Keeping It Real: Finding the Missing Link Between Our Patients and Research
By Christine McDonough, PT, PhD
If consumers are going to "#ChoosePT" to address their movement disorders and functional limitations, they need to perceive physical therapists (PTs) as the preeminent experts in conditions that affect movement, pain, and quality of life. Potential patients and clients look for a strong recommendation from their family, friends, or peers, and perhaps a referral from their treating physician. Physicians and payers—both public and private—want to know that science informs our practice and that our interventions will result in tangible improvements in patient outcomes.
Patient clinical data registries such as the Physical Therapy Outcomes Registry have the power to use large amounts of outcomes data to evaluate outcomes of a given intervention for a specific condition in a specific population—for example, the effects of resistance training on falls in older, community-dwelling women.
This real-world evidence is important, because to provide the best possible patient care and transform the health of our communities, you need to know:
- How to classify patients for accurate clinical decision making
- What interventions work for a particular patient under specific circumstances
- What the prognosis is for patients who undergo treatment
- What kind of home exercise program or behavior changes are necessary to optimize outcomes
Randomized controlled trials (RCTs) are the "gold standard" for establishing the effectiveness of treatments, but their role is changing, as clinical registries enable researchers to understand real-world impacts from patient outcomes data. Evidence developed from clinical data registries such as APTA’s Physical Therapy Outcomes Registry—with their power to use large amounts of data to evaluate outcomes of specific interventions for specific conditions and populations—will fill important gaps in knowledge that will serve PTs and meet the needs of our consumers.
RCTs Are Important but Have Limitations
While RCTs aren’t going away any time soon—and shouldn’t—they are very expensive, and they often take a long time to plan and to complete. It simply is not feasible to conduct RCTs on every intervention available.
Because of the way they are designed, both efficacy and effectiveness RCTs have built-in limitations in determining whether a given intervention will work for the patients you see every day.
Efficacy trials answer the question: "Does the treatment work better than placebo?" If the results of an efficacy trial are positive, researchers can investigate additional questions in subsequent trials. But if the results are negative, the intervention may be abandoned altogether.
These high stakes have an impact on the design of efficacy studies. Because a negative result may mean abandoning a potential intervention after significant financial resources have been devoted to a trial, they are often designed to optimize the possibility of detecting a treatment effect. This is done by designing the trial to answer a focused question, such as "Is this treatment better than no treatment under ideal circumstances?" The studies often include patients with similar characteristics that make them the most likely to benefit, and they exclude more complex patients that might muddy the waters of interpretation.
Effectiveness trials answer the questions: "Is the treatment better than existing treatment?" or "Does the treatment work better when combined with existing treatment?" As with efficacy trials, in order to avoid abandoning effective treatments trial design must balance broad inclusion with detection of treatment effect. Clinical trials are now being designed to more closely represent the real-world environment. These "pragmatic trials" include a broader range of providers and patients, and provide support more aligned with what would occur in real-world implementation.
The Real World Is Not the Ideal World
The effects found in early efficacy and effectiveness studies are not always the same as the effects found in real-world circumstances. There are several important reasons for the differences, including:
- The providers delivering interventions in clinical studies often are different from those providing treatment in the community.
- The patients receiving interventions are more complex than those in clinical studies.
- The range of activities and support provided in clinical trials are not available in actual practice.
So clinical trials are now being designed to more closely represent the real-world environment. These "pragmatic trials" include a broader range of providers and patients, and provide support more aligned with what would occur in real-world implementation.
In a recent blog post on medical registries, Dr Caroline Fife highlighted 10 goals that many registries share. Among them, 6 will be of critical importance to the future of physical therapist practice.
- Provide evidence to support value of interventions and services to stakeholders. The Physical Therapy Outcomes Registry will provide direct evidence of the results of real-world PT services that can be used to communicate our value to clients, patients, payers, and policy makers.
- Submit quality reporting data to CMS (eg, MIPS). For future quality data required of PTs, the Registry can streamline the process.
- Provide benchmarking data to users. Including your data in the Registry will allow you to get real-time feedback to inform and improve clinical decision making and outcomes. High performers can share their success, and everyone will benefit.
- Support quality improvement. Registry data can be used to identify improvement opportunities and to measure the success of quality improvement initiatives.
- Support development of classification and risk stratification tools to improve clinical decision making. As we identify the characteristics that predict response to treatment, we can improve our outcomes and the value of our services
- Provide real-world data for research to fill gaps not covered by traditional research.
There is wide recognition of the value of using data to improve patient care—as it is actually delivered—and help bridge the gap between what is learned in clinical trials and what we need to know. Registries provide this missing link by collecting standardized outcomes data from real-world patients, with all their complexities.
PTs across the country are participating in APTA’s Physical Therapy Outcomes Registry to help bridge the gap. They are pioneering the way for the future of our profession and the future of health care. Visit www.ptoutcomes.com/ to learn more.
Christine McDonough, PT, PhD, is assistant professor in the departments of physical therapy and orthopaedic surgery at the University of Pittsburgh. She also is a member of the Physical Therapy Outcomes Registry Scientific Advisory Panel.