what is an example of a qualitative method for patient preferences?
Example: Focus groups with RA at-risk patients to identify treatment concerns (n=21).
- Lifestyle intervention preferred
- Factors: physician recommendation, evidence base, duration, administration, cost.
what is crucial to ensure when doing patient preference studies and presenting info about a disease or treatment?
Give info without biasing it
e.g. Design informative but non-biased introduction to RA
Discuss pros and cons of treatment
What would you need to know to inform decision
Rank these – semi-quantitative – relative importance of attributes
what are the two main methods for quantitative approaches?
Threshold Technique
Discrete Choice Experiments (DCE)
what is the threshold technique?
cheaper, simpler quantitative method:
- Increase degree of risk until someone changes choice
- Only one risk variable is toggled/altered at a time
- Shows how much risk is accepted for benefit
- lacks full trade-off insights
e.g. how much risk of serious infection is acceptable to patients for 40% reduction risk of disease until they opt out of treatment
what is the discrete choice experiment (DCE)?
gold standard for quantifying preferences
- patients decide between hypothetical treatments with different risk/benefits
- quantifies trade-offs patients make between risk/benefit - estimates amount of risk someone accepts for certain amount of benefits
outputs:
- relative attributable importance
- maximum acceptable risk for given benefits
expensive, large cohort needed, specialist software, time-consuming
what is the IMI-PREFER project?
EU-wide initiative to integrate patient preferences in drug development.
Work Packages (WP):
- WP2: Methodology – literature review, stakeholder consultation
- WP3: Case studies (e.g. RA)
- WP4: Recommendations for EMA and EUnetHTA
Goal: Develop evidence-based recommendations for preference incorporation
what qualitative case study was used in IMI-PREFER? what did they find?
RA prevention
- qualitative study: explore preferences for treatments to prevent RA development
- 16 members of public + 5 first degree relatives (risk of getting RA) - these people don’t currently have RA, so need information
- focus groups and interviews
- preferences for preventative treatment: abatacept, HCQ
- side effects: mild (nausea), serious (pneumonia and risk of hospitalisation)
Findings:
- serious side effects like pneumonia deterred people from treatment if they lacked RA symptoms, but if they did have RA symptoms they would have treatment despite risk of infection
- 40% risk for RA was deemed too low by participants to inform taking a preventative therapy
what are the practical applications of patient preferences?
Used to inform:
o Regulatory approval
o Clinical guideline development
o Shared decision-making tools
Ensures that new treatments are aligned with what patients actually want and accept
summary:
Patient preferences are essential for:
o Enhancing treatment adherence
o Informing trial design and regulatory decisions
o Improving health outcomes
Preference elicitation must be:
o Unbiased
o Context-sensitive
o Methodologically robust
