The idea of incorporating patients’ voices into drug development has been around for more than a decade, but now the US Food and Drug Administration (FDA) is weighing how to provide practical guidance for industry on incorporating such voices. And companies like Pfizer, Roche and Allergan are offering their own perspectives on what that guidance should look like.
Patient voices and perspectives are nearly always relayed to outside experts discussing the approval of new drugs during advisory committee meetings, but rarely do those perspectives conflict with sponsors, and sponsors frequently pay for patients to travel to the meetings and discuss the benefits of a treatment.
According to the sixth iteration of the Prescription Drug User Fee Act
(PDUFA), under which companies pay user fees to FDA to meet certain review deadlines, FDA is required to step up efforts to collect and utilize patient and caregiver input that can more consistently inform drug development and regulatory decision making.
And over each of the next four years
, FDA is tasked with publishing draft guidance on topics related to patient-focused drug development centered on standardized nomenclature and terminologies, methods to collect meaningful patient input, the burdens of disease and treatment and how to facilitate collecting meaningful patient input in clinical trials and clinical outcome assessments, which, when final, will, as appropriate, revise or supplement the 2009 “Guidance to Industry on Patient-Reported Outcome Measures.”
Roche applauded FDA for holding 24 disease-specific meetings over the last five years on understanding patient experiences and said it recognizes that “reasonable” standards are necessary “to better capture the patient voice in benefit-risk assessment evaluation and to foster collection of valid and credible patient-relevant data for dissemination to the patient community.”
In terms of guidance, Roche said FDA “should consider the evolving objectives of patient-focused research throughout drug development and provide practical guidance as to how to choose a fit-for-purpose method to gather patient input for different objectives. The guidance should also provide guidelines that enable Sponsors to engage with patients and their representatives early and throughout the drug development lifecycle and should define clear milestones within the IND process for Sponsors to share patient experience data with the FDA.”
Allergan, meanwhile, called on FDA to better describe how it plans to use information submitted as “patient experience data” so stakeholders can determine the appropriate research questions and study design for the specific context of use.
“For example, to inform FDA’s review of a trial protocol, information from a convenience sample of patients with inclusion & exclusion criteria matching that of the intended clinical trial population may suffice; however, to inform FDA’s risk/benefit assessment, a more comprehensive approach to sampling to ensure the research population is representative and information is generalizable may be required,” Allergan said.
Pfizer also questioned how the agency will incorporate patient experience into benefit/risk considerations.
“For example, it would be helpful to know what level of evidence would be required to incorporate patient experience information into a label, as opposed to, for example, using patient perceptions and experience to inform procedures in a clinical trial protocol,” Pfizer said.
The drugmaker further called on the agency to offer its position on observer-reported outcomes, such as parents or caregivers, which could be particularly relevant for pediatric populations, patients with neuro degenerative conditions, cognitive impairment or any other populations of patients that are not able to self-report.