An article published in the Orphanet Journal of Rare Diseases discusses how rare disease patients and caregivers make benefit-risk assessments for therapeutic options that may be potentially available to them. The survey conducted by the authors show that when faced with a hypothetical treatment, treatments that have the highest chance of working were given the highest priority. The authors have noted the pragmatic nature of the responses, where the patients and caregivers are ready to tolerate a moderate level of side-effects for a hypothetical treatment. In fact, the authors report that the patient and caregivers were willing to trade side-effects for a higher response from the drug.
OrphaNews April 21, 2017
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