• Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) Conference 2015: Patient-Focused Drug Development (PFDD)

      Perfetto, Eleanor M.; Oehrlein, Elisabeth Maria; Anyanwu, Chinenye; Burcu, Mehmet; Gaitonde, Priyanka; Hanna, Maya L.; Ng, Xinyi; Pickering, Matthew; Sieluk, Jan; Vandigo, Joseph Edward (2015)
      A movement to include the patient voice in health care research and decision making is underway. In light of broad stakeholder interest in patient-focused drug development (PFDD), a range of stakeholders are considering approaches to increase the scope of PFDD and enhancing patient engagement. On March 9, 2015, the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), with the support of many partner organizations, held the “M-CERSI Conference on PatientFocused Drug Development.” The objective was to allow stakeholders from patient groups, the US Food and Drug Administration (FDA), the biopharmaceutical industry, payer, and other organizations to voice their views on, activities in, and aspirations for PFDD. During the day-long program, participants discussed the challenges to successful PFDD including regulatory challenges, the patient and patient advocate role, the emerging payer role, along with future directions and opportunities for collaboration. This document summarizes stakeholders’ perspectives on and understanding of the definition and attributes of PFDD as well as its potential for achieving the goal of including the patient’s voice in drug development. The role of various stakeholders and opportunities for their active participation were outlined. The outputs of the conference included a suggested definition, rubric, and framework for PFDD: Definition: Patient-focused drug development is a formal process by which drug* developers and regulators form a partnership with patients to enhance drug* development, research, regulatory, and reimbursement processes with the patient voice. This partnership engages patients to obtain, as critical input, their views, experiences, and preferences throughout a product’s* lifecycle. Rubric: 1. Patients as Partners: Patients, caregivers, and other relevant people (e.g., people who are at risk for a disease, but do not yet have the disease) are recognized as partners in the drug development process throughout the product life cycle. 2. Continuous Patient Engagement: Patient engagement is continuous, throughout the drug development process and product lifecycle; it is not a one-time or sporadic event. 3. Meaningful Patient Engagement: Patient engagement must be meaningful. That is, it must be a real interaction and dialogue, not a “check-the-box” exercise. Patient input should come from thoughtful dialogue and patients should be able to see how the input they provide is used in the specific studies or aspects of processes. 4. The Right Patients are Engaged: Throughout the process, the affected patient population is well represented, and other relevant populations are considered for engagement. 5. Right Time to Engage: Engagement happens at the appropriate time(s) throughout the process. Conceptual Framework: Building upon previously proposed models and the meeting discussion, a conceptual framework for PFDD emerged. Proposed PFDD Conceptual Framework. Adapted from: Clinical Trials Transformation Initiative’s Patient Groups & Clinical Trials Expert Meeting summary; National Health Council’s Dialogue/Advancing Meaningful Patient Engagement in Drug Research, Development, and Approval; and the model proposed by Perfetto et al. Med Care. 2015 Jan;53(1):9-17
    • Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) Conference 2015: Assessing Meaningful Patient Engagement in Drug Development: A Definition, Framework, and Rubric

      Perfetto, Eleanor M.; Oehrlein, Elisabeth Maria (2015)
      A movement to include the patient voice in health care research and decision making is underway. In light of broad stakeholder interest in patient-focused drug development (PFDD), a range of stakeholders are considering approaches to increase the scope of PFDD and enhancing patient engagement. On March 9, 2015, the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), with the support of many partner organizations, held the “M-CERSI Conference on PatientFocused Drug Development.” The objective was to allow stakeholders from patient groups, the US Food and Drug Administration (FDA), the biopharmaceutical industry, payer, and other organizations to voice their views on, activities in, and aspirations for PFDD. During the day-long program, participants discussed the challenges to successful PFDD including regulatory challenges, the patient and patient advocate role, the emerging payer role, along with future directions and opportunities for collaboration. This document summarizes the outputs of the conference including a suggested definition, rubric, and framework for PFDD.