The requirement for demonstrating evidence of efficacy for proposed interventions in rare diseases is similar to common diseases. The intervention(s) should have been previously studied and represent a realistic choice for patients and clinicians. However, if the existing evidence base regarding the intervention(s) for rare diseases is limited, it may be acceptable to show evidence of efficacy for a particular intervention in a different disease where some theoretical and/or biological propositions on such linkage have been developed. In these instances, the investigator should justify how the available evidence would be applicable to the rare disease of interest.
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