Choosing treatment: indication, evidence, benefit, burden and alternatives

MED3 — Choosing treatment: indication, evidence, benefit, burden and alternatives

• Assess whether a medicine has a current indication and meaningful goal.
• Integrate evidence with patient-specific risks and preferences.
• Recognise prescribing cascades and indication drift.
• Compare medicine treatment with non-drug and watchful-waiting options where appropriate.
• Explain uncertainty and support shared decision-making.

Guidelines inform care but do not prescribe for an abstract average patient. Evidence must be interpreted alongside age, frailty, multimorbidity, renal and hepatic function, previous treatment response, treatment burden and what outcomes matter to the patient. The same medicine can be high value for one person and low value for another.

Medicines often outlive the circumstances in which they were started. A proton-pump inhibitor may remain after a short protective indication has ended; a sedative may persist after an acute crisis; an antihypertensive regimen may become excessive after weight loss or frailty progression. Review should therefore ask not only whether the drug appears on the repeat list, but whether the original indication still exists and whether the intended outcome remains relevant.

A prescribing cascade occurs when an adverse effect is interpreted as a new disease and treated with another medicine. Examples include postural symptoms, ankle swelling, constipation, sedation or confusion. Before adding treatment for a new symptom, review whether the existing regimen could be contributing. This is particularly important in older people, where adverse effects may present atypically.

Preventive therapies may offer benefit only after months or years. In advanced frailty or severe competing illness, the likely time to benefit may exceed the period in which the patient is likely to experience that benefit, while adverse effects and monitoring burden occur immediately. This does not justify blanket age-based stopping; it justifies explicit discussion of goals and proportionality.

Burden includes more than pill count. It can include frequent blood tests, complex timing, swallowing difficulty, device technique, dietary restrictions, interactions with other treatment, travel to appointments and the cognitive work of managing a regimen. A clinically modest benefit may not be worthwhile if the burden is high from the patient’s perspective.

Safe prescribing includes considering whether a medicine is needed at all. Non-drug approaches, observation, addressing a precipitant, correcting dehydration or reviewing another medicine may sometimes be preferable. Equally, ‘no treatment’ can have consequences and should be discussed honestly. The patient should understand likely benefits, material risks, reasonable alternatives and what may happen if treatment is not used.

Shared decisions under uncertainty

Uncertainty should not be disguised as certainty. Clinicians can explain what is known, what is uncertain, what outcomes are being prioritised and when the decision will be reviewed. A time-limited therapeutic trial with agreed success and stopping criteria is often more informative than an indefinite prescription.

Sources for this module

• GMC prescribing guidance
• NICE NG5: Medicines optimisation
• AWTTC: Polypharmacy in older people

Educational note: medicine-specific doses, thresholds and rapidly changing regulatory requirements should be checked against current authoritative guidance at the point of care.