The FDA is working to bring patients’ perspectives to its process of evaluating microinvasive glaucoma surgery (MIGS) devices, according to Tieuvi Nguyen, PhD, chief of the agency’s Intraocular and Corneal Implants Branch.
“The next step . . . is to find ways to incorporate the patient’s voice,” said Dr. Nguyen in a panel discussion at the 6th annual Glaucoma 360 New Horizons Forum.
Two years ago, the FDA and the American Glaucoma Society (AGS) convened to discuss the best clinical trial design for MIGS. That process resulted in new guidance promulgated Dec. 15, 2015, Dr. Nguyen said.
In addition, on Aug. 24, 2016, the agency issued guidance on using patient preference information.
This information informs endpoints or effect sizes for regulatory studies, subgroup considerations and labeling changes or expanded indications, Dr. Nguyen said.
In addition, the agency is trying to make more use of patient-reported outcomes for regulatory studies and post-market monitoring in a way that is useful to payers, providers, and patients themselves, she said.
Submissions that include patient-reported outcomes have increased significantly in recent years, both in total numbers and as a proportion of those submissions that got approved, Dr. Nguyen said.
The FDA is collaborating with “centers of excellence in regulatory science and innovation” in a program that currently includes the University of Maryland, Georgetown University, Johns Hopkins University, the University of California, San Francisco (UCSF), and the Yale-Mayo Clinic.
Johns Hopkins is working on quantitative assessment of patient preferences in glaucoma treatment, with a focus on MIGS devices. It is defining characteristics patients can use to describe outcomes, then conducting one-on-one interviews to identify patient-centric outcomes.
It will prepare a quantitative study to assess the compromises that patients are willing to make when weighing treatment options, thereby eliciting the preferences they have for particular attributes and outcomes.
UCSF in collaboration with Stanford University is assessing health-related quality of life in mild to moderate glaucoma. After completing physician and patient focus group discussions, it is now working on a web-based, patient-related outcome measure and plans more interviews of subjects to design a questionnaire that can be used in psychometric evaluations and quantitative validations studies.
The AGS meanwhile is reviewing existing measures of glaucoma patient-reported outcomes and how applicable they are to milder forms of the disease. The society is also providing patients and experts for the university studies, and engaging glaucoma support groups to become more active in clinical trials.
Following Dr. Nguyen’s presentation, Rick Lewis, MD, chief medical officer at Aerie Pharmaceuticals and Gary Novack, PhD, president of PharmaLogic Development queried Dr. Nguyen about endpoints being used for MIGS trials.
Dr. Novack asked whether the FDA would allow comparisons between MIGS devices in the future, as opposed to comparisons between MIGS with cataract surgery to cataract surgery alone.
“Certainly, people can approach it that way,” Dr. Nguyen responded.
Similarly, she said, the agency is open to considering lower IOPs as endpoints that those used in studies up until now. Applicants wishing to use any of these approaches should discuss it in the pre-submission process.
Wiley Chambers, MD, supervisory medical officer of the FDA’s Division of Transplant and Ophthalmology Products was present to answer a question from Dr. Lewis about six drugs that were not approved last year because of manufacturing problems.
“There has been a trend that is different from the past related to chemistry manufacturing,” Dr. Chambers responded.
In the past, new drug applications used to list multiple drug source manufacturers, sterilization facilities, and drug product facilities. Now some applications list only one of each.
“If any one of those three fail, you no longer have a pathway, and if you don’t have a pathway to manufacture a product under good drug manufacturing procedures, we can’t approve the product,” he said.
Finally, Lewis asked how the 21st Century Cures Act based by Congress last year would affect ophthalmic drugs and device approvals. Dr. Chambers responded that the approval process for ophthalmic drugs would not be affected because his division is either already following the procedures mandated by the act, or else not affected by them.
Dr. Nguyen said her division is working to reduce burdens in the approval process.