Top Legislative Priorities Before Election Day

When Congress returns from its extended 7-week summer recess on September 6th, they will have just 17 workdays before adjourning for a pre-election recess. Some refer to this as the "lightning round of legislating," with a lot of work to be done and not much time to do it. 

Childhood Cancer STAR Act

The Childhood Cancer STAR Act will officially record its 250th cosponsor when the House returns from Recess -- but we are running out of time to receive a hearing and secure passage of this bipartisan, comprehensive childhood cancer legislation.

In order for the House Energy & Commerce Committee to consider this bill as one of their last priorities of this Congress, they need to hear from constituents about why this life-saving legislation is so important.

Please take a moment to fill out our Action Alert and tell Congress our kids can't afford to wait until the next Congress for action.

NIH Funding

Congress is tasked with deciding how much funding to provide the National Institutes of Health - and the National Cancer Institute - for 2017, as part of the larger appropriations process. As reported on our blog, a House Appropriations committee approved a spending bill earlier this summer that would increase NIH funding by $1.25 billion, while Senate appropriators would increase the NIH budget by $2 billion.

We are urging advocates to write to their Senators and Representatives and ask them to support the higher Senate number. It is imperative that the NIH and NCI continue to work for new treatments and explore the promise of immunotherapy. While we recognize that Congress faces difficult budget decisions, we also believe that Congress must further prioritize cancer research.

Take action now and tell Congress why each additional research dollar matters for children with cancer.

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Take Action: Pediatric Cancer Families Feel Impact of EpiPen Price Gouging

As many kids go through cancer treatment, one of the supportive care drugs they are provided is epinephrine in an auto-injector - or epi-pen. Epinephrine is a form of adrenalin and is primarily used to treat allergic reactions to food, insects, or other substances. But it's also provided to kids in cancer treatment in case of a reaction to the drugs they're being given.
 
Over the past several yeras, the price of epi-pens has increased by some 400 percent. With the recall of a competing product, the manufacturer of the epi-pen, Mylan Pharmaceuticals, faces little market pressure and used the opportunity to escalate the price. Since the cost of providing epi-pens to parents and patients may often be out-of-pocket, these price increases are especially troubling and represent an added burden to families who are already struggling with the treatment of a devastating disease.
 
Patients from a wide variety of communities are protesting the price-gouging and calling on Congress to intervene. On behalf of the pediatric cancer community, we encourage advocates to join in this appeal by signing on to our petition urging their representatives to speak up for patients and put a lid on profits over patients. 

Sign our petition urging Congress to take action: 
https://actionsprout.io/FC5265

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DC Event Line-Up: Childhood Cancer Awareness Month

Webinar on Psychosocial Impact of Childhood Cancer
September 1
The NCI is hosting an hour-long webinar on the psychosocial and social impacts of pediatric and adolescent cancers -- and how research and therapies are being created to address the unique needs of patients, siblings, parents, friends and others. Use hashtag #AskCCR to pose questions in advance on Twitter, and watch the webinar here.
 
Panel Discussion: Translating Discovery into Cures for Children with Cancer
September 8, 12:00pm-1:30pm, B-340 Rayburn House Office Building
This panel discussion, co-hosted by the Alliance for Childhood Cancer and the American Cancer Society Cancer Action Network, will examine the current state of pediatric cancer research and the unique challenges to developing new drugs to treat children with cancer. 
See invite for RSVP information.
 
CureFest 2016
September 17-18
CureFest is a festival held on the National Mall that brings thousands of childhood cancer advocates and supporters together with the general public and tourists to raise awareness about pediatric cancer. The event features informational booths, games and activities, live entertainment, and an awareness walk. For the first time this year, all events associated with CureFest are free of charge, although participants are still asked to register in advance. 
 
Be sure to stop by the Legislative Initiatives table at CureFest! There will be educational mini-sessions on current legislation and action steps you can take to make an impact.
 
Rally for Medical Research Hill Day
September 22
This annual event brings together nearly 300 national organizations, including the Children's Cause, to ask Congress to make funding for the National Institutes of Health (NIH) a national priority. Learn more.
 
Congressional Childhood Cancer Caucus Summit
September 23, 9-11am, Capitol Hill
Details on the 7th Annual Childhood Cancer Summit will be posted here when available.
 
Alliance Art Show and Luncheon
September 23, 11-1pm, Capitol Hill
Details will be shared when available.

Help us gear up for Childhood Cancer Awareness Month by strengthening our story bank. By sharing your story with us today, you empower us to have a much greater impact this September:

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Recommended Reading: Quick Links

• Children's Cause Responds to FDA's Status Report on PREA: The Food and Drug Administration released a status report in July on the Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA). Several Alliance for Childhood Cancer recommendations for improving PREA made it into the report, including an amendment that would require certain drugs developed for adult cancer indications to be evaluated for a pediatric cancer indication when there is evidence the drug affects specific molecular targets and/or mechanisms shared between adult and pediatric cancers. Read full letter. (CCCA, 8/18/16)
• CAC2 Summit in Philadelphia: The Coalition Against Childhood Cancer held its 4th annual meeting and summit earlier this summer in Philadelphia -- its "largest and most comprehensive educational experience yet." The Children's Hospital of Philadelphia wrote up a terrific overview of the event: Childhood Cancer Advocates Find Strength in Numbers. (CHOP, August 2016)
• Book Recommendation - Cooking for Kids with Cancer: To mark "Book Lovers' Day" earlier this month, we reviewed a new book by Chef Ryan Callahan, Cooking for Kids with Cancer. This book focuses on the food challenges facing children undergoing cancer treatment, and we include an excerpt and a sample recipe on our blog. Read more. (CCCA Blog, 8/9/16)

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New York Reception: November 3

Mark your calendars for the annual New York Reception for the Children's Cause for Cancer Advocacy on November 3. This year's event will take place at the James Burden Mansion in New York City from 7:00-9:30pm.
 
Proceeds benefit CCCA's mission of advocating for less toxic treatments, federal funding for research, and better follow-up care for the unique needs of childhood cancer patients and survivors.
 
Look for email invitation to come in September. If you have questions in the meantime, contact Julie at jtaylor@childrenscause.org.
 

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ALERT: Ask Congress to Fund More Childhood Cancer Research

When Congress returns from their August recess, they will be considering how much funding to provide the National Institutes of Health -- and the National Cancer Institute for 2017. As reported on our blog, a House Appropriations committee recently approved a spending bill that would increase NIH funding by $1.25 billion, while Senate appropriators would increase the NIH budget by $2 billion. We are pleased that both bills include funding for the Precision Medicine Initiative and the Gabriella Miller Kids First Research Initiative.

We are urging advocates to write to their Senators and Representatives and ask them to support the higher Senate number. It is imperative that the NIH and NCI continue to work for new treatments and explore the promise of immunotherapy. While we recognize that Congress faces difficult budget decisions, we also believe that Congress must further prioritize cancer research.

Take action now and tell Congress why each additional research dollar matters for children with cancer.

Then, consider scheduling a district office meeting during this August recess. Face-to-face meetings with your elected officials and their staff in the district office are an extremely effective way to get to know them and express your views on key issues like this one. Our new step-by-step Guide to Congressional District Meetings will help you!

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Rosen Research Award Nominations Due August 15th

The Children's Cause for Cancer Advocacy is pleased to announce the Leonard M. Rosen Memorial Research Award of $10,000, to be granted annually to an individual who has made an outstanding contribution to childhood cancer policy and advocacy. The nomination deadline has been extended to August 15!

Individuals who are engaged in projects in the broad fields of childhood cancer policy and/or advocacy are encouraged to apply or be nominated by colleagues or mentors. Applicants are asked to submit a detailed Curriculum Vitae, including publications, a two-page statement of how the Rosen Award will be used to advance policy and advocacy, and two references.

The inaugural Rosen Award will be made at the Children's Cause annual gathering in New York City on November 3, 2016.

The Rosen Award Nomination Form, along with additional details, is now available on our website at www.childrenscause.org/RosenAward. Thank you for sharing and nominating!

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Policy Spotlight: Pediatric Research Equity Act (PREA)

Making improvements to two drug development laws - the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) - has been a priority of the Children's Cause and the broader Alliance for Childhood Cancer for many years. On our blog this month, we took a closer look at PREA and how the childhood cancer community has been working together to fix this statute's shortfalls.

What is PREA? 

PREA requires pharmaceutical companies who are developing a drug for adult indications to also test the drug in children. Two exceptions to this requirement constrain their impact for children with cancer. First, pediatric studies of a drug can only be required in the same disease for which it is being studied in adults. Since children do not develop lung, breast or prostate cancer, for example, drugs under development for adult cancers do not have to be tested in kids. A second exemption from the PREA requirement applies to any drug being developed for a "rare" disease. Unfortunately, most cancers meet the definition of a rare disease.

These exemptions have resulted in PREA having virtually no effect in stimulating more treatments for childhood cancer. Families and childhood cancer advocates anxious for new treatments have been frustrated with the inadequacy of PREA and have pressed for remedies that stimulate childhood cancer drug development from industry.

A Community Effort

With a legislative opportunity coming up in the next year through a larger must-pass bill, the Children's Cause co-chaired a workgroup of the Alliance and Coalition Against Childhood Cancer (CAC2) that developed a series of policy recommendations to encourage more childhood cancer drug development. These recommendations include changes to the PREA statute.

In anticipation of a new Congress and legislation affecting the FDA, the Children's Cause has begun a broad consensus-building process to solicit additional recommendations and suggestions by stakeholders that will create a bold, effective framework for the creation of new drugs for kids with cancer.

The effort to change the laws that affect pediatric research may be the most challenging and important for our community and will require continued commitment, vision, collaboration and - most importantly - unity.

Full post: Generating New Treatments for Kids -- It's a Team Effort

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Recommended Reading: Quick Links

• ACE Kids Act has its day before the House Energy and Commerce Committee: "It was standing room only at the...hearing on the Advancing Care for Exceptional Kids Act. The July 7 hearing on Capitol Hill drew children's hospital executives, parents of children with medical complexity, health care industry experts and policy leaders for a review of a revised discussion draft of the bill that reflected input the committee and bill's cosponsors received from stakeholders..."Read more. (Children's Hospital Association, 7/8/16)
• Endocrine Abnormalities in Aging Childhood Cancer Survivors: Overall, 44% of survivors in this study demonstrated at least one...endocrinopathy. Survivors of [Hodgkin lymphoma] had the highest frequency of an endocrine abnormality (60.1%), followed by survivors of CNS tumors (54%), leukemia (45.6%), sarcoma (41.3%), non-Hogkin lymphoma (39.7%), neuroblastoma (31.9%), Wilms tumors (28.5%), and bone cancer (27.8%). Read more. (Journal of Clinical Oncology, 7/5/16)
• NIH Awards $55 million to build million-person precision medicine study: "The [NIH] announced $55 million in awards in fiscal year 2016 to build the foundational partnerships and infrastructure needed to launch the Cohort Program of President Obama's Precision Medicine Initiative..." Read more. (NIH, 7/6/16)
• Uncle Sam Wants You -- Or at Least Your Genetic Information: "Government scientists are seeking a million volunteers willing to share the innermost secrets of their genes and daily lives as part of an ambitious 10-year research project to understand the causes and cures of disease..." Read more. (New York Times, 7/23/16)

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Looking Ahead:  Childhood Cancer Awareness Month

Save the Date for CureFest 2016: September 17-18

This year's CureFest on the National Mall will take place the weekend of September 17th. CureFest is a national festival held in Washington, D.C. during Childhood Cancer Awareness Month, bringing thousands of childhood cancer advocates and supporters together with the general public and tourists to raise awareness about pediatric cancer.

The event features informational booths, games and activities, live entertainment, and an awareness walk. For the first time this year, all events associated with CureFest are free of charge, although participants are still asked to register in advance. 

Share Your Story: Help us gear up for Childhood Cancer Awareness Month by strengthening our story bank. By sharing your story with us today, you empower us to have a much greater impact this September.Click here to be a voice.

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Shopping for Charity through Designs that Donate

Designs that Donate is a premier shopping experience that satisfies every gift giving and personal shopping need, while supporting charitable causes. When you make a purchase from Designs that Donate and designate CCCA as the charity of your choice, they donate 12% of your purchase total to the Children's Cause.

Designs that Donate has just launched a brand-new website to provide an even better shopping experience: Visit designsthatdonate.com.

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Now Accepting Nominations for the Rosen Award

The Children's Cause for Cancer Advocacy is pleased to announce the Leonard M. Rosen Memorial Research Award of $10,000, to be granted annually to an individual who has made an outstanding contribution to childhood cancer policy and advocacy.
 
Relevant work should be related to advancing treatment and care for children, adolescents, and young adults and their families. Examples of areas of work include outcomes research, healthcare quality, delivery and cost, economics of drug development, and improved access to therapeutic agents. The award recognizes the importance of the unique challenges associated with the care and treatment of children with cancer that may not be associated with the treatment and care for adults with cancer. 
 
Individuals who are engaged in projects in the broad fields of childhood cancer policy and/or advocacy are encouraged to apply or be nominated by colleagues or mentors. Applicants are asked to submit a detailed Curriculum Vitae, including publications, a two-page statement of how the Rosen Award will be used to advance policy and advocacy, and two references.
 
The deadline for submission of applications in August 1, 2016.The inaugural Rosen Award will be made at the Children's Cause annual gathering in New York City in late October 2016.
 
The Rosen Award Nomination Form is now available at www.childrenscause.org/RosenAward. Thank you for sharing and nominating!
 

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CCCA Capitol Hill Briefing Highlights Survivorship Issues

Briefing speakers (l-r): Dr. Smita Bhatia (Univ. of Alabama), Stacie Ritter, Madeline & Hannah Ritter, Dr. Anne Reilly (CHOP), Dr. Monica Gramatges (Texas Children's), Dr. Julie Wolfson (Univ. of Alabama)

Children's Cause hosted a Congressional briefing this month that featured pediatric oncology experts sharing critical information about the unique needs and challenges faced by childhood cancer survivors over the short- and long-term. The event was attended by Congressional staff, as well as representatives from advocacy organizations and the National Institutes of Health. We thank the great line-up of speakers for sharing their time and expertise.
 
The presenters offered compelling evidence regarding the long-term medical and emotional impact faced by childhood cancer survivors, as well as the medical, geographic and financial challenges in accessing the care that is required throughout childhood and adulthood.
 
The Children's Cause is committed to ongoing efforts like these Hill briefings to raise awareness among Congressional and federal policymakers about the experiences and vulnerabilities faced by childhood cancer survivors throughout their lives. 
 
All of us at the Children's Cause send briefing speakers Madeline & Hannah Ritter our thanks and our congratulations: the twins graduated high school earlier this month! They'll begin their college careers this fall at Arcadia University. Read more about their survivorship journey on our website: Madeline & Hannah's Story.
 

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Pediatric Cancer Policy Updates

Oncology Center of Excellence at the FDA:

The Children's Cause for Cancer Advocacy recently weighed in on the proposed establishment of a new Oncology Center of Excellence at the FDA, part of an agency-wide reorganization effort. In our letter to FDA Commissioner Califf, we gave our support for establishing such a center that is capable of reviewing cancer diagnostics, drugs and biologics for children with cancer. A PDF of our letter is available here.

In breaking news, the FDA's Dr. Richard Pazdur (bio) has been selected to serve as acting director of this new Oncology Center of Excellence. We offer our congratulations to Dr. Pazdur and extend our thanks to Commissioner Califf for continuing to advance this initiative.

Ask Congress to Support the ACE Kids Act:

The Children's Cause for Cancer Advocacy is proud to be supporting the Advancing Care for Exceptional (ACE) Kids Act (H.R.546/S.298), which would improve care for approximately two million children with medical complexities in the Medicaid program. The bill would provide more consistency for children with medical complexity as they often need to travel across state lines for care, which currently results in coverage issues since Medicaid coverage may differ from state to state. We are honored to join the nation’s leading children’s hospitals, along with the Children’s Hospital Association, to support this legislation. TAKE ACTION.

FDA Offers Guidance on Expanded Access to Investigational Drugs: 

The Food & Drug Administration recently released several documents intended to help patients and physicians navigate the process of accessing investigational drugs.  Also called “compassionate use,” the FDA move attempts to streamline the process by which patients – who may have limited treatment options – can secure drugs that are still being tested and not otherwise available outside a clinical trial.

Access to unapproved therapies requires cooperation between the FDA, the company developing the product and the patient’s health care professional.   For its part, FDA released a new form that physicians must complete as part of the process for requesting access to unapproved treatments for their patients.   The new form is estimated to take 45 minutes to complete, instead of the hours the previous form required.  Instructions for completing the updated form along with a question and answer document and guidance for the pharmaceutical industry on how they might charge for access to these therapies were also released by the FDA.

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Research Highlights from ASCO

The American Society of Clinical Oncology (ASCO) held its annual meeting earlier this month in Chicago. Children's Cause was glad to attend this year's meeting, which brings together oncology professionals, organizations and other stakeholders. Here are just a few of the many research highlights out of this year's meeting that relate to childhood cancer survivorship:

• Childhood Cancer Survivors Less Likely to Receive HPV Vaccine: A new study finds that 42% of 13-17 year olds in the general population have received the HPV vaccine, compared to just 22% of childhood cancer survivors in the same age bracket. This is troubling because those with compromised immune systems - including survivors of childhood cancer - are more susceptible to HPV and its complications if infected. (James Klosky, et. al, Abstract #10573) 

  • 1 in 4 Adult Survivors of Pediatric CNS Tumors Unable to Function Independently: This study analyzed the functional independence of more than 300 adult survivors of pediatric central nervous system tumors such as astrocytomas and medulloblastomas. Researchers looked at the survivors' employment status, marital status, driving ability, independent living status, and ability to manage their own personal care and routine needs. Forty percent were classified as independent, while 34% were deemed moderately independent and 26% were non-independent. Non-independent survivors reported worse physical and emotional quality of life. (Tara Brinkman, et. al, Abtract #10524)
  • Parents Report Feeling Under-Prepared for Late Effects & Survivorship: Parents of childhood cancer survivors reported feeling extremely or very well prepared for their child's treatment and daily care needs but feeling less prepared for future limitations their children may experience and for life after cancer in general. Parents reported feeling more confident when oncologists and providers helped prepare them for life after therapy. (Kate Greenzang, et. al, Abract #85)

Also at ASCO, Vice President Joe Biden was on hand to deliver remarks about the president's Moonshot Initiative. "Today, oncologists and cancer researchers realize that they can't [advance cancer progress] alone... What's required today extends beyond any individual or any individual discipline, beyond medicine itself... It requires somewhat of a change in mindset. It requires a lot more openness - open data, open collaboration and above all, open minds," he said. For video of the Vice President's speech, visit ASCO's Virtual Meeting Portal. 

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Conference for Neuroblastoma Survivors: July 14-17

The Children's Neuroblastoma Cancer Foundation is holding a conference for Adolescent and Young Adult (ages 14-22) survivors of high-risk neuroblastoma, July 14-17 in Lombard, Illinois. The event will cover common issues facing these survivors including assuming responsibility for healthcare, transitioning to college, infertility, peer relationships and other psycho-social concerns. Space is still available -- email Jen Click at jsclick@gmail.com for more details and to register.

The AYA event is part of CNCF's 13th Annual Parent and Caregiver Education Conference, which brings together parents, caregivers, and neuroblastoma researchers to share information and learn about new treatment options and current research.

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Save the Date for CureFest 2016: September 17-18

This year's CureFest on the National Mall will take place the weekend of September 17th. CureFest is a national festival held in Washington, D.C. during Childhood Cancer Awareness Month, bringing thousands of childhood cancer advocates and supporters together with the general public and tourists to raise awareness about pediatric cancer.

The event features informational booths, games and activities, live entertainment, and an awareness walk. For the first time this year, all events associated with CureFest are free of charge, although participants are still asked to register in advance.