Germany is drastically squeezing orphan drug pricing while new US laws protect rare diseases drugs from price negotiations. These contrasting moves fuel the longstanding debate over whether orphans still deserve special incentives.

Sanofi/Sobi’s efanesoctocog is in line for US FDA action three months after CSL/uniQure’s gene therapy user fee goal, while BioMarin’s Roctavian resubmission and Novo Nordisk’s concizumab are waiting in the wings.

Designation as a regenerative medicine advanced therapy or breakthrough therapy ‘dramatically increases’ the likelihood of getting a meeting with the agency, Office of Tissues and Advanced Therapies’ Wilson Bryan says; more complex and innovative products, and more challenging questions, also are likely to win sponsors an audience with the FDA.

Standardizing the process for providing clarifications following sponsor meetings, particularly with regard to the written response-only format, is one of several solutions being piloted by the Office of Tissues and Advanced Therapies as part of its ‘growth program,’ which aims to expedite advances in cell/gene therapies while also improving staff satisfaction.