Bulletin — Now at childrenscancercause.org

July Bulletin: Featuring an NIH Funding Alert and Policy Spotlight on PREA

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!

Rosen Research Award Nominations Due August 15th

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!

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

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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Jessica KeanJuly 29, 2016NCI, PREA, Rosen Award, ACE Kids

June Bulletin: Rosen Award, Hill Briefing, Policy Updates & More

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!

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.

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.

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.

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.

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.

Jessica KeanJune 30, 2016

May Bulletin: Action Day brings 200 advocates to DC

RSVP Now: Capitol Hill Briefing on June 23

Children's Cause will host a Congressional briefing with childhood cancer survivors and experts on June 23rd from 12:00-1:30PM.

The afternoon briefing will illustrate and address the access and coverage issues faced by childhood cancer survivors, including recent research findings and best practice recommendations for managing long-term care.

Speakers will include Dr. Smita Bhatia and Dr. Julie A. Wolfson of the University of Alabama, Dr. Anne Reilly of CHOP, and Dr. Monica Gramatges of Texas Children's Hospital.

Learn more here or RSVP to jkean@childrenscause.org.

Childhood Cancer Action Day Propels STAR Act to a New Milestone

The 5th Annual Childhood Cancer Action Days, hosted by the Alliance for Childhood Cancer, took place this month, bringing our community together in Washington to advocate for the Childhood Cancer STAR Act and increased NIH funding.

Approximately 200 advocates attended this year's event, spending a full day in advocacy training followed by a day of pre-arranged visits with Congressional offices. Around the country, advocates joined in from home by asking Congress to #StepUp for the #STARAct.

Your Action Day efforts helped us reach a big milestone: more than half of the House of Representatives now supports this life-saving legislation!

One advocate in attendance, Stacie Ritter, shared her take on the experience with us. Read our blog for Stacie's perspective on this year's Childhood Cancer Action Day.

Children's Cause Endorses ACE Kids Act

The Children's Cause for Cancer Advocacy is proud to be supporting the Advancing Care for Exceptional (ACE) Kids Act, which would improve care for approximately two million children with medical complexities in the Medicaid program.

Children with cancer, like children with other medical complexities, often see multiple specialists and a variety of physicians. There are over 30,000 children with cancer covered by the Medicaid system and the specialized care they require often takes them across state lines. Under the current Medicaid system, parents of children with cancer and other multiple, life threatening disabilities struggle to coordinate the complex, multi-state care of their kids. Only federal legislation can fix the fragmented system for children with medical complexity.

We are honored to join the nation’s leading children’s hospitals, along the Children’s Hospital Association, to support this legislation, which is voluntary for states, families, children’s hospitals and other providers.

Learn more about this bill:

Our Letter of Endorsement to Congress
SpeakNowForKids.org

Quick Links: Recommended Reading

Keeping Cures on the Shelf: Our own George Dahlman, CEO of Children's Cause, wrote a perspective piece on drug shortages in this month's Journal of Clinical Pathways.

"Ultimately, the mitigation of drug shortages will be the result of vigilance on the part of all stakeholders. Maintaining that awareness and managing a quick response system requires that doctors and families become involved in the policy process that makes it work and the rapid reporting of individual instances." (Journal of Clinical Pathways / May 2016)

Rare Tumors May Cause ADHD-like Symptoms: A new study suggests that some kids diagnosed with a hyperactivity disorder who also have high blood pressure may instead have a rare tumor - pheochromocytoma or paraganglioma - causing ADHD-like symptoms. (NIH, May 16, 2016)
Poliovirus Therapy May Treat Glioblastoma: The FDA granted 'breakthrough' designation to a treatment modified from poliovirus that shows promise for treating advanced glioblastomas. The therapy is currently being investigated in adults but researchers at Duke hope to expand their trials to pediatric brain tumor patients by the end of this year. (OncLive, May 16, 2016)

Childhood Cancer in the Spotlight:

From The New York Times Magazine's Cancer Issue: The Improvisational Oncologist & When Do You Give Up on Treating a Child with Cancer?
Humans of New York profiled children with cancer for two weeks and raised $3.7 million in a viral fundraiser for Memorial Sloan-Kettering Cancer Center: HONY Pediatric Cancer Series

Children's Cause Achieves GuideStar Platinum Status

Children's Cause is proud to be one of the first organizations to receive the Platinum Seal of Transparency from GuideStar, the world's largest source of nonprofit information. GuideStar recently launched the Platinum participation level to help nonprofits celebrate their results with new metrics to report on our progress.

We're proud to use GuideStar Platinum to share our full and complete story with the world. We achieved Platinum by completing GuideStar's preceding participation levels of Bronze, Silver, and Gold. Each provided stakeholders with increasingly detailed organizational information ranging from basic contact information to programmatic details.

Check out our updated #GuideStarPlatinum profile here:

http://www.guidestar.org/profile/11-3485631

Jessica KeanMay 25, 2016