Exact(4)
As a reviewer has commented, this notion of enriching a clinical trial with likely responders is very appealing, and should lead to more efficient trials.
A more accurate prediction of the right dose might lead to more efficient trials with a consequent saving in patients and costs (Table 1, IP1).
These provide advantages through increased disease severity and a differing array of symptoms, which allow for more efficient trials utilizing smaller sample sizes without the added need of forced exercise protocols.
With a plethora of new treatments in development and no objective gold standard to define efficacy, a better understanding of what the different endpoints signify would be helpful in designing more efficient trials and in informing clinicians in practice who need to interpret the results.
Similar(56)
In addition, better patient stratification may also allow more efficient trial designs.
The use of more refined outcomes and truly neuroprotective drugs, coupled with more efficient trial design, has the capacity to deliver a new era of therapeutic discovery in this challenging area.
However, there is also a growing consensus that the use of EHRs can provide new and unique opportunities to develop more efficient trial processes.
The Academy of Medical Sciences report on the EU directive may lead to more efficient trial approval across the country, with the creation of the Health Technology Authority (Rawlins, 2011).
This multimodality approach can now better identify those subjects with AD-specific traits that are more likely to present cognitive decline in the near future and that might represent the best candidates for smaller but more efficient therapeutic trials – trials that, through gained and shared knowledge, can be more focused on a specific target or a specific stage of the disease process.
Continuous outcome measures can enable smaller, more efficient randomized trials and randomized trials are generally more informative than single-arm studies with historical controls.
The lessons learned from the completed efficacy trials will enable the development of better designed, potentially more efficient efficacy trials in future.
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