The Compassionate Use Conundrum

In the wake of the amazing story of Josh Hardy, I have been personally contacted by three childhood cancer families desperate to save their child from an almost certain death at the hands of childhood cancer. What happened with Josh Hardy and the amazing social media and advocacy campaign that ultimately worked to save his life has provided hope to other parents that their child can also obtain drugs on a "compassionate use" basis. Behind the scenes, many unsung childhood cancer advocates worked tirelessly in an effort to obtain the drug that Josh's doctors knew would save his life. In the end, this is a story of happiness for the Hardy's and one of heartbreak for so many others.

The problem is that the compassionate use program fails to uniformly provide access for people to drugs that may show signs of efficacy. This case has deeply divided many over the role of advocacy in connection with obtaining drugs through the compassionate use program. I understand the arguments being levied against the widespread grant of compassionate use. The arguments are founded upon scientific, sociologic and economic underpinnings. With that stated, I have also sat in the same place as the Hardy's, and unfortunately for my family, the outcome was not so happy.

My daughter Alexis was diagnosed with an inoperable brain tumor known as DIPG in 2008 and outlived the initial prognosis by close to two years. During the course of her fight, my wife and I, with Alexis in tow, travelled to New York City to have Alexis enroll in a clinical trial. The hope was to begin treatment with a drug that two of the world's top neuro oncologists believed could have some efficacy given her clinical and MRI presentation. After an entire day of tests, the primary investigator, who was not an expert in the field of DIPG, read Alexis' MRI and concluded that she was not eligible. Unfortunately, her reading failed to understand how DIPG, and gliomas in general, present in MRI scans. Alexis was denied enrollment based upon what I believe was the investigator's lack of understanding of DIPG. Unfortunately, we will never know what result this drug may have provided. Due to the quickly progressing nature of DIPG, we had to move swiftly to find an immediate trial and thus did not request compassionate use.

This brings me to the current compassionate use climate. The system disincentives drug manufacturers from agreeing to provide drugs outside of clinical trials. With concerns about the potential for a negative occurrence or toxic impact effecting the ultimate path to FDA approval, drug manufacturers who see the finish line in the clinical trial process often make the decision that they will not provide treatments outside of trials. Given the fact that enrollment numbers for clinical trials are at dismal rates, ultimately not many people are available to gather the proper testing data to begin with.

The good news is that cancer researchers are identifying more genetic targets to focus upon for treatment pathways. These discoveries have allowed researchers to identify common pathways between cancers that were otherwise thought to be genetically dissimilar. The bad news is that in many instances, the drugs that show promise for these newly found targets are not available for that form of cancer treatment. And that brings us full circle. Science is far outpacing the regulatory framework and availability of drugs for children and rare diseases in general.

One legislative approach is HR 2090, The Patient Choice Act. Introduced in 2013 by Morgan Griffith (R-VA), this act would allow drug companies to seek provisional approval of therapies in clinical trial testing that are "adequately safe." Thus, patients facing terminal and life-threatening illnesses with limited treatment options would gain the choice to try a drug that could in fact have some efficacy. The drug manufacturer would be able to monetize this provisional approval thus deferring some of the cost to maintain the clinical trial and bring the drug to market. In the end, the patient has the choice to try a drug that may in fact save or prolong their life; smaller drug manufacturers have the opportunity to compete with larger companies here in the states without having to fully rely upon government grants or venture capital funding. And the process does not impact upon the overall framework of bringing the drug to full approval by the FDA.

I believe that we need to go one step further with respect to children. I believe that regulatory intervention, in the form of legislation, needs to be introduced that guarantees children access to drugs that are not being made available for the treatment of identified genetic targets. Funding through some source (a common fund perhaps) can be made available to defer the cost to the manufacturer. In the childhood cancer world, only one new drug has been specifically approved in the last twenty years. With little focused development of childhood specific treatments for a wide array of diseases, it is children who often suffer the most when facing life-threatening illnesses. Children are being treated with "trickle-down" therapies that in many instances are significantly outdated, have no identified genetic target, and are extremely toxic. Given the current regulatory status, the line of Josh Hardy's will continue to grow taxing the system beyond repair.