The Next Step, April 2012

CCCA Leads Efforts to Improve Key Laws for Childhood Cancer Drug Development

Legislation was recently introduced in Congress that would permanently reauthorize the Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA), two laws that provide incentive and requirements for pharmaceutical companies to study the effects of drugs in children. On March 28, Representatives Mike Rogers (R-MI), Anna Eshoo (D-CA) and Ed Markey (D-MA) introduced the BPCA and PREA Reauthorization Act (H.R. 4274), legislation that extends the programs in advance of the October 1, 2012 sunset.

The Alliance for Childhood Cancer sent a letter to Representatives Rogers and Eshoo last week in response to the release of H.R. 4274. The letter expresses concern that the legislation does not address the shortfalls of PREA for children with cancer. While BPCA and PREA have yielded a wealth of information about the safe and effective use of medications in children, the laws have had a very limited impact on childhood cancer drug development.

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. As a result, companies receive an immediate waiver on virtually all new adult oncology drugs, even though many of those drugs might be beneficial for treating childhood cancers as well.

CCCA continues to advocate for a proposal that would strengthen PREA for children with cancer by utilizing current biomedical advances. The revised PREA language would allow for appropriate pediatric studies based on the scientific relevance of a shared molecular target, rather than based on the organ, origin or name of the cancer.

Over the past several months, CCCA has been working closely with the Children’s Oncology Group (COG) and other stakeholders in the pediatric oncology research and the advocacy community to advance the proposed PREA modification. As reauthorizing legislation moves through Congress, CCCA will continue to advocate for the inclusion of this important proposal, which we believe could significantly advance the treatment of childhood cancer.

Over the past several days, a Children's Cause for Cancer Advocacy action alert has resulted in over 1,000 letters sent from hundreds of CCCA advocates to their Senators and Representatives on Capitol Hill. CCCA worked closely with other childhood cancer organizations, including the American Childhood Cancer Organization, St. Baldrick's Foundation, CureSearch and The Leukemia and Lymphoma Society to coordinate a widespread advocacy campaign throughout the community. For information on how you can add your voice to this effort, please click here.

Supreme Court’s Ruling on Health Care Could Significantly Affect Childhood Cancer Survivors

In late March, the Supreme Court spent three days listening to oral arguments on President Obama’s 2010 health care reform legislation, the Patient Protection and Affordable Care Act (ACA).

Individual Mandate

The biggest question at stake is the constitutionality of the “individual mandate,” the central provision of the legislation that requires all Americans to obtain health insurance by 2014. The legislation provides subsidies and support coverage for individuals who are not otherwise covered by Medicare, Medicaid or their employer. Challengers to the individual mandate argue that the government cannot force people to buy a product they do not want, but the Obama administration contends that health care is a unique product because everyone requires it at some point.

The authors of the ACA included the individual mandate in the health care reform law so that other health insurance reforms could be instituted, such as protections for individuals with pre-existing conditions. Without the individual mandate, healthy people may forgo health insurance or wait to purchase coverage until they are sick, actions which could skew the health insurance market.

If the individual mandate provision is ruled unconstitutional, it is possible that the prohibition against discrimination based on pre-existing conditions could also be stricken. Prior to passage of the ACA, childhood cancer survivors could be denied coverage because of their condition. Requiring everyone to buy into the system makes it possible for insurance companies to afford to cover those with pre-existing conditions at an equitable rate.

Severability

A related issue before the Court is that of “severability,” or the question of whether the rest of the legislation can remain if the individual mandate is struck down. Many legal analysts agree that it’s very unlikely that the Court would rule the entire law unconstitutional, as there are thousands of provisions in the bill, many of which are unrelated to the individual mandate.

An important provision affecting childhood cancer survivors is ACA’s expansion of dependent coverage eligibility for young adults. The legislation requires insurance plans to continue offering coverage to young people under their parents’ plans until age 26. Since enactment of the ACA, 2.5 million young adults have been able to get health care coverage through their parents’ plans.

Medicaid

The Court will also rule on the health reform law’s expansion of Medicaid, one of the nation’s largest providers of health care, especially to children. The constitutional question under evaluation by the justices is whether the Medicaid expansion unfairly coerces the states to participate.

The ACA also expands Medicaid coverage to non-Medicare eligible low-income individuals under age 65. It is estimated that this provision could expand coverage to 16 million new individuals by 2019.

The Medicaid expansion is a core element of the ACA that enables access to health care for our nation’s most vulnerable populations. Striking this provision could have a devastating impact, especially for low-income children and families.

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Although there is no firm deadline for the Supreme Court to rule on the constitutionality of the ACA, it is anticipated that a written opinion will be issued in June 2012. CCCA continues to monitor this case due to its immense potential impact on children with cancer and will report developments in our monthly e-Newsletter.

Advocates Speak Out for Continued Funding for Childhood Cancer Surveillance Activities in FY2013 Budget

CCCA has been at the frontlines advocating for funding for childhood cancer surveillance activities in the 2013 fiscal year (FY) Labor, Health and Human Services, and Education appropriations bill. On March 14, CCCA launched an action alert asking advocates to contact their Senators and urge them to sign on to a letter led by Senators Jack Reed (D-RI) and Johnny Isakson (R-GA).

Thanks in large part to advocate outreach, the Reed-Isakson letter received broad support and was signed by ten Senators. On March 28, the letter was sent to Chairman Tom Harkin (D-IA) and Ranking Member Richard Shelby (R-AL) of the Senate Subcommittee on Labor, Health and Human Services, and Education Committee on Appropriations. The letter urges support for continued funding for surveillance activities, conducted through the Centers for Disease Control (CDC) as called for in the 2008 Caroline Pryce Walker Conquer Childhood Cancer Act.

The CDC program allows researchers to monitor trends and incidence of childhood cancer in order to better understand the disease, ensure appropriate treatment, and track long-term health outcomes. Past funding to the CDC for these activities has helped many states transform the infrastructure necessary to expand electronic reporting, enabling enabled researchers to rapidly report critical information to state cancer registries.

Continued funding is necessary to enable researchers in every state to expand knowledge about childhood cancer patterns – including diagnoses, treatments and survivorship – to ultimately improve care for children with cancer.

CCCA thanks Senators Reed and Isakson for their leadership on this important issue. CCCA also thanks the advocates who participated in the action alert and brought this issue to the attention of their Senators.

Restored Fertility Treatment on the Horizon for Male Childhood Cancer Survivors

New research from the University of Pennsylvania School of Veterinary Medicine provides hope for the one in three male survivors of childhood cancer who face fertility problems as a result of their cancer treatment. The study, a 14-year experiment with animals, examined whether spermatoginal stem cells frozen for a decade or longer could be successfully implanted and lead to sperm production.

Adult men undergoing cancer treatment that might threaten their fertility have the option of freezing their sperm, but that option is not available for pre-pubescent boys. Several children’s research centers have been cryopreserving the spermatoginal stem cells of young boys undergoing cancer therapy. These stem cells, present in boys at birth, later lead to the production of sperm as boys go through puberty. When the reproductive system is damaged by treatments like chemotherapy and radiation, sterility can result.

Many questions remain about the re-implantation of these spermatoginal stem cells, including uncertainty about how long the cells can survive and remain viable.

To answer the question of long-term viability, researchers thawed and labeled spermatogonial stem cells from mice, rats, rabbits and baboons that had been cryopreserved in the mid-90s and implanted them in mice. Researchers found that the cells migrated to the correct location in the testes and embedded appropriately and successfully led to mature sperm.

Researchers were able to successfully fertilize eggs in-vitro to produce offspring, and, in one circumstance, a recipient mouse mated naturally with a female mouse, producing offspring. Most significantly, the offspring from the study had no genetic defects. These early findings show significant promise for similar spermatoginal stem cell implantation to restore fertility in human males.

Late effects of childhood cancer, including fertility problems, can often be a devastating consequence of treatment that can significantly decrease quality of life. CCCA is an advocate for more awareness and research of long-term late effects for childhood cancer survivors. CCCA will continue to follow important research developments.

Registration Now Open for Childhood Cancer Action Day

Join CCCA, a member of the Alliance for Childhood Cancer, for a day of training on advocacy and issues, followed by a day of scheduled Capitol Hill visits with members of Congress and their staff on June 4-5, 2012. The Childhood Cancer Action Day will give the childhood cancer community - parents, children, and others – the opportunity to visit Capitol Hill and advocate for important childhood cancer policy questions currently before Congress.

Registration includes the training, scheduled Hill visits and an option for a room reservation at a rate of $259 at the Hamilton Crown Plaza in Washington, DC. If you do not need a hotel room, there is an option to note that during registration. Transportation to/from Washington DC is the responsibility of those attending.

Registration is accepted on a first come first served basis, so sign up today to reserve your spot!

Visit www.allianceforchildhoodcancer.org/events. Registration closes Thursday, May 3.

Save the Date for CCCA Spring Celebration in Washington, DC

CCCA announces its Capitol Hill spring reception for colleagues, advocates and supporters on Wednesday, June 6, from 6:00 - 8:00 pm at the Phoenix Park Hotel.

This year’s event will highlight current legislative issues before Congress impacting children with cancer. The program will feature the presentation of CCCA’s 2012 Congressional Champion Awards, recognizing Members of Congress for their service and commitment to the childhood cancer community. Honoree and guest speaker John Stewart, a long-time CCCA Board Member, will share his personal connection to childhood cancer.

Save the date so that you may share this unique opportunity to recognize the Champions in our community and support CCCA’s important work on behalf of children with cancer.

Stay tuned for information on purchasing your ticket. For more information on attending or sponsoring this event, please contact us at sjanesko@childrenscause.org.