FDA sometimes is challenged to meet the deadline for completing the review of whether a drug sponsor's clinical trials in children qualify a product for pediatric exclusivity, Office of New Drugs Deputy Director Sandra Kweder indicated May 22.

The Best Pharmaceuticals for Children Act, which provides six months additional market exclusivity for qualifying products, gives FDA 90 days from receipt of results to determine whether the sponsor "fairly responded" to the agency's request for a study on safety and efficacy in children.

During a House Energy and Commerce Committee Subcommittee on Health hearing on pediatric product innovations, several members, including California Democrats Henry Waxman and Anna Eshoo, raised questions about the review provision.

"It is extremely difficult" to reach a conclusion "within 90 days, in many cases," Kweder said in response to questioning by Waxman.

Since 1997, FDA has made 150 exclusivity determinations and granted exclusivity in 136 instances, Kweder said. "In some cases we would have been very happy to have more than 90 days to make that determination." She said there have been instances in which FDA later believed exclusivity should have been denied because it found the study was not well conducted.

The hearings were a prelude to subcommittee markup of legislation to reauthorize BPCA and the Pediatric Research Equity Act, which both expire Sept. 30. The Senate approved reauthorization of the two laws as part of its FDA Revitalization Act (¹ Also see "FDA Reform Bill Leaves Senate With Reduced Safety Powers But Stiffer Fines" - Pink Sheet, 14 May, 2007., p. 16).

Committee leaders intend to draft separate bills to reauthorize the two pediatric programs, aiming to get them through subcommittee and full committee markups and to the House floor around July 4, a staffer said.

The Senate-passed BPCA reauthorization would shorten exclusivity to three months for "blockbuster" drugs, those with annual sales topping $1 billion (² Also see "Rx Safety Bill Produces Restless REMS; Core Of “KEnzi” Is Narrowed But Intact" - Pink Sheet, 23 April, 2007., p. 3).

House subcommittee Chairman Frank Pallone, D-N.J., indicated he is still considering such issues. "There may be a better way to balance the need to provide incentives" for pediatric studies with the consumer's need for lower cost drugs, he said.

But Rep. Tim Murphy, R-Pa., suggested that some diseases might merit longer exclusivity than now provided. For diseases with "awful, tragic consequences ... shouldn't we have different levels, sometimes six months, sometimes longer," so companies can consider making investments in these areas? he asked.

Kweder took no position on altering the exclusivity period, but she did say that if FDA had to consider issues such as company earnings, "we would certainly have to work to attain some expertise in helping us make those" determinations and the process could be delayed.

Some of the impetus for shorter exclusivity for big-ticket products came from a recent study in the Journal of the American Medical Association, which found that companies sometimes see hundreds of millions of dollars in economic benefit during the exclusivity period (³ Also see "Pediatric Exclusivity May Overcompensate Blockbuster Products – JAMA Study" - Pink Sheet, 12 February, 2007., p. 25).

Since BPCA's inception in 1997, resulting pediatric studies have led to labeling changes for 128 products, according to Kweder. The use of 83 products was expanded to a broader pediatric population; 25 drugs had dosing adjustments; 28 were found not safe or effective for children; and 37 had new or enhanced pediatric safety information added.

Asked about the need for more resources to speed up labeling changes, which the General Accountability Office has found can take several years, Kweder maintained that delays are a matter of inadequate data, not lack of resources.

She remarked: "We have gotten much better at ... making labeling changes that we can make early, while we wait for additional data to supplement the labeling later."

- Cathy Dombrowski (c.dombrowski@elsevier.com)

[Editor's note: This story has been changed to correct to the length of exclusivity that the Senate bill offers blockbuster products.]

Pediatric Exclusivity 90-Day Review Can Be “Difficult” Target – Kweder

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