— Vikas Mehta, MD, MPH
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April 2024PROMs are important in assessing the burden of disease and effectiveness of treatment, “because we can’t always rely on objective measures to show that treatments, like surgery and medications, are improving patients’ health,” Dr. Mehta said. Sometimes, he added, “it’s just whether the patient feels better or not. PROMs were created to more accurately measure those things in a standardized and validated fashion.”
As an early faculty member, Dr. Mehta received a master’s in public health at Johns Hopkins University in Baltimore, where he learned the value of PROMs. “From a science perspective, being able to better understand how treatments are impacting patients through quantitative measurements that are reliable, reproducible, and meaningful to patients requires a lot of work in developing, validating, and implementing these measures.”
Many available PROMs haven’t undergone rigorous evaluation to make sure that they correlate with clinically meaningful results. Therefore, any clinician who’s thinking about incorporating one should make sure it has been studied and validated. “We’re a small field, and those of us conducting research know how important it is to have validated research tools and clinical metrics,” Dr. Skirko said. “We want to make sure that anyone helping children and adults has the best access to these tools.”
The Future of PROMs
Dr. Piccirillo is researching the use of ecological momentary assessment (EMA) to capture patient responses more accurately in real time. “Instead of waiting to see the clinician to rate your pain score or symptoms, you could fill out a finite number of questions four times a day, which allows for real-time assessment,” he said. The future will be about how to incorporate electronic digital devices, such as the iWatch or iPhone, for regular assessment outside the physician’s office.
“My recent research has really broadened my understanding of what patient-centered research has become,” Dr. Skirko said. “Patient-centered research is transitioning into an area where not only do we include patient-reported outcomes, but we actually include patients on our research teams. Part of my research now is developing the ability to do patient-centered research in Pierre Robin Sequence,” a rare disorder that involves infant airway obstruction resulting from a small jaw. Included on the team are parents, advocacy groups, speech pathologists, and cleft coordinators. “This enables us to have a more holistic picture when designing clinical research,” Dr. Skirko said.
Electronic digital devices can also help with remote monitoring and telehealth. “Many of our patients are coming from far away, so this could also help with monitoring outcomes,” Dr. Skirko said. “We’re also building teams that are working toward multicenter studies so we can answer some of the harder questions with patient populations that have had broader experiences in a single institution.”
There’s a lot of power in creating registries, Dr. Skirko noted, but a lot of work is required to figure out what’s in the inclusion criteria and who makes the specific diagnosis. “Some conditions are easier to diagnose, but for the rare conditions, there isn’t always uniform consensus on what that diagnosis entails, which can make it really challenging. I think the academy has done a really good job of having some online resources for types of PROMs that have known value and quality.”