January 8, 2018
The Patient Engagement Collaborative is gearing up for a launch in early 2018. The new, external panel is being built by the FDA and the Clinical Trials Transformation Initiative (CTTI), a public-private partnership between the FDA and Duke University. The panel will act as a forum for discussing new methods to increase patient participation in the agency’s decisionmaking process. It will provide input on incorporating patient-preferences into reviews of medical products and trial protocols to make patient engagement an integral part of drug development.
At the agency, the project will be spearheaded by the newly created Patient Affairs Staff, which coordinates agency-wide patient engagement efforts, best practices and outreach, and promotes public awareness of the agency’s work.
CTTI Executive Director Pamela Tenaerts said that engaging patients as equal partners in development is critical to the success of the clinical research enterprise. She believes researchers too often assume what patients think or would like have happen.
“It’s always better to understand each other’s perspectives,” said Tenaerts.
The new Patient Engagement Collaborative, combined with the FDA’s patient-focused drug development meetings and the Patient Preference Initiative, will lead to improvements, according to Tenaerts. The key is to engage with patients in meaningful ways.
At an FDA advisory committee meeting last year, pharmaceutical companies demonstrated how active patient engagement and gathering feedback — including from test-runs of draft trial protocols and mock clinical visits — can lead to fewer and shorter visits, streamlined consent, and fewer dropouts after enrollment.
Currently, the FDA and CTTI are seeking a diverse group of representatives for the 16-member panel, in order to help the agency best understand how to engage across patient communities, according to Rachel Sherman, the FDA’s principal deputy commissioner. About half of the members will be selected from CTTI member organizations and individuals, although self-nominations are accepted.
The panel will be co-chaired by a representative from the FDA and a committee member, who will create a work plan and a list of discussion topics for quarterly, invitation-only meetings, with summaries made public afterward. Invitations for observers will depend on the topics at hand, Tenaerts said.
CTTI intends to host the panel’s first face-to-face meeting in April, at its biannual meeting.
The FDA is looking for candidates with an understanding of clinical research and experience in dealing with a disease, either personally, as a caregiver, or as a representative of a patient group, including advocacy organizations, public health agencies or nonprofit research foundations.
The idea for the panel was born out of public feedback: in November 2014, the FDA requested comments from the research industry to help develop strategies to better consider the patient perspective during its evaluations and regulatory approvals. A common theme in the responses included the creation of an external group to inform efforts across review centers, the agency said.
The FDA has since held the inaugural meeting of its Patient Engagement Advisory Committee last fall, focusing on incorporating patient views into clinical trial designs for medical devices. The committee is the agency’s first to be completely staffed by patients, caregivers and advocates.
The committee tested a new meeting format, with agency moderators leading roundtable discussions with attendees from the public. Discussed by the committee were methods to boost clinical trial enrollment and retention, how to keep patients engaged through extended follow-ups and how researchers should communicate study results to participants.
“It’s a significant step forward in the FDA’s efforts to broaden its engagement with patients — and to deepen the involvement of patients in our regulatory activities,” said FDA Commissioner Scott Gottlieb at the time.
Center for Devices and Radiological Health (CDRH) Director Jeffrey Shuren called the agency’s previous efforts to seek patient voices woefully limited. A portion of past committee meetings allowed members of the public to speak for only a few minutes at a time, and some only accepted written comments from patients.
In addition, FDA reviewers have begun examining companies’ patient engagement efforts during its 30-day reviews of IDE submissions. According to Owen Faris, director of CDRH’s clinical trials program, reviewers are mainly looking to see whether the right patients will be enrolled in the study; if the patients will be willing and able to adhere to the follow-up visit schedule; and whether study success will equal patient success.
CDRH set a goal to give its entire staff the opportunity to interact directly with patients by the end of 2017. If patients are going to be the ones using the devices, the agency should be taking their perspectives into account, Shuren said.
At December’s FDA/CMS Summit, Shuren reported that 96 percent of CDRH staff had met that goal, and that 80 percent of clinical trials reviewed by the agency had incorporated patient-reported outcomes.
The Patient Engagement Collaborative is only the latest in the FDA’s push for patient input in clinical trials, a measure taken to ensure higher patient recruitment levels, patient adherence to study protocol and fewer protocol amendments to clinical trials.