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Background: N-of-1 trials may enhance therapeutic precision by predicting the long-term effectiveness of medical treatment on an individual basis. However, the n-of-1 approach has gained little traction with the clinical community. To learn why, we interviewed physicians and patients, focusing on the perceived benefits and drawbacks of n-of-1 trials and factors influencing these perceptions., Methods: We convened focus groups and individual interviews with 21 physicians and 32 patients, most with chronic conditions. The study employed qualitative interview methods to explore and analyze subjects' views of n-of-1 trials. Analysis involved an iterative process of review and data abstraction after specific topics for coding, definitions of codes, and strategies for abstraction had been established. Previously defined domains and topics were then expanded and enriched, with key themes emerging during the analytic process., Results: Physicians and patients remarked on 4 salient aspects of n-of-1 trials: scientific, relational, clinical, and logistical. Neither physicians nor patients were highly familiar with the n-of-1 concept, but both groups readily grasped the fundamental logic and appreciated the potential scientific benefits. Physicians saw n-of-1 trials as promoting an exciting but possibly threatening paradigm shift in the doctor-patient relationship, while patients viewed the relational consequences as modest. The best n-of-1 candidates were felt to be proactive, cognitively intact, reliable, motivated, and engaged in a trusting physician-patient relationship., Conclusions: Researchers interested in expanding the appeal of n-of-1 trials will need to address these concerns by carefully explaining the approach, emphasizing the benefits, and minimizing the effort required of doctors and patients.

We surveyed 57 caregivers of children with Cornelia de Lange syndrome (CdLS) using a self-report questionnaire designed to elicit their experiences with receiving information regarding health complications associated with this multisystem disorder. Their most frequent sources of information at the time of diagnosis were a health care provider (86%) and the CdLS Foundation (72%). Although most caregivers (82%) indicated a desire to receive information about all possible CdLS related health complications, on average they recalled receiving information on only 64% of their child's current or future health complications. For two of the top three complications (as ranked by caregivers), information was available to fewer than half of the caregivers at diagnosis. Only 40% of caregivers were satisfied with the amount of information received at diagnosis, while only 45% felt that the information they received was useful. Caregivers indicated a preference for receiving information in written format (59%) or verbally (39%). Our results indicate that caregivers of children with CdLS report a high need for information at diagnosis, regarding their child's actual or potential health complications. Dissatisfaction may result both from a deficiency in information provided, as well as a mismatch between issues mentioned and those that caregivers deem most important. Caregivers may benefit maximally from receiving information in person at the time of diagnosis as well as having an additional written source of information. These findings highlight the importance of complementary sources of information for caregivers and are likely to be relevant for other multisystem disorders., ((c) 2006 Wiley-Liss, Inc.)