The Next Step, June 2010

CCCA Presents to Joint NIH-FDA Council on Barriers to Drug Development

On June 2, the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) held a meeting of stakeholders to hear proposals on how scientific breakthroughs can be better translated into new therapies. This meeting is part of a new collaborative partnership between NIH and FDA designed to share information in order to promote “the translation of basic and clinical research findings into medical products and therapies.”

CCCA’s president, Dr. Susan Weiner, authored a presentation given by Lori Salley, CCCA’s new Executive Director, titled “Challenges in Pediatric Cancer Research and Care.”  CCCA offered the only oral presentation that focused on children in need of new and better therapies. Specifically, the presentation laid out the barriers to bringing new molecular, personalized therapies to children with cancer, which include the variety of rare diseases that are childhood cancers; the differences among child and adult cancers; the length of time to complete phase III trials; federal protections that constrain research in children; and the small patient populations that offer little in the way of financial incentives to drug developers in private industry.

CCCA offered several suggestions for addressing these barriers and promoting access to safe, effective, targeted therapies for children with cancer:

• Facilitate public/private partnerships to discover and develop new drugs that can be brought to the NCI cooperative groups for evaluation;
• Establish an interagency task force charged with publicizing NIH resources available for the discovery and development of new pediatric therapies;
• Reauthorize and modernize the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act to reflect contemporary science, including a pediatric incentive for biologics;
• Promote research on minimally invasive biomarkers;
• Increase research on novel statistical strategies to reduce children’s exposure to ineffective treatments and reduce sample sizes for reliable conclusions;
• Ensure FDA personnel assigned to pediatric oncology matters understand the unique issues and complex research constraints for this population; and
• Create a mechanism that fosters collaboration among FDA, NCI, and pediatric oncology experts with authority to advise researchers on strategies to bring modern treatments to children with cancer.

Other groups that made presentations also focused on themes of biomarker qualification, adaptable clinical trial design, and collaboration among agencies, as well as between the public and private sectors.

Health Care Reform Implementation – Rules Issued on “Grandfathered” Plans

The Obama Administration issued additional regulations to implement the Patient Protection and Affordable Care Act (PPACA) that provide guidance on provisions of the law that are designed to allow individuals to keep the insurance they already have – a pledge made by the President during the health reform debate. Specifically, the law “grandfathers” health plans that were already in existence on the date PPACA was signed into law (March 23, 2010) and exempts these plans from many of the law’s requirements. The interim final rule published in the Federal Register on June 17, 2010, explains how “grandfathered” health plans can keep or lose their status.

Under the regulations, health plans can keep their grandfathered status and continue to offer coverage to individuals if no significant changes are made to the plan. Changes that would result in a loss of status include

• Eliminating all or substantially all benefits to diagnose or treat a particular condition;
• Raising co-insurance charges by any percentage;
• Raising co-payments by more than the greater of $5 (adjusted for inflation annually) or the medical inflation rate plus 15 percentage points;
• Increasing deductibles by more than medical inflation plus 15 percent;
• Lowering employer contributions by more than 5 percent;
• Adding or tightening annual limits; and
• Changing insurance companies.

The rules estimate that between 34 and 80 percent of employer plans will lose their grandfathered status by 2013, with small employer plans being more vulnerable than those offered by large employers. Plans that lose their status will have to comply with additional mandates under the law, including free preventive health services, coverage of routine costs incurred by patients enrolled in clinical trials, coverage of emergency services without preauthorization, access to pediatricians and OB/GYNs as primary care providers, establishment of an external review process for claims appeals, and new reporting requirements.
The Administration is accepting public comments on the interim final rule for 60 days.

CCCA Spearheads Letter to Congress in Support of Cures Acceleration Network

CCCA spearheaded a letter to the House and Senate Appropriators in support of full funding for a provision of health care reform called the Cures Acceleration Network (CAN). The letter was cosigned by six other organizations with an interest in pediatric oncology.

The CAN provision in the health care law seeks to address the chasm in funding between basic science and the development of new therapies known as the “Valley of Death.” For diseases that offer no commercial market for drug development, such as pediatric cancers due to small patient populations, this chasm is particularly hard to bridge. CAN establishes a new office in the NIH Directorate to make grants to help facilitate the process of turning new discoveries into new therapies. A board of 24 members from research, FDA, venture capital, patient advocacy groups and others will help coordinate NIH research funded activity with FDA mechanisms of drug approval. Through the CAN funding mechanism, national experts will be able to lend their expertise in a systematic way to solving the complex preclinical development necessary to finance drug development for diseases such as childhood cancers.

The letter, addressed to the Chairmen and Ranking members of the House and Senate Subcommittees on Labor, Health and Human Services, and Education, asks for the full $500 million that is authorized by the new law to be appropriated for CAN in fiscal year 2011 (FY11). Congressional appropriators are currently writing the bills that will fund the federal government in FY11, which will be debated over the summer, with the goal of passing legislation by September 30, when the current fiscal year ends.

New Therapy Shows Promise for Treating Medulloblastoma

On June 5, at the American Society of Clinical Oncology’s (ASCO) annual meeting, researchers reported that a preliminary study (Phase I trial) shows that an experimental drug (GDC-0449) designed to target the genetic make up of tumors may hold promise for treating medulloblastoma, the most common malignant brain cancer in children. The drug attacks tumors by interrupting the “sonic hedgehog” molecular pathway that is linked to 20 percent of medulloblastomas.

While the trial was designed to determine whether the drug can be safely administered to children, the results also suggest signs of efficacy, with some children on treatment almost a year with no progression of the disease. The drug has already been shown to have some effectiveness in adults with recurrent medulloblastoma, as well as with basal cell carcinoma (a type of skin cancer).

Recurrent medulloblastoma currently has a cure rate of less than 5 percent. As a result of the study’s preliminary findings, a Phase II trial of GDC-0449’s effectiveness against recurrent medulloblastomas in children (up to age 21) will start later this year.

CCCA President, Dr. Susan L. Weiner, Participates in Childhood Cancer Survivor Study Meeting

The Childhood Cancer Survivor Study (CCSS) held its annual meeting in Williamsburg, Virginia on June 9 and 10. The CCSS was established in 1993 as a collaborative research project, funded by a National Cancer Institute (NCI) grant, to better understand the long-term effects of cancer and its treatment in order to educate survivors, improve follow up care, and design treatment protocols that increase survival and minimize harm.

The study includes data from 20,436 cancer survivors diagnosed before age 21, between 1970 and 1986, with a control group of 4,000 sibling survivors. The cohort is currently being expanded to include 14,000 survivors diagnosed between 1987 and 1999.

Dr. Weiner serves on the External Advisory Committee of the CCSS, and plays a role in providing guidance on the use and dissemination of the research. She is positioned to share valuable insights about how the data produced by the study can influence public policy to help childhood cancer survivors.

Raise Money for CCCA by Searching Web and Shopping On-Line via GoodSearch

CCCA recently developed a partnership with GoodSearch. GoodSearch is an internet search engine powered by Yahoo! which donates 50 percent of its sponsored search revenue to the charities and schools designated by its users. GoodShop, a component of GoodSearch, is an online shopping mall of world-class merchants dedicated to helping fund worthy causes across the country. Each purchase made via the GoodShop mall results in a donation to the user's designated charity or school – averaging approximately three percent of the sale, but going up to 20 percent or even more. There is no cost associated with using GoodSearch or GoodShop. All the money donated will come from advertisers.

Help CCCA raise money every time you go do a search on the internet or shop online and designate the Children’s Cause as your charity of choice! Go to www.GoodSearch.com for more information.

Save the Date for CCCA’s Rise to the Occasion – New York

Please Save the Date of Thursday, November 11th for CCCA’s annual “Rise to the Occasion” event in New York City. The event will start with a Networking Hour, followed by a Cocktail and Silent Auction Reception. This year’s event will be held at a new location, the lovely Ana Tzarev Gallery, located at 24 West 57 Street in New York City. Please contact Rebecca Gurvich at rgurvich@childrenscause.org for additional information.