Research Points to Inconsistent Use of Patient Experience Data in FDA Approvals
Sanofi is pressing the U.S. Food and Drug Administration (FDA) to keep its patient experience data consistent following a review of approved applications required by the 21st Century Cures Act.
According to the federal program, the FDA is required to include a brief statement of any patient experience data (PED) or other related patient information that was included in New Drug Applications, Biologic License Applications and other applications submitted to the regulatory agency after June 16, 2017. However, an examination of patient experience data included in drug approvals from the FDA in 2018 show a consistent inconsistency, Regulatory Affairs Professional Society publication RAPS first reported. In an article called “FDA Reported Use of Patient Experience Data in 2018 Drug Approvals,” the researchers showed that not all drugs approved last year included the patient experience data and noted that if the data is expected to be a factor in determining efficacy and safety, then standards should be upheld.
According to the research, patient experience data refers to the systematic collection of meaningful data relating to the experiences, perspectives, needs, and priorities of patients. The 21st Century Cures Act requires the FDA to make a public statement about the PED considered in the approval of a drug application, the researchers noted. PED includes the “collection of data concerning the symptoms of their condition and its natural history; the impact of the condition on their functioning and quality of life; patient experience with treatments; input on which outcomes are important to them; patient treatment preferences; and (6) the relative importance of any issue as defined by patients,” the researchers noted.
The Sanofi researchers perused the data related to the 59 approvals of novel drugs and biologics and determined that 48 include a table that summarized whether patient experience data was or was not used during the FDA drug review. Of those 48 approvals, 34 (78%) reported using patient experience data in the review.
“Patient-reported outcomes (PROs) represented the most significant source of PED and were used in 60.4% of approved drug reviews. Additional findings, including PED use by FDA review division and by FDA regulatory designation, are described,” the researchers noted.
In the report, the researchers go on to point out that while PED was used in many of the approvals, there was a lack of consistent formatting between the approvals due to non-specificity in the 21st Century Cures Act requirement.
Some of the findings show that applications with orphan designation were less likely to include PED, as were applications that received both fast track designation from the FDA and were approved under priority review, RAPS reported.
The researchers hope that their findings will encourage the FDA to become more consistent with PED in the future. They also hope that the FDA could make PED more accessible to individuals through the use of a “patient-facing statement describing FDA's use and application of PED for each approval.”
“This analysis should help develop a baseline regarding FDA use of PED and may inform decisions to ensure patients’ experiences are adequately heard in future drug development,” the researchers said in conclusion.