The House will vote tonight (March 13th) on a revised Right to Try Act that was unveiled over the weekend. On a positive note, the new House version appears to reflect some changes FDA Commissioner Scott Gottlieb recommended in October testimony before the House Energy and Commerce Subcommittee on Health regarding a previous version of the bill that passed the Senate by unanimous consent last summer. But the new bill is still nothing more than a Trojan horse positioned as the “first step on a longer path to push the Food and Drug Administration (FDA) to loosen up on its oversight.”
Proponents of Right to Try know precisely how to wage an effective public relations war by tugging at people’s heartstrings. They tout grim statistics, such as the fact that over 600,000 Americans with cancer are projected to die in 2018 – supposedly because they are waiting for access to experimental therapies that haven’t yet been approved. Pitchforks and torches in hand, libertarians use the FDA as a scapegoat for blocking patient access to a mystical treasure trove of investigational treatments.
Politicians and other opponents of Right to Try have been quiet or slow to act. This is because a lack of support for Right to Try can be perceived (albeit incorrectly…) as not being sympathetic to the needs of terminally ill patients. Encouragingly, nearly 40 patient advocacy groups argued that Right to Try would “likely do more harm than good” in a letter sent to House leaders of both parties last month.
The fact remains that under existing regulations, patients can already seek expanded access, sometimes called compassionate use, to experimental therapies that are currently being studied for safety and effectiveness but that have not yet been approved by the FDA. For the past decade, the FDA has received approximately 1,000 such requests for expanded access each year. But with more than 600,000 Americans dying each year from cancer alone, Right to Try supporters argue that red tape and government regulations must be restricting access to promising new treatments for the other 599,000 patients. Hence their desire for an alternative parallel pathway that eliminates these barriers.
However, two key steps are required before the FDA even receives a request for expanded access, which demonstrates that the FDA isn’t the logjam. First, a physician must certify that the patient seeking expanded access has exhausted available treatment options and is unable to participate in a clinical trial involving the desired experimental drug. Second, the physician contacts the drug company developing the investigational drug to ask permission to test the treatment. Assuming that the drug company agrees, the physician then completes and submits paperwork to the FDA. At this point, more than 99 percent of such requests are approved within days (24-hour turnaround for emergency cases), and the FDA even suggests important changes approximately 10 percent of the time to help improve patient safety. Nonetheless, the Right to Try Act being considered by the House eliminates this critical FDA oversight.
Beginning in 2014, Right to Try has now been signed into law in at least 38 states. FDA regulations cannot preempt state laws that preserve constitutionally protected rights, such as the fundamental right to life and medical self-preservation (e.g., the legalization of marijuana in individual states while still illegal under federal law). And yet, there is scant evidence that patients have successfully accessed investigational drugs through state Right to Try programs who wouldn’t otherwise be eligible under the existing expanded access program. This provides further support that the FDA isn’t the bottleneck.
Since the FDA clearly isn’t the problem, why are Right to Try supporters so intent on creating a ‘parallel pathway’ that excludes them? Why not collect more data to understand exactly why there are only 1,000 requests for expanded access each year? More importantly, if Right to Try is the solution – why hasn’t it been successful in state programs over the past several years?
The simple truth is that physicians and drug developers understand better than most the concept of “first, do no harm” that is attributed to the ancient Greek physician Hippocrates. In the case of cancer drug development, the probability of progressing from Phase I to FDA approval is only 5 percent. Accordingly, if all 600,000 terminally ill cancer patients received access to experimental treatments under Right to Try this year, only 30,000 would likely benefit. The other 570,000 patients could suffer toxicities that hasten their death, severely diminish their quality of life, and lead to uninsured medical expenses.
While Right to Try is being positioned as pro-patient, in reality, it’s nothing more than a libertarian Trojan horse designed to weaken the FDA. And once this dangerous precedent is set, I fear it could easily be expanded to include patients with less severe diseases.
No one, especially myself as a terminal cancer patient, wants to deny patients the right to receive potentially beneficial drugs. We already have that right today. Our current expanded access framework is meant to protect patients, and the FDA has a program that approves nearly 100 percent of all expanded access requests it receives. My sincere hope is that the House votes today to put the safety and best interests of patients before politics.