Datamonitor attended this year's meeting of the American Thoracic Society in New Orleans. The 2010 conference was defined not only by the latest clinical trial data, but by new guidelines and an emerging discussion of the role of personalized treatment. Conclusions were also presented from the ISAAC study, highlighting some surprising global prevalence trends.
Following an update in 2009, guidelines from the Global Initiative for Asthma (GINA) now define a patient's level of severity as being either controlled, partly controlled or uncontrolled in recognition of the fact that severity is a product of both the underlying disease and a patient's responsiveness to treatment. Consistent with this, Dr Elis Bel introduced a new 2010 American Thoracic Society (ATS)/European Respiratory Society (ERS) task force definition of severe refractory asthma at this year's ATS meeting. The definition focuses on the maintenance (or lack of) control experienced by a patient receiving high-dose inhaled corticosteroids (ICSs) plus a second controller and/or systemic corticosteroids.
Guidelines received considerable attention at the meeting, particularly in the context of treatment. Of particular focus was the distinction between 'one size fits all' treatment and personalized medicine, with guidelines accused of being too homogenous to be of any real use. Dr E. D. Bateman argued in favor of the GINA guidelines, and was quick to point out that the latest guidelines include a definition of 'difficult to treat' asthma, which allows for a degree of discretion on the side of the treating physician. He maintained that the GINA guidelines are of considerable value, particularly in the first approach for treating patients, with the potential for personalized medicine in the case of refractory patients.
In response, Sweden's Dr L.H. Bjermer took the stage to argue against the guidelines and the 'one size fits all' approach. He noted that the individual characteristics of patients, including even factors such as personality traits, can be fundamental in determining outcomes. To this end, he cited a study in which patients with neuroticism had high levels of compliance and poor outcomes, while extraverts practiced low compliance but were found to be well controlled. He concluded that there is only occasional overlap between guidelines and real life in asthma treatment.
Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines were also debated, with a focus on whether or not their use leads to optimal chronic obstructive pulmonary disease (COPD) treatment. Dr Sonia Buist supported the guidelines, claiming that not only have they led to increased awareness of the disease, but also that they have, or at least will, lead to improved outcomes, pointing out that time is required for increased knowledge to be applied.
In contrast, Dr Dirkje Postma pointed out the flaws of the GOLD guidelines. Significantly, she called into question the use of forced expiratory volume (FEV1) as a tool for both diagnosis and monitoring of the disease, suggesting that spirometry neglects the heterogeneity of the disease and patient outcomes, and leads to overestimation (and therefore over-treatment) at older ages as lung function declines naturally with aging. The fact that the guidelines never allow for adding ICS treatments to mild/moderate patients was also highlighted, as those patients are known to experience some exacerbations, which the drugs could reduce.
These debates have several implications for the industry. In discussing both asthma (via GINA guidelines) and COPD (via GOLD guidelines), the need for considering the individual patient in determining treatment was highlighted. For asthma this was primarily considered for difficult to treat patients, while for COPD the recognition that mild and moderate patients suffer from some exacerbations but are not on maintenance therapy to address them is an example of an issue to tackle. The way in which the majority of asthma/COPD patients are treated is not expected to see much change, but a crucial subgroup - namely, those who are poorly controlled - will continue to make their way to the forefront of both discussions and clinical alterations.
Furthermore, the issues highlighted pertaining to the use of FEV1 as an endpoint for COPD clinical trials illustrate the need for continued innovation and diligence in trial design in order to obtain optimal treatment.
Discussions on the role of guidelines together with the tweaked definitions pertaining to classifications of severity led to considerations of personalized medicine. Dr K.F. Chung discussed the potential movement from standardized to 'boutique' therapy. He highlighted the potential benefits of such a movement, and noted that for successful treatment physicians should have a relationship with the patient for at least three to six months for evaluation. However, he was also quick to point out that cost is and will remain a key barrier to personalized therapy, giving the example of the National Institute for Clinical Excellence's highly restrictive guidelines for treating asthma with Xolair (omalizumab). Datamonitor therefore expects that, for both convenience and cost, personalized medicine only has a future with the most severe patients who are poorly managed on standard of care. Still, for these patients, it could be the key to obtaining control.
Elsewhere at the conference, data from the Phase III ISAAC study (Lai et al., 2009) were discussed. The results show that severe asthma is most prevalent in the most populous countries in the world, such as Asia, Africa and Latin America, indicating that the burden of asthma is increasing. Furthermore, time trend data now available show a surprising decreasing trend in UK and New Zealand prevalence (both areas with very high prevalence), such that global disparities between asthma prevalence seem to be decreasing.