The Next Step, January 2012

Drug Shortage Crisis Stimulates Further Congressional and White House Action

Momentum continues to build to combat the nation’s drug shortage crisis with recent Congressional hearings and the release of a Government Accountability Office (GAO) report and Food and Drug Administration (FDA) interim final rule calling for stronger manufacturer reporting requirements.

CCCA has been actively engaged on this issue over the past several months, co-hosting a Capitol Hill briefing, sharing information with advocates, and educating lawmakers and their staff about the breadth and impact of the shortages in drugs vital to the treatment of children with cancer. Read more about recent activity on this issue below, and visit the drug shortages section of our website for more information.

Pediatric Oncologists Testify at House and Senate Hearings on Drug Shortages

Since September, pediatric oncology experts have testified about the impact of drug shortages on children with cancer at a number of House and Senate committee hearings with jurisdiction over this issue.

Dr. Michelle Hudspeth, Division Director of Pediatric Hematology/Oncology at the Medical University of South Carolina, testified on November 30 before the House Oversight and Government Reform Committee. Dr. Hudspeth stated that “the current situation is nothing short of a national emergency,” and further noted the ramifications of the shortages on clinical research. Without uniform access to the chemotherapy drugs in a clinical research study, further enrollment is prohibited and clinical advances are stymied. Dr. Hudspeth noted that “up to $1.2 million could be wasted each year alone for pediatric cancer trials that are never able to enroll any patients due to chemotherapy drug shortages.”

Dr. John Maris, Chief of the Division of Oncology at the Children's Hospital of Philadelphia (CHOP) and Director of the CHOP Research Institute’s Center for Childhood Cancer Research, testified at the December 15 Senate Health Education, Labor and Pensions (HELP) Committee hearing. Dr. Maris noted the magnitude of the drug shortages at his hospital and highlighted ways that CHOP has responded. CHOP is monitoring the availability of 35 drugs with a four to eight week supply, nine drugs with a two to four week supply, and 10 drugs with less than a two week supply left.

Dr. Maris explained that CHOP has been forced to substitute daunorubicin – a drug that is critical to the treatment of childhood leukemias – with doxorubicin, and that many children experienced severe side effects, such as gut and mouth ulcerations, fever and infections. It is too soon to know whether the drug substitution has had an effect on the success or failure of their treatment. The children that received doxorubicin were all enrolled in clinical trials. Dr. Maris expressed concerns about the impact that the substitutions would have on clinical trial results.

Legislation is currently pending in both the House and Senate that would strengthen requirements that companies notify FDA early about impending shortages. CCCA supports the Preserving Access to Life-Saving Medications Act (S. 296/H.R. 2245) as an important immediate action to address the drug shortages crisis.

Government Accountability Office Releases Report on Drug Shortages

A recent report from the Government Accountability Office (GAO) analyzed trends in prescription drug shortages and outlined FDA’s ability to respond to the issue.

The GAO found that from 2006 to 2010, the number of drug shortages grew by more than 200 percent with a record number of shortages – 196 – reported in 2010. GAO investigators cite manufacturing problems, including manufacturing shutdowns, as the primary cause of most shortages. The report states that FDA is “constrained by its lack of authority to require manufacturers to provide the agency and the public with information about shortages” and calls for the FDA’s authority to respond be strengthened. The GAO report is available here.

FDA Issues Interim Final Rule to Combat Drug Shortages

In response to President Obama’s October 31 Executive Order, FDA has issued an interim final rule clarifying manufacturer reporting requirements. Specifically, the interim final rule requires manufacturers that are the sole producer of certain drugs to report manufacturing interruptions of products that are “life supporting, life sustaining, or intended for use in the prevention of a debilitating disease or condition.” 

Since the President’s Executive Order, FDA has prevented 96 drug shortages. The Administration states that the President’s Executive Order and an FDA letter to industry have raised awareness of the issue, resulting in increased voluntary manufacturer reporting from around 10 voluntary notifications per month, to 61 in the month following the Executive Order.

Public comments will be accepted through February 17, 2012. CCCA will continue to monitor this issue for the potential release of further guidance and other updates.

States Given Flexibility over Essential Benefits Required by Health Care Reform Legislation

The Department of Health and Human Services (HHS) announced on December 16 a plan that allows states to determine which essential benefits insurers will be required to offer enrollees in 2014. Essential health benefits, a core component of President Obama's health care reform legislation, are ten mandated categories of care insurers must offer in individual and small-business policies by 2014.

Under the new HHS plan, individual states can choose from one of the following four benchmark insurance packages: the three largest small-business plans in the state; the three largest state employee health plans; the three largest federal employee plans; or the largest HMO plan offered in the state.

The flexible approach is designed to provide states with the ability to meet the EHB requirements while acknowledging states’ diverse health needs and minimizing the likelihood they will be required to defray the costs of additional benefits. After an initial period of implementation, evaluation and assessment will occur in 2016.

The establishment of essential health benefits is an important issue for children with and surviving cancer and their families. CCCA will continue to follow the EHB implementation process and advocate for robust, meaningful coverage.

Public comments are welcome through the end of January 2012. To read the full bulletin from the Center for Consumer Information and Insurance Oversight, visit this link.

New Drug Therapy Approved for Children with Acute Leukemia

For just the second time in over two decades, a new drug was approved for the treatment of a pediatric-specific cancer. Erwinaze received Food and Drug Administration (FDA) approval in November 2011.

Erwinaze will be given to patients with acute lymphoblastic leukemia (ALL) who have developed a hypersensitivity to the standard treatment protocol, which contains an E.coli-derived agent. An estimated 15-20 percent of ALL patients develop sensitivity during their treatment, amounting to approximately 450-600 children in the United States. The new drug will enable those patients to complete their treatment uninterrupted.

Erwinaze is designed to be taken in conjunction with chemotherapy and works by breaking down a protein that helps overabundant white blood cells grow. In the absence of the protein, leukemia cells die without affecting the healthy cells. Erwinaze was developed by EUSA Pharma Inc., a transatlantic pharmaceutical company based in Langhorne, PA. The company says the drug is available immediately to eligible patients, and they offer a patient assistance program to help expand access to families who may have financial need.

ALL is the most common childhood cancer. Although the disease can affect adults, approximately 60 percent of cases are diagnosed under age 20, according to data from the National Cancer Institute’s SEER Cancer Statistics Report.

NIH Receives $575 Million to Establish National Center for Advancing Translational Sciences

Congress approved the establishment of a new center within the National Institutes of Health (NIH) tasked with translating scientific discoveries into new therapies and speeding high-need cures through the drug development process. The National Center for Advancing Translational Sciences (NCATS) will be housed within NIH and will lead programs such as the Cures Acceleration Network (CAN). CAN received $10 million in appropriations, which will help advance the mission of NCATS.

CCCA advocated for the establishment of NCATS in a letter to NIH Director Francis Collins last year. Most recently, CCCA joined a coalition requesting House and Senate appropriators’ support for funding to create the new center within NIH. CCCA commends Congress for allocating funds to move this critical endeavor forward.

President Obama signed into law the fiscal year (FY) 2012 spending bill, which provides $30.7 billion to the National Institutes of Health (NIH), a 1% increase over FY 2011. The National Cancer Institute (NCI) received an increase of .5% over last year’s spending bill. NCATS and CAN were included in the appropriation increase.

2011: A Year of Growth for CCCA

The Children’s Cause for Cancer Advocacy saw tremendous growth and progress this past year thanks to our generous supporters, advisors and advocates. Because of your generous giving, CCCA was able to serve as an independent, national champion working to bring more and smarter research to make childhood cancer history. We fill a critical gap in the childhood cancer community by ensuring that children with cancer and their families are heard at the national level.

Maureen Lilly became Executive Director early in 2011, and Lauren Neff came on staff as Director of Public Policy. These skilled and energetic individuals enabled CCCA to increase and focus Congressional attention on the issues that deeply affect children and survivors of cancer.

CCCA worked for the better part of the year with Congressional staff to ensure that comprehensive, bipartisan survivorship legislation was introduced in September. This landmark legislation will improve the care and quality of life of the 350,000 survivors of childhood cancer. In 2012 we will focus on advancing this vital legislation in Congress.

Through the leadership of Board chair Lynn Bayard, CCCA’s Board grew by 40 percent, recruiting four top new Board members. The newly-expanded Board worked to set CCCA’s program and policy priorities for 2012.

CCCA expanded its reach and resources in 2011 through a series of successful events in Washington, DC, Chicago, and New York. Most notably, our New York signature event honored Dr. Peter Adamson, chair of the Children’s Oncology Group, and Leonard Rosen, prominent New York attorney and long-time CCCA board member. The event raised over $500,000, the most successful in CCCA history.

Finally, and most importantly, CCCA amplified its programs and direct outreach to children with cancer, survivors and their families. CCCA was proud to co-host the largest and most successful Childhood Cancer Advocacy Day in Washington, when an army of advocates took to Capitol Hill and held nearly 150 meetings with Congressional offices. CCCA played a key role in September’s Childhood Cancer Awareness Month activities, enhancing collaboration among childhood cancer groups and bringing sharp focus and analytic skills to the messages that advocates and childhood cancer organizations brought to members of Congress and their staff.

2012 will bring CCCA’s strong policy analyses, responsible advocacy and collaborative spirit to help build the childhood cancer community. We are committed in 2012 to giving hope and a voice for change to the hundreds of thousands of childhood cancer patients, survivors and their families struggling with these complex, lifelong diseases.