Treating neurodegenerative diseases

What patients want is not what doctors focus on

Parkinson's disease is an excellent example of the challenges of caring posed by people with neurodegenerative disorders. It is insidious in onset, inexorably progressive, of unknown cause, incurable, yet amenable to management with pharmacological and other interventions. With the ageing of the population the prevalence of Parkinson's disease and other such disorders is projected to increase in the years ahead.1 Thus all doctors must be prepared to provide diagnostic and management strategies for this growing population of patients. Medical practitioners must understand the expectations of patients and their families and introduce these perspectives within the framework of scientific understanding and evidence based practice. Conventional medical education has set a tradition of practice based on science, basic and clinical, cemented by a period of postgraduate training in the conventional apprenticeship mode. This has ensured that practices are generally competent and safe and grounded in the best available information. But is this approach consistent with the mission of professionals to build partnerships with patients by means of strategies for care consistent with the knowledge, attitudes, and values of a public most of which is educated.

Do most people believe, for example, that the quality of life of patients with neurodegenerative disorders depends primarily on the severity of disease and the effectiveness of pharmacological interventions? Without a detailed examination of evidence or a familiarity with the risks associated with treatment, patients may have an outlook that differs from that of professionals with respect to health related factors conducive to a better quality of life. Moreover, protocols for the care of patients are likely to derive more from the research interests and focus of investigators than the daily burdens of the people who have the illness.

There is a growing consensus that a lack of congruence exists between what patients and doctors value in terms of the impact of disease on quality of life and what should be done about it. In Parkinson's disease, there is robust evidence in favour of this divergence of perspective which may represent a potential barrier to the effectiveness of protocols for care, guidelines for management, and the most effective and efficient use of health resources.2 When face to face interviews with more than 1000 patients with Parkinson's disease and carers were carried out in six countries only 17.3% of the variation in perceptions of health related quality of life could be explained by the severity of disease and the effectiveness of drug treatment. Such evidence necessarily represents a wake-up call for those health providers who believe that these factors are most important for prognosis and require a large share of professional effort.3

During these interviews, patients were also given the opportunity to complete specially developed questionnaires and validated instruments to identify other domains of care of equal or greater importance which affect the quality of their life. These domains had been identified in pilot studies by the investigators. The salient responses that accounted for approximately 60% of health related quality of life were respondents' mood, satisfaction with the explanation at the time of diagnosis, and current levels of optimism—inclusive of severity of disease and pharmacological interventions. Indeed the mood (depression), as measured by the Beck depression inventory, explained about 40% of variation in the perceived quality of life across this cohort of patients. 2 4 5

Apart from effecting a cure, maintenance and improvement of the health related quality of life are the objectives of any treatment programme for neurodegenerative disorders. The message for clinicians from this and other studies is that, contrary to prevailing opinion, a single minded focus on severity of disease and the effectiveness of drugs will not adequately address the changes in the health related quality of life expected from encounters between patients and doctors. Undergraduate and postgraduate medical education must provide a broader framework that includes the complexities of factors that are interwoven in their efforts to improve quality of life.

Communication skills emerge as paramount tools that together with counselling and behavioural modification contribute substantially to the results of any treatment programme. A recent study assessing the effectiveness of specialist nurses for patients with Parkinson's disease in general practice showed that nurse specialists helped to preserve patients' sense of wellbeing even though health outcomes were unchanged.6 This study emphasises that a major role of the nurse specialist is in counselling and educating patients and carers—that is, as a facilitator of communication. Merely increasing the total number of neurological specialists may not be the best or only solution to these challenging and complex problems. Health planners need to consider the scope and character of diverse and comprehensive skills necessary for improving the quality of life for the growing number of people with neurodegenerative disorders.