Discussion on Right to Try legislation continues, and the federal government could soon change existing rules. Many people wonder whether or not patients should have the right to try potentially life saving, though untested, treatments. Some experts, however, point out that patients already have access to these medications. Keep reading to learn more, or follow the original story here.
There are a small number of patients at University of Michigan’s hospital which are described as severely ill. These are patients with terminal cancers, and rare life-threatening genetic disorders. Some hope that they’ll be able to get access to recently developed treatments. These drugs could be the key to curing them. Of course, some of them could be as ineffective as other treatments, and some could untested drugs can have serious side affects.
These patients are willing to take that risk. These are the kinds of patients Congress and advocates have in mind when they describe a Right to Try bill. Should such a bill pass, it would extend patients across the nation an opportunity to access unproven therapies to treat terminal illness.
Curiously, some say such a system already exists. Over 40 patients at Michigan Medicine gained approval to receive treatment with unproven treatments outside clinical trials in 2016. Kevin Weatherwax leads the team that makes this possible.
Weatherwax and those who work with him make this possible by utilizing the Expanded Access program designed by the United States Food and Drug Administration (FDA). The program enables doctors to submit applications for their patients. These applications are used to gain access to drugs and devices not yet prepared for clinical trials. Alternatively, doctors can use them to access treatments used in clinical trials their patients do not qualify for.
Weatherwax says they’ve been using the Expanded Access program successfully and with other physicians for many years now. 2016 proved exemplary for the program. In many cases, Weatherwax says, those submitted for Expanded Access are life-and-death cases.
He notes that the FDA always shows a willingness to review what his team submits, and that all University of Michigan cases have been approved in a timely fashion.
Furthermore, as Weatherwax points out, the current Right to Try legislation doesn’t cover as much as it seems to. Current existing legislation, and proposed legislation only address drugs and ignore medical devices. The new Right to Try bill also fails to provide the same protections for patients that Expanded Access does. Expanded Access requires examination by a local Institutional Review Board while new legislation only required approval by a patient and physician. The FDA also currently provides patients with a level of cost protection under Expanded Access which Right to Try does not extend.
The difficulty then with gaining access to these experimental treatments is often not the regulations, but the companies producing them. Weatherwax describes convincing manufacturers as the most difficult step in obtaining treatment. Producing experimental drugs can be expensive, especially creating additional batches for individual patients. Some companies are simply too small to carry the burden. Weatherwax says this issue will continue whether or not Right to Try passes.
The hospital at University of Michigan takes great steps to ensure its patients have access to the treatments they need. Not every hospital has a system like this in place, but University of Michigan is prepared to assist in any way it can.
Weatherwax predicts enacting a national Right to Try likely will not increase the efficiency of acquiring experimental treatments. He points to the need for companies to gather clinical trial data rather than information on single patients to explain this.
Weatherwax does, however, acknowledge that the discussion of Right to Try has increased awareness of what is available to patients.
The way forward, Weatherwax says, is instead to focus on best using the system of Expanded Access that currently exists. Michigan Medicine and several other top academic centers already work together on this project. Unified, they hope to share best practices, and experiences to further what can be accomplished within the program.
