U.S. Food and Drug Administration’s Patient-Focused Drug Development Initiative: Experience with Integration of Patient-Experience Data in a New Drug Application for Esketamine Nasal Spray Plus a Newly Initiated Oral Antidepressant for Treatment-Resistant Depression

The Patient-Focused Drug Development initiative of the U.S. Food and Drug Administration (FDA) aims to ensure that the patient experience of disease and treatment is an integral component of the drug development process. The 21st Century Cures Act and Prescription Drug User Fee Act (PDUFA) VI require the FDA to publicly report the type of patient-experience data reviewed in a new drug application (NDA) to inform regulatory decision-making. This report describes a recent approach adopted at Janssen of integrating patient-experience data into the NDA for esketamine (SPRAVATO®) nasal spray with a newly initiated oral antidepressant (esketamine + AD) for treatment-resistant depression. During the development of esketamine + AD, patient-experience data were collected using several patient-reported outcomes, including the Sheehan Disability Scale and 9-item Patient Health Questionnaire (PHQ-9). Additionally, a patient-preference study assessed the relative importance of benefits and harms that patients allocated to different attributes of treatment. Preferences were collected from patients enrolled in phase 3 esketamine trials and from an online panel of primarily ketamine-naive patients. Patient-experience data were integrated into the esketamine NDA, the FDA advisory committee meeting briefing document, and the Sponsor’s presentation. The FDA acknowledged reviewing the patient-experience data and determined that they supported esketamine + AD for treatment-resistant depression. This report highlights the importance of integrating patient-experience methods early in drug development, their impact on assessing patient-relevant benefits and risks, and how they can help improve clinical program design.

PARE0015 UNDERSTANDING THE NEEDS OF PATIENTS WITH OSTEOARTHRITIS TO INFLUENCE THE DEVELOPMENT OF RELEVANT PATIENT-CENTERED PRODUCTS & TREATMENTS

Background:Osteoarthritis (OA) is the most common form of arthritis and affects over 30 million adults in the United States of America.1There is no cure for osteoarthritis, and unlike other forms of arthritis where great treatment advances have been made in recent years, progress has been much slower in osteoarthritis.2However, there is increasing interest in removing barriers to treatment development, and the U.S. Food & Drug Administration has adopted and implemented new patient-focused drug development meetings to ensure the patient voice is informing new treatments.3Objectives:To gain a robust understanding of the barriers, needs and hopes of patients with OA to influence the development of relevant patient-centered products and treatments.Methods:A 34-question web-based survey was distributed between November 1 - 10, 2019. Survey invitations were sent via e-newsletters and e-mails to patients engaged with the Arthritis Foundation. The first survey question screened out patients who had not been diagnosed with OA.Results:2,437 patients diagnosed with OA completed the survey. There are 5 key findings:1.Patients would like pain (90%) addressed more than any other symptom. Other symptoms such as fatigue (50%), sleep (39%), ability to walk (35%), stiffness (32%), ability to stand (16%), and ability to go up & down the stairs (14%) were ranked significantly below pain.2.Patients use a variety of techniques to manage their OA and related symptoms. Physical activity (62%) and heat & cold interventions (62%) are the most prevalent. Approximately half of the patients use topical cream (53%), supplements & vitamins (51%), NSAIDS (50%), and/or acetaminophen (48%). Approximately one-third of patients utilize diet/nutrition (39%), assistive devices (38%), cortisone injections (36%), and/or physical therapy (34%). Other methods, including surgery (26%), were utilized less frequently.3.41% of respondents reported they were “very interested” in participating in OA clinical trials; 40% reported “somewhat interested.” Only 19% reported they were “not interested.” The top 2 reasons patients were not interested included fear of possible risks/unknow effects (48%) and potential impact on other health problems (44%).4.Primary care physicians are diagnosing (44%) and treating (48%) OA patients more than any other health care provider.5.Many daily living tasks are difficult, and patients are interested in in tools/equipment to help. Yard work (66%), opening jars (64%), and cleaning the house (63%) are the most difficult. Over half of patients reported that physical activity (57%) and getting up and down from a chair (54%) are difficult. Patients would like tools/equipment to help them clean the house (54%) and help with physical activity (50%).Conclusion:OA patients use a variety of management techniques yet are still in pain and have difficulty doing everyday tasks. These findings, along with other patient data, will be used to influence product developers to create easier to use products, to inspire researchers to focus on addressing patients’ most pressing needs, and to encourage government agencies to remove barriers and facilitate new patient-centered treatments.References:[1]Centers for Disease Control and Prevention. Osteoarthritis (OA). Last reviewed January 10, 2019. Retrieved fromhttps://www.cdc.gov/arthritis/basics/osteoarthritis.htm[2]Cleveland Clinic. Osteoarthritis: Management and Treatment. Last reviewed November 26, 2019. Retrieved fromhttps://my.clevelandclinic.org/health/diseases/5599-osteoarthritis-what-you-need-to-know/management-and-treatment[3]U.S. Food and Drug Administration. Externally led Patient-Focused Drug Development Meetings. Last reviewed August 28, 2019. Retrieved fromhttps://www.fda.gov/industry/prescription-drug-user-fee-amendments/externally-led-patient-focused-drug-development-meetingsDisclosure of Interests:None declared