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NEI and FDA Address the Use of Patient Reported Outcomes in Medical Product Development

PRO Endpoints</em> Symposium co-chairs Neil Bressler, M.D. (Wilmer Eye Institute/Johns Hopkins) and Rohit Varma, M.D. (Doheny Eye Institute/University of Southern California)
Left to right: PRO Endpoints Symposium co-chairs Neil Bressler, M.D. (Wilmer Eye Institute/Johns Hopkins) and Rohit Varma, M.D. (Doheny Eye Institute/University of Southern California)
On October 13, the National Eye Institute (NEI) and the Food and Drug Administration (FDA) held the third of a series of symposia that engage the vision community in discussions about the types of endpoints appropriate for use in clinical trials that support approvals for new drugs and devices. Entitled Use of Patient-Reported Outcomes in Medical Product Development, the meeting featured representatives from the reviewing divisions within FDAís Center for Drug Evaluation and Research (CDER) and the Center for Devices and Radiological Health (CDRH) that oversee ophthalmic drug and device approvals, respectively. The meeting was organized by the Association for Research in Vision and Ophthalmology (ARVO).

A Patient Reported Outcome (PRO) is a measurement of any aspect of a patientís health status that comes directly from the patient without having been interpreted by a physician or researcher. Because some treatment effects are known only to the patient, PROs are increasingly recognized as an essential component to be considered in the evaluation of drugs and medical devices. Areas commonly assessed by PROs include symptoms, ability to carry out activities of daily living, and perceptions (e.g., concern about the condition or its treatment). A patientís assessment of the impact of an illness and its treatment is referred to as health-related quality of life. These factors are not only important to new medical product evaluation, but are increasingly used in decisions about drug and device reimbursement—and are even being considered in potential health care reform legislation being developed by Congress.

"Simply stated, this meeting will address how patients report their perception of vision in relation to clinical outcomes," stated NEI Director Paul Sieving, M.D., Ph.D., who welcomed attendees and noted the importance of these discussions within the National Institute of Health (NIH). Symposium co-chairs Neil Bressler, M.D. (Wilmer Eye Institute/Johns Hopkins) and Rohit Varma, M.D. (Doheny Eye Institute/University of Southern California) echoed Dr. Sievingís comments, noting the importance of PROs in vision trials and epidemiologic assessments sponsored by the NEI and industry. The issue at hand for this meeting was whether a PRO could eventually be used as a primary endpoint in new product evaluation and, if so, what it would take to develop and validate that PRO, similar to that which is done with clinical data. A corollary issue was the impact of PROs on product labeling claims or other patient information.

Since PROs are increasingly being considered in new product evaluations across the FDA, representatives of its Division of Epidemiology provided insights into their development and use. Many of these concepts are incorporated into a draft FDA guidance document entitled Patient-Reported Outcomes Measures: Use in Medical Product Development to Support Labeling Claims, which will issue in final form shortly on the FDAís Web site. The FDA representatives encouraged attendees to become familiar with the document and to consider meeting early on with the reviewing division in the product approval process to discuss the potential use of PROs in the regulatory submission.

A distinguished panel of vision community representatives subsequently addressed the adequacy of PRO measurement tools within refractive and cataract surgery, glaucoma, and retina. The speakers noted the importance of PROs to vision—such as visual acuity, glare, haloes, night vision, and contrast sensitivity—but acknowledged that quantifying and validating these is not easy, as there is often a disconnect between the functional assessment of vision and that which is reported by patients that must be addressed.

CDER representative Wiley Chambers, M.D., (Acting Director, Division of Anti-Infective and Ophthalmic Products) commented that, for ophthalmic drug approval, there is no requirement for a quality of life study in addition to safety and effectiveness, but if a manufacturer wants to use a PRO in a label claim, then it must be developed and validated. CDRH representative Malvina Eydelman, M.D. (Director, Division of Ophthalmic, Neurological and Ear, Nose and Throat Devices) commented that PROs are not usually primary endpoints, but they are considered in safety reviews of a device, both pre- and post-market, and may be included on separate information sheets to patients. She reported that FDA would shortly announce a collaborative study with the NEI that would use PROs to examine the potential impact on quality of life from Laser-Assisted In Situ Keratomileusis (LASIK), a form of laser eye surgery to improve vision. The study will identify factors that can affect quality of life following LASIK and potentially reduce the risks of adverse effects.

Anne Coleman, M.D. (Jules Stein Eye Institute/University of California-Los Angeles) concluded the formal presentations by identifying challenges to the use of PROs in ophthalmic drug and device approvals, which included developing the appropriate measurements and validating these on an ongoing basis. Dr. Varma concluded the overall meeting by stating that, although the vision community may not yet be prepared to use PROs as primary endpoints, they serve an important current and future role in the evaluation of new treatments.

The PRO Endpoints symposium was developed after a session on quality of life indicators at the second of the joint NEI/FDA meetings, Glaucoma Clinical Drug Trial Design and Endpoints, held in March 2008. The first symposium, Ophthalmic Clinical Trial Design and Endpoints, held in November 2006, focused on new treatments for age-related macular degeneration (AMD) and diabetic retinopathy.

Left to right:  William Rich, III, M.D., F.A.C.S., representing the American Academy of Ophthalmology, NEI Director Paul Sieving, M.D., Ph.D., and Dr. Varma
Left to right: William Rich, III, M.D., F.A.C.S., representing the American Academy of Ophthalmology, NEI Director Paul Sieving, M.D., Ph.D., and Dr. Varma
Left to right: Anne Coleman, M.D. (Jules Stein Eye Institute/University of California-Los Angeles) and NEIís Clinical Director Rick Ferris, M.D.
Left to right: Anne Coleman, M.D. (Jules Stein Eye Institute/University of California-Los Angeles) and NEIís Clinical Director Rick Ferris, M.D.
Left to right: Malvina Eydelman, M.D. (FDA/CDRH, Director, Division of Ophthalmic, Neurological and Ear, Nose and Throat Devices) and Susan Vitale Ph.D. (NEI Division of Epidemiology and Clinical Applications)
Left to right: Malvina Eydelman, M.D. (FDA/CDRH, Director, Division of Ophthalmic, Neurological and Ear, Nose and Throat Devices) and Susan Vitale Ph.D. (NEI Division of Epidemiology and Clinical Applications)
Wiley Chambers, M.D., (FDA/CDER, Acting Director, Division of Anti-Infective and Ophthalmic Products)
Wiley Chambers, M.D., (FDA/CDER, Acting Director, Division of Anti-Infective and Ophthalmic Products)
Left to right:  Robert Weinreb, M.D. (Hamilton Glaucoma Center/University of California-San Diego) and Paul Lee, M.D., J.D. (Duke University) addressed PROs in glaucoma
Left to right: Robert Weinreb, M.D. (Hamilton Glaucoma Center/University of California-San Diego) and Paul Lee, M.D., J.D. (Duke University) addressed PROs in glaucoma
Left to right:  Timothy McMahon, O.D. (University of Illinois at Chicago), Dr. Bressler, and Tracy Valorie (Pfizer Inc)
Left to right: Timothy McMahon, O.D. (University of Illinois at Chicago), Dr. Bressler, and Tracy Valorie (Pfizer Inc)