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