Oncology ‘Beyond Breakthrough’ Takes Shape: FDA Project Will Create Benefits ‘Menu,’ Rescission Process

US FDA’s oncology team has come to realize that a better understanding of how to personalize its ‘all-hands-on-deck’ approach would be even more helpful – especially for first-time sponsors.

Health care success medical research breakthrough and medicine discovery as a doctor stethoscope shaped as a stethoscope breaking a wall to a target as a cure metaphor with 3D illustration elements.
While there is much to like about the program, FDA wants to move away from the idea that Breakthrough is somehow a pre-market “seal of approval” from the agency. • Source: Shutterstock

The US Food & Drug Administration’s ongoing review of the Breakthrough Therapies program will identify best practices from the nearly 10-year-old expedited program and identify ways to make the designation more meaningful to sponsors – including a “menu” of the types of meetings and communications available once the designation has been granted.

Oncology officials acknowledge that the often-used “all hands on deck” description of Breakthrough is “somewhat vague,” and that a better...

Welcome to Pink Sheet

Create an account to read this article

More from Review Pathways

US FDA Commissioner Makary Is Learning To Appreciate His Staff

 

After three months on the job, FDA Commissioner Martin Makary is sounding much more effusive about the quality of the agency staff. Better late than never?

‘Unprecedented Story’: Revoked EU Marketing Authorization Fails To Thwart Translarna Sales

 

PTC Therapeutics is continuing to supply its Duchenne muscular dystrophy treatment, Translarna, in some European markets, despite EU revocation of the drug’s conditional marketing authorization earlier this year.

EMA’s PRIME Scheme Explored: Growing Designations - But Are Accelerated Assessments Slipping?

 

While the first six months of the year saw eight new drugs targeting a range of diseases enter the European Medicines Agency’s priority medicines scheme, and three PRIME-designated treatments go on to win EU marketing approval, use of the accelerated assessment mechanism appears to be limited.

US FDA Asking Questions About Commissioner’s Voucher Program With Details Scarce

 

FDA staff are trying to plan for program implementation in the absence of critical eligibility and other details, although guidance may be coming soon.

More from Pathways & Standards

EMA Says ‘No’ To Roche/Sarepta’s Elevidys, US FDA Investigates Fourth Death

 

The European Medicines Agency recommended against pan-EU marketing authorization of Roche/Sarpeta’s gene therapy Elevidys for the treatment of Duchenne muscular dystrophy as the US reported another death in an Elevidys patient.

US FDA Commissioner Makary Is Learning To Appreciate His Staff

 

After three months on the job, FDA Commissioner Martin Makary is sounding much more effusive about the quality of the agency staff. Better late than never?

Complete Response Letter Trio Raises Prospect Of Tidal Shift In Regenerative Medicine Regulation

 

Replimune's RP1 oncolytic immunotherapy became the third regenerative medicine to receive an FDA complete response letter this month as the agency appears to re-visit previous agreements about accelerated approval.