Approval Bill Includes Rewards for Kids' Drugs
With that in mind, Congress in 2011 set up a program to help promote more pediatric drug research. It gives creators of medicine for rare pediatric diseases a voucher that they can use to have another one of their drugs approved quicker than usual – six months vs. a process that can run a year or often more.
Drug makers can also sell that voucher, which can be a big windfall for a small drug company trying to recoup research and development costs. There have been four vouchers given out since 2014, and one was sold for $67.5 million and a second for $125 million.
The voucher program, which advocates say holds big potential, expires next year. The cures bill seeks to extend it another three years.
“A lot of companies are reluctant to get into pediatric drug development because it’s very difficult if something goes really wrong,” said Alexander Gaffney of the Regulatory Affairs Professionals Society, an association for people involved in overseeing health care or the quality of health care products.
Drugs that are approved for cancer in adults are commonly not approved in kids. “Children are not simply small adults, they metabolize drugs very differently,” Gaffney said.
Critics say that however well-intentioned the voucher program is, it could have some unintended consequences. For example, a company could get a drug approved by the Food and Drug Administration but never bring it to market, if the maker decides it would not generate enough money. Yet the company would still pocket the priority review voucher.
Because of the speed sought by the program, vouchers could be given out without some of the safeguards that come in more traditional testing. For example, the research might not uncover that the drug could be fatal to a child after a few months or years.
Diana Zuckerman, president of the National Center for Health Research, a nonprofit group that seeks to represent children and families on health research policy issues, says the rush in moving drugs through the system can obscure problems. Drug makers “shouldn’t be able to sell it [or use it] unless it works,” she said.