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39. Evaluation of benefits

39. Evaluation of benefits

All drugs have an effect on our body: part of the effect is desired (to prevent, treat or cure a given disease, or ameliorate its symptoms), but part of the effects may be undesired (adverse events). A medicine is authorised to be used in humans when regulators estimate that the desired effects on the disease outweigh the undesired effects on body.

A benefit-risk analysis logically begins with a discussion of the benefits, since the beneficial effects are the basis for use of a drug or vaccine. 

Below are few factors considered but not limited for benefit assessment: 

The epidemiology and natural history of the target disease risk in populations and impact of early intervention on the disease or consequences of no intervention.

Purpose or intended outcome of the treatment: to prevent disease (e.g., vaccine), to prevent the recurrence of disease (e.g., antibiotic prophylaxis for otitis media), treat an acute condition (e.g., streptococcal pharyngitis), treat symptoms of a self-limiting condition (e.g., a decongestant), reduce the risk of a serious outcome (e.g., treatment of hypertension, hypercholesterolaemia or osteoporosis), to prevent progression of disease (thrombolytic therapy), to treat chronic disabling symptoms (e.g., NSAIDs for chronic
arthritis), to reduce or delay morbidity or mortality among patients (e.g., treatment of AIDS or cancer)

Evidence of benefits: Degree of efficacy achieved in clinical trials and effectiveness in clinical practice

If there is no alternative therapy for target disease, what is the effect of no treatment or a non-drug intervention incase of no alternative therapy.

If there are other viable alternative therapies: Data availability on comparable populations from studies such as tolerability, convenience or patient preference.

The type of benefits: Medicine impact on clinical management, patient health, and patient satisfaction in the target population, such as significantly improving patient management and quality of life, reducing the probability of death, aiding improvement of patient function, reducing the probability of loss of function, and providing relief from symptoms. 

The magnitude of the benefit(s) – Assess benefit along a scale or according to specific endpoints or criteria (types of benefits), or by evaluating whether a pre-identified health threshold was achieved. 

The probability of the patient experiencing one or more benefit(s) – based on the data provided, it is sometimes possible to predict which patients may experience a benefit, whereas other times this cannot be well predicted. The data may show that a benefit may be experienced only by a small portion of patients in the target population, or, on the other hand, that a benefit may occur frequently in patients throughout the target population. 

A large benefit experienced by a small proportion of participants may raise different considerations than does a small benefit experienced by a large proportion of participants. For example, a large benefit, even if experienced by a small population, may be significant enough to outweigh risks, whereas a small benefit may not, unless experienced by a large population of participants.

The duration of effect(s) (i.e., how long the benefit can be expected to last for the patient) – some treatments are curative, whereas, some may need to be repeated frequently over the patient’s lifetime. To the extent that it is known, the duration of a treatment’s effect may directly influence how its benefit is defined. Treatments that must be repeated over time may introduce greater risk, or the benefit experienced may diminish each time the treatment is repeated.

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