Science of Patient Input (SPI)

Part 1:  The first of three, in a series about the science of patient input

It is tempting to think of the ultimate goal of healthcare as solving a health problem, curing a disease, or saving a life.  But at its root, healthcare is simply intended to improve a patient’s quality of life in some meaningful way.  Most of the time these goals overlap, but it is a big assumption to believe that all patients have the same priorities or expectations with regard to management of their health conditions.  Details about the specific health conditions, prognoses, treatment availability, cost, and patients’ social cultures all factor in their preferences.  So when designing and evaluating medical devices, what better guide to use than patients themselves?

At first blush, it is difficult to imagine how a typical patient could provide meaningful technical input, when most are not engineers or research experts or scientists, but no one has a better understanding of outcomes of import, or desired functionality and use characteristics (in cases where the patient is also the end user).  Patients can certainly provide useful feedback on device development regarding unmet needs, likes and dislikes of current products, inconveniences, and impracticalities under real-world circumstances.  Patient attitudes toward use of the product can provide valuable insight into its eventual market adoption.  Patients can explain which clinical outcomes matter most to them or even what amount of change would constitute a significant improvement for them.  Most importantly, patients can help us understand the level of risk they are willing to accept in order for a chance at achieving those desired outcomes.

Clinical trial sponsors may use those patient-determined thresholds for significant improvement and acceptable risks to create hypotheses and endpoints to use in trials evaluating medical devices.  FDA has stated a willingness to consider such patient preference data when evaluating the safety and effectiveness of a product under review for marketing approval.  In June 2018, FDA released a draft guidance on the topic, “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders.”  The title of the guidance specifies “drug development”, but the introduction clarifies that the guidance applies to medical product development, which it defines as “a drug, biological product, or medical device.”  The information may be used “for medical product development and regulatory decision making.”  This guidance is intended as the first in a series from FDA to provide recommendations and tools for collecting patient experience data.  For further information, FDA has previously provided two other associated guidances, “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims” and “Patient Preference Information – Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemptions, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling”.  Outside of FDA, valuable tools and best-practices recommendations for patient engagement are also available in materials from other groups such as Clinical Trials Transformation Initiative (CTTI) and Patient-Centered Outcomes Research Institute (PCORI) who offer them for free on their respective websites.

Please check back for additional posts describing the potential uses of patient preference information in designing more efficient clinical trials that are more likely to be successful and thus could save companies significant time and resources.

If you have any questions about how to design more efficient, cost-effective clinical trials, please call us at 855.463.1633 or visit our website


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