In his first year as commissioner of the Food and Drug Administration, Scott Gottlieb has managed to surprise early critics with bold public health moves, such as a plan to limit nicotine in cigarettes, while also keeping many industry supporters and the president on his side.
But Gottlieb may soon face his toughest balancing act: implementing the Right to Try Act. President Trump is expected to sign the bill, which passed the House of Representatives on Tuesday.
The Right to Try Act gives terminally ill patients a direct path to access experimental treatments that aren't FDA approved. Under an existing provision, patient can already apply to the FDA for access, a process called expanded access or compassionate use.
With an estimated 609,640 deaths expected in the United States in 2018 from cancer alone, supporters of the Right to Try Act erroneously point to the comparatively low number of expanded access applications as an indication that the FDA process must be overly cumbersome and time-consuming.
Since 2009, the FDA's expanded access program has been available for people who are fighting life-threatening illnesses, have exhausted all available treatment options and who want to try experimental treatments that aren't on the market. Although clinical trials represent the preferred choice for patients to access these therapies, some people don't meet the criteria to be included and others may not live near a clinical trial center.
Between October 1, 2009, and September 30, 2014, the FDA granted 5,996 of 6,029 expanded access requests, or 99.5 percent, for experimental medicines and noted recent increases in applications. Importantly, the FDA requests modifications to the individualized treatment protocols for these patients to improve safety before allowing treatment to proceed.
Unfortunately, the Right to Try Act won't remove the primary roadblock facing terminally ill patients who want to test unapproved, investigational therapies: access to the drug by its developer.
Under expanded access, the FDA receives and reviews an application only after a physician has determined that use is appropriate and a drug company has agreed to provide the drug. Little is known about the number of expanded access requests denied by manufacturers and, therefore, never submitted to FDA. Neither expanded access nor Right to Try Act mandate cooperation from a drug company.
In testimony before the House Energy and Commerce Committee's Subcommittee on Health, Gottlieb expressed concerns about the Right to Try Act. He recommended narrowing eligibility in from patients who face a "life-threatening disease or condition" to "terminal illness." The concern being that chronic conditions, such as diabetes and heart disease, may be life-threatening but manageable without resorting to unapproved therapies.
In fact, Gottlieb was quoted saying that broadening the definition to include patients who aren't terminally ill risks "undermining the central purpose of this legislation." Will the agency be able to restrict the right to try to include only terminal patients?
Gottlieb also raised the concern that under the Right to Try Act companies and others providing experimental drugs to patients wouldn't be subject to many rules and regulations related to clinical trials, premarket approval and labeling. That could preclude the FDA from taking enforcement actions based on those provisions.
Some of his concerns were addressed in a version of the bill that passed the House in March 2018 but ultimately stalled in the Senate. As a result, the House passed the original measure sponsored by Senator Ron Johnson, R-Wis., that hadn't been revised.
Nonetheless, Gottlieb tweeted Tuesday that he stands ready to implement Right to Try "in a way that achieves Congress' intent to promote access and protect patients; and build on FDA's longstanding commitment to these important goals."
Given the version of right-to-try legislation headed for President Trump's signature, it remains unclear exactly how Gottlieb will address the agency's earlier concerns. The main issue is that the Right to Try Act is intended to run parallel to the FDA's expanded access pathway, providing a route for patients to circumvent the agency.
As a physician, cancer survivor, venture capitalist and government insider, Gottlieb has a unique perspective on the ethical and practical challenges inherent in providing access to experimental drugs. However, implementing the Right to Try Act while ensuring that patients are protected and clinical development of new medicines isn't compromised represents a daunting challenge.
To his credit, Gottlieb appears unfazed. "I feel confident that we're going to have the regulatory tools we need that if we see people taking advantage of patients we'll be able to step in," Gottlieb stated during the CNBC Healthy Returns conference in March.
Michael D. Becker is president and founder of MDB Communications LLC. He previously served as president, chief executive officer, chief financial officer, and director for several publicly traded biotechnology companies, including Cytogen, VioQuest Pharmaceuticals, and Relmada Therapeutics. He describes his fight with stage 4 oropharyngeal cancer in "A Walk with Purpose."