SKC Formally Appeals NICE Decision

The logo for solving kids ' cancer shows a pink butterfly.

FOR IMMEDIATE RELEASE


NICE HEARS APPEAL ON REFUSAL OF CHILDREN’S CANCER TREATMENT

First Appeal Against NICE Decision Led by a Charity

 

September 30, 2016 – New York, NY: The National Institute for Health and Care Excellence (NICE) heard an appeal led by Solving Kids’ Cancer (SKC) against its recent decision to deny children in the UK treatment that is proven to significantly increase the chance of survival for children diagnosed with an aggressive childhood cancer, neuroblastoma.


This is the first appeal against a NICE decision made by a charity acting alone. Solving Kids’ Cancer, a nonprofit based in New York, teamed up with their partner organization in the UK to lodge an appeal in July 2016 when NICE first announced the decision to refuse funding this potentially life-saving drug in England and Wales. NICE accepted that the charity has valid grounds for appeal and the full hearing was held in Manchester on September 30.


The SKC team was made up of both US-based and UK-based parent representatives, all of whom have lost a child to neuroblastoma, and one parent whose daughter survived neuroblastoma as a result of the same treatment NICE is seeking to deny.


John Rogers, a scientist and the father of Stella who survived neuroblastoma after receiving this treatment in the US, said: “Dinutuximab, is the only treatment specifically developed to treat cancer in children and is the standard treatment in the US, Australia, and Canada. It has been tested since the mid-1980s and has been proven to significantly improve survival in these children over the long term. If NICE does not accept this appeal, children in the UK could only hope to access unproven treatments through clinical trials, which is unethical when there is an approved and effective drug available.”


“In 2009, we raised £250,000 to take our 2-year-old daughter, Stella, to the US for dinutuximab therapy. She is now a happy, healthy 8-year-old. Surely children in the UK have just as much right to this chance of survival as children in other parts of the world?”


Solving Kids’ Cancer has five representatives giving evidence, including expert life sciences regulatory lawyer and advocate, Grant Castle. Dr. Castle is a partner in the London office of Covington & Burling LLP, who has provided his services on a pro bono basis, and who presented the charity’s case at the hearing.

Speaking before the hearing, Steve Richards, CEO of Solving Kids’ Cancer UK, said:


“This is the first time that a charity has made a unilateral appeal against a NICE decision, but we cannot stand by and allow NICE to effectively condemn UK children diagnosed with this devastating illness to a reduced chance of survival. By their own assessment, NICE agrees that dinutuximab is a novel approach that saves the lives of some children and extends the lives of others. By sticking rigidly to very narrow appraisal criteria, NICE failed to account for any of the unique contexts, challenges, and considerations that exist within pediatric oncology. The cost of this treatment for the very small number of children in the UK who could benefit was projected by NICE at just £2m.”



“If this decision stands, parents will have to raise in excess of £500,000, as well as enormous reserves of emotional resilience and determination, to take their sick child abroad for the greater chance of survival dinutuximab therapy provides. We are committed to doing everything in our power to ensure that this flawed decision is reversed.”

 

ABOUT SOLVING KIDS’ CANCER

Solving Kids’ Cancer is dedicated to significantly improving survivorship of the deadliest childhood cancers. Since 2008, SKC has supported and managed 26 research projects, introducing 18 new treatment options, and validating 14 agents for further use in combination with other drugs. Solving Kids’ Cancer is a 501(c)(3) public charity.. To learn more about their work, visit solvingkidscancer.org

###

A man and a little girl are sitting in front of a wooden wall.
By duda November 20, 2024
Ahana’s Story of Strength with High-Risk Neuroblastoma Every day, over 1,000 children worldwide are diagnosed with cancer,1 and for many families, that diagnosis means facing an uncertain future. At just 20 months old, Ahana was diagnosed with a rare and aggressive cancer that had already spread through her small body. Her family’s search for life-saving childhood cancer treatments led them from South Africa to Spain and possibly toward a clinical trial in New York. Ahana’s journey shows the importance of supporting organizations like Solving Kids’ Cancer, which works to find, fund, and advocate for advanced treatments that give hope to families fighting against tough-to-treat rare pediatric cancers.
A poster for 2024 year in review for solving kids ' cancer
November 11, 2024
Advancing Childhood Cancer Research: SKC’s 2024 Milestones
A group of children are holding a sign that says
October 1, 2024
Lace Up for Kids: Your Impact on Childhood Cancer Research At Solving Kids’ Cancer, our mission has always been driven by hope — hope for better treatments, hope for breakthroughs, and most importantly, hope for every child fighting cancer. This year’s Lace Up for Kids campaign was nothing short of inspiring. Together, we’ve raised over $264,000 across the entire campaign, funding innovative childhood cancer research and providing more treatment options for children in need.  From coast to coast, nearly 4,000 participants have stepped up to lace up their gold shoelaces and make a difference. Whether it was through schools, sports teams, or community groups, the show of support has been overwhelming throughout the years. Over 7,823 donations have been made to accelerate new treatments, and the hashtag #CareWearShare has been used 13,864 times, spreading the message far and wide!
A girl is standing in front of a car in a parking lot.
September 2, 2024
Gold Laces for Childhood Cancer Awareness Month: Erin’s Story 
A group of children are standing in a circle with their shoes on the floor.
August 8, 2024
Smithtown Goes Gold for Childhood Cancer Awareness Month!
A poster for lace up for kids with a butterfly on it
August 6, 2024
Childhood Cancer Awareness Month: Lace Up for Kids 2024
A man standing on top of a mountain with the words osteosarcoma survivor to surgeon dr. kurt weiss ' story
August 1, 2024
Osteosarcoma Survivor to Surgeon: Dr. Kurt Weiss’ Story
Jacob 's journey : a childhood cancer survivor 's story
May 10, 2024
Jacob’s Journey: A Childhood Cancer Survivor’s Story Every battle has its heroes, and among the bravest are young children like Jacob Mozer, who faced stage 4 high-risk neuroblastoma when he was barely old enough to walk. Diagnosed at only 16 months old, Jacob’s early years were filled with constant medical appointments and intense treatments. Despite these challenges, today, Jacob is a shining example of the remarkable progress in pediatric oncology. He’s not merely surviving; he’s thriving as a pharmacy resident, driven to give back to the medical community that gave him a second chance at life. During National Cancer Survivors Month this June, we honor and celebrate fighters like Jacob who have battled cancer and emerged stronger. This month also serves to amplify the conversation about pediatric cancer survivorship and push for vital research that continues to save lives. Solving Kids’ Cancer (SKC) plays an essential role in this effort, supporting children like Jacob by funding crucial clinical trials that enhance survival rates and improve the quality of life for those fighting fatal childhood cancers.
A close up of a person holding a test tube in a laboratory.
May 2, 2024
Trailblazing Treatments for Pediatric Brain Tumors Picture a fortress built to guard precious treasure but accidentally also keeps out the very allies it needs in a time of crisis. This is the challenge faced by medical researchers when dealing with the brain’s natural defense — the blood-brain barrier — especially in children with brain tumors. At Columbia University Medical Center, Dr. Luca Szalontay is leading the charge to breach this fortress with groundbreaking approaches. Recently, Dr. Timothy Cripe from Solving Kids’ Cancer’s podcast, This Week in Pediatric Oncology (TWIPO) , interviewed Dr. Szalontay to delve deeper into these innovative treatments. This Brain Tumor Awareness Month, we’re shining a light on these important advances in treating children’s cancer. Understanding the Challenge Treating pediatric brain tumors like diffuse midline glioma (DMG) is especially tough because of the brain’s own protective shield, known as the blood-brain barrier. This barrier keeps out most chemotherapy drugs as well as large molecules, like antibodies, making it hard to treat the tumors effectively. Dr. Szalontay describes the dilemma: “Our field hasn’t been able to advance as quickly as it has with leukemias or other solid tumors because the drugs don’t penetrate the brain efficiently.” She adds on the nature of this blood-brain barrier, “It’s evolutionary, very important to protect us from different toxins and harmful agents, but this is a shield which protects the tumor as well.” Even when drugs work well in lab tests, they struggle to get to the tumor in strong enough doses without harming the body. Dr. Szalontay also points out that DMG tumors are found in very sensitive areas of the brain, making traditional surgery or radiation risky because they could damage vital brain functions. This makes finding safe and effective treatments for these tumors even more challenging. So, what’s being done to tackle these obstacles and help kids fight these tumors?
A group of stick figures are holding hands around a globe.
April 4, 2024
World Health Day: Pediatric Cancer’s Global Divide Imagine this: Two children, born at the same moment, oceans apart. In a country brimming with medical resources, one child quickly gets top-notch treatment for cancer. Meanwhile, across the globe, the other child faces a drastically different reality, with limited access to basic care. This isn’t just a thought experiment — it’s the harsh truth of pediatric cancer treatment worldwide. Observed every year on April 7, World Health Day shines a light on the mission to make healthcare accessible for all. This day is particularly significant in the context of pediatric cancer, where vast disparities in treatment availability highlight the global challenge of ensuring every child has the opportunity for health and healing. The Stark Contrast in Survival Rates: A Look at Pediatric Cancer Statistics Every day, more than 1,000 children are diagnosed with cancer, and the chances of survival vary greatly depending on where they live.1 Pediatric cancer survival rates serve as a clear indicator of the disparities between high-income countries (HICs) and low- and middle-income countries (LMICs). In wealthier nations such as the United States and Western Europe, the 5-year survival rate for children with cancer is over 80%, thanks to advanced healthcare systems and access to the latest treatments.2 But, here’s a troubling childhood cancer statistic: in LMICs where 80% of children with cancer live, fewer than 20% survive.1 This gap is due to several factors, including late diagnoses or, tragically, no diagnosis at all. Many children succumb to the disease without access to pain medication, and the burdens of travel and the high costs force most families to abandon care. These challenges are compounded by limited access to necessary treatments,3 as well as a lack of specialized equipment and trained healthcare professionals needed for effective pediatric cancer treatment. Addressing this divide requires a concerted global effort to improve early detection, make treatments more affordable, and enhance healthcare infrastructures, giving every child a fair chance to beat cancer. Navigating the Access Maze Access to cancer care shouldn’t be a treasure hunt. Yet, for many in LMICs, it feels like that — a relentless search for something seemingly out of reach. Here’s the reality revealed in childhood cancer statistics worldwide: many areas lack enough doctors who specialize in children’s cancer4 and the prices of cancer drugs can be sky-high.2 Even getting to a hospital can be an ordeal, with some families traveling hundreds of miles. And the financial strain? Devastating. The cost of care can quickly add up to more than a year’s income, forcing households into deep financial distress. In many LMICs, the lack of comprehensive insurance coverage means families are often left to pay for pediatric cancer treatment entirely out-of-pocket.5 Families often face the impossible choice between their child’s health and their financial survival. Solving Kids’ Cancer: Bridging the Gap Solving Kids’ Cancer (SKC) isn’t standing by; we’re on the front lines, actively working to make a difference in the global pediatric cancer landscape. Beyond funding research, advocating for policy changes, and fostering global partnerships, SKC is committed to educating and empowering the community to take action. We understand that while we might not have a global foothold to address every aspect of the pediatric cancer divide directly, there’s power in collective action. Through our efforts, we aim to catalyze advancements in pediatric cancer treatments and care that could dramatically change outcomes for children worldwide. • Research: We direct funds into cutting-edge studies, searching for breakthrough treatments that promise a brighter future for all kids, regardless of where they live. • Advocacy: We advocate for children’s needs by steering research funding towards unmet challenges and collaborating with experts to prioritize all children, including expanding trials to international sites to maximize access.2 • Global Partnerships: We don’t do it alone. By teaming up with researchers, hospitals, and other organizations across the globe, we’ve helped fund clinical trials that have extended to over 250 institutions across 15 countries worldwide . Uniting for a Cause Solving Kids’ Cancer extends its mission beyond research by fostering community and collaboration. Our signature event, Lace Up for Kids , held annually during September Childhood Cancer Awareness Month, is a powerful testament to this mission, drawing communities together to stand in solidarity against childhood cancer. Additionally, supporters can create their own fundraiser and have a platform to engage their local communities, sparking crucial conversations and garnering support for the cause. These initiatives highlight an important fact: While the challenge of pediatric cancer is significant, each of us has a part to play. By working together and supporting each other, we can fund the research needed to possibly change the direction of pediatric cancer treatment and care worldwide. World Health Day: A Chance for Every Child The fight against pediatric cancer is a stark reminder of the work ahead of us.6 It’s a battle that Solving Kids’ Cancer is dedicated to, but we need more than just our efforts — we need a global movement. This World Health Day, let’s rally for every child’s right to fight cancer on an even playing field. Donate today to help fund more innovative research and one day find a cure for childhood cancer — because every child, no matter where they are born, deserves a chance — a chance to fight, a chance to survive, and a chance to thrive.
More Posts