Recent US Food and Drug Administration Efforts to Integrate the Patient's Perspective in Drug Development and Decision Making

Involve Children and Parents in Clinical Studies

“…That patient involvement in research leads to better outcome measures for research has been demonstrated. The FDA acknowledges the importance of determining the patient perspectives by means of PROMs, as described in the study of Mullin et al …”

“…6 The need to involve children/parents in research design, the preparation of patients' documentation, and information materials to improve research protocols and enhance research participation is widely acknowledged. The US Food and Drug Administration (FDA) wants to integrate the patient's perspective in drug development, a policy described in Mullin et al 7 There are many examples of involvement of children and parents in research. 8 Patient engagement is, for example, arranged via members of the Children's Advisory Network and Young Persons Advisory Groups (YPAGs) 9 with specific tools for involvement.…”

Challenges and opportunities for improving access to approved neonatal drugs and devices

“…To accelerate high-quality neonatal trials and support drug/device innovation, multidisciplinary teams should bring together physicians, nurses, parents, researchers, sponsors, funding agencies, regulators, advocacy groups, and biostatisticians with shared data sources. Parents and patient advocacy groups are critical partners in neonatal drug development from the early stages of trial design through trial completion and the dissemination of research results [ 18 , 19 ]. Families have provided critical insight into inclusion criteria, consent processes, outcome measures, enrollment, participant engagement, retention, and the need for long-term follow-up.…”

Regulation and Innovation: Role of Regulatory Science in Facilitating Pharmaceutical Innovation

“…Regulatory science has different meanings to different audiences but for the purposes of this issue is defined as science and research intended to inform decision making in a regulatory framework. In putting this issue together, we have attempted to cover the very large waterfront of regulatory science, ranging from biomarker validation to novel approaches and acceptability of generation of clinical evidence and regulatory licensure frameworks that now include patient‐focused drug development . We also include regulatory science topics that can improve the effectiveness and efficiency of postmarketing pharmacovigilance, generic drug approval, and strategies to facilitate demonstration of value to support reimbursement .…”