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.
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
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
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
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