April Bulletin: FDA hosts forum; CCCA announces Hill briefing

FDA Hosts Childhood Cancer Forum

An FDA-hosted Childhood Cancer Advocacy Forum last week provided opportunity for groups including the Children's Cause to engage with agency representatives around current drug development challenges and opportunities. The Forum included an update on oncologic drugs approved for pediatric use and clinical trial progress, as well as presentations on BPCA and PREA, compassionate use, pediatric Patient-Reported Outcomes, and more.
 
Among the highlights from the event:

  • On PREA: In a presentation about the effectiveness of the Pediatric Research Equity Act (PREA), the FDA's Dr. Margaret Thompson reported that 15 new oncology drugs were approved in 2015-2016. Of those, only one was approved for treatment in pediatrics: dinutuxinab for neuroblastoma. As Dr. Thompson noted: "This was actually unusual. Most years, all new drugs would be for adult indications." Of the 14 drugs approved for use in adults, none have had the PREA requirement applied. This has led various stakeholders, including Children'sCause, to question how PREA can be improved to make it more effective. Dr. Thompson discussed several possibilities for improving PREA, which include changing the pediatric study requirement from indication-based to mechanism-of-action based.
  • On Trial Eligibility: Dr. Christy Osgood discussed one way to increase pediatric access to adult clinical trials by expanding eligibility criteria to include adolescents in adult trials when the disease and/or target is the same. Opportunities might be also identified where alternative trial designs could allow a younger cohort in early phase trials for older patients. Dr. Osgood said logistical challenges can arise because adults and children are treated in different settings. But Dr. Osgood affirmed that the FDA is "supportive of models that include pediatric patients earlier in trial designs."
  • On Expanded Access: Another member of the FDA team, Dr. Ashley Ward, discussed expanded access or "compassionate use." Benefits to expanded access include the potential to offer hope to children who have exhausted all other options. Barriers mentioned included the additional resource burdens on the manufacturer (time, paperwork, and sometimes limited drug availability). The biggest barrier identified is the manufacturer's fear that an adverse event might disrupt or derail the drug's path to approval. Dr. Ward commented that this fear is largely unfounded based on FDA experience, and stakeholders need to work together to increase manufacterers' confidence in the expanded access process.

Children’s Cause President Susan Weiner co-chairs the Alliance for Childhood Cancer task force on PREA and BPCA and is working with the Alliance on a package of BPCA and PREA recommendations for this year’s reauthorization of the Prescription Drug User Fee Act (PDUFA).

Save the Date: Capitol Hill Briefing & Reception on June 23

Children's Cause will host a Congressional briefing with childhood cancer survivors and experts on June 23rd, followed by a Capitol Hill reception.
 
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. At the evening reception, Members of Congress, staff and the health care community will be invited to join us in honoring the leadership of the Childhood Cancer Caucus and learning about new Children's Cause initiatives.
 
More details will be coming soon, as we finalize the program and speakers.
 
If you are in the National Capitol area and have personal experience with survivorship access issues, we want to hear from you!
Please email us at jkean@childrenscause.org if you're interested in helping us make this event a success.

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There's Still Time to Register for Action Days!

It's not too late to register for the 5th Annual Childhood Cancer Action Days in Washington on May 16 & 17. This two-day Alliance for Childhood Cancer event brings our community together to advocate for important childhood cancer issues currently before Congress. Register here.
 
A strong showing of advocates at this year's Action Days is more crucial than ever, as the Childhood Cancer STAR Act is nearing the critical committee stage in the legislative process. There are now 200 House cosponsors of this bill! Sharing your story in Washington this spring will help us tip the scales toward securing passage of this historic, bipartisan legislation. 
 
Issues and Advocacy Training will take place Monday, May 16th at the Holiday Inn on Capitol Hill, followed by pre-arranged meetings with Members of Congress and their staff on Tuesday, May 17th.
 
Visit the Alliance for Childhood Cancer website for more information and to register.
 

Moonshot Update: Pediatric Oncologists Appointed to Blue Ribbon Panel; NCI Accepting New Ideas

The National Institutes of Health (NIH) announced a Blue Ribbon Panel this month to guide and inform Vice President Biden's National Cancer Moonshot Initiative. We were pleased to see some pediatric experts named to this distinguished panel, including Dr. Peter Adamson of Children's Hospital of Philadelphia and Dr. James Downing of St. Jude.
 
"This Blue Ribbon Panel will ensure that, as NIH allocates new resources through the Moonshot, decisions will be grounded in the best science," said Vice President Biden. The panel will serve as a working group of the National Cancer Advisory Board and will be focused on how to drive advances in several key areas targeted by the Moonshot. Childhood cancer has been specifically identified as one of those key areas of focus.
 
NCI is asking the health community for scientific ideas and suggestions to support the Moonshot. One of the categories the NCI is specifically seeking input around is pediatric cancer.
 
If you have a suggestion, visit the NCI's online platform Cancer Research Ideas before July 1. We'll be weighing in, and we hope you will, too.
 
Related reading: 

Guest Blog: Why DIPG Needs an Awareness Week

This month we featured a guest blog from Janet Demeter, founder and CEO of Jack's Angels Foundation. Janet recently secured the introduction of a House Resolution (H.Res.586) that would designate the fourth week in May as DIPG Awareness Week.
 
An excerpt: 
 
"H.Res.586 was introduced in January by Congressman Steve Knight (CA-25) and calls for elevated consideration for low-surival-rate cancers and years-of-life-lost in the research grant process with the NCI. [This resolution] is the first legislation to directly recognize DIPG but our goal is to raise awareness for all the hundreds of types and sub-types of pediatric cancer. By calling attention to one type of childhood cancer, we aim to elevate the profile of all childhood cancers."
 
Read on and then take action.

Remembering Leila Polintan, Former CCCA Staff

We are deeply saddened to announce the passing of Leila May Polintan, 39, a former Children's Cause staff member who battled breast cancer for the past several years. Leila managed programs and development for the Children's Cause before moving on to become Communications Director for Grantmakers in Health. Leila is remembered by those who love her as an involved community member, an avid reader, a devout Catholic, and beloved wife, daughter, sister and friend.
 
We remember Leila with affection as a delightful, charming and passionate young woman who played an integral role in the formative years of the Children's Cause. Our thoughts are with her family and loved ones during this difficult time.
 
Leila's obituary is available here.
 

Are you ready for mother's day?

Mother's Day is quickly approaching, and we want to take the opportunity to remind you of a few ways you can support Children's Cause when you shop online:

If you're shopping on Amazon, considering using AmazonSmile instead. You get the same item at the same price, but we get a donation from Amazon!

Shopping somewhere else online? Check out Goodshop first to see if your shopping merchant is one of the thousands of stores in their network. Goodshop's site will help you find great coupons -- and then donate a percentage to us!
 
>>> Sending flowers? Use Goodshop and they'll donate 7-8% of your 1-800-Flowers or ProFlowers total to us!

When you make a purchase through our Bravelets page, Children's Cause receives a $10 donation. Products including jewelry, watches and scarves are available in yellow or gold (the colors of childhood cancer awareness) and enscribed with the words "be brave." Shop Bravelets.

As always, you can make a donation directly to the Children's Cause in honor or memory of a loved one. Let us know it's a Mother's Day gift in the comment field of your donation, and we'll be sure to let the honored woman know. Thank you!
 

Jessica Kean
March Bulletin: Mobilizing Childhood Cancer Advocates

KIDS ACTION NETWORK: NOW ACCEPTING MEMBERS!

We are excited to have a new grassroots advocacy program in development called the Kids Action Network (KAN). As we prepare to launch this new program, we invite you to become a founding member.  Members of KAN will receive tools and training to become engaged and effective advocates in Washington, in local communities, and online.

To promote and recruit this new program, we've spent some time this month talking about the power of advocates working together to find safe, effective, affordable treatments and cures for our kids:

CURE Magazine profiled our work through the lens of one family advocating on behalf of their daughter, a three-year-old survivor of a rare and typically fatal brain tumor: Children's Cause Gives Families a Voice in Pediatric Cancer Policy Debates.

George Dahlman, Children's Cause CEO, appeared on Childhood Cancer Talk Radio this month to discuss current policy issues in Washington and the power of collaboration in the childhood cancer community. A short clip is at right, and the full hour-long podcast is available here.

We have exciting plans in store for the KAN program and we are grateful for these opportunities to invite more passionate people to join our growing ranks of childhood cancer champions. As this program fully takes shape over the coming weeks and months, we look forward to keeping you posted on its first activities and achievements! 

Become an Advocate: Sign up now to become a founding member of the Kids Action Network!

CHILDHOOD CANCER STAR ACT: TURNING THE MAP GOLD

The Childhood Cancer Survivorship, Treatment, Access & Research (STAR) Act continues to gain bipartisan support in Congress with nearly 200 House and Senate co-sponsors, but we need even more in order to advance this bill to the Committee phase of the legislative process.

We've made it easier for you to engage around this bill with an interactive map and write-to-Congress tools all on the same page. If you see a red telephone icon in your district, then your Congressman sits on the critical Energy and Commerce Committee and needs to hear from you! We were glad to help bring this new tool to life and we hope it helps all of you engage around this bill.

Related: The Huffington Post Parents Blog asked their readers to think about the terrifying reality of childhood cancer in a piece titled "Childhood Cancer - How You Can Help." The post linked to our action alert, bringing brand-new supporters to our cause and new voices to Congressional offices. Thank you, Huffington Post!

TAKE ACTION:  Check the map, then write your Rep!

NIH AND NCI FUNDING: ALLIANCE WEIGHS IN

Through the Alliance for Childhood Cancer, we submitted comments this month to Appropriations Committee leadership on Fiscal Year 2017 funding levels and priorities. In this letter, we call for $34.5 billion for the National Institutes of Health (NIH) and $5.9 billion for the National Cancer Institute (NCI) in FY2017.

We also call for childhood cancer to become a greater priority within Congress and the NIH. Toward that aim, we included specific requests for report language around the Pediatric-MATCH Trial, pediatric immunotherapy advances, and more.

Read the full letter here.

REGISTER NOW FOR CHILDHOOD CANCER ACTION DAYS

There's still time to register for the 5th Annual Childhood Cancer Action Days in Washington on May 16 & 17. This two-day Alliance for Childhood Cancer event brings our community together to advocate for important childhood cancer issues currently before Congress. Register here.

Issues and Advocacy Training will take place Monday, May 16th at the Holiday Inn on Capitol Hill, followed by pre-arranged meetings with Members of Congress and their staff on Tuesday, May 17th. A block of rooms has been secured at the training location with a group rate deadline of April 15th.

Visit the Alliance for Childhood Cancer website for more information on registration, scholarships (still available!), and hotel reservations.

RESEARCH ROUND-UP

  • FERTILITY: Male survivors of childhood cancer are three times more likely to suffer infertility as a late effect of their treatment compared to female survivors. The new research, which examined the impact of 14 commonly used chemotherapy agents, found that 50 percent of male survivors would later become fathers compared to 80 percent for their siblings. The study's authors reinforced that boys diagnosed post-puberty should bank their sperm. Female survivors studied were significantly less likely to suffer infertility problems, but problems with fertility accelerated after age 30. (The Lancet Oncology)

Related: Cincinnati Children's is offering a Live Q&A on Fertility Preservation for children with cancer on April 20. Learn more and register.

  • HEART HEALTH: A two-decade clinical trial has proven the effectiveness of the cardioprotective agent dexrazoxane when administered following doxorubicin therapy (associated with toxic cardiac effects) for children with certain blood cancers. The study followed 600 patients and found that children who received dexrazoxane during their treatment had normal hearts, while many of those who did not receive dexrazoxane had significant cardiac late effects. (Journal of Clinical Oncology)
  • MEDULLOBLASTOMA: Early research in mice has identified a new and less toxic combination therapy to potentially treat the most aggressive form of medulloblastoma (Group 3 tumors). Patients with Group 3 medulloblastoma have just a 40 percent survival rate. Researchers hope to quickly advance this novel drug combination to a clinical trial (Cancer Cell)

ADVANCING HOPE ACT PASSES OUT OF SENATE HELP COMMITTEE

The Advancing Hope Act, a provision to reauthorize a pediatric priority review voucher program, passed out of the Senate's Health, Education, Labor & Pensions Committee this month by a vote of 18-2. This legislation is part of the broader Innovation for Healthier Americans Act, which is the Senate response to the House's 21st Century Cures bill (H.R.6, passed in July 2015).

The Senate Committee voted to extend the Advancing Hope Act program by ten years, which would give the program a 2022 sunset, with a 2027 extension for drugs and biologics that receive a rare pediatric disease designation by 2022.

SURVIVOR SPOTLIGHT: MEET AIDEN

In recognition of March as National Kidney Month, we highlighted a young survivor of Wilms' tumor to help spread awareness that children get kidney cancer, too. This March also marked three years since Aiden, now 6, was diagnosed with the disease.

"Over the course of the last 3 years, I have met many child patients, or warriors, and their families. I count them among some of the bravest people I know. Many of their stories don't end happily. We need to do more. More for these kids, and their families," his mom writes. "I can tell you that there is not a day that goes by that I don't feel a flash of terror at the thought of his cancer returning, even though the statistics say it won't. I have seen too many beautiful children succumb to these disease and become the wrong statistc."

Thank you for reading and sharing.

Have a story of your own? We want to hear from you.

HELP US UPDATE OUR RESOURCES

As part of a recent website relaunch, we are conducting a full review and update of our online resources. We want to hear from you: What organizations, publications, links or other resources would you recommend to families navigating childhood cancer? 

We're looking for resources that span the spectrum of care, from diagnosis and clinical trials to palliative care, transitions of care, survivorship and more. Thank you for helping us provide information that is current, comprehensive, and useful to families!

Leave your suggestions here, or email us at jkean@childrenscause.org.

Jessica Kean
February Bulletin: Updates from Washington

CHILDREN'S CAUSE DEBUTS BRAND NEW WEBSITE

Thank you for visiting our freshly re-designed website at www.childrenscause.org!

We hope this new site will make it easier for you to:

We would love to hear your feedback.

Become an Advocate: With the launch of the new website comes a chance to join our grassroots action team! Sign up now to be among the first to hear about upcoming advocacy opportunities.

WHITE HOUSE HOSTS PRECISION MEDICINE SUMMIT, ANNOUNCES NEXT STEPS FOR INITIATIVE

We learned concrete details about the next steps for the Precision Medicine Initiative from a White House summit yesterday, which featured remarks by NIH Director Francis Collins and President Obama.
 
"My hope is that [the Precision Medicine Initiative] becomes the foundation, the architecture, whereby 10 years from now we can look back and say that we have revolutionized medicine in areas like cancer or Alzheimer's or some of the diseases that cause so much pain and suffering for so many families all across the country," said the president in panel remarks.
 
Among the key actions announced:

  • Direct Volunteers Pilot Studies Program: NIH announced a new grant to Vanderbilt University and Verily (formerly Google Life Sciences) to explore the optimal approaches for enrolling participants in the PMI Cohort Program, a first step toward reaching its goal of 79,000 cohort volunteers by the end of this year. Dr. Collins also announced the establishment of a PMI Cohort Program Institutional Review Board to ensure responsible oversight of the program.
  • Sync for Science: Working with electronic health record developers, this program will pilot the use of “open, standardized applications to give individuals the ability to contribute their data to research, including for the PMI cohort.”
  • precisionFDA Challenge: FDA launched its first “precisionFDA challenge,” a call to the genomics research community to achieve more consistent and accurate DNA testing results by using the new precisionFDA collaboration platform. This first ‘community challenge’ runs through April 25th.
  • Data Security Policy Principles: The White House released for comment its draft guidelines and framework for ensuring data security within the Precision Medicine Initiative.

Children's Cause is energized by the potential of these initiatives to improve the lives of pediatric cancer patients. We will be carefully reviewing these new developments and urging the Administration to include children with cancer and their families in the framework and the developing cohort. We will work to ensure that valid insights from precision medicine translate into the development of new therapies for children with cancer as soon as possible.

 

RELATED: NIH Director Francis Collins, MD, devoted a blog post this month to reporting on a new study that holds significant promise for the future of precision medicine in pediatric oncology. The study found genetic clues with the potential to refine care and guide treatment in approximately 40 percent of the 150 children with cancer studied.
 
Dr. Collins wrote: "While much more work remains to determine how genomic analyses can be used to devise precise, new strategies for treating kids with cancer, the study provides an excellent sample of the kind of research that NIH hopes to accelerate under the nation's new cancer 'moonshot.'" (NIH Director's Blog)

REGISTRATION NOW OPEN FOR CHILDHOOD CANCER ACTION DAYS

You’re invited to join Children's Cause and the Alliance for Childhood Cancer for the 5th Annual Childhood Cancer Action Days. This two-day event in Washington brings our community together to advocate for important childhood cancer issues currently before Congress.
 
Issues and Advocacy Training will take place Monday, May 16th at the Holiday Inn on Capitol Hill, followed by pre-arranged meetings with Members of Congress and their staff on Tuesday, May 17th. Registration is now open!
 
Visit the Alliance for Childhood Cancer website for more information on registration, scholarships, and hotel reservations.
 
A strong showing of advocates at Action Days 2016 is more crucial than ever, as we anticipate the Childhood Cancer STAR Act will be at a critical point in the legislative process. Sharing your story in Washington this spring will help us tip the scales toward securing passage of this historic, bipartisan legislation, which now has over 180 House and Senate cosponsors!

FROM THE CEO'S DESK

George Dahlman, new CEO of the Children's Cause, takes to the blog to share insights and perspectives from his first month on the job:

Meet George: An Introduction -- Learn more about George's story, his background, and why he believes in the work of the Children's Cause. Plus, learn how you can help us succeed in our mission of finding cures and improving the lives of survivors.

“While cancer research has strong bipartisan support in Washington, the fight for dollars will require a concerted effort by the cancer community - and especially cancer advocates - to keep the heat on their legislators.”

Read on for George's perspective on the climate in Washington as he explores two big questions: What does the National Moonshot Initiative really mean for children with cancer, and how much can be accomplished in an election year? 

IN CASE YOU MISSED IT [QUICK LINKS]

  • DRUG SHORTAGE RECOMMENDATIONS: Pediatric oncologists have issued an "ethical framework" for allocating life-saving oncologic drugs facing shortages. The guidelines aim to help clinicians conserve oncology drugs in times of shortages and guide difficult decisions about prioritization and rationing. “In the absence of a much-needed national advisory statement on how best to allocate scarce drugs, and until policymakers and stakeholders can prevent future shortages, the guidance articulated here supports reasoned decision-making in the face of an actual drug shortage and aims to minimize bias as might occur when individual clinicians or institutions are forced to make difficult, and at times tragic, rationing decisions for children with cancer,” the authors write. (Journal of the National Cancer Institute)

 

  • LATE EFFECTS: An important study about detecting secondary thyroid cancer in childhood cancer survivors finds that physical exams alone may miss a substantial number of nodules. Thyroid cancers account for about 10 percent of secondary cancers in pediatric cancer survivors. "When you have a high-risk population, particularly those who have had head and neck radiation, thyroid ultrasound is really what is needed to screen those patients," said the study's senior author, Sarah O'Brien, MD. (Journal of Pediatric Hematology/Oncology)

 

  • CHILD4CHILD MUSIC VIDEO: On International Childhood Cancer Day (February 15), our friends at the American Childhood Cancer Organization debuted a new music video, We Are One. The powerful song is a compilation of more than 900,000 children and teens who participated by recording themselves singing along. (ACCO.org)

 

  • SURVIVOR STORIES:  On social media this month, we shared stories of two amazing young survivors. Meet Arielle, who was diagnosed with an aggressive and typically fatal brain tumor at just 8 days old, and Emmalea, diagnosed with ovarian cancer at age 11.

 

  • POLICY LETTERS: Children's Cause has signed our name to a variety of policy letters in the past month including support for increased research funding and a thank you letter to Vice President Biden for his leadership of the National Cancer Moonshot. Read these and other recent policy letters here.

GO GOLD WITH BRAVELETS: A NEW WAY TO SUPPORT CCCA

Now you can support the Children's Cause and spread awareness about childhood cancer! When you purchase any item from our Bravelets fundraising page, we'll receive $10 as a donation.

Thank you for shopping and sharing!

Jessica KeanComment