Re-Introduction of the STAR Act in the 115th Congress
More on Creating Hope Act of 2011
With the passage of the 21st Century Cures Bill in the U.S. House, cancer research received a major boost while critical childhood cancer provisions have passed while others were extended.
The House passed the 21st Century Cures Act to increase funding for the National Institutes of Health (NIH) and reauthorize the Creating Hope Act which will incentivize the development of new treatments for children with rare pediatric diseases, such as cancer.
The sponsors of the Creating Hope Act applaud the approval of the first drug ever to treat high-risk neuroblastoma and call for the law to be made permanent.
Progress in Developing First Pediatric Cancer Treatments in Decades Highlights 4th Annual Childhood Cancer Summit
Progress into research and development of the first new pediatric cancer drugs in nearly 30 years was announced at the 4th Annual Congressional Childhood Cancer Summit on Capitol Hill on September 19, 2013.
President Obama signed a bill today that will provide incentives to drug companies to research and develop drugs for rare diseases. The Creating Hope Act grants pharmaceutical companies that create drugs for diseases like childhood cancers a voucher giving speedier review of any other drug they submit for approval. The Oval Office signature was a hard-fought victory for children like Mollie Ward, 11, who survived a rare form of pancreatic cancer thanks to an experimental drug, and for other families who have fought childhood cancers. Nancy Goodman, founder of Kids v. Cancer, which is devoted to pediatric cancer research, lost her son Jacob Froman, 10, to a rare form of brain cancer nearly four years ago. He’d been diagnosed at the age of 8. She was instrumental in getting the bill passed. She and others said that drug companies had little financial incentive to develop new treatments because childhood cancers are so rare. “The reason I started working on the Creating Hope Act was that I found very early on that there are just very few drugs to treat kids with cancer,” she told ABC News. “We created a big, fat carrot. The carrot is a voucher.” While 50 new drugs for adult cancer have been released in the last 20 years, just one expressly for pediatric cancer has gotten initial Food and Drug Administration approval, doctors say. The track record for drugs for other pediatric rare diseases is even worse. “The reason that companies don’t make pediatric drugs is all market force driven,” said Dr. Henry Friedman, an neuro-oncologist at Duke University Medical Center. “Companies have an obligation to their shareholders to make money. The pediatric diseases by and large are such low numbers. … The incentive to make pediatric drugs is very small.” Goodman said the measure was smart legislation and an attempt to ensure that more families don’t experience what she has. “I don’t think the American public realizes how devastating pediatric cancer is,” she said. “It’s far worse than any other adult cancer. There’s something unfair and unjust about the death of a child.”
Incentivizes Drug Companies to Develop New Treatments for Children with Rare Pediatric Diseases
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. firstname.lastname@example.org
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.
This was supposed to be the year for Jacob Froman's bar mitzvah, except that a rare and fatal brain cancer reached out for him first. Just 10, Jacob died in 2009, two years after a diagnosis of medulloblastoma, a rare brain cancer with no drug designed specifically to treat it. During the arduous months of ultimately unsuccessful treatments, his mother, Nancy Goodman, discovered that few pharmaceutical companies are fully invested in developing drugs specifically targeted toward children's cancer and other rare diseases. She established Kids v. Cancer, her hope for children suffering from pediatric diseases that lack drug treatments. A national organization, it aims to create a pediatric brain cancer tissue bank for researchers as well as a specific pediatric cancer subsection of the National Cancer Institute to provide funding for pediatric cancer research and incentives to pharmaceutical companies to create more drugs. Her persistence led her to Texas Republican Rep. Mike McCaul, who has sponsored the Creating Hope Act that, if passed, would offer drug companies the ability to expedite more profitable drugs through the Food and Drug Administration review process in exchange for the creation of new drugs to combat rare pediatric diseases. "Until we change the culture of drug companies to find a cure, we're never going to win this battle," McCaul declared. Market not lucrative According to Texas Children's Hospital, only about 12,000 children and teens are diagnosed with cancer each year, so the market for pediatric cancer drugs isn't very lucrative. "There's no profit in childhood cancer," McCaul said. "You would think childhood cancers would get the most attention, but just the opposite. It's been neglected for decades." With cancer, the drugs and therapies used to treat adults often don't affect children the same way, and can lead to health issues or new cancers down the road. Because children's cells multiply at a quicker rate than those in adults, chemotherapy - which attacks rapidly multiplying cells to stamp out the malignant ones - tends to affect children negatively in ways that adults don't experience. "There are kids with scores of other neurological diseases and they need help, too," said Goodman. The legislation is designed not only for children with cancer, but for "kids with other terrible rare diseases who … have no new drugs developed for their diseases." Texas resident Shannon Hayes, of Spring, stood behind McCaul last week when he announced the bill. Baring a bald head in memory of her daughter, she is part of 46 Mommas, a group of woman touched by pediatric cancer who shave their heads to raise awareness and money for the St. Baldrick's Foundation. Bill's prospects good Hayes' daughter, Delaney Starcher, died in 2008 at age 5 from a rare brain cancer. The disease builds around the brain stem like a "handful of sand thrown into a field of grass," she said, making it difficult to treat. McCaul is founder of the Childhood Cancer Congressional Caucus; his wife sits on the board of Houston's University of Texas M.D. Anderson Cancer Center, one of the world's foremost cancer treatment centers. He and 19 other House members, including Texas Reps. Michael Burgess, Henry Cuellar and Pete Olson, co-sponsored the bill, which McCaul said he expects will pass easily. And Goodman hopes that, eventually, every child with cancer will have the chance to throw a big bash when he or she turns 13.
FDA Law Blog: Rep. McCaul Seeks to Reinvigorate Interest in the Creating Hope Act of 2011; Introduces Companion Bill in the House
Last week, Representative Michael McCaul (R-TX), along with Reps. G.K. Butterfield (D-NC), Sue Myrick (R-NC), and Chris Van Hollen (D-MD), introduced H.R. 3059, the Creating Hope Act of 2011. The bill is a companion bill to S. 606, which was introduced in the Senate earlier this year by Senator Robert Casey (D-PA), along with co-sponsors Sens. Scott Brown (R-MA), Sherrod Brown (D-OH), Al Franken (D-MN), and Johnny Isakson (R-GA). Both the House and Senate versions of the Creating Hope Act of 2011, which are almost identical, are substantially similar to the 2010 version of the bill, S. 3697, which was introduced by now-retired Sen. Sam Brownback (R-KS). The Creating Hope Act would amend FDC Act § 524 to change the transferable Priority Review Voucher (“PRV”) program created by the 2007 FDA Amendments Act (the so-called “treat and trade” program), and would amend the PRV program to extend it to applications for a “rare pediatric disease.” Under the PRV program, sponsors of certain new drugs and biologics for “tropical diseases” that have received priority review may receive a PRV entitling the holder to a 6-month priority FDA review of another application that would otherwise be reviewed under FDA’s standard 10-month review clock. We previously reported on the Creating Hope Act of 2010 here, and we refer you to that post for a summary of the bill. The 2011 version of the bill, which we reported on here, makes some revisions to the 2010 version. The most significant change appears to be the conditions under which FDA may refuse to issue a PRV upon the approval of a rare pediatric disease product application – the so-called “good faith intent to market determination.” Under both the 2010 and 2011 bills, FDA may consider several factors in determining whether to refuse to issue a PRV, including “the history of such sponsor of producing rare pediatric disease products for which such sponsor received a [PRV], orphan drugs for which the sponsor received exclusivity under [FDC Act § 527], or pediatric drugs for which the sponsor received an additional 6 months of exclusivity under [FDC act § 505A].” Added to the 2011 version of the bill is the requirement that FDA issue guidance before making a good faith intent to market determination. Rep. McCaul, who co-chairs the House Childhood Cancer Caucus with Rep. Van Hollen, introduced H.R. 3059 on the day of the 2nd annual Childhood Cancer Summit. (The caucus website includes links to several stories on the Creating Hope Act and an endorsement video from actor Dennis Quaid.) In a Dear Colleague Letter signed by Rep. McCaul and the bill co-sponsors, they urge support for the bill, stating that it “would expand and strengthen the cost-neutral FDA [PRV] program, giving pharmaceutical companies an incentive to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions.” In addition, the letter notes that the possibility of a shortened review time offered by a PRV “is estimated to be worth hundreds of millions of dollars.” The true value of a PRV is debatable. BIO Ventures for Global Health has estimated that, based on certain assumptions, a PRV could be worth a few hundred million dollars. The group cautions, however, that “[o]ne factor that may reduce the market value of priority review vouchers is risk tolerance. For example, companies may not be willing to invest as much in obtaining a priority review voucher if there are any doubts as to whether or not their product will be approved.” To date, FDA has issued only a single PRV – with the approval of Novartis’ COARTEM (artemether; lumefantrine) for the treatment of acute, uncomplicated malaria infections in adults and children weighing at least five kilograms (see our previous post here). Novartis traded in the PRV (instead of selling it) to obtain priority review for a supplemental Biologics License Application for ILARIS (canakinumab) for the treatment of gouty arthritis attacks in certain patients, but an expedited review did not pan out for the company (see our previous post here). Thus, to date, the PRV market is untested. Adding further uncertainty to PRV value is the high PRV redemption user fee FDA has set. FDA recently set the Fiscal Year 2012 PRV redemption fee at $5,280,000 – and that amount is in addition to the Fiscal Year 2012 full application fee of $1,841,500.