The FDA had more than 700 orphan drug designation requests last year. As a result, the FDA recently announced a new pilot program designed to make the request process easier. Along with a new form for the submissions process, FDA will make more resources available to sponsors.
Orphan Drug Submissions
FDA released an on-line tutorial to guide sponsors through the submissions process. There is also a new inter-FDA consult process which streamlines and standardizes communications. FDA has also pledged to eliminate its backlog of orphan drug designations and recently held a public meeting to discuss scientific and regulatory issues related to cancer treatments.
FDA also entered into an understanding with the National Organization for Rare Disorders to conduct outreach with patient affairs staff. The goal is to enhance the incorporation of patient experience into regulatory discussions.
Specifically, FDA explains the Memorandum of Understanding (MOU) will define “the framework for collaboration between the Food and Drug Administration (FDA) and the National Organization for Rare Disorders (NORD). FDA and NORD share a common goal to improve the safety of the American public when using FDA-regulated medical products. FDA and NORD will collaborate to promote scientific progress in innovation, patient education, drug safety communications, training, research, including risk evaluation and mitigation strategies (REMS), and adverse event reporting. Before any specific collaboration is initiated or implemented, the parties shall develop separate written agreements for collaborations. Where applicable, the agreements shall incorporate by reference this MOU.”
“As part of this process, we’re planning a joint series of pilot listening sessions on rare diseases. We recognize that early and iterative engagement can improve clinical and regulatory understanding of diseases and conditions; provide a common understanding of the most urgent patient needs; and inform drug development programs,” Gottlieb said.
Gottlieb continued: “Over the course of 2018 we’ll continue our efforts to increase the consistency and efficiency of our reviews of rare disease products. We remain committed to supporting rare disease research on diagnostics, therapies, and potential cures. We’ll also continue to evaluate how to best support investment in rare diseases; and to encourage the development of drugs that target rare, unmet patient needs. A lot of devastating and rare conditions still lack approved therapy. During this Rare Disease Week, it’s gratifying to review the steps we’ve taken, and to commit to more progress in the future, and making sure that our framework supports the needs of patients.”