Differences in the House and Senate versions of the Food and Drug Administration Safety and Innovation Act were resolved by lawmakers on Monday, and the bill is expected to be voted on today in the House and next week in the Senate.
The reconciled version includes CCCA’s request for a special FDA analysis and report on whether and how BPCA (Best Pharmaceuticals for Children Act) and PREA (Pediatric Research Equity Act) have been effective in improving new therapies for children with cancer. Letters to Congress supporting special attention for childhood cancer in the bill were sent from the Alliance for Childhood Cancer and from the Cancer Leadership Council, coalitions of over 60 national cancer patient, research and professional associations.
BPCA and PREA, provisions that work together as an incentive and a requirement for drugs to be evaluated for use in children, will be permanently reauthorized. FDA’s Pediatric Subcommittee of the Oncologic Drugs Advisory Committee will also become permanent as the public forum in which FDA discusses how to improve therapies for childhood cancer.
The bill further requires that the Secretary of Health and Human Services hold at least one public meeting on how to encourage and accelerate the development of new therapies for all pediatric rare diseases, which would include childhood cancers. A strategic plan is a required result of this meeting.
The FDA bill includes a new program for rare diseases, whereby a company that develops a drug for use solely in a pediatric rare disease, pays fees and meets other requirements could receive a voucher to obtain FDA priority review for a different drug. Companies could sell the voucher, which may serve as an incentive for industry to develop brand new agents for pediatric rare diseases. Originally called The Creating Hope Act, the voucher program creates a new and to be determined factor in developing drugs when there is no economic promise of a larger adult market. The rare diseases voucher program is modeled on a program targeted at neglected tropical diseases, which to date has had limited effect. The Government Accountability Office (GAO) will be required to report on the effectiveness of the rare disease priority voucher program after a third voucher is issued.
Childhood Cancer Action Days, on June 4-5, hosted by the Children’s Cause and other members of the Alliance for Childhood Cancer, brought over 150 advocates to participate in 170 meetings on Capitol Hill. Virtual advocates from around the country joined the effort online.
An onsite training day educated participants on childhood cancer issues currently before Congress and how to be effective advocates on the Hill. CCCA's long-time Board Member John Stewart spoke as part of the Issues Panel, along with several Capitol Hill staffers. The following day families asked their Congressional representatives to support provisions in the pending FDA reform bill that can benefit children with cancer.
Advocates’ request to members of Congress to co-sponsor the Childhood Cancer Survivorship Act resulted in four new Senators and eight House members signing on to a bill designed to improve research and follow up services for children surviving cancer. Advocates also urged members to join the Congressional Childhood Cancer Caucus, resulting in eight additional House members volunteering in a bipartisan coalition to bring greater attention to the needs of patients and survivors. About 40 children, including patients and survivors, walked the halls of Congress with the adult advocates. Seven-year-old Brooke and her mother were profiled in this local DC television story. The Hill’s online events page, The Washington Scene, also covered the event. Photos below courtesy of photographer Mark Saunders (click thumbnail to enlarge).
Advocates, colleagues and supporters gathered on June 6th for CCCA’s “Capitol Reception for Children with Cancer” at the Phoenix Park Hotel in downtown Washington, D.C. Senator Jack Reed (D-RI) and Congressman Michael McCaul (R-TX) received the 2012 Congressional Champion Awards from CCCA for their work to help children with cancer. Awards were presented by survivors Jake Cunniffe and Ursula Samson-Daly.
Senator Reed thanked CCCA and advocates for their efforts on behalf of children with cancer. “You have truly transformed your experience into something extremely powerful, particularly here on Capitol Hill. Your efforts continue to be critical in underscoring the importance of not only improving childhood cancer treatments and increasing awareness, but also identifying and responding to the social and emotional impacts of cancer on children and their families. And it is working.”
In accepting his award, Representative McCaul stated, “The cause of fighting on behalf of children with pediatric cancer has perhaps been the most fulfilling issue that I have had the opportunity to work on. It has given me a sense of purpose that is unparalleled to other work that I do in the Congress.”
Long-time CCCA Board Member and childhood cancer advocate John Stewart was honored with CCCA’s Leadership and Service Award. John spoke about the powerful impact that the voices of children and their parents have when they share stories of their cancer experience.
CCCA thanks Gold Ribbon sponsors Biotechnology Industry Organization (BIO), Turner & Goss, LLP, and generous supporters and event attendees for their contributions toward our important advocacy on behalf of children with cancer. Photos below courtesy Brian Silver of DC Hot Spots (click a thumbnail for full-size image).
Dr. Michael Link, renowned Stanford pediatric oncologist and retiring president of the American Society of Clinical Oncology (ASCO), focused his presidential address at ASCO’s 48th Annual Meeting in early June on key lessons from pediatric oncology that can enhance treatment of cancer in adults.
“Progress in the management of children with cancer is one of the great success stories of modern medicine,” said Dr. Link. “There has been a dramatic improvement in outcome for almost every category of childhood malignancy.”
Dr. Link attributes this success to the genetic makeup of childhood cancers, the treatment tolerance of young people, and the visionary leaders who pioneered early research in pediatric oncology. Perhaps most important, said Dr. Link, has been the multidisciplinary research collaboration approach through the pediatric clinical cooperative groups. Cancer clinical trial enrollment rates for children far exceed those for adults, and these high enrollment rates have directly facilitated the steep increases seen in childhood survival.
Consistent with his presidential theme of “Collaborating to Conquer Cancer,” Dr. Link urged research cooperation in tumor sampling and analysis in order to gain insights into subtypes of cancers. He emphasized a need to focus on the long-term survivorship implications for patients when considering treatment options.
“Our children have taught us that cure is not enough. We must understand the consequences of therapies we prescribe and continuously modify them to eliminate agents associated with the most important late effects,” said Dr. Link. New research about breast cancer risk for girls treated with radiation presented at the ASCO meeting is a clear example of Dr. Link’s concern. (See article below).
Dr. Link also discussed some of the toughest challenges currently facing the oncology community, including the drug shortage crisis, the high cost of care, and the plight of the uninsured. To watch or read Dr. Link’s full address, visit here.
Researchers are learning more about late effects facing childhood cancer survivors, including second cancers that can arise later in life as a result of treatment. There have been several recent reports of such research developments:
Breast cancer risk for girls treated with radiation: Women treated with chest radiation as children face a six to seven times greater chance of developing breast cancer by age 50, according to a study from Memorial Sloan-Kettering Cancer Center, presented at the June Meeting of the American Society of Clinical Oncology (ASCO). Breast cancer occurred in approximately one in four female childhood cancer survivors who received chest radiation.
For girls who were treated for Hodgkin lymphoma, the rate was even higher at 30 percent. This study is the first to note that the breast cancer rate for these survivors is similar to that for women who carry the high-risk BRCA1 genetic mutation but have no history of cancer in childhood.
Even when girls were given lower doses of radiation, their risk of breast cancer was still significant. The Children’s Oncology Group recommends that women who received chest radiation as children begin breast cancer screenings at age 25 or eight years after finishing treatment, whichever comes later.
Leukemia and brain tumor risk from childhood CT scans: Children who receive multiple CT scans face a slightly elevated risk of developing leukemia or brain cancer in the decade following the first scan, according to a National Cancer Institute (NCI) study of more than 175,000 children and young adults. The study did not distinguish between healthy children receiving scans for an injury versus sick children receiving scans for a life-threatening illness such as cancer.
This research is the first to demonstrate a direct link between CT radiation exposure in children and cancer risk, albeit a small risk. The study estimated that for every 10,000 CT scans performed on children under the age of 10, there occurred one more case of brain cancer and one more case of leukemia than otherwise expected during a child’s subsequent decade of life. The risk increased with cumulative doses of radiation from CT scans. Researchers emphasized that the benefits of CT scans often outweigh the future cancer risks.
Colon cancer risk for childhood cancer survivors: Survivors of childhood cancer face a five-fold greater risk of developing colon cancer and other types of gastrointestinal cancers later in life compared with the general population, according to a new study from the University of Chicago’s Childhood Cancer Survivor Center. At particularly high risk – 11 times greater than the general population – were children who received radiation to the abdomen. Survivors of Hodgkin lymphoma or Wilms tumor, and those who received certain chemotherapy drugs, were also found to be at increased risk.
The Children’s Oncology Group currently recommends that childhood cancer survivors who received significant radiation begin regular colon cancer screening at age 35 or 10 years after the radiation treatment, whichever comes later. The Chicago study found an average of 22 years between the initial childhood cancer diagnosis and a later diagnosis of gastrointestinal cancer. Some survivors in the study were diagnosed with gastrointestinal cancer as early as 5.5 years after their childhood cancer diagnosis.
In an unparalleled release of pediatric DNA data, St. Jude Children’s Research Hospital announced that a catalogue of complete genome sequences from 260 childhood cancer patients will be made available to the global scientific community, doubling the volume of genome data from other currently available sources. Researchers working as part of the Pediatric Cancer Genome Project (PCGP) in partnership with Washington University hope the shared data will lead to more rapid identification of the causes of childhood cancers and the development of new therapies.
By the end of 2012, PCGP researchers aim to have decoded the genomes of more than 600 childhood cancer patients. For each patient, the genomes of both normal and cancer cells are sequenced in order to compare genetic differences and identify problem spots. This research has already led to a number of key discoveries about childhood cancers of the retina, brainstem and blood. The PCGP differs from other genome projects because it examines the genome data of entire DNA sequences instead of focusing on individual genes thereby providing a more complete picture of genetic mutations.
On Sunday, August 12, CCCA will be one of the benefitting charities at the Pro-Am Charity Doubles Invitational, a tennis tournament to be held at the Old Westbury Golf and Country Club on the North Shore of Long Island. Amateur players of all abilities will be paired with professionals for a fun day on the courts. The event begins with a cookout in the beautifully landscaped Old Westbury Country Club. Tickets are $350/player and can be purchased from CCCA. Spectators are welcome to attend the lunch and watch the matches with a suggested donation of $50. For more information, please contact Adam Rosen at email@example.com.