Who says Congress can’t act in a decisive and bipartisan way?
The Senate, at least, appeared to achieve this recently when it unanimously approved the Right to Try Act.
The measure, to be considered by the House of Representatives now that Congress has returned from its summer recess, allows seriously ill patients to request access to experimental drugs after all other available treatment options have been exhausted. These are drugs that have successfully completed Phase I clinical trial testing to evaluate safety and the potential for serious side effects and are being evaluated by the federal Food and Drug Administration.
Such so-called “expanded access” has existed since the early 1960s and has worked like this: A doctor sends a request on a patient’s behalf to the manufacturer of a drug. If the company complies, as sometimes happens, then the physician asks the FDA to approve the experimental treatment. Under the Senate bill, introduced by Republican Sen. Ron Johnson of Wisconsin, the FDA is removed from the process. Drug company approval would be the only OK needed. The Republican-led Senate sees this as an effective way to increase access by stripping away bothersome federal involvement. (Never mind that the FDA approves 99 percent of all requests.)
For the Senate, passage of the act symbolizes a health care “win” that has been elusive this year in Washington. For the patients it claims to help, the bill delivers something much less. It fails to address the biggest obstacle facing people who seek access to drugs that are still in clinical trials: Pharmaceutical manufacturers have complete discretion over whether to grant or deny such requests. And denials are the norm.
A manufacturer’s willingness to provide a drug is necessary because the government cannot compel companies to make clinical therapies available to the public. This sole discretion of the drug maker is a common frustration among patients desperately trying to gain access. The problem is exacerbated by the fact that patients have no idea what criteria the companies use when they give the thumbs-up or, more typically, the thumbs-down to a request. No standard for evaluating patient requests exists in the industry.
The Right to Try Act would have amounted to more than a mere symbol if it had actually compelled the drug companies to grant reasonable emergency requests, with appropriate legal protections and incentives for the manufacturers.
Senator Johnson and his colleagues would have us believe they acted out of a concern for people who might be saved by access to experimental drugs. If the senators actually support expanded access, why doesn’t their bill supply it? For the most part, it echoes the right-to-try laws that 37 states have passed — none of which, it should be noted, has been a factor in inducing a pharmaceutical company to grant drug access to a terminally ill person. Like the Senate bill, these state measures are toothless when it comes to compelling access.
The “right to try” label cleverly implies that the laws enable patients to try an experimental treatment. All they really provide is a right to request drug access. They do nothing to oblige the drug companies to grant the requests.
To be fair, the reluctance of the companies is not difficult to fathom. They worry that if a patient is harmed by an experimental treatment, then the negative result could lead the FDA to deny approval of the drug. A more careful crafting of the Senate’s bill, then, would have included protection for the companies and their products from such an outcome, along with ways of rewarding companies that are more generous with drug access, plus full-disclosure provisions that help patients understand the inherent risks of taking trial therapies.
Instead, the Right to Try Act is an opportunity lost. Politically, it looks like a win, something that Congress and the Trump administration can point to as a step forward on health care legislation. But rather than providing a pathway to access, the Senate bill weakens the existing structure by removing the FDA’s oversight from the process. At the end of the day, it keeps all control of drug access in drug manufacturers’ hands, doing little to aid seriously ill Americans.
Stacey Lee is an associate professor at the Johns Hopkins Carey Business School, where she teaches courses on legal issues in medicine and business. She can be reached at firstname.lastname@example.org.