Patient Focused Drug Development An FDA Perspective Theresa

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Patient Focused Drug Development An FDA Perspective Theresa Mullin, Ph. D. Director, Office of

Patient Focused Drug Development An FDA Perspective Theresa Mullin, Ph. D. Director, Office of Strategic Programs FDA Center for Drug Evaluation and Research September 19, 2017

Patient Perspective Can Inform BR Assessment at Multiple Levels Benefit-Risk Summary and Assessment Dimension

Patient Perspective Can Inform BR Assessment at Multiple Levels Benefit-Risk Summary and Assessment Dimension Analysis of Condition Current Treatment Options Benefit Risk Management Evidence and Uncertainties Conclusions and Reasons Patient Focused Drug Development Provides therapeutic context for weighing benefits and risks Clinical Outcome Assessments (e. g. , PROs) Incorporates expert judgments about the evidence of efficacy and safety, and efforts to further understand or mitigate risk 2 2

Further integrating patient perspective into medical product development and decision making Need to build

Further integrating patient perspective into medical product development and decision making Need to build in the patient’s perspective starting in the translational phase What impacts (burden of How to better integrate disease and burden of treatment) matter most to patients and how to measure them? What aspects of clinical trials can be better tailored to meet the patients who (might) participate in the trial? Translational Clinical Studies • How do we ensure that we get input representative of the whole disease population? • Do endpoints planned for the trial include the ones that matter most to patients? Are the desired endpoints feasible? • What symptom or functions matter most to people with this disease? • Does the protocol facilitate (or discourage) enrollment or continued participation? Could that be improved? • How to best measure? (endpoints, frequency, mode of reporting, etc. ) • Do informed consent and other processes within the trial reflect the needs and preferences of people with that disease? patient reported outcome data or elicited patient preferences into BR assessments? Pre-market review • How can the patientreported evidence be integrated and evaluated? • How to utilize elicited patient preference studies? • How to factor in key uncertainties? • How could individual differences in patient experience (or preference) of benefit versus harm be considered? How to best communicate the information to patients and prescribers? Post-market • How to convey info that helps facilitate patients’ and clinicians’ informed decision making? • How to convey uncertainty to inform and support clinical decision-making? 3 3