The Miami Herald

Rare disease drug-approval provision clears Congress

The Senate on Tuesday approved a proposal from Sen. Kay Hagan, D-N.C., that will quicken the approval process for drugs intended to treat patients with rare diseases.

The provision passed the Senate as part of a wider bill that updates and reworks the user fees paid by drug companies that are seeking Food and Drug Administration approval for their products.

The FDA Safety and Innovation Act passed the Senate 92-4; it’s already cleared the House of Representatives and now moves to President Obama for his signature.

Hagan originally proposed her provision as a standalone piece of legislation in February. It’s intended to expedite the review of treatments for patients who have certain serious or life-threatening diseases.

The provision is significant for patients suffering from diseases that don’t have adequate treatment options or might have no treatment options at all, Hagan said Tuesday in a conference call. She said the provision would enhance the uniformity and consistency of the FDA’s accelerated-approval system; since 1992, the agency has allowed faster approval for drugs that treat certain serious illnesses.

Fewer than 250 of the 7,000 known rare diseases have FDA-approved medications, according to Diane Dorman, the vice president for public policy at the National Organization for Rare Disorders.

In a statement, Hagan said the bill’s passage “represents tremendous progress in bringing medical advances to the patients who need them most. . . . I am hopeful that the bill will bring renewed hope to these patients and their families.”

The wider FDA bill revises and extends user fees that pay for the prescription-drug and medical-device approval process; it also creates user-fee programs for generic drugs and biopharmaceutical products. The drug and medical device industries have paid such fees for FDA reviews since 1992.

Public Citizen, a consumer advocacy group that’s often critical of the drug industry and the FDA, opposes the user-fee law and the new approval process Hagan proposed.

“The problem with the accelerated approval process is that it does set a lower bar for bringing things to market,” said Dr. Michael Carome, the deputy director of Public Citizen’s Health Research Group. In addition, Carome said, user fees create a conflict of interest for the FDA and the drug and medical device industries.

“We’ve long opposed user fees because we believe it sets up and alters the balance and relationship between the regulator and the industry that’s regulated in a way that’s not good for consumers,” he said. “This tilts the agency toward making decisions in favor of the industry.”

Hagan and Sen. Tom Harkin, D-Iowa, who sponsored the user-fee legislation, emphasized the bipartisan nature of the bill.

“Well over a year of diligent bipartisan work has gone into the legislation before us today, and I believe we have hit the sweet spot,” Harkin said in a statement Monday before the bill passed the Senate. “Neither Democrats nor Republicans got everything they wanted in this bill. . . . Where we could not achieve consensus, we did not allow our differences to distract us from the critically important goal of producing a bill that everyone could support.”




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