My grandson, at 15 months of age, was diagnosed with high risk neuroblastoma. He is a survivor. Although, he endured an incredible six year long journey, this story is not about him, his family, the changes in their lives, or even the obstacles any of them endured and overcame. This is a story about all the changes that have taken place over the same six years in funding for childhood cancer. This parallel story also is incredible and even more difficult to believe. We begin in 2006, about the same time my daughter heard the news, “Your son has cancer.”
By 2006, the only child specific cancer drug approved by the FDA was clofarabine (Clolar, Genzyme) used for refractory pediatric acute lymphoblastic leukemia (ALL). In 2004, it was the first drug approved since 1948 when the FDA was first founded. It was original and not a spinoff of a previously approved adult drug. Prior to this, in 56 years since the creation of the FDA, only 15 adult drugs were approved for use in children. New adult cancer therapies numbered 120 during the same period. One pediatric drug stands in contrast to 120 adult drugs; this is reflective of the challenges that were faced by pediatric oncology prior to my grandson’s diagnosis of neuroblastoma.
In a 2006 article in Molecular Therapeutics*, Dr. Jessica Boklan answers the following questions, “Why has pediatric drug development been the poor stepchild of cancer drug development? What are the major challenges currently confronting pediatric cancer drug development? What are pediatric oncologists and other children’s advocates doing to overcome these barriers?”
“In general,” she continues, “the major obstacles faced by pediatric oncologists arise from the same underlying issues faced by adult oncologists but are significantly amplified in pediatrics-limited funds, limited time, limited numbers of investigators, limited numbers of patients, and limited political clout.”
Dr. Boklan noted that “cancer was the most common cause of nonviolent death for children in the United States. One in 300 children will be diagnosed with cancer by the age of 20 years. Yet, the total number of new pediatric cancer diagnoses is minuscule compared with the total number of new adult cancer diagnoses. Whereas 12,000 to 13,000 new cases of pediatric cancer are diagnosed in the United States yearly, a staggering 1,368,030 adults were diagnosed with cancer in the United States in 2004. For additional perspective, there are more cases of breast cancer diagnosed in New York State each year (15,190 in 2004) than there are new pediatric cancer diagnoses nationwide. Once pediatric cancers are broken down by individual diagnoses, their numbers relative to adult cancers become exceedingly small.” Keep in mind that 7 years have passed since Dr. Boklan made these observations and we are still quoting the same statistics that she quoted in 2006. Very little has changed in seven years!
Dr. Boklan continues, “With the average cost of research and development to bring one drug to market at $802 million and given that 1 in 1,000 new compounds that enter preclinical testing ever make it to human testing and only 1 in 5 agents that enter human trials receive FDA approval, it is little wonder that pharmaceutical companies would hesitate to invest in pediatric cancer treatments. Basic economics clearly favors investment in a treatment for 215,990 adults yearly diagnosed with breast cancer over a treatment for a mere 425 children yearly diagnosed with rhabdomyosarcoma” Again, not much has changed, and in addition, this situation also influences the future of research because bright, young laboratory investigators avoid a field of study where the chances of having research reach the patient is almost impossible. They instead choose other fields of research.
In 1997, the FDA tried incentives by offering an extra 6 months of patent protection to drug companies under certain conditions on new agents or studies requested by the FDA. The Pediatric Exclusivity Provision of the FDA Modernization Act of 1997, renewed in 2002, produced 10 adult oncology drugs suitable for use in children by 2006. The provision was renewed again in 2007 as part of the 2007 Best Pharmaceuticals for Children Act (BPCA).
That approach, though, has led to only 15 such cancer medicines being allowed for use in children since 1997. The new focus on getting adult cancer drugs to youngsters may be aided a little by legislation that took effect in January, 2013. It requires drug makers to discuss pediatric studies earlier than normal for appropriate populations of younger patients. Now, pharmaceutical companies are required by law to study their products in children if the disease exists in the pediatric population. The key here is the wording, “if the disease exists in the pediatric population.” Such cancers as neuroblastoma and other purely pediatric cancers that never appear in adults will not benefit at all under this provision.
On July 9, 2012, The Creating Hope Act was signed into law. The Hope Act works by offering vouchers as “big fat carrots” as Nancy Goodman, creator of the legislation, likes to describe them. “The vouchers offered to drug companies come with rights to a speedier FDA review of another drug they are trying to get to market, possibly a blockbuster drug, and it does not require taxpayer funding.” If a company can shave 6 months off the normal review time, it could be a huge financial gain for them. Nancy is actively working with the FDA to get the full benefit of the new incentive for drug companies.
Dr. Jessica Boklan describes the 2006 situation in this way: “A substantial segment of the U.S. population is disenfranchised because of their age. Simply stated, kids cannot vote. Without a voting block to get the attention of Washington, children must rely on others to make their case for them come budget time. Historically, pediatric cancer has not been a budget priority. For example, the total budget for National Cancer Institute (NCI) for fiscal year 2004 was $4.7 billion. Of that, only $166 million (3.5 percent) was devoted to pediatric cancers in any form, including funding for prevention, treatment, and long-term follow-up.” Today in 2013, nine full years later, the NCI budget remains at 3.6 percent for pediatric cancer, treatment, prevention and long term side effects.
Now, sequestration has taken place. The budgets of NIH and NCI already cut by 10% in 2006 have been cut another 5.1% again as of March 1, 2013. This was the sentiment expressed by Dr. Boklan in 2006 when NCI funds were cut. Does this sound familiar? “These cuts translate into fewer clinical trials and scientific inquiries for children with cancer at a time when clinical trial enrollment and resulting access to novel drugs have never been more critical. In addition to short-term issues of survival, increased capital is likewise essential to address the long-term effects faced by childhood cancer survivors from today’s toxic treatments. As these children now live into their thirties, forties, and beyond, chronic illness and disability are emerging as major challenges. Increased funding is needed both to develop less toxic treatment regimens to minimize late effects and to address those effects when they do occur.” Dr. Boklan was correct then and she effectively predicted what we are seeing today. It sounds like a broken record! That is EXACTLY what we are saying today! Nothing has changed yet.
Seven years ago, Jessica Boklan remained hopeful in her 2006 article and was very optimistic about a new bill that was introduced at about the time her article was being completed. She wrote, “Despite worsening budget constraints, there is a glimmer of hope. One encouraging new development is the Conquer Childhood Cancer Act of 2006. Introduced in March 2006 to the Senate and House of Representatives jointly by Senators Norm Coleman (R-MN) and Jack Reed (D-RI), Congresswoman Deborah Pryce (R-OH), and Congressman Michael McCaul (R-TX), the Act would authorize $100 million for over 5 years to support translational research into pediatric cancers. It is too soon to tell whether this bill will successfully pass and whether it heralds a change in priority by our government toward curing pediatric cancer.” Two years later, after a tremendous amount of work, the bill did pass with unanimous consent on the Senate floor and a vote of 416 to zero in the House of Representatives! Known as HR 1553, The Carolyn Pryce Walker Conquer Childhood Cancer Act of 2008, named after Congresswoman Deborah Pryce’s nine year old daughter who lost her battle with neuroblastoma. It was signed into law on July 29, 2008 by President George W. Bush. Before the signing, President Bush revealed that he had lost his three year old sister in 1953 to leukemia. The bill authorized even more money than Dr. Boklan saw in the original 2006 version of the bill. The newly passed version authorized $30 million annually over five years to create a population-based national childhood cancer database and to further improve public awareness and communication regarding available treatment and research for children with cancer and their families. It also required that NCI continue to enhance, expand and intensify pediatric cancer research. There were no restrictions on using some of the funding to expand clinical trials if NCI deemed it appropriate.
You may have to restrain yourself as you read on. Sadly, of $150 million approved for the bill, Congress only appropriated $5 million to the Centers for Disease Control (CDC) to carry out the law’s provisions of creating a childhood cancer registry and printing a publication for families. While the CDC never stated that it did not need further funding and would have gladly accepted any funding that Congress provided, the NCI said they already had enough money in their regular budget. The following statement was issued by the appropriations committee, “The National Cancer Institute reports that it is meeting the funding level identified for pediatric cancer research in the Caroline Pryce Walker Conquer Childhood Cancer Act of 2008 within its base budget. The conferees commend NCI for its attention to this issue.”
To clarify, NCI said, “No thanks,” to appropriations in 2009, 2010, 2011, 2012 and 2013 for an additional $30 million each year for use in pediatric cancer, the CDC never requested more funds and Congress never followed up to see if the money was being appropriated as intended in the bill.
Maybe the problem was that the name of the bill was not fashionable for the the times. The Carolyn Pryce Walker Conquer Childhood Cancer Act may have been better received if it had been called the The Carolyn Pryce Walker Bail Out Childhood Cancer Act. During this five year period, the government could not spend money fast enough. They bailed out AIG, Fanny Mae, Freddy Mac, GM, Chrysler, Citi Bank, Bank America and countless others for billions and billions of dollars!
This appears to be not just a conundrum, but what many may call a cluster conundrum!
Author: Joe Baber
Editor’s note: Since this article was first published, sequestration ended and a bill was re-introduced in Congress under the same name, but with major revisions and the amount requested was only about 1/3 the amount of the approved original bill. The new version never passed. Another bill, The Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act was passed and signed into law in 2018 after 2 years of hard work by advocates. It is for $150 million over 5 years. The first year appropriation of $30 million was also approved. The bill is intended to expand opportunities for childhood cancer research, improve efforts to identify and track childhood cancer incidences, enhance the quality of life for childhood cancer survivors, and ensure publicly accessible expanded access policies that provide hope for patients who have run out of options. Click Here for details.
*Dr. Jessica Boklan’s 2006 article, “Little patients, losing patience: pediatric cancer drug development,” may be found at http://mct.aacrjournals.org/content/5/8/1905.full
Fascinating. Disturbing. WHY WON”T MAINSTREAM MEDIA PICK THIS UP!!! Sorry – frustrated – can’t even imagine how frustrated families battling this beast must be…..thanks for reporting on this! Your posts are very informative and eye opening.
Love this article!! Not what is showcases, which is disappointing to say the least, but how. Very informative. I too hope things change soon.
There must be some magicians behind all these!!! Where is the money? Show me the money! One life is too many if that life is your child. I hope the magicians realize this. What if it is your child who will be afflicted …