news Take a breather: Biotech billionaire: Forgotten patients: Inhaled medicines Physician-entrepreneur Mental health offer quicker, more promotes data sharing trials tackle suicide potent drug delivery for cancer research prevention 183 187 190 Congress vouches for of childhood disease

A recently introduced piece of legislation in the US aims to foster new treatments for rare pediatric diseases by extending an existing voucher program offered by the country’s Food and Drug Administration, but some critics say the approach is fundamentally flawed. Priority review vouchers, or PRVs, were originally passed by the US Congress in 2007 to encourage pharmaceutical companies to develop new treatments for neglected tropical diseases such as and . Under the scheme, when a drug company’s qualifying neglected disease treatment is approved, it can receive a PRV redeemable for an accelerated

review of another for any Alan Crosthwaite condition. The company must also pay a user Vouched for: Congress backs priority review vouchers to spur drug development. fee to the FDA to redeem the PRV—for the 2012 fiscal year, the fee is nearly $5.3 million. Ideally, voted 11-1 against approving the drug for this The legislation adds language specifying that using the PRV reduces the usual ten-month indication last June. companies can earn PRVs for approval process to six months. Kesselheim points out that Coartem is hardly treatments already available outside the US “Providing reasonable incentives can help a novel malaria treatment, having been on the only if they’ve been on the market for two the drug industry enter a market where market market outside the US for more than a decade. years or less—making sure that it engenders forces haven’t provided enough of an incentive” “This didn’t actually incentivize any new new research. by themselves, says Peter Adamson, chief of research,” he says. Kesselheim says that although this reform, clinical pharmacology and therapeutics at David Ridley, a health economist at Duke which he dubbed “the Coartem corollary,” is a © 2012 Nature America, Inc. All rights reserved. America, Inc. © 2012 Nature the Children’s Hospital of Philadelphia, who University in Durham, North Carolina and good step in addressing problems with PRVs, specializes in new drugs for childhood cancer. one of the authors of a report that served as it doesn’t change his critique of the overall Both the Senate and the House of the outline for the PRV system, says that it is mechanism. There is research to suggest npg Representatives last year introduced versions too early to pronounce the program a failure that accelerated FDA reviews are linked to a of the Creating Hope Act, which would allow (Health Aff. 25, 313–324, 2006). Since it takes higher risk of safety problems down the road, companies to also earn PRVs for new drugs around seven years to get a new treatment to according to Kesselheim. aimed at rare diseases affecting children, such phase 3 clinical trials, he’s hardly surprised that A better way to encourage drug development as pediatric cancers and cystic fibrosis. Both there hasn’t been a bumper crop of PRVs nearly for rare and neglected tropical diseases is to bills are still being reviewed by congressional four years since it began. enhance the US National Institutes of Health’s committees. Ridley says he’s already seen evidence that (NIH's) funding for basic research science, However, the impact of the program PRVs have spurred interest in new research. Kesselheim says. However, with austerity on remains debatable for some. “PRVs would He pointed to NanoViricides of West Haven, the lips of every Washington pundit, advocating be a questionable policy even if they did Connecticut, which has identified a new for even maintaining existing funding levels work,” says Aaron Kesselheim, who studies molecule to combat —a tropical for research is not likely to earn points for any pharmacoeconomics at the Harvard School of disease that currently has no treatment beyond politician. Public Health in Boston. “But it turns out they fluid replacement and acetaminophen—in part Adamson says that although increasing don’t even seem to work.” thanks to the possibility of attaining a voucher, funding for NIH research is obviously desirable, Thus far, the FDA has granted only one PRV he claims. it’s difficult to develop therapeutics without voucher: in April 2009 to the Swiss drug giant Ridley acknowledges that the voucher collaborating with the pharmaceutical industry. for its malaria treatment Coartem system, as described in his original paper and in He says incentives are the most effective solution (artemether/lumefantrine). The company then its original implementation by Congress, is far to bring drug companies aboard, though PRVs used the PRV to speed up the review for an from perfect. “Some of those deficiencies reflect do need some fine-tuning, admittedly. expanded use of its anti-inflammatory antibody my naiveté as an academic,” he says. “They’re still something of an experiment,” Ilaris (canakinumab) for the treatment of gouty But the Creating Hope Act, Ridley says, Adamson says. arthritis, but an FDA advisory committee corrects several flaws in the PRV mechanism. Roxanne Palmer

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