The field of medical product development is constantly evolving, with patient-centered approaches gaining importance. Patients’ experiences and perspectives are invaluable in understanding the efficacy and impact of medical interventions. Traditionally, clinical trials and research studies have relied on clinician-reported data and objective measurements. However, in recent years, inclusion of patient-reported outcomes (PROs) has emerged as a valuable resource to inform all stages of the product development life cycle. What is the significance of patient self-reported data in supporting medical product development and how it can positively impact key stages of the development process?
What are Patient Reported Outcomes (PRO)?
A patient-reported outcome (PRO) is data obtained directly from patients and is based on a patient’s perception of a disease and its treatment. PROs are important because they provide a patient perspective on a disease/treatment that might not be captured by a clinical measurement. This information is collected through validated questionnaires or surveys, where patients self-report their experiences; these responses come directly from the patient, without interpretation of the patient’s response by a clinician or anyone else. Traditionally, PROs have been collected using a paper-based questionnaire, but as with most paper forms, many labs have recently migrated to electronic forms, also known as electronic Patient Reported Outcomes (ePRO). PROs offer a unique perspective as they reflect the patients’ feelings, preferences, and day-to-day experiences with a medical condition or treatment. Some examples of PROs include pain levels, physical functioning, mental health, and overall satisfaction with treatment.
How are Patient Reported Outcomes used in the Product Development Life Cycle?
- Conceptualization (Pre-clinical):
The product development life cycle starts with conceptualization. In this phase, ideas for new medical products and treatments are brainstormed, evaluated, and selected to move on to the clinical development phase. By engaging patients early in the process, medical developers can gain valuable insights into patient perspectives, preferences, and priorities, ensuring that the product’s concept aligns with actual patient demands. Incorporating patient self-reported data during this phase can be instrumental in identifying unmet patient needs and provide a good foundation for creating a desirable final product that patients will want.
- Feasibility Assessment:
During the planning stage, the design is conceptualized. The concept is based on customer needs and technical requirements. The feasibility of the concept is assessed. Product developers evaluate the practicality and viability of their proposed medical product. There is a prototype analysis, various go-to-market strategies are developed, and an initial testing takes place in order to collect user feedback. The patient’s perspective regarding the product’s design is taken into consideration before moving on to further development steps.
- Design and Development:
Patient self-reported data plays a pivotal role in the design and development phase. PROs better capture negative, uncomfortable, and impactful symptoms from the patient’s perspective, such as nausea and anxiety. Therefore, they are better at identifying potential risks and side effects, enabling developers to proactively address safety concerns. Incorporating patient perspectives can enhance the usability, functionality, and overall patient experience of the medical product.
- Validation/Clinical Trials:
Clinical trials are critical in determining a medical product’s safety and efficacy. By utilizing patient self-reported data in these trials, developers can assess patient outcomes and experiences with the product under real-world conditions. PROs measure the subjective elements of patients’ conditions, including health-related quality of life, pain intensity, activity limitations, participation restrictions, satisfaction or adherence to treatment and help to evaluate the burden of disease and treatment from patients’ perspectives. Integrating PROs into clinical trial data not only enhances the data’s comprehensiveness but also validates the product’s efficacy from a patient’s standpoint.
- Launch and Regulatory Approval:
Regulatory bodies such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are increasingly recognizing the significance of patient self-reported data in evaluating product efficacy and safety. They should still be supported by objective or functional outcomes but by including PROs in clinical trial data submissions, companies can provide regulators with a more comprehensive view of the product’s benefits and potential risks.
6. Post-Market Surveillance:
Once a medical product enters the market, the company must continually monitor its safety and continue to gather patient feedback. Once on the market, it will be sold and used by a more diverse population and therefore may lead to unexpected results. PRO is one kind of data that researchers can use to track adverse events, product shortcomings, and areas for improvement. Analysis of this feedback can be used for continuous product updates that provide better results for unmet patient needs.
By capturing patients’ experiences and perspectives, patient self-reported data can enrich a clinician’s understanding of the patients’ experience with unique information that could not be gained from biomedical outcomes alone. Integrating PROs into the product development life cycle allows for improved clinical outcomes, accelerated regulatory approvals, and faster product improvements. Companies should embrace patient-centric approaches as they will not only lead to more effective and user-friendly treatments, but also foster stronger patient-company relationships built on trust and empathy.
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