Patient-Reported Outcomes in Clinical Trials of Rare Diseases
نویسندگان
چکیده
منابع مشابه
Patient-reported outcomes in neurofibromatosis and schwannomatosis clinical trials.
OBJECTIVES Neurofibromatosis (NF) is a genetic disease with multiple clinical manifestations that can significantly impact quality of life (QOL). Clinical trials should include patient-reported outcomes (PROs) as endpoints to assess treatment effects on various aspects of QOL, but there is no consensus on the selection and use of such measures in NF. This article describes the PRO Working Group...
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There is general acceptance of the importance of incorporating patient-reported outcome (PRO) measures including health-related quality of life (HRQOL) into clinical trials, and there are now a number of guidance documents available on how to use PRO's for regulatory authorities and in comparative effectiveness research. The methods used to collect, analyse and report PRO data in clinical trial...
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Primary brain tumors and their treatment are associated with a significant impact on function and quality of life (QOL). Patient-reported outcomes (PROs) are measures that allow report of the impact directly from the patient. Instruments to measure both QOL and symptom burden have been developed for use in the primary brain tumor patient population. Use of these instruments coupled with tumor r...
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Every cancer treatment, irrespective of its clinical effectiveness, has an impact on patients' quality of life (QoL). Even recently developed targeted therapies might have side effects and significantly impact patients' QoL. Thus, understanding the advantages and disadvantages of different treatments from the patient's standpoint has become a must in clinical research and is highly valued by ma...
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BACKGROUND Assessment of symptoms or disease improvement by study participants is an important aspect of assessing new dermatological therapies in clinical trials, especially for chronic skin diseases that lack objective severity markers. OBJECTIVES We sought to determine the frequency and prominence of reporting of participants' subjective efficacy outcomes in dermatological clinical trials....
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ژورنال
عنوان ژورنال: Journal of General Internal Medicine
سال: 2014
ISSN: 0884-8734,1525-1497
DOI: 10.1007/s11606-014-2892-z