Pediatric Rule Update on Capitol Hill May 2003

A bipartisan group of senators, led by the chair of the Senate HELP Committee, recently introduced legislation to codify the FDA's pediatric rule. The new bill, S. 650, received HELP Committee consideration and passed from Committee on a unanimous vote. Senate floor consideration has not yet been scheduled. During Committee consideration, Sen. Gregg offered an amendment to sunset the legislation in conjunction with the sunset in the ?Best Pharmaceuticals for Children Act? (October 2007). The amendment passed on a party-line vote. Champions of the legislation did not support the amendment; however it is recognized by those who voted for the amendment that in the current political landscape, the sunset provision gives the bill a better chance of passage. Sen. Judd Gregg (R-NH), HELP chair, introduced the bill, along with Senators Hillary Clinton (D-NY), Mike DeWine (R-OH), and Christopher Dodd (D-CT).

Pediatric Rule Update on Capitol Hill October 9, 2002

October 9, 2002
PHONE: 202-225-2015

Congresswoman Pryce Introduces Legislation to Codify the Pediatric Rule
Measure reflects compromise passed in Senate Health Committee

WASHINGTON, D.C. - House Republican Conference Vice-Chairman Deborah Pryce (R-OH-15) teamed up with Congresswoman Connie Morella (R-MD-8) today to introduce legislation that would codify the Food and Drug Administration's "Pediatric Rule" requiring drug companies to conduct safety tests of adult medicines most likely to be given to children.

The "Pediatric Rule" was first adopted in 1998 to urge improvements in information about drugs for children. Prior to establishment of the "Pediatric Rule," drugs were rarely tested on children and doctors were forced to guess how dosage levels for adults should be adjusted for children. The legislation will codify the rule to ensure manufacturers of certain new and marketed drugs and biological products will continue to test the safety and effectiveness of medicines for children and put this information on the product's label.
The impetus for codification of the rule stems from a March 18, 2002 announcement by the FDA that it would revise and suspend the rule for two years in order to study its necessity. Approximately one month later, the FDA announced it would continue to enforce the rule as it simultaneously takes steps to review it.
Pryce said that while she was relieved to learn the FDA would continue to enforce the rule, codification is necessary to ensure that the rule will be in place permanently.

"Many people have the misperception that children's bodies are just smaller versions of adult bodies, when that is just not the case. Simply reducing the dosage of medicine for the treatment of a child is not always effective and is definitely not always safe," Pryce said. "By protecting the 'Pediatric Rule' and continuing to provide incentives for testing medicines for kids, we will give doctors the information they need to provide our children the best quality health care possible."

The legislation introduced by Morella and Pryce mirrors a bipartisan compromise recently passed by the Senate Health, Education, Labor and Pensions Committee.

Pediatric Rule Update on Capitol Hill August 1, 2002

On August 1, the last day before the Senate recessed for the month of August, the full Senate Health, Education, Labor and Pensions Committee (HELP) unanimously reported out by voice vote the bipartisan bill S. 2394, which codifies the 1998 Pediatric Rule.

There was one consensus amendment included in the reported version of the bill. The amendment requires that the FDA continue its current practice of asking manufacturers to voluntarily conduct such studies for drugs already on the market before invoking the pediatric testing rule.
We are very pleased that such a strong and bipartisan vote came out of HELP Committee. This bodes well for future efforts this year on the bill.

On the House side, discussions have begun with Representative Tauzin's staff to strongly urge him to cosponsor and champion this legislation in the House. You will recall that H.R. 4730 was introduced in the House but currently has no republican cosponsors. We need to remedy that situation and the Senate HELP committee's unanimous bipartisan vote will certainly assist with those efforts.

Organizations representing children's health issues will continue to plug away at this during the August recess but we will ask for your renewed enthusiasm and action to push this bill forward on the Senate and House side when Congress returns in September.

In addition to thanking your local representatives, if you have a moment to drop the following Senators and their staff a note of thanks for their support of bill S.2394, I know it would be much appreciated: Senators Dodd (Debra Barrett); DeWine (Abby Kral); Clinton (Christina Ho); Kennedy (David Dorsey); Gregg (Vince Ventamiglia) and Frist (Shana Christrup).

Again, thank you for everyone's efforts through writing letters of concern to your representatives in the House and Senate. It couldn't have happened without your help!

Working together to provide better healthcare for our children.

Ruth Hoffman Executive Director, National Office Candlelighters Childhood Cancer Foundation

Pediatric Rule Details

On March 18 2002, the FDA announced to a U.S. District Court in Washington, D.C., that it intends to suspend the pharmaceutical industry?s requirement to do pediatric testing of drugs for the next two years. This decision effectively overturned a 1998 regulation where the FDA had mandated pediatric studies of a drug, if there was evidence suggesting that the drug might be beneficial to children. This regulation, known as the Pediatric Rule, was intended to encourage pediatric labeling for those products that were being submitted for an adult use, if the disease or condition existed in children and if the product was a therapeutic advance over existing approved products or was likely to have substantial use (greater than 50,000 children per year). Such testing was designed to encourage the development of drugs for children, as well as address issues of safety, dosing and labeling. Sadly, approximately 75 percent of medicines currently used in the United States still have not been tested for use by children. Most often, this results in under-dosing of medicine (which can mean the potential loss of therapeutic benefit) or overdosing of medicine (with attendant adverse side effects for our children). Now, after being in place less than 3 years, the FDA is considering suspending the Pediatric Rule which ensures that the pediatric population has crucial and important safety and efficacy information for new drugs, as well as some drugs that are currently on the market.

The recent announcement of a possible two year suspension followed a suit that was filed against the FDA a year ago by three organizations: The Competitive Enterprise Institute, Consumer Alert and the Association of American Physicians and Surgeons. Their charge was that the FDA did not have authority to require drug manufacturers to conduct pediatric tests of products. Of note, the lawyer hired by The Competitive Enterprise Institute, Mr. Dan Troy who initially filed the suit against the FDA on this matter, was appointed a year later by the current Administration, to be the Chief legal counsel of the FDA.

As background to explain the significance of this Federal initiative to pediatric oncology, there have been two important programs implemented that promote development of drugs for children. The first is a financial incentive program that began in 1997, which was renewed this year through the Best Pharmaceuticals for Children Act. This voluntary program rewarded pharmaceutical companies for pediatric drug development, by giving them an additional six months of patent and marketing ?exclusivity.? The second initiative, the above-mentioned Pediatric Rule was nonvoluntary, and forced drug companies to develop pediatric labeling for drugs that showed potential benefit to children. The critics filing suit against the FDA claimed that the first incentive program was sufficient on its own to entice industry to do pediatric drug testing. However, the original two-part program was designed to work hand in hand by both mandating industry to do pediatric testing of drugs (Pediatric Rule), as well as provide financial incentives to companies (Exclusivity). BOTH initiatives were needed to ensure that pediatric information about drugs is obtained. The key variables include:

• The Rule is mandatory; the incentives are voluntary.
• The Rule includes biological products; the incentives do not. (Biologics include: blood products, cancer vaccines, monoclonal antibodies, growth factors and numerous other products relevant to cancer research and drug development).
• The Rule is ongoing; the incentives are time-limited and must be renewed periodically. (The current incentive program sunsets in 2007).
• The Rule captures drugs that the incentives do not. The incentives program can only be used once, and if additional uses of a drug are discovered, then the Pediatric Rule is the only remaining mechanism to force study among children, when a public health need arises.

In response to the FDA?s recent actions, Candlelighters has joined with numerous pediatric organizations to express our concern, by signing on to a letter addressed to the Acting Commissioner of the FDA (Mr. Lester Crawford) and to the Honorable Tommy Thompson, the Secretary of the U.S. Department of Health and Human Services (HHS). It is our belief that any suspension, and possible abolishment of the Pediatric Rule could greatly impede pediatric oncology drug development.

How can you help?

Families are encouraged to express their personal concerns regarding FDA?s recent actions. A template letter has been added to our website (www.candlelighters.org) to assist those interested in writing a letter to their Senators, Representatives and to the secretary of the HHS, asking for a reversal of the FDA?s proposed suspension of the Pediatric Rule. Families are encouraged to add a personalized message about the importance of on-going drug development, safety testing and labeling of drugs for children. The following websites will assist you with locating contact information for your congressional representatives:

• U.S. House of Representatives: Write your Representative Service
• United States Senate: Contacting the Senate - Senators of the 107th Congress
• Project Vote Smart