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'Creating Hope Act' Now Law

Incentivizes Drug Companies to Develop New Treatments for Children with Rare Pediatric Diseases

Houston Chronicle: Pharmaceutical firms to get incentives for child cancer drugs

WASHINGTON - When all else failed, the promise of corporate profits for pediatric cancer drugs did what cajoling to save children could not. Legislation by Texas Rep. Michael McCaul, soon to be signed by President Barack Obama, will offer drug companies multimillion-dollar incentives to pioneer medications for rare childhood diseases that afflict too few kids to make a profit. The legislation is meant to remedy a chronic mismatch in which the FDA has approved dozens of new drugs to combat adult cancers since 1980 - and only one for the treatment of childhood cancer. "We're giving companies incentives to make money because the free market has failed to develop these medications," says McCaul, a five-term Austin Republican and father of five who founded the 94-member Congressional Childhood Cancer Caucus. The measure "fundamentally transforms the way drug companies look at rare pediatric diseases and compensates for market failures that have prevented any new treatment for pediatric cancer from being developed in a generation," he added. Dr. Eugenie Kleinerman, head of pediatrics at the University of Texas M.D. Anderson Cancer Center, said clinicians have been frustrated for years by the absence of state-of-the-art anti-cancer medications tailored to children. M.D. Anderson treats 2,000 pediatric patients a year including 200 in clinical trials. "But this is absolutely a move forward," says Kleinerman. "It will shine a light on the fact that we really haven't had access to the latest new therapies." Development of such drugs still will take five to seven years. Some 12,500 children up to the age of 18 are diagnosed with cancer each year - a fraction of the hundreds of thousands of adults who confront cancer diagnoses each year. Helping other families Families hit by childhood cancer embraced the promise of tailor-made drugs even though it is too late for their own kids. "There's been no excuse for having no drugs to treat children because there's more money to be made treating adults," says Donna Culliver, of Brenham, whose 4-year-old son, Adam, died in 2003 from fast moving acute myelogenous leukemia. She and her husband started Adam's Angels Ministry to help families and began lobbying for ways to encourage drug companies to do more. "We can't help our son, but we can help other families that receive a diagnosis of childhood cancer," Culliver said. "To us this legislation is a huge blessing - Adam's death wasn't in vain, it was for a reason." In return for developing drugs for the small and unprofitable market of rare childhood diseases, cooperating pharmaceutical manufacturers could earn vouchers for faster Food and Drug Administration approval of new and potentially more profitable drugs. By cutting FDA review time by 40 percent from an average of 10 months to six months, participating drug manufacturers could reap rewards of up to $500 million in additional sales on patent-protected medications that reach the competitive marketplace first, Congress estimates. Drug companies also could sell the vouchers to other companies jockeying with rivals to get their drug to market first. The legislation provides for three vouchers to be issued in coming years, followed by an audit on the effectiveness of the approach by Congress' watchdog Government Accountability Office. Drug companies Kate Connors, spokeswoman for the Pharmaceutical Research and Manufacturers of America, said drug manufactures poured almost $50 billion into developing and discovering new drugs last year and will support any provisions that "encourage research into how better to treat children." However, she added, the industry organization had not assessed yet the incentives included in the new law. Advocacy organizations and physicians say they will be reaching out to drug manufacturers in hopes of piquing interest and speeding development of medications targeting rare childhood diseases. "This will be a sea change in drug development," forecasts Nancy Goodman, who founded Kids v. Cancer after the death of her son Jacob at the age of 10 in 2009 from a pediatric brain cancer. "We will work with companies to help shepherd them through the FDA review process," said Goodman, who worked with doctors and researchers at more than 35 institutions across a dozen countries in an effort to find a medication to save her son's life. "We do so much up here on Capitol Hill that shows we're kind of spinning our wheels on things that never get done," said McCaul. "This is refreshing - it will make a real difference in the lives of children." McCaul's wife, Linda Mays McCaul, a multimillionaire heiress to her family's Clear Channel Communications, is a member of the board of M.D. Anderson. stewart.powell@chron.com

Houston Chronicle: Pharmaceutical firms to get incentives for child cancer drugs

WASHINGTON - When all else failed, the promise of corporate profits for pediatric cancer drugs did what cajoling to save children could not. Legislation by Texas Rep. Michael McCaul, soon to be signed by President Barack Obama, will offer drug companies multimillion-dollar incentives to pioneer medications for rare childhood diseases that afflict too few kids to make a profit. The legislation is meant to remedy a chronic mismatch in which the FDA has approved dozens of new drugs to combat adult cancers since 1980 - and only one for the treatment of childhood cancer. "We're giving companies incentives to make money because the free market has failed to develop these medications," says McCaul, a five-term Austin Republican and father of five who founded the 94-member Congressional Childhood Cancer Caucus. The measure "fundamentally transforms the way drug companies look at rare pediatric diseases and compensates for market failures that have prevented any new treatment for pediatric cancer from being developed in a generation," he added. Dr. Eugenie Kleinerman, head of pediatrics at the University of Texas M.D. Anderson Cancer Center, said clinicians have been frustrated for years by the absence of state-of-the-art anti-cancer medications tailored to children. M.D. Anderson treats 2,000 pediatric patients a year including 200 in clinical trials. "But this is absolutely a move forward," says Kleinerman. "It will shine a light on the fact that we really haven't had access to the latest new therapies." Development of such drugs still will take five to seven years. Some 12,500 children up to the age of 18 are diagnosed with cancer each year - a fraction of the hundreds of thousands of adults who confront cancer diagnoses each year. Helping other families Families hit by childhood cancer embraced the promise of tailor-made drugs even though it is too late for their own kids. "There's been no excuse for having no drugs to treat children because there's more money to be made treating adults," says Donna Culliver, of Brenham, whose 4-year-old son, Adam, died in 2003 from fast moving acute myelogenous leukemia. She and her husband started Adam's Angels Ministry to help families and began lobbying for ways to encourage drug companies to do more. "We can't help our son, but we can help other families that receive a diagnosis of childhood cancer," Culliver said. "To us this legislation is a huge blessing - Adam's death wasn't in vain, it was for a reason." In return for developing drugs for the small and unprofitable market of rare childhood diseases, cooperating pharmaceutical manufacturers could earn vouchers for faster Food and Drug Administration approval of new and potentially more profitable drugs. By cutting FDA review time by 40 percent from an average of 10 months to six months, participating drug manufacturers could reap rewards of up to $500 million in additional sales on patent-protected medications that reach the competitive marketplace first, Congress estimates. Drug companies also could sell the vouchers to other companies jockeying with rivals to get their drug to market first. The legislation provides for three vouchers to be issued in coming years, followed by an audit on the effectiveness of the approach by Congress' watchdog Government Accountability Office. Drug companies Kate Connors, spokeswoman for the Pharmaceutical Research and Manufacturers of America, said drug manufactures poured almost $50 billion into developing and discovering new drugs last year and will support any provisions that "encourage research into how better to treat children." However, she added, the industry organization had not assessed yet the incentives included in the new law. Advocacy organizations and physicians say they will be reaching out to drug manufacturers in hopes of piquing interest and speeding development of medications targeting rare childhood diseases. "This will be a sea change in drug development," forecasts Nancy Goodman, who founded Kids v. Cancer after the death of her son Jacob at the age of 10 in 2009 from a pediatric brain cancer. "We will work with companies to help shepherd them through the FDA review process," said Goodman, who worked with doctors and researchers at more than 35 institutions across a dozen countries in an effort to find a medication to save her son's life. "We do so much up here on Capitol Hill that shows we're kind of spinning our wheels on things that never get done," said McCaul. "This is refreshing - it will make a real difference in the lives of children." McCaul's wife, Linda Mays McCaul, a multimillionaire heiress to her family's Clear Channel Communications, is a member of the board of M.D. Anderson. stewart.powell@chron.com

Washington Post: Out of tragedy, some hope

This is the saddest story you will ever read about how a bill becomes law. It should also make you feel better about our dysfunctional political system. The story begins on a Sunday in February 2007, when a doctor suggested that Jacob Froman, then 8, had brain cancer. An MRI on Monday confirmed the grim diagnosis: metastatic medulloblastoma. Jacob was in surgery at 7 the next morning. 84 Comments Weigh InCorrections?Recommend Tweet Personal Post Ruth Marcus An editorial writer specializing in politics, the budget and other domestic issues, she also writes a weekly column and contributes to the PostPartisan blog. Archive @RuthMarcusFacebookRSSGallery Tom Toles on health care: A collection of cartoons on the debate. You may also like... Dana Milbank Ruined by closed-primary rule Matt Miller GOP to the uninsured: Drop dead .“And then,” says his mother, Nancy Goodman, “he was never the same.” After the surgery came radiation and chemotherapy. But within two weeks, it became clear the treatment wasn’t likely to work. Jacob’s doctors stuck with it — because there weren’t other options. When she embarked on a quest to save her son, Goodman, an international trade lawyer with degrees from Harvard (public policy) and Chicago (law), discovered that in the past 30 years there have been no major changes in treating medulloblastoma, which primarily affects children. Only one pediatric cancer drug has won initial approval from the Food and Drug Administration in 20 years. The National Cancer Institute allocates 4 percent of its budget to pediatric research. Children account for far less than 4 percent of cancer cases but, then again, cancer wreaks particular devastation on a child. Ordeal is too mild a word to describe what Jacob and his family endured. He spent nine of his remaining 23 months in the hospital. Jacob died at age 10 on Jan. 16, 2009. The next morning, “I put my laptop on the dining room table and started working,” Goodman says of the organization she launched, Kids v Cancer. “I was alive and Jacob wasn’t.” The battle against childhood cancer is sometimes framed as a success story. Five-year survival rates from acute lymphoblastic leukemia, which accounts for three in four cases of pediatric cancer, are now 90 percent — up from 10 percent in the 1960s. But those encouraging numbers obscure painful truths: That surviving for five years means something different at 8 than at 58. That the after-effects of treatment for children can be terrible. And, most pertinent, that the economics of pharmaceutical research disfavor new treatments. The small number affected, combined with the risk of pumping toxic chemicals into tiny bodies, discourages investment. Children with cancer are mostly relegated to “hand-me-down” drugs for adult cancers. Kids v Cancer aims to transform that bleak landscape. One approach Goodman quickly identified was to copy an existing voucher program designed to encourage research into neglected tropical diseases. The concept behind what came to be known as the Creating Hope Act was to dangle a fat carrot: Companies that obtained FDA approval for new pediatric cancer drugs would also receive vouchers guaranteeing speedier review of other, potentially more blockbuster drugs. This valuable commodity, possibly worth millions, could be sold. Manufacturers would pay fees for the faster review. Goodman isn’t exactly a political naif. Her husband, Michael Froman, is an assistant to President Obama, his law school classmate. But this is not a tale of Washington connections. Goodman cold-called a staffer for Sen. Sherrod Brown, an Ohio Democrat who had worked on the tropical disease bill, and made her pitch. Goodman found an unlikely pairing for the liberal Brown in Kansas Republican Sen. Sam Brownback. And when the bill did not become law in 2010, Goodman found another conservative Republican, Texas Rep. Michael McCaul, to carry the torch the next year, along with North Carolina Democrat G.K. Butterfield. Goodman and assistant Adrienne Westcott, a 24-year-old leukemia survivor, found staffers uniformly willing to hear them out. They enlisted compelling lobbyists: Jacob’s younger brother Ben, Jacob’s school friends. Brianna Commerford, a 13-year-old survivor of Hodgkin’s lymphoma, recruited House Minority Leader Nancy Pelosi (D-Calif.) and Rep. Ron Paul (R-Tex.) as co-sponsors in a single day. Despite the heart-tugging subject, the measure was an uphill climb. The FDA wasn’t thrilled about being told what drugs to prioritize. Some lawmakers worried that the pharmaceutical industry would take the vouchers without producing. The most likely vehicle for getting the measure passed was the FDA reform bill, but there was pressure to limit amendments. But when the FDA bill was finally passed last week, the Creating Hope Act remained in. “She did an amazing thing,” recalled Ellie Dehoney, the former Brown staffer. “It was a real long shot and she just met with people and met with people and met with people.” Other grieving parents have turned tragedy into legislation: Megan’s Law, Amber Alerts. But in our partisan, gridlocked, money-drenched politics, Jacob’s story stands out, almost as gratifying as it is sad.

Washington Post: Out of tragedy, some hope

This is the saddest story you will ever read about how a bill becomes law. It should also make you feel better about our dysfunctional political system. The story begins on a Sunday in February 2007, when a doctor suggested that Jacob Froman, then 8, had brain cancer. An MRI on Monday confirmed the grim diagnosis: metastatic medulloblastoma. Jacob was in surgery at 7 the next morning. 84 Comments Weigh InCorrections?Recommend Tweet Personal Post Ruth Marcus An editorial writer specializing in politics, the budget and other domestic issues, she also writes a weekly column and contributes to the PostPartisan blog. Archive @RuthMarcusFacebookRSSGallery Tom Toles on health care: A collection of cartoons on the debate. You may also like... Dana Milbank Ruined by closed-primary rule Matt Miller GOP to the uninsured: Drop dead .“And then,” says his mother, Nancy Goodman, “he was never the same.” After the surgery came radiation and chemotherapy. But within two weeks, it became clear the treatment wasn’t likely to work. Jacob’s doctors stuck with it — because there weren’t other options. When she embarked on a quest to save her son, Goodman, an international trade lawyer with degrees from Harvard (public policy) and Chicago (law), discovered that in the past 30 years there have been no major changes in treating medulloblastoma, which primarily affects children. Only one pediatric cancer drug has won initial approval from the Food and Drug Administration in 20 years. The National Cancer Institute allocates 4 percent of its budget to pediatric research. Children account for far less than 4 percent of cancer cases but, then again, cancer wreaks particular devastation on a child. Ordeal is too mild a word to describe what Jacob and his family endured. He spent nine of his remaining 23 months in the hospital. Jacob died at age 10 on Jan. 16, 2009. The next morning, “I put my laptop on the dining room table and started working,” Goodman says of the organization she launched, Kids v Cancer. “I was alive and Jacob wasn’t.” The battle against childhood cancer is sometimes framed as a success story. Five-year survival rates from acute lymphoblastic leukemia, which accounts for three in four cases of pediatric cancer, are now 90 percent — up from 10 percent in the 1960s. But those encouraging numbers obscure painful truths: That surviving for five years means something different at 8 than at 58. That the after-effects of treatment for children can be terrible. And, most pertinent, that the economics of pharmaceutical research disfavor new treatments. The small number affected, combined with the risk of pumping toxic chemicals into tiny bodies, discourages investment. Children with cancer are mostly relegated to “hand-me-down” drugs for adult cancers. Kids v Cancer aims to transform that bleak landscape. One approach Goodman quickly identified was to copy an existing voucher program designed to encourage research into neglected tropical diseases. The concept behind what came to be known as the Creating Hope Act was to dangle a fat carrot: Companies that obtained FDA approval for new pediatric cancer drugs would also receive vouchers guaranteeing speedier review of other, potentially more blockbuster drugs. This valuable commodity, possibly worth millions, could be sold. Manufacturers would pay fees for the faster review. Goodman isn’t exactly a political naif. Her husband, Michael Froman, is an assistant to President Obama, his law school classmate. But this is not a tale of Washington connections. Goodman cold-called a staffer for Sen. Sherrod Brown, an Ohio Democrat who had worked on the tropical disease bill, and made her pitch. Goodman found an unlikely pairing for the liberal Brown in Kansas Republican Sen. Sam Brownback. And when the bill did not become law in 2010, Goodman found another conservative Republican, Texas Rep. Michael McCaul, to carry the torch the next year, along with North Carolina Democrat G.K. Butterfield. Goodman and assistant Adrienne Westcott, a 24-year-old leukemia survivor, found staffers uniformly willing to hear them out. They enlisted compelling lobbyists: Jacob’s younger brother Ben, Jacob’s school friends. Brianna Commerford, a 13-year-old survivor of Hodgkin’s lymphoma, recruited House Minority Leader Nancy Pelosi (D-Calif.) and Rep. Ron Paul (R-Tex.) as co-sponsors in a single day. Despite the heart-tugging subject, the measure was an uphill climb. The FDA wasn’t thrilled about being told what drugs to prioritize. Some lawmakers worried that the pharmaceutical industry would take the vouchers without producing. The most likely vehicle for getting the measure passed was the FDA reform bill, but there was pressure to limit amendments. But when the FDA bill was finally passed last week, the Creating Hope Act remained in. “She did an amazing thing,” recalled Ellie Dehoney, the former Brown staffer. “It was a real long shot and she just met with people and met with people and met with people.” Other grieving parents have turned tragedy into legislation: Megan’s Law, Amber Alerts. But in our partisan, gridlocked, money-drenched politics, Jacob’s story stands out, almost as gratifying as it is sad.

3rd Annual Childhood Cancer Summit

Childhood cancer advocates go to Capitol Hill to fight for improved care

Joint Childhood Cancer Caucus--High Tech Caucus Briefing with Dell and TGen

The Congressional Childhood Cancer Caucus and High Tech Caucus held a briefing on "Personalized Medicine: How Technology is Driving Innovation." This briefing featured a discussion: •The world’s first personalized medicine clinical trial for pediatric cancer. •How technology is helping researchers identify a greater depth of personalized treatment strategies. •Challenges to developing new treatments for children with cancer. •The future of personalized medicine. Panelists include: •JAMIE COFFIN, VICE PRESIDENT AND GENERAL MANAGER, DELL HEALTHCARE & LIFE SCIENCES •SPYRO MOUSSES, PROFESSOR AND DIRECTOR, CENTER FOR BIOINTELLIGENCE AND VICE PRESIDENT, OFFICE OF INNOVATION, TRANSLATIONAL GENOMICS RESEARCH INSTITUTE (TGEN) •NANCY GOODMAN, EXECUTIVE DIRECTOR, KIDS V CANCER

Joint Childhood Cancer Caucus-High Tech Caucus Briefing

40 Years of Progress in Cancer Research: Transforming Patient Care through Innovation