The United States House of Representatives is about to take a vote today (March 13, 2018) on a piece of legislation that is known as “The Right to Try Act of 2017.” HR 878 will come to the floor for a vote at approximately 6:30 pm. Over the summer the Senate passed S. 204 which, by the House moving on the legislation which is likely to pass tonight, will then be ripe for the Senate to once again move the legislation forward for signature by the White House and thus Right to Try will be codified on the federal level in the United States.
As a father who lost his first born child to a type of pediatric brain cancer with no known or effective therapy, I have come full circle in my thinking on Right to Try laws. As I have devoted my life personally and professionally to helping children with cancer and the families that stand so close to the suffering, I have helped several obtain drugs and treatments that have not yet been FDA approved. I believe that those families had a right to seek access to those drugs.
But that is rather different than having an almost unfettered right to access experimental drugs in the manner in which the Right to Try legislation provides. Those who misunderstand the collateral damage that is about to occur following the passage of this legislation, I believe, are setting pediatric cancer drug development further back, in terms of years, and we already have a pretty dismal record thus far.
With only 3 pediatric cancer specific drugs being given Food and Drug Administration (FDA) approval over the course of the last 20+ years, for those of us in the community it is essential that we try to fully understand just how detrimental the Right to Try is to this process.
Too Good to be True
Right to Try laws sprung up on the state level (38 states now have right to try laws) and became a pet project of the current administration through the Vice President. The federal legislation, in essence, is floated upon the air of a “feel good” story that lacks sufficient oxygen to allow it to breathe on its own. On its face, the federal legislation makes all of us feel like there is hope. The fiction told to patients by the purveyors of this legislation is that people with life-threatening illnesses should be given access to any drug that they want to try in an effort to “save their life” without the FDA delaying this process. Those who push this legislation, including the Koch Brothers who have utilized their billions of dollars to influence politics in the United States and the Goldwater Institute, market this legislation as a fix to the regulatory impediment that dooms terminally ill people to death. As the rhetoric goes, they are going to die anyway, why not give them access to something that may help. And thus, people without an understanding of the mechanism by which a drug undergoes FDA approval and safety testing, hear that dying people are being precluded from access to life saving drugs because of the government and the logical emotional path is to then codify legislation that grants unfettered access. As with many stories though, they are often too good to be true. And more importantly, for purposes of children with cancer, the Right to Try legislation on the federal level will likely do more to keep promising drugs out of the clinical pipeline than it will do to provide kids access to the drugs.
Expanded Access / Compassionate Use
There already exists a viable (not perfect) mechanism for patients to access drugs that are not FDA approved. Expanded access, or “compassionate use” to which it is often referred, is a process by which a patient, through their treating physician, can file an application with the FDA to approach the drug manufacturer to obtain the drug. The FDA approves approximately 99% of all requests under this system. In fact, FDA physicians are available 24 hours a day to approve requests. Yet, those pushing the Right to Try legislation foment a climate of misinformation by perpetuating a myth that the FDA is the impediment to access. Rather, the FDA, in considering these applications, will actively participate when able, to provide some guidance to clinicians in the administration of the drug being sought. In addition, the FDA seeks to maintain safety and efficacy data on the drug that is administered through this mechanism. Despite this working solution, the Right to Try legislation will fast approach the House floor and likely sail through.
Insurers Can Opt Out of Paying
Several aspects of the Right to Try legislation about to hit the House floor are significantly troubling. The legislation allows insurers to opt out of paying for experimental drugs that a patient obtains through Right to Try. Many will argue that insurance carriers currently fail to approve coverage for many treatments and diagnostic tests patients seek, so this is not a new issue. Unfortunately, desperate patients seeking to obtain drugs that are not covered by insurance will likely spend small fortunes, when able, seeking treatments that in the end have no hope of working. This slate of laws creates the belief that effective treatments exist and due to regulatory controls patients are not given access.
Removal of Legal Redress
Right to Try additionally removes the legal redress an individual may have, should they be the victim of malpractice or should the person’s death be directly related to the drug. Under the current FDA regulations, patients can sue clinicians for malpractice and file claims against drug manufacturers. Institutional review boards that seek to maintain safety standards in connection with drug administration monitor cases filed by patients under the current framework. This is an added layer of safety in the process. The House version of the Right to Try legislation essentially insulates everyone in the process from legal liability, which on its face may seem positive but in reality it allows for more unscrupulous operation and drug administration to patients desperate for any hope.
No More Reporting of Patient Outcomes
Additionally, under the Right to Try legislation being taken up by the House, the FDA is barred from reporting patient outcomes from the use of experimental drugs. What this means, in a chilling twist of misunderstood zealotry, is that if a patient suffers a serious adverse reaction, including death, the FDA cannot utilize this information in its consideration of whether or not to approve the drug. Removal of this critical FDA reporting is chilling to those seeking treatments that are in late phase clinical trial testing and development.
Lack of Motivation for Drug Manufacturers
One major point that should be highlighted, regardless of any of the emotion and smoke surrounding this legislation, is that there is no compulsion for the drug manufacturer to release the drug or treatment pursuant to the Right to Try legislation. Ultimately, I simply cannot ever imagine a time when Congress would be in a position to compel drug manufacturers to release drugs to any patient with a life-threatening disease or that drug manufacturers would allow this to happen. (The House bill narrows the eligibility criteria from the Senate bill. Per the House bill, patients eligible to seek drugs pursuant to Right to Try will be limited to anyone with a reasonable likelihood of death). Ultimately, the reality is that the Right to Try legislation does nothing to provide patients access to drugs as it is devoid of any real teeth. The reality is that there is a lack of evidence from the 38 states maintaining right to try laws to suggest that they are effective or ironically that anyone that has obtained a treatment in this manner would not have been able to access the drug through the existing expanded access mechanism.
What will it Mean for Pediatric Cancer Drug Development?
I believe that the Right to Try legislation will likely create an increased climate of dismal pediatric drug development. Many of us in the childhood cancer community have worked tirelessly to create regulatory mechanisms to encourage and compel pediatric cancer drug development. Through the Creating Hope Act, the RACE for Children Act and the reformation of existing regulations, the climate for drug development and drug discovery for pediatric cancers based upon genetic or molecular targets has never been more promising. Removal of FDA oversight into experimental drugs provided to “dying” patients, including data on adverse reactions, creates a climate of increased fear for drug manufacturers in the rare disease space or for pediatric disease populations in general. Drug manufacturers will likely be more judicious with their funds and resources when it comes time to select oncologic targets for drug development rather than initiate their drugs into clinical trials that may expose them to requests leading to anecdotal data from patient populations regarding the lack of efficacy.
False Hope for Desperate Parents
Creating a climate of false hope for desperate parents also increases the likelihood of increased disappointment and anger. I believe the federal Right to Try legislation will provide the impetus and springboard for more desperate parents to seek access to drugs without the benefit of the FDA aiding in the process, including advising them upon safety and dosage. Despite the FDA being precluded from logging adverse reactions, including death, patient populations will continue to rely upon social media channels as a vocal forum to weigh in on drug efficacy creating a dangerous unregulated and informal data repository. This scenario will likely have one of two results: first, either the drug manufacturer will become far less willing to provide access to a particular drug or class of drugs, or, the early “unscientific data collection” and probable emotional patient criticism for lack of efficacy of a treatment will cause the cessation of clinical progress for many drugs or treatments.
Data Collection Sidestepped
The drug development process is dependent upon the ability to gather and collect data which allow experts to understand how to create effective and safe therapies. Allowing this process to be sidestepped by emotion through the removal of the FDA is the wrong way to proceed. Rather, maintaining the current system of expanded access, although not perfect and susceptible to reformation, provides the best path forward for those of us in positions to advocate for an increase of drug development. Information and data collected on experimental drugs simply provides clinicians and researchers a way to further expand the number of patients that may benefit from a drug.
My Own Horror Story
As I noted earlier, my thinking has come full circle for many reasons. When my daughter Alexis was diagnosed with diffuse intrinsic pontine glioma (DIPG), a pediatric brain cancer with a 99% mortality rate, I was as naïve as I was the first day I walked into my first year torts class in law school. Upon learning that Alexis’ tumor could not be surgically removed, that there were no effective therapies in existence and that horrifically, she was marked for death in 9-12 months, I was fully convinced that somewhere in a lab or government building the “cure” was being squirreled away. It was 2008, Neal Armstrong, who, in an odd coincidence, lost his daughter to DIPG in 1963, walked on the moon 39 years before and there was nothing that could be done for my daughter. As a then 37 year old trial lawyer with a propensity to get angry, the horror story that was unfolding for my daughter did not sit well with me. And thus, I crusaded against the government, the FDA and the pharmaceutical companies that were “hiding the drug that could cure” Alexis. What has unfolded since that time, including the death of my daughter in 2011, is a far deeper understanding of the regulatory framework, the operation of the FDA and the manner in which drug development occurs. And thus, whereas I would have tried anything to keep Alexis alive, and in fact her mother and I did just that, the answer did not lie in desperately throwing any experimental drug down her throat because she was going to die anyway. I maintain the belief that more investment into pediatric cancer drug development is beyond necessary.
A Better Way
I have since come to believe, through an extensive education that includes an appointment to an NCI committee that reviews adult and pediatric clinical trials, co-founding a research laboratory, working with hundreds of researchers and several other ventures, that there are enough channels available, and more for us to create, that will allow for more robust investment into pediatric cancer drug development. While many of us work on these “channels,” for those parents seeking to access experimental drugs for their children, the mechanism of expanded access may not be perfect, but it does work to grant access 99% of the time. There is significant danger to the federal Right to Try legislation and how it will impact this delicate balance many of us have worked hard to acheive. It seems that most members of Congress, including those on the Congressional Childhood Cancer Caucus do not even appreciate or understand this negative potential.
Ultimately, the goal of the Right to Try legislation is not to support true development of effective therapies. Rather, it has the aim of weakening the FDA and rolling back many of the protections and built in mechanisms that support drug development for disease populations like the childhood cancer community. The result will not be more children surviving their diagnosis due to a greater number of treatments being made available. Rather, clinical trial enrollment will suffer and far less drugs will be made available for testing or clinical use.
Jonathan Eric Agin, JD, is the Executive Director for the Max Cure Foundation and the co-founder of the Children’s Cancer Therapy Development Institute, a non-profit childhood cancer research biotech located in Beaverton, OR. He was appointed to and elected to sit as a Patient Advocate on the National Cancer Institute Brain Malignancy Steering Committee. He is also the Cancer Knowledge Network (Canadian Oncology Journal) Childhood Cancer Awareness and Advocacy Section Co-Editor and frequent contributor to the Huffington Post. Jonathan is an attorney by training and a former trial lawyer from Washington, DC. He is one of the most recognized names in the childhood cancer community. He has testified before the United States Congress on issues of identity theft impacting the childhood cancer community, which ultimately led to the introduction of bipartisan legislation named after his daughter Alexis (HR 2720, The Alexis Agin Identity Theft Protection Act of 2013). This legislation was later enacted into law as part of the overall budget deal of 2013. Jonathan’s legislative advocacy has proven effective in the passage of several bills in a climate of congressional stagnation and he continues to work closely with members of the community and beyond on legislative initiatives impacting the rare disease community. He has provided public comment before the FDA pedODAC Committee on the topic of biopsy in children with DIPG (an inoperable and almost universally fatal pediatric brain tumor). Jonathan became involved in the childhood cancer community following the diagnosis of his daughter Alexis at the age of two with DIPG in April 2008. Alexis battled heroically for thirty-three months until January 14, 2011. Jonathan frequently interacts with members of Congress and their staff, the White House, as well as various regulatory agencies and other cancer organizations in an effort to improve the plight of children with cancer. He is an original founding steering council member of the DIPG Collaborative. Jonathan resides in Falls Church, Virginia and has four children, Alexis (1-31-06 to 1-14-11), Gabriel age 9, Trevor age 5 and Kylie 4 years. Jonathan maintains his own website for his advocacy activities: www.jonathanagin.com and can be followed on Twitter @jonathanagin. In his spare time he also competes in endurance events like running marathons and triathlons.