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Shire’s Replagal Withdrawn: A “Non-Traditional” Review Ends Badly

This article was originally published in RPM Report

Executive Summary

Shire’s decision to take its Fabry Disease therapy and go home sets up two possible narratives. One accentuates FDA’s apparent inflexibility and is sure to reinforce calls for legislative changes to the approval standard. The other highlights a recalcitrant or even petulant-sounding sponsor. Neither is likely to sound good to Fabry’s patients.

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Despite a “yes” vote from an advisory committee, FDA sent Chelsea’s Northera back to the development phase with a complete response letter asking for data from another trial. There may have been nothing Chelsea could have done to get a better result—but the company could have held off on a post-panel victory lap.

Dr. Woodcock, Orphan Drugs and Congressional Reform – Be Careful What You Ask For

FDA’s top drug review official is wary of calls for legislative changes affecting the approval standard. The co-founder of the Abigail Alliance, Steven Walker, sees it differently. Here is his response to an article published by The RPM Report about Woodcock’s remarks at the US Rare Diseases Conference.

The Drug Shortage Problem: Good Solutions are as Hard to Find as Some Critical Products

Drug shortages are top-of-mind topic for government officials and legislators in Washington DC. But effective action will likely require changes in regulation, reimbursement, legislation and business practices involving many different stakeholders. This is definitely a case where the problem is easier to talk about than to fix.

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