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India's patent law does not define "efficacy" or say how it should be measured.
To the Editor: The issue can be reduced to a simple question: How does one define efficacy in cancer treatment?
Outcome assessments are used to define efficacy endpoints when developing a therapy for a disease or condition.
A structured dose escalation design can reveal dose-dependent effects and adverse effects, crossover studies can demonstrate change, presurgical studies can define efficacy, interictal discharges and photosensitivity models can explain changes in seizure features, transcranial magnetic stimulation evaluates hyperexcitability.
Patients may define efficacy in terms that are individual, largely variable from one patient to the other, and may encompass several attributes of a drug (e.g., speed of efficacy, duration of efficacy, tolerability, ease of use, efficacy on associated symptoms, etc).
For example the use of one species (i.e. rodents) to define efficacy and a second species to define safety (i.e. the dog) precludes assessment of therapeutic index until an agent actually enters early phase human studies.
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Data were extracted by study methodology and defined efficacy measures.
The issue is whether a thus defined efficacy allows predicting the outcome of a therapy in the individual patient.
Secondly, IVT-AFL 2q8 versus dexamethasone 0.7 mg implants using an indirect analysis (Bucher) with defined efficacy (gain ≥ 10 letters) and safety (macular edema, reduced visual acuity, vitreous hemorrhage, eye pain, increased intraocular pressure, and cataract) outcomes.
Randomized controlled trials are more appropriate for defining efficacy and assigning causality, but their external validity or generalizability can often be low [ 34] and they are rarely sufficiently large or long running to identify all adverse events [ 35, 36].
It is of paramount importance to recognize that how one defines efficacy – whether one measures only reduction in symptoms or also improved academic achievement, improved peer and family relations, improved classroom behavior, etc. – can determine which treatments one considers effective.
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Justyna Jupowicz-Kozak
CEO of Professional Science Editing for Scientists @ prosciediting.com