How Folia Health is aligned with FDA guidances on Patient-Focused Drug Development
The FDA published in April 2023 the draft of the last of a four-part guidance series on “Patient-Focused Drug Development (PFDD)”, intended to enhance the incorporation of the patient’s voice in drug development initiatives. Guidance 4 addresses methodologies, standards, and technologies for the collection, capture, storage, and analysis of clinical outcome assessment (COA) data.
In the pursuit of patient-centric healthcare, the FDA has emphasized the importance of considering various aspects of health when evaluating treatment benefits. This recognition is particularly relevant for diseases with diverse clinical manifestations that affect patients differently. Folia Health is at the forefront of aligning with FDA PFDD guidelines, specifically methods discussed in Guidance 4 related to implementing personalized endpoints that prioritize individual patient experiences and unique health concerns. In this blog post, we will explore the advantages of personalized endpoint measurement in complex diseases, how Folia Health's methods align with the FDA's guidance, and the advantages this approach bring’s to life sciences research.
Understanding Personalized Endpoints:
Traditionally, clinical trials often rely on standardized assessments measured through the development and use of validated COA/PRO instruments. However, in conditions where disease manifestations vary widely, such as autoimmune, neurological, behavioral, and many rare diseases, the FDA has recognized that a one-size-fits-all approach will not adequately capture the diverse impacts on patients' health. Standardized assessments are just not built to tell the full story of disease burden and treatment impact. In fact, the currently accepted method for developing these assessments actually artificially restricts measurement in the targeted disease population they are intended to measure. Concepts are removed or discarded from the final “validated” survey directly due to the high patient burden that would be required to answer the volume of survey questions required to capture wide variations of symptoms, different organs and areas impacted, and outcomes most important to the individual. In Guidance 4, the FDA acknowledges the need to consider multiple aspects of health and to weigh the strengths and limitations of various approaches in evaluating treatment benefit.
The Advantages of Personalized Endpoint Measurement:
Screenshot from FDA’s PFDD Guidance #4: The Advantages of Personalized Endpoints
Personalized endpoints place patients at the heart of decision-making, ensuring that their unique concerns are captured and considered during the trial. For instance, why ask all patients about fatigue through a standardized fatigue specific assessment in a study, when some patients with the disease do not care about or experience fatigue? Additionally, by taking this one-size-fits-all approach to outcomes measurement, those patients who are not experiencing fatigue are actually diluting the meaningful changes experienced by patients whose fatigue is a primary symptom of concern. An approach that measures individual endpoints legitimizes patients’ unique experiences, thus increasing engagement during the data collection period.
Another advantage to personalized endpoints is that they will enable a comprehensive assessment of treatment benefits in the aggregate study population that doesn’t exist in the current one-size-fits-all COA/PRO methods used. When we focus measurement on the most meaningful changes each individual cares about the most, researchers will see the full, multi-faceted impact and value of that therapy in the target disease population. When capturing meaningful changes to each patient’s most bothersome symptoms, multiple dimensions of disease impact can be measured, symptom based phenotypes will become apparent, and researchers can finally have a path to meaningfully measure population heterogeneity and varying symptoms within rare and/or complex disease populations.
A question still remains for researchers though. How can we capture and measure the comprehensive experiences and varying symptoms that represent the full burden of disease when evaluating treatment efficacy?
Folia Health's Approach:
Folia Health recognizes the significance of personalized outcomes, as they will reflect what is truly important to each individual patient. We have specifically built a technology platform and novel data capture methodology that prioritizes each individual’s personal perspectives of changes in health that are experienced over time. These methods have been informed by years of allowing patients to tell us what matters most to them, personally. By allowing patients to identify and monitor their most bothersome aspects of their individual disease experiences they become the experts to identifying changes in health and therapy effectiveness, instead of answering survey assessments that are designed with methods that artificially restrict measurement concepts for target populations or are not relevant to the changes experienced.
Folia’s approach of focusing on how individual patients experience changes over time to their specific baseline symptom burden has allowed us to develop advanced analytics focusing on both the n-of-1 as well as full population outcomes analysis. These methods ensure that study insights and results will encompass the full burden of living with a disease. Therefore researchers will now be able to measure the comprehensive impact of novel treatments in the study population. By enhancing the accuracy and relevance of the data collected and analyzed for each individual, Folia’s unique ability to measure personalized outcomes endpoints will ultimately lead to more patient-centered results in drug development.
Folia Health's commitment to Patient-Focused Drug Development is revolutionizing the way that evidence for new therapies is created and evaluated. By implementing personalized endpoints that reflect the unique experiences and concerns of individual patients, Folia Health ensures that outcomes are patient-focused, comprehensive, and meaningful. Through our proprietary and innovative analytical methods methods, Folia Health is advancing patient-centric drug development and ushering in a new era of healthcare that truly prioritizes the needs and perspectives of patients.