How do the revised rules in the Bipartisan Budget Act of 2015 affect you and your business?
Dubbed “right to try,” the legislation makes it easier for patients with terminal illness to access drugs not yet approved by the Food and Drug Administration, but that have passed the FDA’s safety requirement.
The bill was sponsored by state Rep. Joann Ginal, D-Fort Collins, who spent more than 20 years in the pharmacology industry before entering politics. Giving patients the option to try experimental treatments has the potential to save lives, she said.
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Ginal introduced the bill in February and has heard from many Colorado families expressing support. It passed with bipartisan support, but remains controversial nationwide. Louisiana, Missouri and Arizona are considering similar measures.
It takes more than 10 years and $1 billion to get a drug approved by the FDA, Ginal said, and terminally ill patients rarely have enough time to wait a decade. “Right to try” allows patients to apply directly to the pharmaceutical companies that manufacture the drugs to try to get them before they get FDA approval.
The FDA testing process has doubled since the 1970s, Ginal said. At that time, getting a drug approved took only four or five years as opposed to 10.
A federal law already allows patients to get access to experimental drugs in terminal cases, but they must apply to the FDA, Ginal said. Getting approval takes three to six months and, again, terminal patients may not have that kind of time. Allowing patients to go directly to the manufacturer will decrease the wait time, Ginal said.
Patients would have to confer with their doctors to make sure that using an experimental drug is the best route, and would be fully aware of potential side effects or lack of efficacy, Ginal said.
Drugs that have been through Phase 1 of FDA testing are deemed safe, but the second two rounds of testing check for a drug’s effectiveness.
Using unapproved drugs provides a choice for those patients who are too sick to enter clinical trials, which allow some patients to get access to new treatments. Terminal patients are not chosen for clinical trials, Ginal said.
When conducting a clinical trial, researchers want to find the healthiest possible candidates with the condition a new drug is meant to treat, said Charles Siler of the Goldwater Institute, a libertarian-conservative think tank that helped write the legislation.
Only 3 percent of people who apply for a clinical trial are accepted, Ginal said.
Critics of the legislation say that accessing experimental drugs will create a false sense of hope in terminal patients or that unscrupulous people or companies may attempt to pass off bad drugs.
But patients will only be able to access drugs that have made it through the first round of FDA testing, Siler said, and their physicians must have honest conversations with patients to make sure their expectations are realistic.
Other criticisms include that expanding access to experimental drugs will negatively impact the existing clinical trial and drug testing structure, but data could be collected from patients who take drugs under “right to try” that could be used to augment data retrieved from traditional testing processes.
While “right to try” will give patients another option for treating their diseases, there is no guarantee that the drugs will work, Siler said.
Access to these drugs probably will save only a fraction of people, but that fraction is worth it, he said. About 1.6 million people a year die following a terminal illness in the United States, according to the Centers for Disease Control and Prevention.
“I can’t say this is a cure-all,” Ginal said, “but it gives people a choice.”
Molly Armbrister can be reached at 970-232-3129, 303-630-1969 or email@example.com. Follow her on Twitter at @marmbristerBW.