After months of debate, Congress enacted legislation yesterday that establishes a pathway for terminally ill individuals to gain access to investigational products. Even though FDA has a well-established and effective expanded access/compassionate use program that assists very ill patients in obtaining not-yet-approved therapies from sponsors, agency critics have long demanded stronger action to remove roadblocks to experimental treatment.
FDA commissioner Scott Gottlieb, MD, and previous agency leaders have maintained that this legislation is not needed and may expose desperate individuals to harmful or ineffective treatment. They also have concerns that the latest bill undermines FDA authority to vet and approve important new medicines based on safety and efficacy. Under pressure from the Trump administration, Gottlieb recently softened his opposition, stating that FDA can implement the new policy without harming agency operations or safeguards.
The Right-To-Try (RTT) bill initially sponsored by Sen. Ron Johnson (R-Wis) was approved by the Senate in August 2017 in a deal to move forward must-pass legislation to renew FDA user fees. The House passed its own version of the Senate measure in March 2018, which added provisions to address patient safety and limit liability of prescribers and drug companies, as sought by patient groups and manufacturers. But the Senate failed to approve the House measure, prompting RTT advocates to press for House approval of Johnson’s original bill. The House now passed the Senate bill by a strong margin, largely along party lines, sending it to the White House for President Trump’s signature.
RTT legislation has been promoted by the libertarian Goldwater Institute of Arizona with strong backing from the Koch brothers. While its stated goal is to provide life-saving treatment for seriously ill individuals, it also challenges the FDA approval process for biopharmaceuticals by reducing incentives for patients to participate in clinical trials and for manufacturers to finance long clinical testing programs. Almost 40 states have enacted their own right-to-try laws, but its backers have long sought federal legislation. The Goldwater Institute also is promoting state laws that permit off-label promotion by pharmaceutical companies, which has recently been successful in Arizona and Tennessee. These bills prevent states from taking action against pharmaceutical sales reps or healthcare institutions engaged in truthful promotion of off-label drug uses.
Manufacturers at risk
The federal RTT law permits terminally ill patients to go directly to drug companies to request access to experimental drugs that have moved to Phase II testing, bypassing FDA’s compassionate use process, which regularly authorizes 99% of requests from patients and physicians for early access to unapproved drugs. In both the current and the new process, a biopharmaceutical company developing a new therapy can decide whether or not to provide an experimental product to an individual. Such decisions are particularly difficult for small companies that often lack sufficient supplies outside clinical trials. Sponsors have concerns that very ill patients will suffer serious adverse events that can compromise a drug development program and FDA approval of the new therapy. And industry fears liability claims from patients who fail to respond or decline under treatment.
Friends of Cancer Research and a long list of medical research organizations and professional societies strongly objected the bill. The opponents claim it creates serious risks for patients, undermines an effective drug regulatory process, and raises false hopes for individuals seeking help. Industry organizations have been quiet on the issue but will face challenges in dealing with new requests.