Investigators Seek Optimized Ophthalmology Clinical Trial Outcomes


A team of clinicians and investigators are working to define core outcome sets that it hopes will increase the utility of randomized clinical trials and stimulate research.


Investigators from Brown University and Johns Hopkins University shared new considerations from 3 groups working to standardize Core Outcome Sets (COSs), to increase the utility of results from randomized clinical trials for 3 eye conditions: age-related macular degeneration (AMD), refractive error in children, and dry eye.

This new editorial comes from the mindset, lead author Ian J. Saldanha, PhD, MPH, MBBS, assistant professor at Brown, told MD Magazine®, that there is something within ophthalmology care standards that can be fixed.

“We wrote this editorial to inform the world about an initiative we've undertaken to help solve this problem, starting with 3 disease areasa—age-related macular degeneration, refractive error in children, and dry eye,” Saldanha said.

Several other diseases already have standardized COSs, which have expedited research, and helped physicians better use that research in making treatment decisions for patients. The editorial discussed a specific example where after the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) endorsed COSs for rheumatoid arthritis, there was a substantial increase in the proportion of randomized clinical trials that reported these outcomes.

“By promoting COSs developed through stakeholder engagement, regulatory bodies such as the FDA can expedite innovation and product approval,” the authors wrote.

The working groups, made up mostly of ophthalmologists and optometrists in the US, first met during the American Academy of Ophthalmology (AAO) Annual Meeting in November 2017. There was 1 group dedicated to each of the 3 conditions, though some individuals did participate in more than 1 group. Each group was led by a clinician and 3 methodologists. The AMD group included 27 individuals, the refractive error in children had 24, and the dry eye had 39.

“The outcomes working groups we convened include various stakeholders: clinicians who need outcome data from RCTs to treat patients effectively; clinical trialists who choose outcomes in the face of budgetary and other realities; systematic reviewers and guideline developers who are frustrated when inconsistent outcomes among RCTs means that meta-analyses combine data from only a select few RCTs; editors of major ophthalmology journals; the FDA; and the NEI,” the authors wrote.

Over the course of this year, the groups discussed methodological, practical, and disease-specific considerations for developing these COSs. The methodological considerations describe the details that each endpoint should include, such as consistent metrics, measurements, and time points. In terms of practicality the outcomes should take into account the subtypes and severity of each disease, the potential technological advances in measurements, and the possibility that different stakeholders (such as patients as opposed to clinicians) may give varied importance to each measure.

Lastly, the disease-specific considerations must take into account the age of patients and their ability to report outcomes and the inclusion of outcomes for which there are no treatments yet — which may facilitate the development of treatments for important symptoms.

“We are in the early stages of this process,” Saldanha said. “Doctors should know that an important aspect of core outcome development and eventual adoption is making sure that the chosen outcomes are clinically relevant (in addition to being relevant to patients and others who have a stake in this). To that end, we will need to get buy-in from clinicians and make sure that the outcomes chosen for research are those that are relevant to doctors in today's clinical practice.”

Saldanha expressed hope that, once a common metric is found and shared, treatment evaluation will be improved, and recommendations and guides for clinical practice could follow suit.

The article, “Choosing Core Outcomes for Use in Clinical Trials in Ophthalmology: Perspectives from Three Ophthalmology Outcomes Working Groups,” was published online in the American Academy of Ophthalmology Journal.

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