Topic Archives: Rare Disease

October 25, 2021 - FDA, Pharma, Rare Disease, Regulatory, United States

FDA Finalizes Guidance on Interpretation of Orphan Drug “Sameness” for Gene Therapy Products

By: Rachel Park and Brigid DeCoursey Bondoc

On September 30, 2021, the U.S. Food and Drug Administration (FDA) announced final guidance titled “Interpreting Sameness of Gene Therapy Products under the Orphan Drug Regulations.” The guidance finalizes the January 2020 draft guidance and provides FDA’s current perspective on certain criteria that …›

March 1, 2021 - Biotech, COVID-19, FDA, Healthcare, Pharma, Rare Disease, Regulatory

Rare Disease: Sustained Progress in Development Regardless of the Pandemic

By: Catherine M. Polizzi

Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare disease drug development by sponsors and FDA throughout 2020. In addition, a number of policies implemented in response …›