A bill introduced in the US Senate on Tuesday becomes the latest legislative effort to expand the ability of terminally ill patients to gain access to experimental medicines. The legislation, which joins a companion bill that was introduced in the House last summer, would prohibit the federal government, including the US Food and Drug Administration, from taking any action to prevent patient access.
The bill, which was introduced by Senator Ron Johnson (R-Wis.), comes after 28 states have passed so-called “Right to Try” laws. These allow patients to leapfrog a drug-development process that takes years before new treatments become available. And the laws reflect rising frustration with an FDA program called expanded access, in which people who are seriously ill can obtain a drug under development, even though they aren’t enrolled in a clinical trial.
Under federal law, if terminally ill patients are not eligible to obtain an experimental therapy through a clinical trial, they can apply to the FDA for what is known as compassionate use. The agency actually approves the vast majority of these requests, but as we have noted previously, critics say the federal program is arbitrary and cumbersome.
And so, a nationwide movement has been under way to seek to cut the FDA out of the process of approving the use of experimental drugs in such circumstances. The effort has been propelled by the Goldwater Institute, a Libertarian think tank that helped draft the state laws. In fact, these measures have been introduced in another 15 states.
As a practical matter, though, the numerous state laws, so far, do not appear to have accomplished very much. A spokeswoman for the Goldwater Institute claims that 40 patients have received medicines, but she could not provide the names of the physicians or companies involved. Critics, meanwhile, say there are a host of problems with the laws that make the concept untenable.
For one thing, patient protection is uncertain, since the FDA is precluded from providing oversight. Also, the laws do not require drug makers to make their medicines available. And while insurers are not required to pay for treatment, most states have failed to create funding to help patients cover medical expenses.
The Right to Try laws are “feel-good legislation that terminally ill patients are only too eager to believe,” Art Caplan and Lisa Kearns of New York University’s Langone Medical Center’s division of medical ethics wrote last winter in an editorial in The Hastings Center Report. Caplan, who also oversees a pilot program in compassionate use for Johnson & Johnson (JNJ), wrote us that the Senate bill “budgets nothing to help desperate patients nor does it provide any incentive to companies to grant compassionate use.”
Moreover, there are unanswered legal questions about whether state laws can trump federal regulations. This helps explain the effort to introduce legislation in Washington. Although momentum continues at the state level, the Goldwater Institute and many patient groups believe the issue can best be solved with federal legislation.
“In an era of unprecedented medical innovation, we have to do more to ensure that patients facing terminal illnesses have access to potentially lifesaving treatments. These patients don’t have the time to wait — often years and years — for the FDA to grant final approval,” said Johnson, who introduced the bill, in a statement. “Now is the time to give more patients a chance to save their lives.”
To what extent the legislation will accomplish its goal, though, is unclear. The House bill, which was introduced last July, has generated bipartisan sponsorship, but has languished in a committee ever since.