FDA guidance ‘a step in the right direction’
The US Food and Drug Administration (FDA) recently published the second of four patient-focused drug development (PFDD) documents as part of its efforts outlined in the 21st Century Cures Act and the FDA Reauthorization Act of 2017 (FDARA) Title I.
With the final two yet to be published, the first guidance discussed potential sampling methods for collecting ‘comprehensive and representative’ patient input.
The second guidance specially addresses how to collect patient data to ensure the information is ‘asked, captured, and measured’ in a way that will lead to proper conclusions and insight, said Erica Prowisor, global head of patient recruitment and retention at Iqvia.
“Patients must be at the center of research,” she added. “To provide the most benefit to patients, it is critical to understand what it is truly like to live with a disease, for patients, their caregivers, and loved ones.”
Identifying what is important to patients is the focus of the second guidance, which also discusses survey methods and qualitative research topics to help avoid misleading results.
Applying these methods to appropriately collect data, adapting to address the needs of specific patient populations, Prowisor said, “the depth and breadth of knowledge can lead to breakthroughs in drug development that would otherwise not be known or possible.”
“With a more complete and accurate understanding of the areas that can improve the everyday lives of patients, stakeholders involved in drug development can prioritize and focus efforts to fill voids and make a more meaningful impact to improve outcomes,” she added, noting that the goal of the guidances is to highlight patients as a key stakeholder and provide methods of engagement.
“Researchers must gain a more in-depth understanding about the populations they are working with to ensure they are focused on the areas that will drive the greatest impact,” Prowser said. “True insights can only occur if the data is captured in an unbiased and representative way that accurately embodies the full patient experience.”
As Prowisor noted, ‘patient centricity’ and ‘big data’ are ubiquitous terms in the discussions about drug development – but it is important to collect the right data to drive the best decisions, she said, calling the guidance ‘a step in the right direction.’
Said Prowisor, “By sharing their current thinking on methods, and eventually how the information could be used for approval decisions, this guidance will help accelerate the science and the use of patient input to inform clinical development programs.”
