FDA Plan for Issuance of Patient Focused Drug Development Guidance

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Patients who live with a disease have a direct stake in drug development and in the outcome of the FDA review process for new drugs. Patients are also in a unique position to contribute to an understanding of benefit and risk considerations throughout the medical product development process. Under the 2012 FDASIA reauthorization of the Prescription Drug User Fee Act (PDUFA), FDA pioneered the use of patient focused drug development (PFDD) meetings2 to help address the need for systematic collection of direct patient input. The twenty-two PFDD meetings we have held so far have each focused on a different disease area and have identified key findings including that patients living with a disease are experts on what it is like to live with the condition. In addition, the meeting highlighted that what patients care most about may not always be factored into clinical trials or approved labeling.
However, PFDD meetings alone will not address gaps in information on the patient perspective. Our next step is to bridge from PFDD-type meetings to methodologically sound fit-for-purpose tools to systematically collect key information about patients’ experience (e.g., in clinical trials) including the burden of disease, benefit and burden of a therapy. Ideally, these patient-identified disease impacts, and potential measures of benefit and burden, would be explicitly considered from the early stages of drug development. In addition, these patient-identified key impacts and elements of disease experience could be translated into a measurement set that is validated for clarity to patients, reliability in capturing their reported experience, and responsiveness of the reporting scale to reflect changes in experience. For a given disease the set of elements used in different clinical studies would ideally reflect those that patients have identified as mattering most to them.

Description

Patients who live with a disease have a direct stake in drug development and in the outcome of the FDA review process for new drugs. Patients are also in a unique position to contribute to an understanding of benefit and risk considerations throughout the medical product development process. Under the 2012 FDASIA reauthorization of the Prescription Drug User Fee Act (PDUFA), FDA pioneered the use of patient focused drug development (PFDD) meetings2 to help address the need for systematic collection of direct patient input. The twenty-two PFDD meetings we have held so far have each focused on a different disease area and have identified key findings including that patients living with a disease are experts on what it is like to live with the condition. In addition, the meeting highlighted that what patients care most about may not always be factored into clinical trials or approved labeling.
However, PFDD meetings alone will not address gaps in information on the patient perspective. Our next step is to bridge from PFDD-type meetings to methodologically sound fit-for-purpose tools to systematically collect key information about patients’ experience (e.g., in clinical trials) including the burden of disease, benefit and burden of a therapy. Ideally, these patient-identified disease impacts, and potential measures of benefit and burden, would be explicitly considered from the early stages of drug development. In addition, these patient-identified key impacts and elements of disease experience could be translated into a measurement set that is validated for clarity to patients, reliability in capturing their reported experience, and responsiveness of the reporting scale to reflect changes in experience. For a given disease the set of elements used in different clinical studies would ideally reflect those that patients have identified as mattering most to them.

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