03/19/2013 11:27 am ET | Updated May 19, 2013

Engaging the Pharmaceutical Industry in the Fight Against Childhood Cancer - Piece No. I

This is the first piece in a four piece series that I hope will lay out a plausible road-map for the fight against childhood cancer. Please share your ideas with me as we journey together.

The fight against childhood cancer is not possible without innovative drugs that are available for delivery without significant restrictions. This statement, given the lack of development of childhood specific cancer drugs is difficult to mesh with the current state of affairs, yet I think is completely true. There are obvious problems with respect to the overall access to these drugs both for testing in the laboratory as well as in the realm of patient availability. In addition, there is little movement in the development of childhood specific drugs. Part of these issues lie squarely with the Food and Drug Administration (FDA), or with institutional review boards who think they are protecting our children by failing to grant access. That's a discussion for down the road in this series.

We hold the pharmaceutical industry in this country in a unique place. In many instances, we deal with and approach them at arm's length and never try to engage them on the ground as advocates. This is a mistake. The childhood cancer community needs the pharmaceutical industry and the companies as partners, not as foes. First, we need to engage them to ensure that they are willing to allow compounds into the laboratory for testing. This is a significant issue in the brain tumor community in general. One of the methods researchers have for finding breakthroughs is to be provided with drugs to test. Yet, there seems to be the sense that this is not occurring at a fast enough pace or that access is not being provided in general. Part of the reason for this is that the drug manufacturers are concerned over the viability of compounds for commercial sale and use. And thus, if a drug fails in a research setting, that could suggest that the drug will not be viable in patient trials. Therefore, manufacturers are afraid to allow further testing and release compounds. This is completely understandable. For profit companies are entitled to continue to pursue business models that generate profits. Given this conundrum, we must discuss this lack of access amid the fear that failure will negatively impact the bottom line of the company. In addition, as suggested below, we must try and partner funding with drug manufacturers to obtain the compounds for testing in labs.

It is costly to bring a drug to the market. This cost, given the perception that a low patient population does not equate to a profit model is one of the largest stumbling blocks for the development and marketing of new childhood cancer specific treatments. Therefore, the childhood cancer community must demonstrate to the pharmaceutical companies that creation of child specific drugs can be used for upstream profits in the more populated adult cancer community, as well as other disease specific communities. Because childhood cancers do act differently than most adult cancers, and children are sometimes better able to withstand the toxicities and rigors of treatments, there is the theory that focusing on children could create greater numbers of efficacious drugs for adults. Therefore, patience in the creation phase of new drugs could pay off in the end for greater profitability. Children will always get the "hand me downs" but if we can demonstrate success in the creation of drugs that help children but also help larger adult populations, we can provide adults with "hand me ups." Of course, this involves drug manufacturers taking a certain leap of faith, but it also embraces the theory that there are greater rewards through greater risks.

There have been several attempts to legislatively impact this lack of drug development and access. The most recent is the Creating Hope Act. This act, that Nancy Goodman and many in the childhood cancer community worked so hard to obtain passage of, creates incentives for drug manufacturers to develop new compounds for childhood specific diseases, including cancer. It is a milestone in the legislative landscape and should prove to be a significant tool for the advancement of our cause. Unfortunately, since its passage, I am unaware of a single drug manufacturer taking advantage of this legislation despite the ability to utilize it to increase profits by obtaining favorable fast-track review for another compound. We in the community must find ways to push the pharmaceutical companies to utilize this tool. Its existence simply is not sufficient. We must seek to meet with the executives of these companies and engage them in a truly personal manner. This has worked in the past for various compounds including one called Xerecept, a steroid alternative that was withheld from pediatric trials until the community confronted the executives of the manufacturer with evidence of the horrors of steroid use in children with cancer. Ultimately, with the help of the FDA, the compound was placed in childhood trials and it is proving effective. We must attempt to open the doors and discuss the landscape and the incentives. These discussions necessarily should include the ultimate discussion of profits from the creation of childhood specific drugs.

Finally, we must approach and treat the drug manufacturers as partners. This can be done by trying to join forces with private monetary interests to help ensure that clinical trials do not losing funding, and, more importantly, matching researchers with compounds and then supporting those research trials by removing the fears that the repercussions of failure bring. By combining forces, we can create a lasting partnership that keeps in mind the objectives of both the childhood cancer community as well as the drug manufacturers.

This is a long and evolving issue that is not going to change overnight. For those kids fighting cancer at the present time, and children like my daughter Alexis who lost her battle, this portion of the road map comes too little too late. For many, that will not be palatable. I completely understand and frankly I do not disagree. However, if the childhood cancer community is going to be the tip of the spear that thrusts itself broadside into the issue of drug development and access, then we must exercise a mixture of steadfast resolve and patience at the same time. Opening the doors is not an easy task, and, in all honesty I do not have all the answers. I am fortunate to have this platform. I simply know that at some point, the door will open and we must be prepared to walk through with the ideas. Altruism and profitability are not that far removed from one another.

The collective voice of the community is rising. I have witnessed this happen over the last number of years. It is through the motivation and singular conviction of all of these amazing parents and children that the balance is shifting. I just do not know whether the drug manufacturers have heard our voices enough.

To be continued in Part II of IV.....