In the ever-evolving landscape of healthcare and clinical research, patient-centricity has become increasingly paramount. Patient Reported Outcomes (PROs) are at the heart of this paradigm shift.
Patient Reported Outcomes are health outcomes that come directly from patients, without interpretation by clinicians or any other third party. These can include subjective reports on symptoms, quality of life, functional status, and treatment satisfaction. PROs provide a unique window into the patient experience, one that objective clinical outcome measures alone cannot capture.
A Shift Towards Patient-Centricity
Healthcare has traditionally relied heavily on outcomes measured by clinicians and biomarkers to assess novel treatments. While these are undoubtedly important, they often fail to capture the full picture of a patient's experience with a condition or its treatment. Take for example a new treatment that may control the shaking and stiffness for patients suffering from Parkinson’s disease. However, if the treatment causes tremendous drowsiness to the point where patients may not be alert enough to interact with their family, is that considered a success? The move towards patient-centricity acknowledges that patients are experts in their own experiences and that their perspectives are invaluable in developing effective treatments.
There are several reasons why PROs are considered critical inputs in developing true patient-centered treatments:
- Capturing the Patient Experience: PROs allow us to understand aspects of health that are known only to patients. For instance, pain levels, fatigue, or the impact of a condition on daily life are subjective experiences that can't be fully measured by external observation. By incorporating PROs, researchers ensure that treatments address what truly matter to patients
- Improving Treatment Efficacy: When the broader picture of patient experience is better understood, researchers can develop more effective treatments. PROs can reveal side effects or quality of life impacts that may not be readily apparent through traditional clinical outcome measures. This can guide modifications to treatments, making sure they are more tolerable and effective from the patient’s perspective
- Facilitating Comparative Effectiveness Research: PROs allow for more comprehensive comparisons between different treatment modalities. By including patient-centered metrics, researchers can evaluate treatments not just based on their clinical efficacy, but on their overall impact on patients' lives
- Supporting Regulatory Decisions: Regulatory bodies like the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) [RS1] are increasingly recognizing the value of PROs in evaluating new treatments. PRO data can provide compelling evidence of a treatment's benefits in terms that are directly relevant to patients, potentially influencing approval decisions
- Identifying Unmet Needs: With the use of PROs, researchers can uncover aspects of diseases or treatments that may be overlooked when relying solely on traditional clinical measures. Such insights can help drive innovations that address previously unrecognized patient needs
- Better Patient Engagement and Satisfaction: Incorporating the use of PROs in clinical trials and treatment assessment allows patients to have active participation in their healthcare. This engagement can lead to increased satisfaction with care, better adherence to treatment regimens, and ultimately, improved health outcomes
- Assisting Clinical Decision Making: PROs can help inform shared decision-making between patients and healthcare providers. When clinicians have access to PRO data, they can make more informed decisions that align with patient preferences, values and experiences
- Increasing the Value of Healthcare: By recognizing what matters most for the patients and enabling patients and healthcare providers to better communicate about treatment goals and expectations. At a higher level, provisions can be made to address gaps in delivery system, such as anticipation of complementary services or changes in care settings such as counseling before surgery, or at-home rehabilitation.
Challenges and Considerations When Implementing PROs
While the benefits of PROs are clear, their implementation is not without challenges. Ensuring the validity and reliability of PRO measures, standardizing data collection, integrating PRO data with other clinical information and appropriate interpretation of findings are ongoing areas of development in the field. Particularly, the interpretation of PRO data requires careful consideration due to cultural differences, individual patient perspectives, and the differing contexts of data collection. As reliance on PROs is increasing across multiple sectors of medical research and clinical care, robust methodologies for their collection and analysis should be established.
In summary, PROs bridge the gap between clinical efficacy and real-world effectiveness, ensuring that treatments not only work from a medical standpoint but also meaningfully improve patients' lives. As researchers continue to advance treatment development, PROs will undoubtedly play an increasingly central role in shaping a healthcare system that truly puts patients first.
By enabling, encouraging and supporting the use of PROs, Mapi Research Trust is proud to help foster a patient-centered approach to treatment development, and build a future where treatments are not only clinically effective, but also profoundly improve in the lives of those they aim to help.