This is part 2 of the Jonathan Agin series, “Childhood Cancer – My Road Map”

Being sent to an emergency room to have a CT scan performed on your two-year-old child is frightening. Being brought into a small cramped room on a pediatric oncology ward and told your child has six to nine months to live is life-altering and devastating. Finding out that there are no viable treatments for your child and then learning about the process of engaging in a clinical trial is overwhelming. Welcome to the world of childhood cancer.

We were officially given our “welcome” on April 10, 2008. It happens to other families each and every day in this country and all across the planet. What I learned during the long 33-month journey that Alexis spent in the world of childhood cancer is that oftentimes, our children are not provided with access to drugs and treatments, and development of compounds are stifled in the name of protecting the patient. As the incidence rate of childhood cancer continues to rise and childhood cancer maintains the unenviable position of being the number one disease killer of children in this country, I believe that there must be change on all levels, and the Food and Drug Administration (FDA) is one of the entities that we must engage to alter the landscape for the better. There are some ways that we as a community can engage the FDA in this dialogue.

First, the FDA has a patient advocate program that allows for the engagement of information exchange between patients and the FDA. Through this Patient Representative Program, the individual can share with the FDA information regarding the issues that face either themselves or their family members that are impacted by a life-threatening illness. This is a great way to be able to communicate — at a grassroots level — the concerns that impact the childhood cancer community, and disease specific sub-types. As our children cannot be a voice for themselves, it is our responsibility to fill that role. The Patient Representative also serves on committees that review products and therapies for treatment. Patient Representatives must be nominated to the FDA. I personally am not aware whether we currently have members of the childhood cancer community serving in this role. There may in fact be some individuals who represent our interests at this level. This path presents a chance to engage with more people in this role and provide the message that our needs in the community are not being met. The more people who are out there engaging at this level, the more our message gets shared with those who can effectuate change. I believe we must place more advocates in this role to ensure that the communities needs are being understood and communicated.

Another concern for the childhood cancer community is the access to drugs on a compassionate use basis. Compassionate use means being provided an investigational drug outside of a trial. The process is complicated and oftentimes ends tragically, as the child is either not provided the drug or the approval comes too late in the battle. To obtain compassionate use, the patient and the doctor have to apply for the drug and arrangements need to be made with the manufacturer for approval. Ultimately, it is the FDA who grants or withholds access. Herein lies the problem: The ordeal takes too long, and oftentimes the drug manufacturer is not willing to engage in the process. I believe that the FDA needs to have a fast-track approval system with clear guidelines for any application for compassionate use. They may in fact believe this exists; however I can attest that on the practical level it does not. In this regard, patients should know of a clear and quick path to be able to obtain drugs that may in fact alter the outcome of their prognosis. Of course, this is all controlled pursuant to federal regulations which are often complicated and difficult to navigate. I understand the overall need for protections to be in place. With that said, I do not believe that any parent whose child is faced with a life-threatening illness would choose a path that they know would harm their child with little to no efficacy. Rather, as we did with Alexis, we chose to balance her treatment protocols to ensure that her quality of life was as high as possible under the circumstances.

Unfortunately, there were several drug compounds that we knew were available in clinical trials that Alexis was not eligible to receive for one reason or another. In discussing this with her doctors, there was often reluctance to even try to obtain compassionate use for these drugs due to the process involved and the potential risks. For those of us in the childhood cancer community who are provided with little to no hope, we must be granted every opportunity to try and save our child. The regulatory scheme in place at the present time is not assisting our children in fighting their battles. (Try understanding these regulations.) Accordingly, the community needs to engage with members of congress and the FDA to alter the regulatory scheme to make this option more viable. This is obviously not an easy task, I understand.

Finally, I believe that we must act to partner with the FDA and work with them to connect with the pharmaceutical industry to ensure that the childhood cancer community is not overlooked for drug development and clinical trial availability. This may be the hardest of the three avenues for change, but it may prove to be the most lucrative for our community. The FDA and pharmaceutical industry coexist in a space of necessary involvement. The process of drug development is long and costly, and the FDA exists to ensure that the final product is safe for the consumer population. Fair enough. One of the main problems in this equation, and this goes back to my last piece, is that the pharmaceutical industry oftentimes does not engage the pediatric population for fear of failure and lack of profits.

The problem with changing this conundrum has been addressed by Congress on several occasions. It is quite complicated and difficult in practice, and it does not appear to be utilized with much practical success. It exists though, and as a patient group we must band together to engage both sides, the FDA and the pharmaceutical industry, to utilize the existing pathways for both drug use and drug development. In many instances, it starts with us as advocates educating our physicians on these pathways and expanding the overall knowledge base of these channels. This is often difficult for a parent who is in the throes of the battle trying to balance saving their child with making every moment count. The reluctance to push the envelope is problematic given the continued lack of success in many forms of childhood cancer. It is yet another burden that we must shoulder so that those children battling today and those who will come tomorrow are better armed to battle this indiscriminate killer.

Changing the way that the FDA operates in relation to childhood cancer is probably one of the most difficult of all of my proposed routes on the road map. The complexities involved with the regulatory framework are significant. If we focus on only one of the three ideas above, creating a larger group of Patient Representatives who are singularly focused upon childhood cancer, I think we can begin to have our voices heard behind the doors of the agency with more frequency. Where that takes us, I do not know. I do know that we have to start somewhere in this David vs. Goliath battle that we find ourselves embroiled within.

Ultimately, I do not regret one decision my wife and I made with respect to Alexis’ care and treatment. Although the cancer Alexis was diagnosed with was an absolute terminal prognosis, we were fortunate to have the opportunities to research and make the best decisions possible. With that said, there were opportunities that were unavailable as a result of the FDA, the pharmaceutical industry and regulatory scheme, that could have benefited Alexis. For these reasons, I seek change.

Author: Jonathan Agin    

Additional articles of interest on this subject are also available on Four Square Clobbers Cancer:

“We need more than just research.”  This is a story of one father’s struggle to get a “compassionate use” designation for a drug to be used on his son with terminal cancer.

“Cancer Drugs, The Valley of Death,” An overview of FDA drug development in relation to childhood cancer drugs. The costs, and associated pitfalls for development of pediatric cancer and other rare disease drugs are discussed in detail.

Click Here for Part I of this series

Click Here for Part III of this series

Click Here for Part IV of this series

Part 4 will be available soon, stay tuned!