Compassionate Use | Who Decides When its a Child’s Life?
Each year on the Jewish High Holy day of Rosh Hashanah, which is the beginning of the Jewish New Year, I read a prayer in Temple that states that on that day, it is written in the book of life who in the succeeding year will live and who will die. That phrase has taken on a less abstract and more secular meaning as I venture into the world of childhood cancers and as I do so, am invited occasionally to take an active role in which children with cancer would live and which would die. These were issues foreign to me over my 40 year career as a litigation lawyer for a large regional law firm in the Northeast, including a period of five years in the late 1970’s into the early 1980’s. During that period I represented one of the target defendants in what has come to be known as the Asbestos Litigation. My firm was retained to manage 54 law firms in 48 States, plus the District of Columbia. I was the representative of the firm who travelled the country, working with local trial counsel in an effort to defeat the claims of those claiming to be sick, some with cancer, as a result of exposure to the products containing asbestos manufactured by my client. And then in 2007, my grandson, Max, was afflicted at age 4 with cancer, a rare form of B Cell Lymphoma in the bone. Max is now 11 years old, being considered a 7-year survivor of this potentially deadly disease. He is looking forward to a long and healthy life. I, in turn, retired from my law firm as of December 31, 2008, and with Max’s parents formed The Max Cure Foundation.
Others have commented on the irony that as an attorney I fought against folks with cancer seeking to blame my client for their contracting the disease and now, in my retirement from the practice of law, I have taken on the mission of advocating for children with cancer. The mission of Max Cure Foundation, when formed, was to fund research. We soon added family assistance to our mission, namely, assisting low income and military families battling cancer in their children, and then in 2011, added advocacy as one of the goals of the Foundation. As a former trial lawyer, I have taken the lead on behalf of Max Cure Foundation in advocating for children with cancer and attempting to raise awareness to the needs of the most innocent among us, our children with cancer.
I had not heard the term “Compassionate Use” until earlier this year. I was asked by a parent if I could identify several childhood cancer doctors from the hospital that treated Max who would support their request for a Compassionate Use Waiver from a specific drug company. Their son, Zachary, age 11, had a rare form of cancer considered to be terminal. They were told by a noted scientist/pediatric cancer researcher that he believed an experimental drug (one that had not yet received approval to be marketed by the Federal Drug Administration [FDA]) when combined with two other drugs on the market, could assist Zachary and potentially cure him. I knew my former law firm represented the drug company and made some phone calls that resulted in the immediate granting by that drug company of permission to combine its experimental drug with the two drugs on the market. To get the drug to Zachary, however, required the issuance by the FDA of what I was told was a Compassionate Use Waiver. The FDA immediately issued such a waiver and Zach received the drug. I then received a phone call congratulating me in obtaining the first Compassionate Use Waiver for a combination of drugs for a pediatric cancer patient. My response was that all I did was make a few phone calls but thanked the person for calling that to my attention.
Then in early March, 2014, I was asked by the President of the Coalition Against Childhood Cancer (CAC2) to be part of the group that would advocate for Josh Hardy, a 7 year old boy from Virginia who had fought Kidney Cancer his entire life and in January, 2014, had a bone marrow transplant at St. Jude in Tennessee. Josh, with his immune system totally compromised, was diagnosed in mid-February with a virus that threatened his life. St. Jude knew of an experimental drug manufactured by a bio-tech company in North Carolina known as Chimerix. St. Jude had participated in a clinical trial of that drug (Brincinofir) and believed it would save Josh’s life. Chimerix, despite repeated requests by St. Jude and by the family, refused to make the drug available to Josh, stating it was involved in phase 3 clinical trials, had discontinued in 2012 consenting to give the drug to those that asked and was devoting all of its resources to bringing the drug to market. From 2012 through March, 2014, when CAC2 got involved, it denied 300 requests for the drug. The FDA would not consider granting a Compassionate Use Waiver if the drug manufacturer did not ask it to do so and hence, when CAC2 was contacted, Josh was given by St. Jude one week to live. The President of CAC2, as noted, immediately asked me to get involved. Through the Max Cure Foundation social media network, which alone reached 1.5 million persons advocating for Josh, and with the assistance of others working behind the scenes, Chimerix, on March 12th , gave the drug to Josh as part of a newly formed phase 3 clinical trial for 20 children (Josh was the first patient) established by Chimerix with the assistance of the FDA. Josh’s virus went away shortly after taking the drug. He is now at home in Virginia with his family having survived due to the issuance to him of the experimental drug. On the same day that I was told Josh would receive the drug, I was also told that Zachary, identified in the prior paragraph, had died. This demonstrates that although there exists a reasonable basis for the giving of the experimental drugs to patients who would ordinarily be terminal, the results are not always what we would want.
Following the media coverage resulting from the Josh Hardy experience, the Max Cure Foundation had been asked to assist other children with cancer who had not responded to standard treatments to obtain experimental drugs, first assisting in obtaining the consent of the drug manufacturer and then if that were obtained, seeking a Compassionate Use Waiver from the FDA. Over the four month period since Josh was given the drug I had turned down requests to get involved for children who were dying of cancer. In each instance I refused the request because their doctors did not conclude in writing that there was a reasonable basis the particular experimental drug sought would assist the child. I also as a layman concluded after reviewing information regarding the child’s condition that the condition was terminal and that death was inevitable. It was heart wrenching to me to turn down the requests to help save their children’s lives but as I indicated, I had to maintain our credibility and only “fight” for children who I conclude, after some investigation, would benefit from the drug.
Recently I was told of the plight of Nathalie Traller, a 15 year old from Portland Oregon who had a rare form of cancer known as ASPS. Nathalie needed an experimental drug to survive. I was told by Nathalie’s parents that time was of the essence. Following my investigation, I concluded, with the input of Nathalie’s treating doctors and others whose opinion I rely on for such things, that there was a reasonable scientific basis to conclude that one of two drugs manufactured by three pharmaceutical companies could help Nathalie defeat her cancer. The first order of business is to get one of the drug companies to consent to give the drug to Nathalie and then to obtain, based on that consent, a Compassionate Use Waiver from the FDA. Or, alternatively, because the drugs are in clinical trials, to try to get Nathalie into one of those trials (which currently are for adults only and which have an age limitation, which appears to be arbitrary, of 18 years). Clinical trials for these two drugs for children may be had in the future – too late for Nathalie. CNBC, through its investigative reporter, Meg Tirrell, throughout the day on August 5th ran segments on the issues that arise, and the obstacles that exist, when one seeks a Compassionate Use Waiver (after, in the first instance, seeking the consent of the manufacturer of the experimental drug, a condition to the Compassionate Use Waiver being granted).
One of the obstacles that confront industry in the first instance is that it is being asked to decide, “Who shall live and who shall die.” There are many considerations that go into that decision even after receiving information from the treating doctors that the experimental drug would help the person (and in our case, the child). Those considerations include the resources involved. Insurance does not cover experimental drugs nor will the government through Medicaid or other sources fund the treatment of the patient who may be given the experimental drug. So, it is left to the family and/or to the manufacturer to bear the cost. The government does fund, at least in part, some clinical trials but I am here focusing on the receipt of an experimental drug through a Compassionate Use Waiver. Also, if the person does not respond positively to the drug, the issue exists as to how that would impact on the ultimate approval to market the drug. Further, industry is concerned that if it gives the drug to one patient, it would set a precedent that would potentially open the floodgates to others seeking the drug under a Compassionate Use Waiver.
The former President of Chimerix, in connection with its giving the drug to Josh Hardy and saving the life of this 7-year old boy, told me that Chimerix was pressured into giving the drug to Josh through the social media campaign orchestrated by Max Cure Foundation and others. The “Josh Hardy matter”, as it has become known, has caused a great deal of soul searching among the government, industry and patient advocates as to how to treat requests for Compassionate Use Waivers, including consideration of the ethical issues confronting primarily industry when companies are asked to give an experimental drug to a dying patient. When I attend High Holy Day services next month on Rosh Hashanah and read the prayer that tells us that it is written on that day “who shall live and who shall die”, it will take on new meaning to me.
Having said the above, it is my goal for now to be part of the effort to save the life of Nathalie Traller and to attempt to convince one of the three drug companies that Nathalie’s life is worth saving and that there is a reasonable scientific basis to conclude that they can play a significant role in that effort – they can become the hero that Chimerix became for Josh, with the input of the FDA.