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savejosh (3)I have seen the good and the bad in the entire debate and process of the issue of compassionate use, or expanded access. Many families who have children diagnosed with cancer without accepted protocols at one point or another find a drug that they believe would be of benefit to their child. It begins from desperation. A parent facing the unthinkable: the loss of their child. Countless hours spent on the internet looking at available clinical trials, complimentary care, new investigational drugs and snake oil alike. It becomes a search borne from despair in a valiant attempt to save their child. It sometimes comes to a point where, together with the child’s treating physician, the complex process of seeking to obtain a drug via compassionate use is seen as the only option. Those of you reading this who have followed the work done through the Max Cure Foundation know this scenario all too well as it played out in a very public manner through the Josh Hardy situation. Max Cure Foundation Vice-Chairman Richard Plotkin has made this issue one of his raison d’etre. He successfully spearheaded the Josh Hardy situation that ultimately saved Josh’s life.

When my wife and I were riding the waves of the childhood cancer boat trying to find a treatment, any treatment, that may have any type of efficacy on the inoperable brain tumor (DIPG) our daughter Alexis battled, we often thought about seeking this outlet to obtain a drug. There was even a time when we anecdotally heard about a drug that was being manufactured by a small pharmaceutical company that people believed could allegedly cure DIPG. The drug was undergoing preclinical testing and was years from ever seeing a clinic, and then probably years from that point from ever seeing children in a clinic. The speculation and sparse information did not stop many of us in the DIPG community from seeking information. I began thinking of ways to get into the company and get my hands on the drug. How could I not be willing to risk everything to save my daughter?

Alexis Jonathans daughterJPGThe truth is that I do not know what became of this “promising” drug. Talk of it stopped at some point. I never broke into a pharmaceutical lab, and my wife and I never submitted any requests for compassionate use waivers for any drugs. Alexis lived with DIPG following her diagnosis for a long thirty-three months. For most of that time she had an amazing quality of life. She was on a total of four drug related clinical trials throughout the course of her journey and she graced us with her strength, wisdom and inspiration here on earth until January 14, 2011.

The problems and perils with the current compassionate use framework are significant on both sides of the equation. In the triangular systemic relationship that exists between the treating physician, the Federal Drug Administration (FDA) and the drug manufacturer, there is significant built up animosity as well as inherent risk involved. In the normal course of the exercise, once the treating physician makes the request, the FDA has to approve the appeal and then the ball rests firmly within the court of the drug developer as to whether they will provide the drug. It is at this stage that most frequently there is more reticence in the process.

Looking at it from the perspective of the drug manufacturer, they have invested literally hundreds of millions of dollars, if not billions to try and bring a new drug to market. The pressure to protect this investment and the momentum through the clinical trial system is immense. The entire system to bring a drug to market could take upwards of ten years while the drug goes through the various phases of clinical administration and study.

The risk for the drug manufacturer arises if they do in fact provide the experimental drug to a child on a compassionate use waiver and there is an adverse occurrence, or even worse, death. Any such impact could derail the process of commercialization of the drug and thus impact investors, cost the company significantly more time and money or shut the process down altogether and the drug never sees the clinic. Those are risks that are very real and difficult to surmount when you are a parent desperate to try a treatment. These risks continue to cause a lack of compassionate use provision for childhood cancer indications as well as drug development in the pediatric oncology space. It is not as simple as an impassioned plea from a desperate parent. Rather, it is a delicate balance between such elements as business, compassion and regulatory intricacies.

thinking-outside-the-boxThere are a number of new legislative attempts aimed at changing or correcting some of the issues highlighted herein. I do not intend to indulge upon a legislative survey in this piece. That may form the basis of another post soon to come. With that said, the point I am trying to make is to provide perspective on this issue in an effort to arm stakeholders with a greater understanding of the pressure points and concerns of all involved. Perspective will allow everyone in the debate the ability to effectively engage in a dialogue that ultimately leads to a permanent fix. In order to arrive at a better solution and more predictable outcome, the stakeholders have to agree upon a middle ground that protects as many of the overall interests as possible while also providing desperate parents the opportunity to utilize experimental drugs.

A parent facing the loss of their child will seek any avenue available to save their child. Sometimes we as parents think about avenues that are not necessarily available or above board perhaps. It is borne from love; it is borne from desperation. These are difficult issues and considerations that continue to test everyone involved in the debate.

Author: Jonathan Agin, Director, External Affairs
Follow Jonathan on Twitter @jonathanagin