uring 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.”