Patient experience data: Sanofi researchers call for more consistency from FDA
Researchers at drugmaker Sanofi are calling on the FDA to make its publication of patient experience data (PED) in review documentation more consistent after reviewing applications approved in the first full year after a requirement of the 21st Century Cures Act compelled the agency to do so.
Under Section 3001 of the 21st Century Cures Act, the FDA is required to “publish a brief statement on any patient experience data or related information that was part of the application” for applications submitted after 16 June 2017.
In 2018, the FDA’s Center for Drug Evaluation and Research (CDER) approved 59 novel drugs and biologics. Of those, 48 applications (81.4%) included a Sec. 3001 PED table within the review documentation. Among the 11 products that did not include a PED table, seven were submitted to the FDA prior to the implementation date for Sec. 3001.
Among the 48 applications that included a PED table, the researchers found that 34 (70.8%) reported using PED during the review. Of those 34 applications, 29 (60.4%) included only sponsor-submitted PED, four (8.3%) included sponsor- and nonsponsor-submitted PED and one (2.1%) included only nonsponsor-submitted PED.
The sources of the nonsponsor-submitted PED included the FDA patient-focused drug development meeting summaries, a meeting between the FDA and patient stakeholders and an FDA advisory committee meeting.
The researchers also reviewed the use of PED across products with special regulatory designations, including orphan drug designation, breakthrough therapy designation, fast track designation, priority review and accelerated approval.
Contrary to their expectations, the researchers found that applications with orphan designation were less likely to include PED in their review documentation, with 60.7% of applications with orphan designation including PED compared to 85% of non-orphan applications utilizing PED as part of their review.
The researchers observed a similar trend for applications that received fast track status or a priority review, noting that 61.9% of applications with fast track status and 64.9% of priority reviews included PED, while 77.8% of non-fast track and 90.9% non-priority review applications included PED.
On the other hand, products granted breakthrough therapy designation or accelerated approval were more likely to include PED in their review documentation.
“Inclusion of the PED table within the FDA Review Documentation—which are by nature highly technical and accessed primarily by sponsors and academics—would limit their visibility to stakeholders less familiar with the FDA regulatory process (such as patient groups and healthcare providers),” the researchers write.
The researchers also point out that while the format of the PED tables included in the review documentation were “largely consistent throughout these 48 drug product applications, the use of the table by FDA reviewers varied significantly,” noting that in just over half of the applications (58.3%) the PED table specified the application section where PED was discussed. Additionally, the researchers say that only nine of the 29 applications that included patient reported outcome (PRO) data specified which PRO instrument was used.
“Further, it appears that FDA did not consistently publish a PED table for supplemental applications, which are also approved under FD&C Act 505(b) or PHSA 351(a)—while this information sometimes can be accessed by other means, there is no consolidated, ‘publicly available’ access to this information,” the researchers write.
The researchers highlight the fact that clinical outcome assessments (COAs), including PROs, accounted for nearly three-quarters (73.2%) of the sources of PED in the applications.
While the researchers note that this may be due to both sponsors and the FDA having more experience with COAs compared to other sources of PED. “Based on the results of this review, both the FDA and sponsors should ensure that all forms of PED, beyond COAs, are appropriately being incorporated into drug development,” they write.
Looking ahead, the researchers say they expect the FDA to be more consistent in its handling of PED across its review divisions and argue that the requirements under Section 3001 of the Cures Act should apply to supplemental applications as well.
Additionally, the researchers say that the FDA could make its use of PED more accessible by including a plain language “patient-facing statement describing FDA’s use and application of PED for each approval,” similar to how the agency provides its drug trial snapshots.
The researchers also argue that the FDA should include or link to its use of PED in its reviews of applications in approved labeling. “Without direct inclusion in labeling, or at least a meaningful and accessible linkage of the information to labeling, true publication of FDA’s use of patient input arguably has not been effectuated for the benefit of all users,” the researchers write.
RAPS: First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.