Orphan Drugs & Their Impact on Rare Childhood Cancers

Having a child with rare cancer that has no established treatment protocol can leave you feeling like you have nowhere to turn. But as science continues to progress, new and innovative cancer treatments are becoming more available. These treatments are called orphan drugs, and in this article, we explain what they are, how the US Food and Drug Administration (FDA) makes them available to the public, insurance questions, and more.

What Are Orphan Drugs?

According to the National Cancer Institute, orphan drugs are medications or treatments used to “treat, prevent, or diagnose an orphan disease” — a disease the FDA says affects “less than 200,000 people in the United States.” Each type of childhood cancer is rare, with about 15,000 children diagnosed annually in the United States in individuals younger than 20 years old.

 

What Are Ultra-Orphan Drugs?

Ultra-orphan drugs treat the rarest diseases in the world, which often impact less than 1 in 50,000 people globally and are typically the most serious, potentially debilitating, and fatal.

A person wearing purple gloves is putting pills in a petri dish.

The Orphan Drug Act of 1983

Developing medications and treatments for rare diseases is not lucrative for pharmaceutical companies. The cost of research, development and approval for drugs to treat rare diseases can be astronomical. That’s why, for many years, there were very few advancements in the realm of pharmaceutical treatment for rare diseases. 

However, in 1983, the U.S. Congress passed the Orphan Drug Act, a law that incentivizes the pharmaceutical industry to research and develop more treatments for rare diseases. Since the law’s passing, the FDA has approved hundreds of orphan drugs and granted thousands of FDA breakthrough drug designations — providing more novel treatments for diseases like rare childhood cancers and more hope for patients and their families.

 

4 Ways the FDA Makes Orphan Drugs Accessible to the Public

When it comes to potentially life-saving drugs, the FDA has four ways to expedite their availability, including: 

• Fast Track: A process to create and get drugs to patients faster. Treatments must fill an unmet medical need, such as being the first of its kind to treat a disease or provide a superior outcome or fewer side effects, for example. Pharmaceutical companies that develop fast-tracked drugs may also receive an expedited FDA review process to gain approval. 

• Breakthrough Therapy: A process to create and get drugs to patients faster     when the drug shows a superior outcome over existing treatment. Pharmaceutical companies who develop breakthrough therapies receive the same FDA benefits as fast-tracked drugs, plus additional guidance.

• Accelerated Approval: A process to create and get drugs to patients faster when a drug is believed to forecast a clinical benefit. Researchers can demonstrate benefits through various mediums such as lab results and diagnostic imaging. Accelerated approval with the FDA is based on evidence that a patient’s outcome may improve. 

• Priority Review: A process to create and get drugs to patients faster via an expedited and dedicated reviewal process (typically within six months). For a priority review, the drug must prove a significant improvement in patient outcomes based on various evidence points.

 

How to Access Orphan Drugs

Parents should speak to their child’s medical oncologist for more information and recommendations on the best course of treatment. Typically, an oncologist will prescribe and submit the request for treatment to insurance companies on the patient’s behalf. 

Tip: Try researching highly specialized healthcare institutions (tertiary or quaternary care institutions), as they tend to be on the cutting edge for treating rare diseases and running pediatric clinical trials

For more information on currently available orphan drugs, the Genetic and Rare Diseases Information Center (GARD) provides a list of FDA orphan drugs that can be sorted by rare disease names.

 

Does Insurance Cover Orphan Drugs?

Unfortunately, for various reasons, these types of drugs remain significantly more expensive than everyday pharmaceutical drugs and treatment. Per the American Journal of Managed Care, “Health plans restrict access to orphan drugs approximately one-third of the time, and restrictions vary considerably across plans,” with insurance companies generally restricting the most expensive orphan drugs. 

But all is not lost. That means two-thirds of the time, orphan drugs are approved by insurance companies. And it can also be well worth it to reach out directly to pharmaceutical companies to see if they are willing to subsidize or waive the cost of treatment.

 

The Impact of Orphan Drugs on Rare Childhood Cancers

Orphan and ultra-orphan drugs provide hope and a fighting chance for children battling rare childhood cancers — children who have dreams and aspirations — just like any other child. In addition, novel medications and treatments can, in some cases, improve a cancer patient’s outcome and quality of life. 

But there is so much more to be done. Funding effective treatments and breakthroughs for childhood cancer has never been more urgent. That’s why nonprofit and medical organizations, researchers, and medical oncologists continue to advocate for research into childhood cancers with low survival rates. 

At Solving Kids’ Cancer, we help accelerate new, next-generation treatments, including immunotherapy, cancer vaccines, and new drug combinations by carefully applying an understanding of the entire childhood cancer research landscape to invest in the most promising, innovative projects…. and clinical trials. 

When you give to Solving Kids’ Cancer, you help positively impact the lives of children living with rare cancers today and in the future by making better treatment options possible — because Every Kid Deserves To Grow Up®.

A pink button that says `` fund a new treatment today ''.
By Kristi McKay December 2, 2024
Ways to Give Back During the Holidays
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
By Kristi McKay July 4, 2024
A Quick Guide to Your Role as a Parent Advocate 
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.
More Posts