New legislation introduced this week would reauthorize the US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program, which is currently set to end after triggering a sunset clause in its authorizing statute. Background Priority review vouchers are incentives meant to catalyze the development of new therapies for historically under-served disease areas, such as rare pediatric diseases affecting fewer than 200,000 children in the US...

The US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program is slated to end in one year, after the agency awarded its third-ever rare pediatric voucher, thereby triggering a little-known provision in the voucher program. Background Regular readers of Regulatory Focus will be familiar with FDA's priority review voucher programs. We've written about them extensively over the years, and the vouchers are the focus of our most rec...

A new drug being co-developed by drugmakers Sanofi and Regeneron could, according to new data , dramatically decrease low-density lipoprotein (LDL) cholesterol in patients and lessen cardiac events. But it's a reduction in something else that could be most important for Sanofi and Regeneron: the time it might take the US Food and Drug Administration (FDA) to review the new drug, Praluent. Background In July 2014, Sanofi and Regeneron announced they had purchased what...

Drugmaker United Therapeutics has become just the second company in US history to obtain a new type of voucher which allows a company to potentially get its drug approved by the US Food and Drug Administration (FDA) in 40% less time than it normally takes. Background The voucher in question is known as a rare pediatric disease priority review voucher (PRV), and is modeled off a similar program intended to help spur the development of new drugs for so-called "neglected" ...

President Barack Obama has signed a new bill into law allowing any pharmaceutical or biotechnology company that successfully develops a product to treat the Ebola virus to obtain a special voucher potentially worth millions—and even billions—of dollars. The law, Adding Ebola to the FDA Priority Review Voucher Program Act , was signed into law by Obama on 16 December 2013. The law focuses on FDA's neglected tropical disease priority review voucher (PRV) program, a pro...

The US House has quickly passed legislation making changes to the  Neglected Tropical Disease Priority Review Voucher  (PRV) system in the hopes of making it more enticing to pharmaceutical companies, and in particular to those developing new treatments for the Ebola virus. S.2917 , the  Adding Ebola to the FDA Priority Review Voucher Program Act , passed the US House of Representatives by unanimous consent on 3 December 2013, just one day after the same bill was pas...

The US Senate has passed legislation that would overhaul the Neglected Tropical Disease Priority Review Voucher  (PRV) system in the hopes of making it more enticing to pharmaceutical companies, and in particular those developing new treatments for the Ebola virus. S.2917, the Adding Ebola to the FDA Priority Review Voucher Program Act , passed the US Senate by unanimous consent on 2 December 2013. "Today the Senate took a strong step to support that mission by pass...

Both the US House of Representatives and Senate are moving forward with identical pieces of legislation meant to overhaul the Neglected Tropical Disease Priority Review Voucher (PRV) system in the hopes of making it more enticing to pharmaceutical companies, and in particular those developing new treatments for the Ebola virus. Bill Background The legislation, the Adding Ebola to the FDA Priority Review Voucher Program Act , would make three notable changes...

The Canadian pharmaceutical company Knight Therapeutics has reportedly sold its Neglected Tropical Disease Priority Review Voucher to Gilead Sciences, making it the first time a tropical voucher has ever been sold. Background FDA maintains two types of priority review voucher programs, which allow companies to have their products reviewed by FDA in just six months instead of 10. The first and oldest of the voucher types is intended to facilitate development of products ...

The US Food and Drug Administration (FDA) has released a long-anticipated guidance document intended to explain how a new incentive program known as the Rare Pediatric Disease Priority Review Voucher system works. Background Created in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA) , the Rare Pediatric Disease Priority Review Voucher is modeled closely off a similar program known as the Tropical Disease Priority Review Voucher system. T...

Legislators in the US Senate have unveiled a new piece of legislation meant to incentivize companies to go after the Ebola virus by making critical and long-sought changes to a US Food and Drug Administration (FDA) regulatory program. Background As Focus explained in October 2014 , a recent outbreak of Ebola has led to a scramble for new drugs and vaccines to help treat and prevent the virus. At present, there are no recognized treatments for the disease. Some publi...

Bipartisan members of the US Senate's healthcare oversight committee plan to introduce legislation that would amend a little-used regulatory program in the hopes of spurring the development of treatments for the Ebola virus. Background The program, known as the neglected tropical disease priority review voucher program, was created in 2007 under the Food and Drug Administration Amendments Act (FDAAA) . The voucher program, first envisioned in a 2006 Health Affairs po...