In July, 2012, President Obama signed into law the Creating Hope Act for the purpose of incentivizing pharmaceutical companies, biotech firms and universities in seeking cures for what are known as Rare Children’s Diseases. Childhood Cancer is considered such a Rare Children’s Disease. Companies that are successful receiving approval from the FDA of drugs that meet the criteria of the Act receive vouchers that can be valuable to the companies in their efforts to bring other drugs to the market or, if they wish, can result in the sales of the vouchers by the companies to other companies at substantial prices. The passage of the Act was supported by The Congressional Caucus on Pediatric Cancer formed in 2009 by Michael McCaul, Congressman from Texas.
The Caucus’ Summit on Capitol Hill in September, 2011, was devoted to discussions regarding the Act, seeking support from legislators, industry, healthcare providers, pediatric cancer advocates and others. Among the speakers at the September, 2011, Caucus was 8-year old, Max Plotkin, whose cancer was then in remission and who addressed the audience of over 500 persons, telling them what it was like being diagnosed at age 4 with cancer. He told of having to endure two years of chemotherapy and having to experience all the unpleasant side-effects from the cancer and the treatments to cure the cancer, including his “daddy shaving his own head so that he, like Max, would be bald.” Max received a standing ovation from the audience when he concluded his remarks. Following the September Summit, the pediatric cancer community was active in seeking support from Congress. The pediatric cancer advocates in the forefront of seeking such support included Max Cure Foundation’s Richard Plotkin, Vice Chairman, and Jonathan Agin, Director, External Affairs.
The story of the creation and approval of new drugs for children with cancer in the United States has simply been one written on primarily blank pages. Until March 10, 2015, only two new childhood cancer specific drugs had received Food and Drug Administration (“FDA”) approval in the last twenty years. Step back from that statement and the number and just consider that fact for a moment. According to the FDA’s own report, in 2014 alone, forty-one (41) new drugs for all different indications gained approval. Seven of the drugs listed are specifically oncology drugs for various cancer indications. This is simply from 2014. (Click here for an interesting historical analysis of the total number of drugs approved in the history of the FDA). Yet, in comparison, twenty years ticked by with only two drugs given FDA approval for a childhood cancer indication.
Significant work is occurring behind the scenes engaging with members of Congress, the FDA, the National Institutes of Health (“NIH”) and other regulatory agencies as well as private industry and interest groups, in an effort to alter the dismal statistics that continue to ensure that children with cancer obtain trickle down drugs or outdated and toxic treatment modalities. So, when the announcement hit the newswires on March 10, 2015 that a new drug aimed at high-risk neuroblastoma received FDA approval, there was cause for a little sigh of relief that current efforts were showing progress.
Thanks to the Rare Disease Priority Review Voucher Program, more commonly referred to as the Creating Hope Act, which was championed by Kids v. Cancer’s Founder and Executive Director Nancy Goodman, United Therapeutics was awarded a priority review voucher for Unituxin, an antibody drug that is used in combination with surgery, chemotherapy and radiation. Clinical trials have demonstrated that there is a positive response for children with high-risk neuroblastoma who have added this drug to their treatment regimen. As a result of the use of this drug, the overall survival rate for children after three years in these clinical trials increased by approximately fifteen percent. Of course the drug is not without side effects, but we have to take this one step at a time.
Legislatively, the Creating Hope Act provides financial incentives for pharmaceutical manufacturers to develop new drugs for rare or orphan disease indications. The sponsor of the treatment must request that the FDA label the specific disease for which they are seeking approval for the drug or biologic as a “Rare Pediatric Disease” and then the sponsor may request that the drug or biologic agent be designated a “Rare Disease Product Disease Application.” If the FDA chooses to make such designations, and the drug or biologic is approved, the sponsor obtains a priority review voucher. The sponsor obtaining the priority review voucher would then utilize it to obtain faster review of a drug or biologic that ostensibly has a greater commercialized potential. The sponsor who obtains the voucher may also transfer the voucher to another sponsor or drug company that intends to utilize it for priority review for a drug or biologic by the FDA. The voucher ultimately makes the overall process of submission and approval to the FDA quicker with the ultimate hope of getting the drug to market faster.
Some drug manufacturers may come along for the ride willingly, and some may have to be provided with large incentives to sweeten the pot. Still others may have to be shepherded to the end goal by the front end of a sharp stick. Children with cancer simply deserve to have more treatment options that provide actual cures while decreasing the long-term side effects and impacts from those currently being utilized. It is a circular issue that needs to be addressed in as safely and quickly a manner as possible.
In the end, it is innovation in approach, thought process and pursuit that will lead to the increased number of pediatric drug approvals for oncology drugs. Organizations like the Max Cure Foundation are right there in the mix, utilizing advocacy skills in collaboration with so many others in an effort to enact positive change. The theory of “trickle down treatments” has not warranted cause for excitement, nor has it amounted to new cures. Rather, there is still a waiting game underway by so many parents who struggle with the ticking clock of their child’s diagnosis. For my wife and I, this ticking clock was set between nine months and a year for our daughter Alexis. Almost eight years ago to the day that I am writing this piece, April 11, 2008, we were told that Alexis had DIPG, an inoperable and incurable pediatric brain tumor. There were no curative treatments and all available clinical trials at the time failed miserably. Alexis battled for a long thirty-three months and during that entire time we hoped and prayed for some breakthrough treatment to come along. Be it a new discovery or some drug that was used for an adult indication trickling down to her, we lived with this optimism. Our hopes evaporated completely on January 11, 2011 when we watched Alexis take her last breath on earth.
As genetic sequencing for the analysis and understanding of the underlying mutations and drivers for many types of childhood cancer becomes more the norm, the hope is that many of the existing drugs and treatments available in the adult oncology world will show efficacy for children with cancer that possess the same mutation or genetic markers. The issues related to the access of those specific drugs for pediatric preclinical testing and ultimately clinical availability are currently the subject of legislative scrutiny and possible fixes. This is the topic for another day though. The point being, progress is not always linear. Rather, sometimes it takes the motivated and collective efforts of parents, grandparents and caregivers who have walked through the horrors of their child or grandchild’s cancer diagnosis, or worse loss, to advocate for change that in a perfect world would occur as a matter of course. We all know that there is no such thing as a perfect world, so sometimes you simply have to Create Hope in other ways. The childhood cancer community can now say that in the last twenty-years, three new childhood cancer specific drugs have been approved by the FDA. We can all agree that this fact is nowhere close to being sufficient enough, but three is better than two.
Author: Jonathan Agin, Director, Executive Director
Follow Jonathan on Twitter @jonathanagin