Bill Would Change Priority Review Voucher Program

A bipartisan bill was introduced late last week that would give drugmakers additional incentives to develop medications for rare pediatric diseases. TheCreating Hope Act would entice drugmakers by offering a 'priority review voucher,' which would entitle them to a six-month priority review by the FDA of another drug in exchange for developing a med aimed at treating a rare pediatric illness. In other words, a drugmaker would have a potentially big seller reviewed several months faster than would occur otherwise, since the standard FDA review time is 10 months.

"We are falling woefully and inadequately short in our efforts to cure and treat rare and neglected pediatric diseases and conditions," says Sherrod Brown, an Ohio Democrat who co-sponsored the proposal, in a statement. "The Creating Hope Act is aimed at doing exactly that - creating hope - for the millions of American children suffering from rare and pediatric diseases by increasing the incentives for pharmaceutical companies to expand research and development into these devastating illnesses."

Essentially, the bill would amend an existing law that offers the same accelerated review for drugs to treat certain tropical diseases. However, that program, which went into effect two years ago, was quickly criticized when Novartis sought FDA approval for Coartem, an existing malaria drug that combines two other meds and was already marketed in other countries. Basically, Novartis won FDA approval for a drug that offered no new benefits to existing patients and, in the US, may appeal to overseas travelers. However, the drugmaker could sell the voucher for whatever the market bears.

To some, the episodesuggested the value of a voucher depends on the success of having a blockbuster in the pipeline (read the background). In hopes of linking innovation with incentives and avoiding a repeat, the new proposal requires that a drug must be an "innovative treatment" to be eligible for a voucher and the drug must not have been approved to treat a tropical disease outside the US for more than two years before FDA approval is sought.