More on Creating Hope Act of 2011

Roll Call: Creating Hope for Kids With Rare Diseases

Imagine your child fighting for his life, taking massive doses of highly toxic radiation, chemotherapy to kill the cancer in his body before it kills him. After years of pain and uncertainty, he beats the disease, only to find out that he will likely develop serious medical conditions related to the treatment that saved him. While survival rates are up for some types of pediatric cancer, for more than half who beat the odds and proudly call themselves “survivors,” the fight is not over. Three out of five are later afflicted with life-altering and life-threatening conditions, including secondary cancers, as a result of harsh treatments developed for adults. Since the 1980s, the Food and Drug Administration has approved only one new treatment for any type of childhood cancer. This is compared with 50 approved treatments for adult cancers during the same time period. So why, despite this significant unmet medical need, is more not being done? The problem, as is often the case, is money. Pharmaceutical companies have been reluctant to develop drugs for rare pediatric diseases because it requires making an investment in products that do not cover the high costs associated with their research, development, marketing and distribution. The good news is that there is now hope for children living with cancer and other rare pediatric diseases. The Creating Hope Act, which we introduced last week, would give pharmaceutical companies an incentive to develop treatments by strengthening the FDA priority review voucher program. In short, it would allow pharmaceutical companies to receive faster FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases, at no cost to taxpayers. Pharmaceutical companies can receive a priority review voucher now if they develop novel treatments for neglected tropical diseases, such as malaria and leprosy, entitling the company to a priority six-month review of another new drug application that would otherwise be reviewed under the FDA’s standard 10-month period. The Creating Hope Act would expand this program to offer pharmaceutical companies the same shortened review time in return for development of new treatments for children with rare diseases. This shortened review time, which can lead to earlier market entry, is estimated to be worth hundreds of millions of dollars. While survival rates have greatly improved for many rare pediatric diseases, too many families lose children to diseases for which no treatments, or no age-appropriate ones, exist. More than 2,400 children die of cancer each year, making it the No. 1 disease killer of American children. Children who suffer from rare pediatric diseases such as cancers, 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. Until now, pharmaceutical companies have been able to conveniently dismiss this unmet medical need as too costly and as a threat to business. Not anymore. The Creating Hope Act will give these children a voice in Congress. It is time for pharmaceutical companies to come to the table and to work on their behalf. Congress is listening — we must pass the Creating Hope Act. Rep. Michael McCaul (R-Texas) is the founder and co-chairman of the Childhood Cancer Caucus, of which Rep. G.K. Butterfield (D-N.C.) is a member.

ABC News: Getting Big Pharma to Treat Childhood Cancers

At age five, some kids have won pee-wee sports championships or perhaps a class spelling bee, but for Luke Fochtman of Okemos, Mich., his fifth birthday marked his victory over a much larger foe -- in a life-or-death battle with childhood cancer, Luke has come out on top. "Through 72 weeks of treatment, chemotherapy, 105 fevers, he never said 'no' to us," says Luke's mom Monica Fochtman, 36. "That to me was very inspiring and humbling. To be in the presence of that kind of grace gave me the courage to keep going," she says. Though Luke's type of pediatric sarcoma has a high recurrence rate, he is currently cancer free. In honor of her little hero, Fochtman shaved her head this week alongside 45 other mothers of children with cancer as part of a larger effort to raise awareness for pediatric cancer. This "Shave for the Brave" event took place on Wednesday in the nation's capital as part of a series events leading up to Friday's the Second Annual Childhood Cancer Summit and meeting of the Congressional Pediatric Cancer Caucus. ABCNEWS.comView Full Size ABCNEWS.com Breast Cancer Easier to Spot With New Technology Watch Video When Bone Marrow Donors Back Out Watch Video Vladimir Putin Belts Out 'Blueberry Hill' Watch Video One of the biggest issues on the docket this year is the dire need for better pediatric cancer treatments. Because there are so many different types of pediatric cancers, the market for any particular drug would be small, making it highly unlikely that pharmaceutical companies will take up the cause. As a result, the vast majority of drugs used on pediatric cancer patients today were created for adults 30 or even 50 years ago. Luke's chemotherapy was developed 25 years ago, says Fochtman, and was so harsh that it caused repeated bacterial infections in her son as his immune system was completely knocked out by the chemo rounds. "We are desperate for new treatments. We have not had a single meaningful improvement in pediatric cancer medication in decades and the children have paid the price," says Dr. Peter Adamson, chief of the Division of Clinical Pharmacology & Therapeutics at The Children's Hospital of Philadelphia. "Even though we cure four out of five pediatric cancer patients, even those who survive often go on to have lifelong side effects from the treatment we give them." Adamson will be presenting to the Caucus Friday, arguing for changes in legislation to make developing pediatric cancer drugs more financially appealing to pharmaceutical companies. Getting Big Pharma to Fund Pediatric Cancer Drugs Friday's summit will be geared towards members of congress, hill staff, and pediatric cancer advocates with a panel on "The Future of Childhood Cancer Drug Development." Two previous legislations concerning pediatric drugs are up for modification and a new legislation, known as the Creating Hope Act, will be presented to the caucus. The bill will constitute the first federal incentive to pharmaceutical companies to specifically research and market pediatric cancer drugs. "We live in the country with the world's leading researchers and scientists, and yet almost none of that talent is being directed toward drugs for our children. The problem is that the size of the drug markets for pediatric cancer drugs is too small for drug companies to enter," said Nancy Goodman, founder and director of Kids v Cancer in a statement for the Summit. Goodman, whose son Jacob passed away five years ago from pediatric brain cancer, has been instrumental force in the creation of the Creating Hope Act. "There have been a number of legislative efforts to promote new drug development, but frankly, they have not been effective for pediatric cancer drugs," Goodman said. The previous laws, the Best Pharmaceuticals for Children Act (BTCA) and the Pediatric Research Equity Act (PREA), both deal with encouraging pharmaceutical companies to do clinical trials for their adults drugs on kids to see if there are important pediatric applications of a new drug. The Creating Hope Act however, will allow companies that research and develop a pediatric cancer drug to expedite the FDA review of another drug they are developing. The idea, explains Adamson, is that selling a pediatric cancer drug is not very lucrative, because a single type of pediatric cancer may only have a few hundred new patients each year, as opposed to, say, a diabetes drug which would have millions. This act would make investing in pediatric cancer research more economically viable by allowing them to get another, more lucrative drug they produce, to market faster. "Instead of nine months in FDA review, it will happen much faster," says Adamson.

FOX News: Lobbyists Take to Capitol Hill for Childhood Cancer

WASHINGTON - 8-year-old Ryan Darby marched right up on a stage in the Capitol Visitor's Center, stood on a ladder and talked about his dream. "Maybe one day, every kid that gets diagnosed with leukemia will beat it, like me." Ryan has beaten leukemia, but one in five kids with cancer do not survive. "There are 13,500 children that are diagnosed with cancer each year. That sounds like a lot, but actually there are a lot of different types of childrens' cancer, so when you start to break it down, individual types of cancer are so rare that there is not a lot of incentive for pharmaceutical companies to get involved in developing and discovering drugs for childrens' cancer," says John Lehr of CureSearch Children's Cancer. Only one drug for pediatric cancer has been developed in the last 20 years. Nancy Goodman's son, Jacob, died of brain cancer when he was 10-years-old. "Within two weeks of Jacob's diagnosis, Jacob's medical team had information that the drugs they were intending to give him were unlikely to work. And they continued giving him those drugs because there were no alternatives available," says Goodman, now executive director of KidsvCancer. Goodman lives in D.C. Ryan is from Bethesda. Both made the short trip to lobby on Capitol Hill for something called the "Creating Hope Act." Here's how it would work. When a company wants to get a new money-making drug on the market, it has to wait in line at the FDA. But if that company were willing to manufacture a less lucrative pediatric cancer drug, both that drug and the big money-maker drug would be moved to the front of the line. Ryan says his doctor, Aziza Shad, saved his life at Georgetown University Hospital. She wishes she could do that more often. "I think the hardest part of my job is when you come to what we call the end of the line. When you've given every possible treatment to a child and there is nothing new to offer. I think that will change because of this act," says Dr. Shad. Read more: https://www.myfoxdc.com/dpp/news/local/lobbyists-take-to-capitol-hill-for-childrens-cancer-092311#ixzz1ZByeDjMD

The Creating Hope Act of 2011

Creating Hope Act Press Conference

Austin American-Statesman: Hope for children with cancer

Imagine your child fighting for his life, taking massive doses of highly toxic radiation and chemotherapy to kill the cancer in his body before it kills him. After years of pain and uncertainty, he beats the disease, only to find out that he will likely develop serious medical conditions related to the treatment that saved him. Though survival rates are up for some types of pediatric cancer, for more than half of those who beat the odds and proudly call themselves "survivors," the fight is not over. Three out of five are later afflicted with life-altering and life-threatening conditions as a result of harsh treatments developed for adults, including secondary cancers. Since the 1980s, the Food and Drug Administration has approved only one new treatment for any type of childhood cancer. This compares with 50 approved treatments for adult cancers during the same time period. So, why, despite this significant unmet medical need, is more not being done? The problem, as is often the case, comes down to money. Pharmaceutical companies have been reluctant to develop drugs for rare pediatric diseases because it requires making an investment in products that do not cover the high costs associated with their research, development, marketing and distribution. The good news is that there is now hope for children living with cancer and other rare pediatric diseases. The Creating Hope Act, which we introduced in the U.S. House of Representatives this week, would give pharmaceutical companies an incentive to develop treatments by strengthening the FDA priority review voucher program. In short, it would allow pharmaceutical companies to receive faster FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases, at no cost to taxpayers. Presently, pharmaceutical companies can receive a priority review voucher if they develop novel treatments for neglected tropical diseases, such as malaria and leprosy, entitling the company to a priority six-month review of another new drug application that would otherwise be reviewed under the FDA's standard 10 month review period. The Creating Hope Act would expand this program to offer pharmaceutical companies the same shortened FDA review time in return for development of new treatments for children with rare diseases. This shortened review time, which can lead to earlier market entry, is estimated to be worth hundreds of millions of dollars. Though survival rates have greatly improved for many rare pediatric diseases, too many families lose children to diseases for which no treatments, or no age-appropriate treatments, exist. More than 2,400 children die of cancer each year, making it the number one disease killer of American children, more than asthma, diabetes, cystic fibrosis and AIDS combined. Children who suffer from rare pediatric diseases such as cancers, 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. Children are not old enough to vote and they do not have lobbyists fighting for them in Washington — and yet many of them are sick and in need of new treatments. Until now, pharmaceutical companies have been able to conveniently dismiss this unmet medical need as too costly and as a threat to business. Not anymore. The Creating Hope Act will give these children a voice in Congress. It is time for pharmaceutical companies to come to the table and to work on their behalf. Congress is listening. We must pass The Creating Hope Act. McCaul, R-Texas, is founder and co-chairman of the Childhood Cancer Caucus. Butterfield, D-North Carolina, is a member of the caucus.

Dennis Quaid Supports the Creating Hope Act

Houston Chronicle: Creating hope for children with rare diseases

Imagine your child fighting for his life, taking massive doses of highly toxic radiation and chemotherapy to kill the cancer in his body before it kills him. After years of pain and uncertainty, he beats the disease, only to find out that he will likely develop serious medical conditions related to the treatment that saved him. While survival rates are up for some types of pediatric cancer, for more than half who beat the odds and proudly call themselves survivors, the fight is not over. Three out of five are later afflicted with life-altering and life-threatening conditions as a result of harsh treatments developed for adults, including secondary cancers. Since the 1980s, the Food and Drug Administration has approved only one new treatment for any type of childhood cancer. This compares to 50 approved treatments for adult cancers during the same time period. So why, despite this significant unmet medical need, is more not being done? The problem, as is often the case, comes down to money. Pharmaceutical companies have been reluctant to develop drugs for rare pediatric diseases because it requires making an investment in products that do not cover the high costs associated with their research, development, marketing and distribution. The good news is that there is now hope for children living with cancer and other rare pediatric diseases. The Creating Hope Act, which we introduced in the U.S. House of Representatives this week, would give pharmaceutical companies an incentive to develop treatments by strengthening the FDA priority review voucher (PRV) program. In short, it would allow pharmaceutical companies to receive faster FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases, at no cost to taxpayers. Presently, pharmaceutical companies can receive a priority review voucher if they develop novel treatments for neglected tropical diseases, such as malaria and leprosy, entitling the company to a priority six-month review of another new drug application that would otherwise be reviewed under the FDA's standard 10-month review period. The Creating Hope Act would expand this program to offer pharmaceutical companies the same shortened FDA review time in return for development of new treatments for children with rare diseases. This shortened review time, which can lead to earlier market entry, is estimated to be worth hundreds of millions of dollars. While survival rates have greatly improved for many rare pediatric diseases, too many families lose children to diseases for which no treatments, or no age-appropriate treatments, exist. More than 2,400 children die of cancer each year, making it the No. 1 disease-killer of American children - more than asthma, diabetes, cystic fibrosis and AIDS combined. Children who suffer from rare pediatric diseases such as cancers, 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. Most of our nation's children are not old enough to vote, they do not have lobbyists fighting for them in Washington, and yet they are sick and in need of new treatments. Until now, pharmaceutical companies have been able to conveniently dismiss this unmet medical need as too costly and as a threat to business. Not anymore. The Creating Hope Act will give these children a voice in Congress. It is time for pharmaceutical companies to come to the table and to work on their behalf. Congress is listening - we must pass The Creating Hope Act. McCaul, a Texas Republican, is founder and co-chair of the Childhood Cancer Caucus, of which Butterfield, a North Carolina Democrat, is a member.

My FOX NY: Pediatric Cancer: David and Max Plotkin

MYFOXNY.COM - David and Max Plotkin are looking for a cure... for pediatric cancer, that is. Max was diagnosed with a rare form of cancer when he was four years old. Today, Max has been cancer-free for more than two years. David -- who left a lucrative career in finance to focus on his son's recovery-- has created the Max Cure Foundation.

Caucus Hosts Second Annual Childhood Cancer Summit

The House Childhood Cancer Caucus hosted its Second Annual Childhood Cancer Summit on September 23, 2011 on Capitol Hill. The Summit featured a panel discussion entitled "The Future of Childhood Cancer Drug Development."