In the past week, major steps have been taken in Congress to reauthorize the Prescription Drug User Fee Act (PDUFA), which is set to expire on September 30, 2012. Two important components of PDUFA, the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA), have been the focus of intense CCCA activity for months in an effort to make these laws more effective for children with cancer.
Under current law, PREA requires pediatric studies for drugs developed only for diseases that occur in both adults and children. Common adult cancers – such as breast, colon, lung and prostate – typically do not affect children, which makes the legislation ineffective for children with cancer. CCCA has proposed new language calling for evaluation of the impact of BPCA and PREA on pediatric cancer therapies.
Read on for the latest updates surrounding the Senate and House versions of this important legislation.
Final Senate Legislation Introduced Tuesday
The Senate bill, S.3187, introduced on Tuesday calls for a public meeting of the Food and Drug Administration (FDA) within 18 months of enactment to examine ways to encourage and accelerate the development of new therapies for pediatric rare diseases. A report is to follow within 180 days of the meeting.
CCCA worked with Senate leaders to provide technical input on the public meeting and report language, which will present a critical opportunity to highlight the constraints of the law as it applies to children with cancer.
It is anticipated that the PDUFA legislation will move to the Senate floor for a vote before the end of May.
In a show of remarkable bipartisan support, the House Energy and Commerce committee approved the PDUFA legislation last Thursday by a unanimous 46-0 vote.
The House legislation contains language proposed by CCCA that requires an FDA public meeting to examine the effectiveness of BPCA and PREA on children with cancer. The meeting is to occur no later than December 31, 2013.
The legislation also mandates an internal FDA report examining the impact of the laws on studying drugs for children with cancer, and calls for recommended modifications to the programs to facilitate the development of new and better therapies for children with cancer.
The full legislation must now go before the House of Representatives for a floor vote, expected to take place before the end of the month. Once both chambers have passed the bill, the differences between the two versions will be negotiated in a Conference Committee likely occurring in June. Lawmakers in both chambers have expressed hope that the final legislation will be presented to President Obama for his signature by the Fourth of July.
Language to combat drug shortages was also included in the PDUFA legislation. Please see the sidebar at right for these important drug shortage provisions.
CCCA has spent the past several months working with key Senate and House offices to address the shortfalls of BPCA and PREA. CCCA commends the dedication of Representative Anna Eshoo (D-CA), Representative Mike Rogers (R-MI) and Senator Jack Reed (D-RI) and their staffs for their hard work on the reauthorization and their interest in ensuring that it represents the needs of children with cancer.
The Cancer Leadership Council, comprised of approximately 30 national organizations, is sending a letter of support for the user fee legislation to leaders in both chambers of Congress, endorsing key provisions and supporting more effective pediatric cancer research laws.
CCCA thanks advocates across the country who have shared their stories with their legislative representatives and leaders in the pediatric oncology research community who have shared their expertise. CCCA also thanks our partners in advocating for this important legislation, including the Alliance for Childhood Cancer, American Childhood Cancer Organization, Cancer Leadership Council, CureSearch, Leukemia & Lymphoma Society, Rally Foundation, and St. Baldrick's Foundation.
Food and Drug Administration Commissioner Margaret Hamburg recently posted a progress report online about prescription drug shortages, six months after President Obama issued an Executive Order to help ease the crisis.
Commissioner Hamburg reported that in the past six months, 128 drug shortages have been prevented and fewer new shortages have occurred. So far, drug shortages in 2012 have been cut by more than half as compared to the same time in 2011, dropping to 42 from 90. Drug companies’ early notification to FDA appears to have had the biggest impact on this improvement. After the president’s Executive Order was issued, FDA requested that drug manufacturers voluntarily report potential disruptions in drug supply, resulting in a six-fold increase in the average number of monthly early notifications.
Of particular concern for the childhood cancer community has been the limited supply of methotrexate, a critical drug used in the treatment of childhood leukemia. Because of quick action from advocates, manufacturers and FDA, Commissioner Hamburg stated that “supplies of methotrexate…are currently meeting all demand, and we do not expect any further supply issues.”
Dangerous shortages remain, noted the Commissioner. For example, Leucovorin injection, sometimes given along with methotrexate for children with leukemia, faces a limited supply. FDA is working with Teva Pharmaceuticals to increase production in the coming weeks in order to prevent a disruption in its supply.
Drug shortages are a complex problem that requires a “multifaceted approach involving industry, regulators, payers, and others,” said Dr. Hamburg. FDA is currently working with Congress on bipartisan legislation to require early notification of anticipated shortages. Please see the sidebar box for more on this legislation.
CCCA continues to monitor legislative action surrounding this important issue for children with cancer. Drug shortages will be a key issue that advocates discuss with their members of Congress on the June 4-5 Childhood Cancer Action Days in Washington.
For some cancers, black children benefit less than white children from improved childhood cancer treatment, according to a recent analysis published April 30 online in the Journal of Clinical Oncology. Researchers from St. Jude Children’s Research Hospital (SJCRH) analyzed data from the National Cancer Institute's Surveillance, Epidemiology, and End Results (SEER) program and from SJCRH for the periods 1992 to 2000 and 2001 to 2007.
Over the 15-year period, the survival gap narrowed between black and white children for some cancer types, including acute lymphoblastic leukemia and Hodgkin lymphoma. However, for other types of pediatric cancers, including acute myeloid leukemia and neuroblastoma, the survival rate of black children worsened over time as compared to white children.
Comparing SEER data with SJCRH data, investigators found that patients treated at SJRCH showed almost no difference in survival rates between blacks and whites. Researchers suggest that unequal access to comprehensive medical care and psychosocial support may be to blame for the differences between survival rates found in the SEER vs. the SJCRH data.
The study compared survival outcomes for 19 forms of pediatric cancer in over 4,000 children at SJCRH and almost 24,000 children at other U.S. medical centers. Data on only black and white patients were analyzed and not that from children of other racial groups.
CCCA joins members of the Alliance for Childhood Cancer for Childhood Cancer Action Days on June 4-5, 2012, an opportunity for the childhood cancer community to advocate for key childhood cancer policy questions currently before Congress. Action Days will include training on advocacy and issues, followed by scheduled Capitol Hill visits with members of Congress and their staffs.
The 2012 advocacy agenda will include the drug shortage crisis, survivorship issues, and drug development challenges and incentives. Advocates will also be asking House members to join the Congressional Childhood Cancer Caucus.
Registration is now closed, and nearly 250 parents, children and other passionate supporters are signed up to participate. Those not able to make the trip to Washington, DC, can still have the opportunity to make a difference from home. CCCA will be sending out an online action form at the start of the event to enable remote participants to easily reach out to their Congressional representatives. CCCA will also provide updates through Facebook and Twitter and will recap the events in our June newsletter.
Representative Michael McCaul (R-TX) and Senator Jack Reed (D-RI) have accepted an invitation to speak at CCCA’s Capitol Hill spring reception at the Phoenix Park Hotel on Wednesday, June 6, from 6:00 - 8:00 p.m.
The Spring Celebration will feature CCCA’s 2012 Congressional Champion Awards, recognizing Members of Congress for their service and commitment to the childhood cancer community.
Colleagues, advocates and supporters will gather to honor John Stewart, long-time, dedicated CCCA Board Member and childhood cancer advocate. Enjoy hors d’oeuvres and drinks as you bid on must-have silent auction items and mingle with other families and friends.