Measuring outcomes: an ethical premise in management of childhood disability

Abstract

The theme of the 22nd Annual Meeting of the European Academy of Childhood Disability (EACD), which takes place in Brussels from the 27th to 29th May 2010, is ‘Measures of Progress – Evaluating management outcome in childhood disability’. In recent years, measuring and monitoring outcomes has become a critical issue in this field. The scope of evaluation has broadened considerably in the context of the World Health Organization’s classification of functioning, disability, and health, and also as a result of increasing recognition of individual child- and environment-related factors in modifying outcome. In addition, major conceptual and technological advances in the management of children with neurodevelopmental disabilities have brought about new challenges with respect to assessment, intervention, and research in habilitation outcomes. Indeed, realistic expectations based on objective knowledge of the efficacy of proposed treatment should be an ethical imperative when devising a management plan for a child with disabilities. In the opening lecture, delivered (significantly) at the European Parliament, Nigel Paneth thus stresses that the first obligation of the physician is to establish that the treatment works. A number of practices are still carried out because it is assumed that they ‘work’. Many are based on questionable theories and established either by tradition or by hope placed in novelty. The phrase ‘it works’ is oft-repeated as a wishful, almost self-evident mantra. What works? On what? How does it tell us which children might benefit from intervention, what types of interventions should be organized, and what outcomes of intervention might be anticipated? This cannot be accepted unchallenged lest management be based on (self-) deceit. Outcome assessment is further complicated in childhood disability by the fact that the objectives of habilitation are very rarely normalization. Treatment planning often relies on a stepwise process that involves a complex relationship between a normative situation and carefully defined groups of ‘atypical’ children. The sequence of questions can be articulated as follows: (1) How do typical children function in the considered area? (2) Why do they do so? (3) How do the atypical children function in this area? (4) Why don’t they do as the typical children? (5) Why do they do as they do? (6) What would be better for them in the long term? And finally, (7) how can they be helped to achieve this? Appropriate tools for measuring the impact of intervention are needed. What is their clinimetric validity (e.g. Ashworth scale, goal attainment scaling)? Which items are relevant and when should they be assessed with respect to intervention (e.g. computer-assisted gait analysis, neuroimaging)? How is this information integrated in a multidisciplinary and real-life setting? As Martin Bax points out, multidisciplinarity must involve not only health professionals but also those with training in education and social science and, crucially, the individual with disabilities and the family. Perhaps even more than other journals, Developmental Medicine & Child Neurology can pride itself on a long tradition of presenting papers addressing the many aspects of the ‘Measures of Progress’, from specifically designed questionnaires, observational studies, and clinical scales to biochemical, neurophysiological, or kinematic approaches, and functional and structural neuroimaging techniques. Many subjects are covered, including assessment of neurological development, cognition, behaviour, communication, motor control, participation, and quality of life. Evaluation is thoroughly discussed and much evidence is presented. Clearly, the greatest challenge posed by evidence-based practice lies in the application of evidence into clinical practice. But it must be recognized that sound evidence is still lacking in most domains of childhood disability. As Hans Forssberg, chairman of the EACD, notes in the concluding lecture, study groups are often small and heterogeneous. Moreover, there is an urgent need for more adequate, robust, validated outcome measures to produce the evidence. It is often difficult to control the many intervening factors as well as to apply a life-course perspective. So we are all left with the same duty of evaluating intervention in order to guide clinical decision-making, and optimize social participation and quality of life of the child and family. Evaluation is neither a goal nor an outcome in itself, nor should it be a one-off action. It should be an unremitting, essentially participatory process, as it is a necessary instrument for the continuous improvement of quality in the management of childhood disability.