Take it from the PROs

NEW ORLEANS—In a disease like diabetes, in which patientcompliance with the treatment regimen is critical, one would expect the patientto be involved in the design of the clinical trial.

"It seems obvious that clinical trials need to have measuresto talk to patients about treatment, capture the patient's voice and work withthe patient to implement procedures," says Yin Ho, founder and CEO of ContextMatters Inc., a startup healthcare information and data analytics platformcompany based in New York City.

Despite rising interest in real-world outcomes, a recentstudy performed by Context Matters, which uses data analytics to focus on riskassessment metrics for pharmaceutical and biotechnology products, and PfizerInc., a multinational pharmaceutical company with a heavy emphasis on research,demonstrates that less than 10 percent of diabetes clinical trials conducted inthe past decade included patient-reported outcomes (PROs) as part of theirprotocols. The study began when Ashley Jaksa, director of data and analytics atContext Matters, was working with Alexandra Barsdorf, associate director of thePRO Centre of Excellence at Pfizer, to do in-house technology assessments.

"Working with Context Matters has been a very valuableexperience. In the past, it was difficult and time-consuming to collect thisinformation, but the company's data curation procedures and analytics platformhas allowed us to gain a quick, clear understanding of the use of PROs indiabetes clinical trials in the past decade," Barsdorf says.

The objective of the study was to assess the prevalence andtypes of PROs used in type 2 diabetes clinical trials. The data analytics tooldeveloped by Context Matters was designed to access and understand complex datathat has never before been quantified or aggregated in a tailored, needs-basedapproach.

"PROs are related to the quality of life and the use ofresources," explains Emily Rubenstein, director of research at Context Matters."In diabetes, where the endpoints are biomarkers, it's important to knowwhether the patient will stick with the medication for a long time."

Presented in May at the 18th Annual International Societyfor Pharmacoeconomics and Outcomes Research conference in New Orleans, thestudy analyzed 622 Phase III and Phase II/III diabetes clinical trials from theclinicaltrials.gov database. Among these trials, only 7.4 percent (47 trials)included PROs as part of their assessment protocols. There were no PROs at allin 2001 and 2002, according to the study results.

"One of the current trends in the pharmaceutical industry isto track real-world outcomes. However,in the area of diabetes, one of the most prevalent diseases in the UnitedStates, patient-reported outcomes, which are a commonly used real-worldassessment tool, were included in less than 10 percent of clinical trialprotocols since the year 2000," says Jaksa.

"These patients are undergoing very intense treatments, andmanaging the process of resolving glucose levels is not easy," she adds. "Weneed patient input and PROs to determine whether patients are becoming more orless compliant. Historically, that has not been the case."

The researchers explained that the disease, which often hitsminorities, is expected to grow exponentially, and "something needs to be doneto 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 andchallenges that increase with the patient's age.

The study concluded that the development of a standardizedclinical trial-specific tool that helps measure patient-reported outcomes mightlead to wider incorporation of PROs in clinical trial protocols, in line withthe interest in real-world outcomes. Specifically, it said that although thereare a host of diabetes-specific PROs available, the development of a clinicaltrial-specific, user-friendly instrument might be needed, given the increase inmedicines, especially injectables, coming to market in the future.

"We keep developing new tests and instruments, but it'simportant to have consistent use of them and make sure the instrument ismeasuring 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 wedon't know how to use the information and how to implement it," she adds.

According to Jaksa, "The answer is going to come frompeople being more aware that there isn't a consistent way of using PROs todevelop a consistent way of measurement. When there is general awareness of that,things will change."