Patient-Reported Outcomes and the Elephant in the Conference Room

Did your hospital do anything with your patient reported outcome of your stroke rehab? YOU MEAN YOU DIDN'T HAVE ANYONE ASKING YOU ABOUT HOW WELL YOUR STROKE PROTOCOLS GOT YOU TO 100% RECOVERY?

HOW FUCKING INCOMPETENT IS YOUR HOSPITAL?

Patient-Reported Outcomes and the Elephant in the Conference Room 

Patient-reported outcomes (PROs), defined as outcomes reported by patients without filtering or interpretation by others, are an important part of patient-centered research and care. The applications of PROs are varied and complex. They sit like an elephant in a room full of people, each of whom is touching his or her small part. No person can see beyond what is directly in front of him or her to the full picture.
At FasterCures, we see great potential in more effectively using PROs to advance the science of patient input. This year we are launching a new project under our Patients Count program to help provide a wider angle view of PROs and to reveal the big picture of their potential. The time is right for this approach.
While many agree in theory that PROs used in research and clinical care should be based on what patients report as being most important to their disease management, health and well-being, this has often not been adequately considered. For example, a PRO might capture whether a patient with arthritis can dress him or herself. In the patient’s experience, however, the time it takes to dress is a better measure of how well he or she is doing. This misalignment of measures hurts efforts to create patient-centered research and care.
Making PROs themselves truly patient-centered is one element of the challenge ahead. Another challenge is aligning various stakeholders’ interest in, and use of, PROs. As a starting point, we have identified the relevant parties and their interest or stake in PROs.
Patients
Patients and caregivers have the most “skin in the game.” There is no substitute for patients being able to base treatment decisions on outcomes that a) matter to them, b) are gathered from large numbers of patients like them and c) are specific to the treatment options available. Patients are eager for information collected from them to be used to improve and accelerate patient-centered discovery, design, development and delivery of medical products.
Drug and Device Companies
Companies want the PRO data they collect to be used in regulatory decision-making and the information they communicate to health-care providers, payers and patients about their medical products. Companies include PROs in clinical trials to help understand efficacy, safety, adverse events, tolerability and other potential benefits and harms. In spite of this investment, a recent study documented a steady decline in PRO data on labels for new drug applications. While companies are collecting PROs, the information about a product’s effect on these measures is not routinely making it to patients and providers for care decision-making.
Clinics
Hospital systems and provider groups use PROs and other tools to understand clinical care outcomes, or the way particular care approaches affect patient health and health-care utilization at the population level. As the health-care system as a whole moves toward value-based health care, there is a greater focus on engaging patients and measuring clinical outcomes that matter to them, rather than simply billing for services rendered.
Clinicians
Physicians and other health-care providers frequently use PROs to assess how well individual patients feel or function as a basis for shared decision-making about care options. And they use PROs to monitor how well an individual is doing on his or her course of therapy.
Payers
There is growing recognition that PROs can be useful in establishing the value and comparative effectiveness of medical interventions, both of which are important factors for payers determining medical coverage and reimbursement policies.
Researchers
Researchers apply their expertise to develop PROs that capture the domains of interest and meet scientific standards sufficient to be used in decision-making.
Regulators
The Food and Drug Administration (FDA) encourages sponsors to use PROs to help demonstrate the safety and efficacy of drugs and medical devices, both in clinical trials and after products are made available to patients. In spite of numerous efforts, including a 2009 guidance outlining a process to validate PROs, many companies feel the expected standards are too rigid and the process too lengthy and unpredictable. There is great interest in resetting expectations, and FDA has committed to revisiting its guidance by 2021.
Patient Organizations
Many patient organizations are contributing to efforts to create patient-centered PROs specific to their condition. They also assist in collecting PROs through patient registries and other means that can describe the natural history of the condition, inform research and regulatory decisions, and improve patient care and access.
With so many parties taking a stake in PRO development and utilization, and each from a different vantage point, it’s easy to understand why this particular elephant is tough to get in full view. Our objective at FasterCures will be to invite stakeholders to step back, take a broader look and approach the challenges from a more holistic perspective. From there we hope to identify opportunities to streamline the path to making PROs a stronger foundation for the science of patient input.