During his first State of the Union address, President Trump urged Congress to pass a bill aimed at giving terminally ill patients access to experimental drugs. As an individual with terminal cancer and a former biotechnology industry executive, I know that the “right to try” isn’t merely a theoretical concept. And while I can relate to patients’ desperate hopes and desires to try new therapeutic approaches, I believe that passage of the federal Right to Try Act would be a disaster.
Right-to-try laws have already been approved by many states. On their surface, they seem to be kind and egalitarian: let terminally ill Americans who have no other options try experimental therapies that haven’t been approved by the Food and Drug Administration. But it’s actually an approach that could do more harm than good for people who seemingly have “nothing left to lose.” I’m also not sure why such a law is needed, since people like me already have access to experimental therapies.
Under the Federal Food, Drug, and Cosmetic Act, patients can seek expanded access, sometimes called compassionate use, to investigational therapies, meaning those that are being studied for safety and effectiveness but that have not been approved by the FDA. Since expanded access was enacted in 2009, the FDA receives approximately 1,000 requests for expanded access each year. It approves more than 99 percent of these requests, and makes meaningful changes approximately 10 percent of the time to improve patient safety.
The FDA retains oversight of expanded access treatments. It can use the results of these treatments to judge the safety and effectiveness of the experimental therapy. As currently drafted, the federal right-to-try bill eliminates or reduces that oversight — a concern noted by FDA Commissioner Scott Gottlieb in testimony before the House Energy and Commerce Committee’s Subcommittee on Health.
Put simply, under the right-to-try bill, a death caused by the use of an experimental drug could not be considered by the FDA in deciding whether to approve or reject the therapy. While this may help address pharmaceutical and biotechnology industry concerns that expanded access programs could jeopardize ongoing studies, it is simply unethical at its core.
I’m also worried about the financial, legal, and medical protection afforded to patients and their families under the proposed right-to-try bill. Do patients undergoing right-to-try therapies lose their coverage for hospice? Would insurers be absolved of any responsibility for covering further medical expenses once a patient starts a drug under right-to-try? What if the experimental drug causes hospitalization or leads to additional treatments — who would pay for that?
In his testimony, Gottlieb also raised the concern that, if the federal right-to-try was enacted without changes, sponsors and others providing investigational drugs to patients would not be subject to a number of rules and regulations related to clinical trials, premarket approval, and labeling. That would, in essence, preclude the FDA from taking enforcement actions based on those provisions. In other words, cutting out the FDA as a gatekeeper increases the risk of patients being harmed through the peddling of false hope and snake oil.
As I am learning firsthand, being diagnosed with a terminal disease is extremely challenging for patients and their families. Introducing significant new financial, legal, and medical uncertainties via the Right to Try Act would make matters worse.
For nearly a decade, the FDA’s expanded access program has been available for people like me who are fighting life-threatening diseases and who want the option to have access to new therapeutic approaches in a responsible and ethical manner, especially when there is no other FDA-approved treatment option.
And that means we already have a right to try, one that offers greater protection for the already vulnerable individuals who will use it than the new law the president promoted in his State of the Union address. The federal Right to Try Act isn’t a boon for patient rights. In fact, it could dismantle the very safety system that currently protects us — and that would be a disaster.
Michael D. Becker is president and founder of MDB Communications LLC. He previously served as president, chief operating officer, chief financial officer, and director for several publicly traded biotechnology companies, including Cytogen, VioQuest Pharmaceuticals, and Relmada Therapeutics. He describes his fight with oropharyngeal cancer in “A Walk with Purpose: Memoir of a Bioentrepreneur.”
I hear people are feeding bleach to their autistic kids because they think it will help. Right to fry.
As a stage IV patient with no cure I am eligible for Phase I trials. What bothers me, is that often there are serious side effects that “pharma” say isn’t because of their brand new med yet I see it is . . . Thus putting the vulnerable patient at more risk. It is a tough decision. But for me . .. I’d rather my death come without any harsh side effects.
That’s the beauty of America. Right to try, is NOT a requirement to try. Each individual can chose to “try”, or not to try. I once knew a woman in her 50’s who chose to forgo ALL treatment for stomach cancer. She died in a year, her family was heart broken, or course, but they respected her decision. The freedom to choose, what a beautiful thing.
How could the patients & their families be sure that “snake oil or actually harmful” remedies, proportional to the risks, are being pushed at them??
After all, being terminal, the people have little to lose or other alternatives…
My late brother spent years & all his money chasing “non-traditional” remedies that did nothing but hasten his dementia & death!!
The problem here is that dying patients DO NOT have access to therapies. The clinical trials are incredibly small and slow , not keeping pace with the new genetically based treatments . Check out the low numbers of pts in a 5 – 7 year trial and compare it with the annual death rate from that disease. The few thousand compassionate use requests granted are pathetically few in a supposedly modern society of 314 Million . My wife was ” terminally ill ” in 2010 , would have died by early 2011. She did get the drug after a crazy struggle. See ” Lorraines’ Story about RTT on the Goldwater Institute website. The FDA was the main obstructor of her drug Kadcyla, and other drugs at that time, Iclusig , Lemtrada . The slowness of the system means continued pt. pressure on the system . Rather than hypothetically crying that the ” sky will fall “, how about trying to really fix the system and save lives now. Eliminate any delay by the FDA , simply notify them of a comp. use decision under RTT . Then if they have concerns they can step in . rather than discouraging dr.’s and pt.s from even trying. RTT will help . at least try it , given the stakes. Comp. use is almost always for drugs already in trials. The ” snake oil ” ” thalidomide”, ” Laetrile ” scare tactics have already been exposed . After RTT , then put the focus on the pharma co.’s and their lack of compassionate use , putting $ before lives. and the IRB’s, benefits managers and Ins. co.’s who also support a corrupt , broken approval system. Costing lives. During the 3 extra years the FDA took to finally approve Kadcyla, 45,000 Americans died from that sub- type of breast cancer . As I have said in the past – wait until your miracle drug comes along , and for many it will – then see how ” easy ” it is to get …
Phil – Yes, the terminal patients do already have access. The fact is that ~1,000 patients per year for the past decade have received expanded access under existing regulations. That’s ~10,000 patients.
Mr. Becker, I’m new to this issue. A coworker’s mother had to travel to Germany for treatment that she couldn’t get here. It’s awful that someone so weak would need to travel to the other side of the world for a chance to save their own life. It’s worked, so far, and bought her another Christmas, and her daughter had her for another birthday. To me the argument for this is pretty clear and I’m having trouble with the argument against. You say ~1000 terminally ill patients a year get experimental drugs, but a quick google search suggests ~ 545,000 cancer deaths a year (and of course that isn’t the only disease that you can die from). 1 in 545 is NOT access. Is the argument against this really money, legal liability and the FDA’s data set?
Thank you for taking the time and energy to post this well-thought-out article.
My pleasure, Kari. It’s an important topic…