- Patients may get more access to experimental drugs
- The law does't require insurance companies to cover cost
- The law protects doctors from liability
Before it approves a medicine for human use, the federal Food and Drug Administration requires rigorous clinical trials to ensure that it is safe and effective. These clinical trials can take many years. But what about people who have life-threatening illnesses now, and who might benefit from an experimental treatment that is still a long way from approval?
Currently, such patients have two options. One is to enter one of the clinical trials, but this is often impossible due to the patient’s geographic location or stage of illness.
The second option is to apply under the FDA’s “expanded access” program. These requests are almost always granted; between 2010 and 2014, only 33 of 5,849 patients were turned down. The problem is that the application process is burdensome and discouraging – the application alone can require more than 100 hours to complete.
Recently, five states, including Michigan, have passed laws to give terminally ill patients greater access to experimental drugs, and 25 more are considering such legislation. (The states that have passed the laws are Arizona, Colorado, Louisiana, Michigan, and Missouri.)
Gov. Rick Snyder recently has signed a law giving Michigan patients the chance to try experimental drugs that haven’t gotten final approval for general use from the U.S. Food and Drug Administration.
Governor Snyder the Right to Try Act lets patients with “extremely difficult” medical situations potentially try alternative treatments that could extend or save their lives.
The law allows drugmakers to give patients with advanced illnesses experimental drugs, biological products and devices that have successfully cleared Phase 1 of an FDA-approved trial. Doctors are shielded from sanctions based solely on recommending treatment with experimental drugs.
The law doesn’t require insurance companies to cover costs related to experimental treatment.
However, there may be legal challenges on the horizon, as some argue that these laws might well be unconstitutional, because states can’t pass laws that directly contradict federal laws. In addition, manufacturers are not allowed to ship an experimental drug across state lines without the FDA’s approval. And health insurers are not required to pay for experimental treatments, which can often be prohibitively expensive.
At a minimum, these state laws have called attention to the issue, and other help might be on the way. The FDA recently announced that it would introduce a new application form for its expanded access program, which it estimates can be completed in just 45 minutes. And a bill now in Congress, H.R. 5805, is aimed at improving the expanded access program in other ways.