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Bill to Speed FDA Approvals Includes Rewards for Drugs Designed for Kids

Agency entices pharma companies with 'priority review' vouchers

Advocates for children with rare diseases are watching a congressional effort to streamline the nation’s drug-approval process because the bill includes a provision extending a federal program that rewards companies making remedies for these patients, according to a report from Kaiser Health News.

The reward program, the advocates say, offers hope to families that often have few options. Approximately 15 million children are diagnosed with rare diseases, and 35% of deaths in the first year of life are caused by them.

“Treatments aren’t getting to kids, and kids deserve more than the leftovers,” said Nancy Goodman, founder and executive director of the advocacy group Kids v Cancer whose 10-year-old son died from brain cancer. She helped push for the original reward program in the hope that children like her son would have access to a wider range of treatments.

The extension of that program is part of the bipartisan 21st Century Cures bill, which seeks to rewrite the rules for drug development to make innovative treatments available faster. The overall bill has generated support on Capitol Hill, but some critics contend that it has the potential to undermine drug safety and to generate profits for drug makers.

Children’s advocates say there is a shortage of effective therapies for rare and often deadly pediatric diseases, which include cancer, skull deformities, and enzyme deficiencies. Pharmaceutical companies have historically been hesitant to test drugs in children because of concerns about potential negative outcomes, the children’s inability to consent to treatment, and the perception that the market for these drugs was limited. Doctors, therefore, are often left to use adult-tested drugs on sick children without study data that show pediatric safety or efficacy. But drugs used on adults don’t always work on children in the same way because of differences in metabolism and maturation of organs.

The advocates say more research needs to be conducted with children, but that kind of testing can be costly, and it requires extra care because there are more-stringent ethical protocols to protect minors. Bad results — either injuries or deaths –– can set back research efforts and have financial consequences for the company.

With that in mind, Congress in 2011 set up a program to help promote more pediatric drug research. The program gives developers of medications for rare pediatric diseases a voucher that they can use to have another of their drugs undergo “priority review” within 6 months compared with a process that could 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. Four vouchers have been given out since 2014; one was sold for $67.5 million, and the other for $125 million.

The voucher program, which advocates say holds big potential, expires next year. The 21st Century Cures bill seeks to extend it another 3 years.

Critics say that however well-intentioned the voucher program is, it could have some unintended consequences. For example, a company could have a drug approved by the FDA but might never bring it to market if the maker decides it would not generate enough money; and 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 with more-traditional testing. For instance, the research might not uncover that the drug could be fatal to a child after a few months or years.

Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, an advocacy group pushing for drug companies to spend more money on drug development, wants the pediatric-drug voucher program extended. She notes that the program is still too new for officials to evaluate whether it is effective.

One problematic part of the current House version, she said, is that it extends the program for only 3 years, whereas drug companies generally need 10 years to find investors and to begin research on a new drug. The potential reward of an expedited drug review might not be enough to motivate companies to make financial plans for drugs based on the program, Jenkins said.

The 21st Century Cures bill covers more than 60 health problems, including a $10 billion boost in funding for the National Institutes of Health and $550 million in extra money for the FDA over the next 5 years. Other provisions include creating a database of genomic information from a million U.S. patient volunteers and allowing the FDA to approve drugs without a pivotal clinical trial, instead using smaller observational studies or clinical experience.

The bill passed the House Energy and Commerce Committee unanimously in May and is expected to come up for a vote in the full House. Senators are in the early stages of working on a similar bill.

Source: Kaiser Health News; June 8, 2015.

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