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First, although NEAR is a similar approach to a binary composite endpoint defining "efficacy without ADR", it is not exactly equivalent.
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].
We have included this discussion, even though sibutramine has been suspended from use, because it can teach us a great deal about the difficulties encountered in defining efficacy criteria for the development of new drugs.
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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.
Data were extracted by study methodology and defined efficacy measures.
In the analysis (see further below) both the continuous variable PRR and the binary variable of ≥ 90% parasite reduction were used, the former to define efficacy at 12 hours, and the latter to define efficacy at 24 hours.
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Justyna Jupowicz-Kozak
CEO of Professional Science Editing for Scientists @ prosciediting.com