“… Looking forward, we will build on existing rare disease collaborations and strengthen the information technology process in the FDA’s Office of Orphan Products Development (OOPD). Today, we are pleased to announce two actions that support our efforts:
The FDA will host a public meeting on February 24: “FDA Rare Disease Day 2020: Supporting the Future of Rare Disease Product Development.” The meeting will focus on several topics. First, the FDA wants to hear from rare disease stakeholders, including patients and caregivers, clinicians, and researchers, on strategies to optimize registry and natural history data collection. Second, the FDA wants to hear from stakeholders about opportunities and challenges when developing medical products for diseases or conditions that only affect one person, or just a few. FDA staff will also offer their perspectives on these topics. We invite anyone who is interested to participate in this public meeting.”
“The FDA will strengthen the information technology processes in OOPD in an Orphan Drug Technology Modernization effort. This effort will build on the FDA’s Orphan Drug Modernization Plan from June 2017 and will streamline the orphan drug designation request process by moving from a paper-based process to a new cloud-based online submission portal. The new online portal will allow sponsors to submit orphan drug designation requests electronically. We expect the portal to be available later this year. This effort is an example of the FDA’s commitment to broader efforts in overall technology modernization across the FDA.”
“Looking forward, we will build on existing rare disease collaborations and strengthen the information technology process in the FDA’s Office of Orphan Products Development (OOPD). Today, we are pleased to announce two actions that support our efforts:
The FDA will host a public meeting on February 24: “FDA Rare Disease Day 2020: Supporting the Future of Rare Disease Product Development.” The meeting will focus on several topics. First, the FDA wants to hear from rare disease stakeholders, including patients and caregivers, clinicians, and researchers, on strategies to optimize registry and natural history data collection. Second, the FDA wants to hear from stakeholders about opportunities and challenges when developing medical products for diseases or conditions that only affect one person, or just a few. FDA staff will also offer their perspectives on these topics. We invite anyone who is interested to participate in this public meeting.
The FDA will strengthen the information technology processes in OOPD in an Orphan Drug Technology Modernization effort. This effort will build on the FDA’s Orphan Drug Modernization Plan from June 2017 and will streamline the orphan drug designation request process by moving from a paper-based process to a new cloud-based online submission portal. The new online portal will allow sponsors to submit orphan drug designation requests electronically. We expect the portal to be available later this year. This effort is an example of the FDA’s commitment to broader efforts in overall technology modernization across the FDA…” See details here.
Source: FDA Harnesses Technology and Collaboration to Support Rare Disease Product Development – January 17, 2019. FDA.
