November 15, 2024
By Dwight Akerman, OD, MBA, FAAO, FBCLA, FIACLE
Photo Credit: Getty Images
In the article “Putting efficacy into context: do controlled trials on efficacy of myopia control translate to a clinical population?” authors Stephanie Kearney and Mhairi Day address significant concerns over the rising prevalence of juvenile-onset myopia and the effectiveness of various interventions aimed at controlling this condition. The increasing rates of myopia, particularly in young children, are alarming, as high myopia is associated with serious eye conditions such as myopic macular degeneration, which can lead to visual impairment. This context underscores the urgency of evaluating whether the findings from randomized controlled clinical trials on myopia therapies can be effectively applied in real-world clinical settings.
The article begins by outlining the growing global interest in myopia control and the diverse range of optical and pharmacological interventions available. These interventions seek to slow axial elongation, the primary cause of myopia. However, the authors highlight a crucial challenge — the stringent inclusion and exclusion criteria that are typically used in clinical trials. These criteria often limit participation to children with specific characteristics, such as those with lower levels of myopia and minimal astigmatism. Moreover, very young children (under 6 years) or older teens (over 12 or 13 years), as well as those with specific binocular vision problems, are frequently excluded.
This restricted participant profile raises questions about the generalizability of trial results. Kearney and Day emphasize that the real-world population of children with myopia is more diverse and may not fit these criteria. As a result, the efficacy demonstrated in randomized controlled trials may not fully translate to broader clinical practice. The authors bring attention to issues such as compliance and motivation, which tend to vary significantly outside of trial settings. In controlled environments, adherence to treatment protocols is closely monitored, and participants are usually more motivated by the trial’s structure. However, in routine clinical practice, the commitment level of parents and children could differ markedly, potentially impacting treatment outcomes.
The authors delve deeper into the implications of these factors on treatment effectiveness. They point out that while clinical trials provide critical insights and data, the variations found in a more relaxed, real-world setting could lead to different treatment effects. For instance, the effectiveness of interventions that rely on daily adherence — such as contact lenses or atropine eye drops — might be diminished if compliance is not monitored. The findings from the research underscore the necessity for practitioners to consider how these factors may influence individual cases when applying trial data to clinical practice.
Kearney and Day also review existing literature on the translation of trial results into clinical settings. They highlight a previous case series that explored the practical application of clinical trial data on myopia control, revealing significant variations in outcomes based on patient demographics and treatment adherence. Such studies stress the significance of understanding the context in which treatments are applied, as well as the diverse range of patient responses to myopia control measures.
An additional focus of the paper is on the portrayal of efficacy in literature. The authors discuss how various studies report positive outcomes in controlled settings. However, they caution that this portrayal can create a potentially misleading perception of how well such interventions will perform in a typical clinical population. The disparity between controlled trial conditions and the realities of everyday clinical practice can lead to overconfidence in treatment efficacy.
Through their research review, Kearney and Day advocate for a broader consideration of patient demographics and treatment environments when evaluating myopia management strategies. They emphasize that enhancing treatment efficacy in the clinical population may require strategies that go beyond those detailed in controlled trials. Such strategies could include tailored interventions that account for individual patient needs and varying compliance levels — a move towards more personalized myopia management.
The article concludes with calls for further research to better understand the real-world applicability of trial findings. Kearney and Day suggest that future studies should strive to include a more diverse population, reflecting the various ages, levels of myopia, and other relevant factors commonly encountered in practice. By conducting research that mirrors clinical situations, investigators can provide more accurate and valuable data for practitioners.
Abstract
Putting Efficacy into Context: Do Controlled Trials on Efficacy of Myopia Control Translate to a Clinical Population?
Stephanie Kearney, Mhairi Day
Concerns surrounding the global increase in the prevalence of myopia have led to the development of various optical and pharmacological interventions that aim to slow axial elongation within the myopic eye. This is of particular importance when considering that myopia, particularly high myopia, increases the risk of diseases such as maculopathy and subsequent visual impairment. Children recruited to clinical trials must meet a set of stringent inclusion and exclusion criteria. Typically, children with high amounts of myopia and moderate to high amounts of astigmatism are excluded. Additionally, preschool (younger than 6 years) and post-primary school (older than 12 or 13 years) children are typically excluded from trials, as are those with binocular vision issues. This leads to the question of how applicable research trials are to a more diverse clinic population, that is, the real world, particularly when compliance is not monitored as a stringent requirement as within clinical trials and where motivation and commitment among parents and children may be less than in a clinical trial. The application of clinical trial data in myopia management is a topic that has been investigated within a case series of patients and with a larger clinic population further presented and undergoing further analysis. Here, we want to consider how efficacy is portrayed in literature, the variation it results in for treatment effect, and the applicability of research on the efficacy of intervention from clinical trials to clinical practice.
Kearney, S., & Day, M. (2024). Putting efficacy into context: do controlled trials on efficacy of myopia control translate to a clinical population?. BMJ open ophthalmology, 9(1).
DOI: 10.1136/bmjophth-2024-001921
