Representatives McCaul and Van Hollen send letters to the FDA and Teva Pharmacueticals to seek more information about the ongoing shortage of daunorubicin, an essential therapy in the treatment of leukemia in children.
The Creating Hope Act of 2011
McCaul, Butterfield, Myrick, Van Hollen Introduce The Creating Hope Act of 2011
Incentivizes Pharmaceutical Industry to Develop New Treatments for Pediatric Cancers
‘Creating Hope Act’ Has Broad Bipartisan Support
WASHINGTON, D.C.— Congressmen Michael McCaul (R-TX), G.K. Butterfield (D-NC), Sue Myrick (R-NC), and Chris Van Hollen (D-MD) introduced legislation that would incentivize pharmaceutical companies to develop new drugs for rare pediatric diseases, such as childhood cancers, that have not been developed in decades because they are not profitable. Senators Bob Casey (D-PA) and Scott Brown (R-MA) introduced a companion bill, S. 606.
The Creating Hope Act of 2011, introduced on the day of the 2nd annual Childhood Cancer Summit, would expand the cost-neutral Food and Drug Administration priority review voucher (PRV) program, allowing pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases. Since 1980, the FDA has approved only one drug for treatment of childhood cancer, compared to 50 for adults.
Pediatric cancer remains the number one disease killer of American children, with about 35 children diagnosed every day. While survival rates have greatly improved for some types of pediatric cancers, 2,300 children are lost to this disease every year and thousands more encounter complications related to the harsh adult treatment. Furthermore, the lack of age-appropriate treatment options means that three out of five children who are lucky enough to survive these grueling battles will sufferfrom life-altering and/or life-threatening consequences resulting from harsh treatments developed and intended for adult patients.
“They win the war against cancer, only to battle the onset of other serious conditions the rest of their lives,” said Congressman McCaul, the founder and co-chairman of the House Childhood Cancer Caucus, which hosts the Summit. “The Creating Hope Act offers the best chance of encouraging pharmaceutical companies to develop treatments for children at no cost to taxpayers.”
“Encouraging the development of innovative and effective treatments for patients diagnosed with rare diseases, especially children, is vitally important,” said Congressman Butterfield. Whether a disease is rare or common, the need for effective care and potential cures is the same. The Creating Hope Act of 2011 provides a much needed incentive to promote research and development that will ultimately save lives.”
“I’m proud to join my colleagues in introducing the bipartisan Creating Hope Act. The goal of this legislation is to kick start private sector investment in new treatments for rare diseases affecting children, including childhood cancers,” said Congressman Chris Van Hollen (D-MD), co-chair of the Childhood Cancer Caucus. “It is time we help put the health care industry to work researching and developing treatments to help children and families who are struggling with these dreaded diseases.”
The cancers in children are different from cancers in adults, such that drugs developed to treat an adult malignancy, such as colon cancer, may not be useful for a cancer that occurs in children, such as neuroblastoma.
“A growing number of promising new drugs are being developed to treat cancers that occur in adults, but the biopharmaceutical industry rarely develops new drugs specifically to treat cancer in children,” said Dr. Peter Adamson, Chairman of the Children’s Oncology Group.
Despite this significant unmet medical need, pharmaceutical companies have been reluctant to develop drugs for rare pediatric diseases because it requires making an investment in products that are unlikely to cover the high costs associated with their research, development, marketing and distribution. Developing products for children is particularly challenging because of the difficulties associated with conducting clinical trials on this patient population.
The Creating Hope Act of 2011 will:
· Expand the Priority Review voucher program to include treatments for pediatric rare diseases, such as childhood cancers.
· Close a loophole in current law to prevent companies from receiving a voucher for products thatthey already market in other countries.
· Offer unlimited transferability of vouchers to create a more easily traded asset.
· Provide greater certainty to sponsors by permitting them to seek a designation from FDA before they submit their new drug application that the drug, if approved, will qualify for a voucher.
· Strengthen reporting and marketing requirements by requiring that the sponsor submit a statement of good faith intent to market the eligible drug.
“My son, Jacob, died of brain cancer two years ago because there were no effective drugs available to him. The Creating Hope Act gives us all hope that one day, children with brain cancer will not have to suffer Jacob's fate,” said Nancy Goodman, Founder of Kids v. Cancer.
Children, who suffer from rare pediatric diseases such as cancers, pediatric AIDS, cystic fibrosis, Tay-Sachs and sickle cell disease, make up the vast majority of the 30 million Americans who suffer from rare diseases, according to the National Organization for Rare Disorders (NORD).
Creating Hope Act Original Cosponsors:
Michael McCaul (R-TX)
G.K. Butterfield (D-NC)
Sue Myrick (R-NC)
Chris Van Hollen (D-MD)
Dr. Michael Burgess (R-TX)
Jackie Speier (D-CA)
Mike Kelly (R-PA)
Hank Johnson (D-GA)
Brian Bilbray (R-CA)
Danny Davis (D-IL)
Pete Olson (R-TX)
Edolphus Towns (D-NY)
Frank Wolf (R-VA)
Donna Christensen (D-Virgin Islands)
Dr. Charles Boustany (R-LA)
Bobby Rush (D-IL)
Cathy McMorris Rogers (R-WA)
Henry Cuellar (D-TX)
Geoff Davis (R-KY)
Bill Keating (D-MA)
Francisco "Quico" Canseco (R-TX)
Nancy Pelosi (D-CA)
Ileana Ros-Lehtinen (R-FL)
Steven Rothman (R-NJ)
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The following groups and businesses have endorsed the legislation:
Aeras Global TB Vaccine Foundation, American Childhood Cancer Organization, American Porphyria Foundation, American Society of Clinical Oncology, American Society of Pediatric Hematology and Oncology, American Society for Tropical Medicine and Hygiene, Association of Pediatric Oncology Social Workers, Bio Ventures for Global Health, Biotechnology Industry Association (BIO), Chai Lifeline, Children’s Brain Tumor Foundation, Children’s Cause for Cancer Advocacy, Children’s Hospital Association of Texas, Children’s Hospital of Philadelphia, Children’s Hospital of Pittsburgh at UPMC, Children’s Medical Center Dallas, Children’s Oncology Group (COG), CSL Behring, CureSearch, Friends of Scott Foundation, Genzyme, GlaxoSmithKline, Kakki’s Every Life Foundation, Kids v Cancer, Leukemia and Lymphoma Society, Madison’s Foundation, Max’s Ring of Fire, Medicines for Malaria Venture (MMV), Merek, Metronomix, National Adrenal Disease Foundation, National Association of Children’s Hospitals, National Brain Tumor Society, National Children’s Cancer Society (NCCS), National Tay-Sachs and Allied Diseases Fund, National Organization for Rare Diseases (NORD), Novartis, People Against Childhood Cancer (PAC2), Pennsylvania BIO, Rady Children’s Hospital, Rally Foundation, The Sarcoma Foundation of America, Seany Foundation, Shire, St. Baldrick’s Foundation, TB Alliance, Texas Children’s Hospital