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The Next Step, May 2013


House Bill Would Help Childhood Cancer Survivors
CCCA Launches Action Alert

Bipartisan legislation was introduced in the House of Representatives this week to improve the treatment, care and research for the nation’s more than 360,000 childhood cancer survivors. Representative Jackie Speier (D-CA) introduced the Childhood Cancer Survivors Quality of Life Act (H.R. 2058) on Tuesday with the Co-Chairs of the Congressional Childhood Cancer Caucus, Representatives Michael McCaul (R-TX) and Chris Van Hollen (D-MD).

The bill would establish vital programs to enhance and expand the care for childhood cancer survivors. Specifically, the Childhood Cancer Survivors’ Quality of Life Act will:

  • Support research on late effects of childhood cancer treatments and health care disparities;  
  • Create pilot programs to evaluate model systems of care and identify the most effective ways to provide transition of care between providers;  
  • Establish clinics to provide comprehensive, continuous, long-term follow-up care; and
  • Strengthen psychosocial care for childhood cancer survivors.

The provisions of this survivorship legislation are consistent with the recommendations of the landmark IOM report, “Childhood Cancer Survivorship: Improving Care and Quality of Life.” Since the release of that report, which CCCA’s President Susan Weiner co-authored, the Children’s Cause has been leading advocacy efforts to draw attention to the needs of children who survive cancer among policymakers. CCCA was proud to lend our support and expertise to the development of this legislation.

CCCA launched an action alert on this legislation after its introduction, providing you the opportunity to quickly and easily tell your Representative why this bill matters. Please take a moment to share your story with Congress, and ask your friends and family to do the same.

Family and survivors coming to the Alliance for Childhood Cancer’s Action Day in June will ask their Representatives to co-sponsor the survivorship bill in their meetings on Capitol Hill. The Alliance has already endorsed the bill in a letter to Rep. Speier.


FDA Asks Patients for More Input

The FDA Safety and Innovation Act (FDASIA), signed into law in July 2012, called for the Food and Drug Administration (FDA) to increase patients’ participation in the regulation of medical products. The FDA intends to use new formats and forums to include patients’ perspectives in regulatory decisions. FDA also aims to be more transparent and provide more useful information about its decisions.

Families of children with cancer have a special perspective to offer FDA on benefit-risk considerations when new drugs are being reviewed for use in children. Factors such as the severity of a disease, its impact on daily life, and the available treatment options and their risks and side effects are key to families’ perspectives on the usability of new agents.

FDA’s past mechanisms for obtaining patients’ and families' input were restricted to discussions of specific drugs under review or through open participation at FDA Advisory Committee meetings. As a result of FDASIA, several new efforts are underway to expand opportunities for patients’ input.

Patient Network Website: FDA recently re-launched its Patient Network website, an interactive tool for educating patients, advocates, and consumers about how drugs and devices are reviewed and approved by FDA.

The new site has information on opportunities for early patient involvement in the regulatory process, clinical trials, and treatment options. It also has information about medications and warnings or recalls of drugs and devices. The site includes important information about how advocates can serve as patient representatives on FDA Advisory Committees, how to submit comments on disease-specific issues and how to stay updated and engaged in the drug development process.

Benefit-Risk Assessment Draft Implementation Plan: FDASIA required FDA to publish a draft five-year plan describing the agency’s approach to creating structured benefit-risk assessments in the drug regulation process. Assessment of a drug’s benefits and risks involves a carefully weighted analysis of the severity of a condition and current treatment options available for a given disease. In March, FDA released the “Structured Approach to Benefit-Risk Assessment in Drug Regulatory Decision-Making,” (pdf) which attempts to further clarify and make more transparent the factors FDA weighs in evaluating a drug for approval. CCCA’s comments submitted to FDA highlighted special considerations that families of children with cancer take into account when determining their children’s care. The draft plan provides insight into the FDA’s benefit-risk assessments and provides greater transparency regarding the factors that regulatory staff consider when reviewing a new drug. These factors include the analysis of a condition and its current treatment options, possible benefits of the new product, possible risks, and risk management strategies.

FDA plans to implement the draft plan over the next four years, with full implementation for all new drug applications in FY2017. In the interim, the agency will hold two public workshops on benefit-risk considerations beginning in FY2014.

Patient-Focused Drug Development: FDA will also seek patients’ input in at least 20 meetings over the next four years, each focused on a specific disease area where the agency believes it could benefit from greater patient perspectives. The Patient-Focused Drug Development Initiative will attempt to identify new outcome measures for specific diseases and treatments. FDA will issue a report on each meeting to capture insights on benefit-risk assessments for drugs that treat specific disease areas.

CCCA led the Alliance for Childhood Cancer in recommending to FDA that they hold a meeting on childhood cancer as part of this initiative to encourage FDA to focus on the needs of children with cancer. FDA did not select childhood cancer as a disease area of focus in its initial meeting schedule for FY2013-2015. The agency will seek input from patient advocates as they determine the disease focus areas for FY2016-2017. CCCA will continue leading advocates in the push to focus on childhood cancer in the next round of meetings.


Parents Need More Guidance in Administration of At-Home Medication

A new study revealed a high rate of errors by parents in giving cancer medication at home to their children. Parents are assuming greater responsibilities for administering therapies at home, tasks that once were the responsibility of trained nurses. Over a third of the mistakes observed in this study could have been prevented with better communication between families and medical teams, according to the research published in the May issue of Pediatrics.

Nurses observing 92 children receiving 242 at-home administrations of cancer treatment noted 72 accidental medication mistakes, including 4 that caused injuries and 40 that were potentially harmful. Two of the errors were classified as life-threatening. The overall weighted rate was 70.2 errors per 100 patients. On average, the children being observed were taking about 10 medications, with some taking as many as 20 drugs a day.

Many of the injuries stemmed from parents misunderstanding complicated labels, leading the study’s authors to call for better labeling and improved drug information sheets. In some cases, the label dosage or instructions were themselves incorrect.

Some errors resulted from parents deriving their own strategies to get their reluctant children to take their medication, such as sprinkling crushed chemotherapy on food when it was supposed to be taken on an empty stomach. Dosages were altered inadvertently by families using knives rather than pill cutters. Researchers recommended that parents receive more information and support to correctly manage their child’s treatment at home. They suggested that better communication and prevention strategies, such as text message reminders, might help to reduce administration errors at home.


Save the Date for a DC Reception on Tuesday, June 25

CCCA is pleased to announce our summer reception in Washington, DC, on Tuesday, June 25th. The event will take place at Hogan Lovells’ law firm at 555 13th Street, NW, and will benefit our ongoing advocacy work for children with cancer.

Please join us for drinks, hors d’oeuvres and an evening with friends. Attendees will have the opportunity to hear a survivor’s story and learn about CCCA’s current advocacy activities. Tickets are $25, and can be purchased here.