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The Next Step, February 2012


Feature: What Is the Solution to the Nation's Growing Drug Shortage Crisis?

Childhood Leukemia Drug in Critically Short Supply

Alarming news reports surfaced this week of hospitals across the country experiencing a severe shortage of methotrexate, a drug critical to successfully treating acute lymphoblastic leukemia (ALL) in children.

Just days ago, some hospitals began preparing for the likelihood that their supplies of the crucial drug would be depleted within two weeks, potentially forcing them to delay treatment for children with ALL.

Co-chairs of the Congressional Childhood Cancer Caucus, Representatives Chris Van Hollen and Michael McCaul, sent a letter to Margaret A. Hamburg, Commissioner of the Food and Drug Administration (FDA),  on Wednesday seeking answers on the methotrexate shortage and offering their support to the FDA as the Agency takes action to address the crisis.

On Tuesday, a federal regulator within the FDA told the press that the three manufacturers of the drug have all given assurances that they will be sending out new shipments before the end of the month. The short-term solution will ease the immediate emergency but leaves many questions unanswered about ensuring a long-term, uninterrupted supply.

According to a statement released by Dr. Peter Adamson, Chair of the Children’s Oncology Group: “A near-term solution to the shortage is emerging. This indeed is very encouraging news. However, until we are confident that every center that cares for children with cancer who require treatment with methotrexate has drug available at their center, members of the Children’s Oncology Group will not consider the crisis fully resolved.”

Legislation Introduced in House of Representatives to Combat Worsening Crisis

Methotrexate is one of 283 drugs facing limited supply in the ongoing crisis – a number that has tripled since 2005.

Legislation to address the issue was introduced in the House of Representatives on January 31 by Congressmen John Carney (D-DE) and Larry Bucshon (R-IN). The Drug Shortage Prevention Act of 2012 (H.R.3839) would require the FDA to:

  • Develop a Critical Drug Shortage List to identify drugs with a shortage vulnerability, and develop an opt-in system to notify doctors and patients when a drug is added to the list;
  • Notify distributors of critical drug shortages, so distributors are better positioned to watch out for “grey market” sellers. The FDA would work with the Attorney General to prevent illegal stockpiling and price gouging;
  • Expedite the review process for applications or requests that relate to drugs on the critical shortage list;
  • Communicate concerns about a critical drug within one business day from the regulatory office to the drug shortage office and within five business days to the drug manufacturer; and
  • Work with the Drug Enforcement Agency and the Attorney General to determine whether to increase the allowed quota for any drugs on the shortage list that are regulated under the Controlled Substances Act.

The legislation would also require the Secretary of Health and Human Services (HHS) to study the feasibility of creating a critical drug shortage contingency plan, including a national stockpile of drugs.

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. Last month, CCCA released a policy brief highlighting the drug shortage issue.

Senator Klobuchar Proposes Attaching Early Notification Legislation to Transportation Bill

One year ago, Senator Amy Klobuchar (D-MN) introduced legislation in the Senate that would require drug manufacturers to provide early notification to the FDA about anticipated drug shortages. The legislation had bipartisan support but stalled in a gridlocked Congress.

This week, Senator Klobuchar, along with Senators Bob Casey (D-PA) and Susan Collins (R-ME), attempted to revive that legislation by offering an amendment to a transportation funding bill currently under consideration by the Senate. The Preserving Access to Life-Saving Medication Act would require drug companies to alert the FDA of any incident that would likely result in a drug shortage. The FDA has said that such early notification measures helped prevent 99 shortages last year alone.

The Preserving Access to Life-Saving Medication Act would provide a short-term solution to just one of the many complex factors at the root of the issue, which Senator Klobuchar acknowledges. “The amendment is more getting at the short-term problem. But you have to get at the short-term problem, because otherwise people are going to die.”


CCCA Urges Improvements to Key Laws on Pediatric Drug Development

Two laws designed to encourage pharmaceutical companies to study the effects of drugs in children are set to expire on September 30, 2012. Designed to work in tandem, the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) are the focus of intense CCCA activity in an effort to make these laws more effective for children with cancer.

CCCA has partnered with pediatric oncologists and others in the childhood cancer research community to identify policies that could advance the development of new and less toxic therapies for children with cancer. Though legislation reauthorizing the laws has not yet been introduced in Congress, CCCA has been at the frontlines advocating for the adoption of thoughtful policies that will strengthen the two programs.

Often known as “the stick,” PREA allows the Food and Drug Administration (FDA) to require companies to conduct pediatric studies of new drugs for adult diseases when they are the same as children’s diseases. Companies developing oncology drugs, however, have received waivers of this requirement from FDA because adult cancers are different from childhood cancers. Pediatric oncology researchers have long wanted better and earlier access to new important oncology agents when the biological targets of a drug in adults are highly relevant to the targets in a childhood cancer.

CCCA’s work with members of Congress and the pediatric oncology research community has focused on changing the language of PREA to allow FDA to require pediatric studies of new oncology drugs when they are highly likely to be effective in treating any type of childhood cancer.

BPCA (often known as “the carrot”) awards companies six-months of marketing exclusivity for all uses of a drug, if pediatric studies are carried out according to FDA specifications. BPCA to date has been moderately successful in generating pediatric oncology safety and efficacy studies of agents typically used in adults. However, such studies are entirely voluntary on the part of companies. CCCA is urging that FDA’s Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee (ODAC) be permanently reauthorized to enable pediatric oncology researchers and advocates to continue to have a public opportunity to advise FDA on its BPCA decisions and other pediatric oncology drug development issues.

A series of Congressional hearings have been scheduled to examine elements of additional FDA-related legislation up for reauthorization this year, including the Prescription Drug User Fee Act (PDUFA), Medical Device User Fee Act (MDUFA), and BPCA and PREA. On February 1, the Health Subcommittee of the House Energy and Commerce Committee held its first hearing on these issues.

Committee members and witnesses expressed strong support for the reauthorization of BPCA and PREA, noting the significant advances the laws have made in children’s health. Witnesses from the biopharmaceutical industry called for the permanent reauthorization of BPCA and PREA, stating that the sunset periods create an uncertain regulatory environment for pediatric drug development. Other witnesses advocated for proposals that aim to improve accountability and increase transparency of the two laws.

Margaret Hamburg, M.D., Commissioner of the Food and Drug Administration, the National Organization for Rare Disorders (NORD) and the American Academy of Pediatrics (AAP) also testified. Witness testimonies are available here.

As the reauthorization of BPCA and PREA moves through Congress, CCCA will advocate for policies that strengthen the laws and enhance access to new agents to improve the lives of 13,500 children newly diagnosed with cancer each year in the United States.

CCCA Featured in PBS NewsHour Story on Health Reform

CCCA Executive Director Maureen Lilly was quoted in a story examining the impact of health care reform on children and families affected by childhood cancer. “Health Reform: Changing the Game for Pediatric Cancer Patients?” profiled Cooper Cochran, diagnosed at age two with acute lymphoblastic leukemia, and discussed his family’s ensuing financial struggles.

Maureen outlined a number of provisions in the Patient Protection and Affordable Care Act that provide important safeguards for childhood cancer patients and survivors. These benefits include protections that prohibit insurers from denying coverage based on pre-existing conditions or imposing lifetime caps on coverage.

Maureen also raised the serious challenges that face the growing population of childhood cancer survivors. CCCA continues to advocate for the unique needs of survivors through the Pediatric, Adolescent and Young Adult Cancer Survivorship Research and Quality of Life Act, bipartisan legislation that would improve and expand access to comprehensive, long-term follow-up care.

PBS NewsHour’s special report on childhood cancer also featured Dr. Michael Link, pediatric oncologist and President of the American Society of Clinical Oncology (ASCO). Dr. Link outlined research gains made in the treatment of pediatric cancer since 1971. An article on teenagers with cancer and a photo board on the social networking site Pinterest rounded out the PBS report.

St. Jude Launches Website on Genomic Research Results

Published research results from the Pediatric Cancer Genome Project (PCGP), a joint project of St. Jude Children’s Research Hospital and Washington University, are now freely available at The Explore website aims to accelerate research advances by making the PCGP’s research more accessible to the entire research community in a user-friendly way.

The PCGP, launched in January 2010, is a three-year project to sequence the entire genomes – both normal and cancerous – of 600 children diagnosed with aggressive childhood cancers. As of January 2012, more than 250 complete sets had been sequenced. Recent papers in Nature reported novel discoveries in acute lymphoblastic leukemia and retinoblastoma.

Most recently, the PCGP yielded new information about a brainstem tumor found almost exclusively in children - diffuse intrinsic pontine glioma (DIPG). One of the deadliest of childhood cancers, DIPG has a 90 percent fatality rate within two years. St. Jude and Washington University announced on January 29 the discovery that nearly 80 percent of the DIPG tumors have mutations in genes that were not previously tied to cancer. DIPG tumors cannot be cured by surgery and do not respond to traditional therapies. The study’s authors hope that these findings will finally lead to new targeted therapies for this disease.

Save the Date: June 4-5 for the Alliance Childhood Cancer Action Day

Members of the Alliance for Childhood Cancer, including CCCA, will host a two-day Childhood Cancer Action Day on June 4-5, 2012. The event will provide the childhood cancer community – parents, children and others – with the opportunity to visit Capitol Hill and advocate for the important childhood cancer issues currently before Congress.

Childhood Cancer Action Day will include advocacy training and pre-arranged Capitol Hill visits with members of Congress and their staff. Online registration opens on April 2, when a block of hotel rooms at the Hamilton Crown Plaza in Washington, DC, will also begin accepting reservations on a first-come first-served basis. Hotel information and event participation registration links will be sent out via email on April 2. If you have any questions in the meantime, please contact CCCA at or 202.336.8375.