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Take it from the PROs
NEW ORLEANS—In a disease like diabetes, in which patient compliance with the treatment regimen is critical, one would expect the patient to be involved in the design of the clinical trial.
"It seems obvious that clinical trials need to have measures to talk to patients about treatment, capture the patient's voice and work with the patient to implement procedures," says Yin Ho, founder and CEO of Context Matters Inc., a startup healthcare information and data analytics platform company based in New York City.
Despite rising interest in real-world outcomes, a recent study performed by Context Matters, which uses data analytics to focus on risk assessment metrics for pharmaceutical and biotechnology products, and Pfizer Inc., a multinational pharmaceutical company with a heavy emphasis on research, demonstrates that less than 10 percent of diabetes clinical trials conducted in the past decade included patient-reported outcomes (PROs) as part of their protocols. The study began when Ashley Jaksa, director of data and analytics at Context Matters, was working with Alexandra Barsdorf, associate director of the PRO Centre of Excellence at Pfizer, to do in-house technology assessments.
"Working with Context Matters has been a very valuable experience. In the past, it was difficult and time-consuming to collect this information, but the company's data curation procedures and analytics platform has allowed us to gain a quick, clear understanding of the use of PROs in diabetes clinical trials in the past decade," Barsdorf says.
The objective of the study was to assess the prevalence and types of PROs used in type 2 diabetes clinical trials. The data analytics tool developed by Context Matters was designed to access and understand complex data that has never before been quantified or aggregated in a tailored, needs-based approach.
"PROs are related to the quality of life and the use of resources," explains Emily Rubenstein, director of research at Context Matters. "In diabetes, where the endpoints are biomarkers, it's important to know whether the patient will stick with the medication for a long time."
Presented in May at the 18th Annual International Society for Pharmacoeconomics and Outcomes Research conference in New Orleans, the study analyzed 622 Phase III and Phase II/III diabetes clinical trials from the clinicaltrials.gov database. Among these trials, only 7.4 percent (47 trials) included PROs as part of their assessment protocols. There were no PROs at all in 2001 and 2002, according to the study results.
"One of the current trends in the pharmaceutical industry is to track real-world outcomes. However, in the area of diabetes, one of the most prevalent diseases in the United States, patient-reported outcomes, which are a commonly used real-world assessment tool, were included in less than 10 percent of clinical trial protocols since the year 2000," says Jaksa.
"These patients are undergoing very intense treatments, and managing the process of resolving glucose levels is not easy," she adds. "We need patient input and PROs to determine whether patients are becoming more or less compliant. Historically, that has not been the case."
The researchers explained that the disease, which often hits minorities, is expected to grow exponentially, and "something needs to be done to bring patients into the mix," according to Rubenstein. Diabetes, she adds, is the consequence of a lot of other conditions, such as obesity, body mass and challenges that increase with the patient's age.
The study concluded that the development of a standardized clinical trial-specific tool that helps measure patient-reported outcomes might lead to wider incorporation of PROs in clinical trial protocols, in line with the interest in real-world outcomes. Specifically, it said that although there are a host of diabetes-specific PROs available, the development of a clinical trial-specific, user-friendly instrument might be needed, given the increase in medicines, especially injectables, coming to market in the future.
"We keep developing new tests and instruments, but it's important to have consistent use of them and make sure the instrument is measuring what needs to be measured," Rubenstein says.
She believes that if regulatory agencies looked at PROs, pharmaceutical companies would be more concerned.
"Everyone is trying to bring diabetes rates down, but we don't know how to use the information and how to implement it," she adds.
According to Jaksa, "The answer is going to come from people being more aware that there isn't a consistent way of using PROs to develop a consistent way of measurement. When there is general awareness of that, things will change. "