Is it possible to match research outcome measures with patients' expectations?

Article type
Year
Authors
Celani MG1, Bignamini A2, Baiocco L3, Papetti R3, Macone S1, Bassi C1, Candelaresi P1, Cusi C1, Cuzzubbo S1, Giannandrea D1, Mahan K1, Melis M1, Motto C1, Nardi K1, Oppo V1, Pellegrini A1, Piras V1, Siniscalchi A1, Tremolizzo L1, Cantisani TA1
1Cochrane Neurosciences, Italy
2Milan University, Pharmaceutical Sciences, Italy
3Perugia Hospital, Dpt Neurosciences, Italy
Abstract
Background: It is crucial to select outcome measures and their rating scales to assess the meaningful results in chronic and disabling neurological diseases.

Objectives: To address the mismatch between what clinical researchers do and what patients need.

Methods: Epilepsy, amyotrophic lateral sclerosis and traumatic and non-traumatic spinal cord injury were considered. For each disease we performed a systematic review of all randomized control trials (RCTs) to evaluate any intervention published in any language over a period of five years in order to analyse the quality and methodological aspects of clinical research. For each trial, 13 different neurologists filled out a computerized form saved in a database. The evaluation of patient and career needs and emotions were performed with focus group discussions, transcribed into text-files, blindly elaborated into key semantic meanings and analyzed in a semi-quantitative way using 'Concordance' software.

Results: We present preliminary results of our study regarding epilepsy only. We examined 949 published papers; only 167 were truly RCTs. We excluded 322 as not focused on epilepsy, 226 were not randomized, 104 focused on animal and phase I/II trials, 110 were on-going, open label studies, meta-analysis, and 23 were duplicated papers. A total of 61 patients and carers participated (40% of people contacted), they were organized into five groups of patients, and six groups of caregivers. Most frequently expressed needs were 'assistance', expressed three times more frequently by carers than patients, and 'experience sharing' and the 'need for knowledge' expressed twice as frequently by patients. The need for assistance was directly proportional with disease severity, while the need for knowledge was inversely proportional. Emotions most frequently expressed were anger and fear, which were proportional with disease severity, but also hope, resignation and acceptance.

Conclusions: This approach could provide useful strategies to collect end-users of treatments' perspectives, ideas and values to reach an agreement between different stakeholders needs and to promote valuable clinical research.