Agency Announces Rare Disease Day Meeting

Rare Daily Staff

The U.S. Food and Drug Administration later this year will begin accepting applications for Orphan Drug designation through an online portal, the agency announced in a blog post.

The agency said the effort will build on the FDA’s Orphan Drug Modernization Plan from June 2017 and will streamline the orphan drug designation request process.

The Orphan Drug Technology Modernization is intended to create a more connected information technology system, advanced analytics, and improvements in facilitating knowledge management. In addition, it will provide external sponsors direct communication with the FDA.

As part of the same blog post, the agency announced that it will host a public meeting on February 24 in honor of Rare Disease Day 2020. During the meeting, the FDA wants to hear from rare disease stakeholders on strategies to optimize registry and natural history data collection. It would also like to hear 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 provide their perspectives on these topics.

“To optimize rare disease product development, we must collaborate not only across diverse scientific and medical disciplines but also with different stakeholder groups such as patients, researchers, and regulators,” wrote FDA Principal Deputy Commissioner and Acting Chief Information Officer Amy Abernethy, and Director of the Office of Orphan Products Development Janet Maynard. “To optimize rare disease product development, we must collaborate not only across diverse scientific and medical disciplines but also with different stakeholder groups such as patients, researchers, and regulators. We must develop new strategies to collaborate in all phases of biomedical discovery and development.”

Photo: Janet Maynard, director of the office of orphan products development

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