Texas is getting closer to becoming one of a handful of states to allow terminally ill patients, through their doctors, access to experimental but potentially life-saving drugs not yet on the market, but which have cleared initial review by the U.S. Food and Drug Administration.
House Bill 21, whose primary author is Rep. Kyle Kacal, R-College Station, is based on a concept known as “Right to Try”, which advocates at the national level for many years have been championing as a humane and necessary option for gravely-ill patients who have no other legal last resort in Texas.
HB 21 was approved by the House of Representatives on Wednesday, April 22.
HB 21, coauthored by Rep. Terry Canales, D-Edinburg, would allow a dying person, with the consent of their physician, to receive promising new medication that has passed the first phase of the FDA’s rigorous review process, but which has not received final approval.
“During this long process, people may die waiting for the FDA to approve potentially life-saving medicines,” said Canales. “HB 21 would help protect physicians and drug manufacturers from legal liability. But most importantly, it would provide a person another chance at life when there is no hope, which is what all of us would want for ourselves and our loved ones.”
This legislation seeks to establish a remedy to this situation, he contended, explaining that the proposal “seeks to cut through the FDA’s red tape as the FDA is determining whether a drug is safe enough to bring to market.”
Canales, who had also filed similar legislation, joined forces with Kacal to help successfully move the issue through the House of Representatives.
Arizona voters on Tuesday, November 5, 2014, passed Proposition 303, a state ballot measure giving terminally ill patients the right to try investigational medicines that have passed the first phase of FDA approval but still may be years away from reaching pharmacy shelves.
“Right to Try” is already law in Colorado, Louisiana, Michigan and Missouri, where it passed with overwhelming bipartisan support in all four states. Arizona is the first state to pass the law by voter initiative.
A similar measure, Senate Bill 694 by Sen. Paul Bettencourt, R-Houston was approved by the Senate on Thursday, April 9, and is awaiting action in the House of Representatives.
Under current law, the FDA has a compassionate use exception policy that allows for terminally-ill patients to receive drugs and treatments prior to being FDA approved if the treating physician certifies that certain conditions are met.
Under HB 21/SB 694, the FDA’s lengthy and unduly burdensome application process that takes approximately 30 days or longer is eliminated and replaced with a more efficient mechanism that results in the patient receiving the needed medication in a much more expeditious manner. The time saved can make the critical difference – in living instead of dying – for a patient because it allows them to start treatment when they are stronger and their bodies are healthy enough to fight and succeed with safe, ground breaking treatments.
“In these fairly uncommon situations, time is a predator, that it is unconscionable that government is standing in the way of a potential cure,” Bettencourt said.
In explaining the intent of the “Right To Try Act”, Canales outlined the key processes required by the FDA before a new drug is approved.
“There are four phases in the drug review process by the FDA, which can take longer than decade to complete, far too long for critically-ill people who have not been helped by available medications and treatments,” the South Texas lawmaker said.
Phase I alone, on average takes almost two years to complete.
Phase 1 studies are usually conducted in healthy volunteers, according to the FDA. The goal here is to determine what the drug’s most frequent side effects are and, often, how the drug is metabolized and excreted. The number of subjects typically ranges from 20 to 80.
Phase 2 is given to a larger group and is further evaluated for its safety.
Phase 3 is given to an even larger group that confirms its effectiveness, monitors side effects and compares it to similar commonly used treatments.
Phase 4 studies the long-term effects of the drug, and gathers information about the drugs effect on various populations.
The current process to test, approve, and bring a new drug to market under federal regulations can take a decade or more, which is longer than patients with a terminal illness can wait, the House Research Organization confirmed in its bill analysis of HB 21.
The House Research Organization is the research arm of the House of Representatives.
“Under the bill, manufacturers would not be required to provide investigational drugs,” the House Research Organization noted it is bill analysis of HB 21. “The bill would encourage manufacturers to make the drugs available by specifying that the bill did not create a cause of action for a lawsuit after the patient signed an informed consent form and consulted with the patient’s physician.”
Also according to the House Research Organization review of HB 21:
HB 21 would be known as the “Right To Try Act.” The bill would specify that the Legislature intends to allow for patients with a terminal illness to use potentially
A “biological product” is defined in federal law under 42 U.S.C. sec. 262 to include a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, or protein applicable to the prevention, treatment, or cure of a person’s disease or condition.
Under the bill, a patient with an terminal illness would be eligible to access and use an investigational drug, biological product, or device if the patient’s physician had considered all other treatment options currently approved by the U.S. Food and Drug Administration (FDA) and determined that those treatment options were unavailable or unlikely to prolong the patient’s life and the physician had recommended or prescribed in writing that the patient use a specific class of investigational drug, biological product, or device.
The bill would not require a manufacturer to provide an investigational drug, biological product, or device to an eligible patient. Under the bill, a manufacturer could choose whether to charge a patient for the cost of the manufacture of the investigational drug, biological product, or device. A health insurance plan could, but would not be required to, provide coverage for the cost of an investigational drug, biological product, or device.
The bill would not create a private or state cause of action for a lawsuit against a manufacturer of an investigational drug, biological product, or device or against any other person or entity involved in the care of an eligible patient for any harm done to the patient as a result of the treatment.
Under the bill, a state of Texas official, employee, or agent could not block or attempt to block an eligible patient’s access to an investigational drug, biological product, or device. The Texas Medical Board could not revoke, fail to renew, suspend, or take any action against a physician’s license based solely on a physician’s recommendations to an eligible patient regarding access to or treatment with an investigational drug, biological product, or device, as long as the care and recommendations the physician provided to the patient met the standard of care and requirements of the bill.
“Right to Try” has bipartisan political support, including from the Goldwater Institute of Phoenix, Arizona, which was founded in 1988 with the blessings of the late Sen. Barry Goldwater, a national conservative leader and Republican presidential nominee in 1964. He served in the U.S. Senate for 30 years until his retirement in 1987.
According to the Goldwater Institute, even a program by the FDA to allow a critically-ill patient, with the consent of his/her physician, to use experimental drugs takes too long.
The FDA, whose extensive duties include ensuring that medicine is safe and effective for human use, is a huge federal agency with almost 15,000 employees and a $14 billion operating budget. Such a massive government organization makes it difficult to overcome overwhelming bureaucratic red tape and related delays.
As a result, the Goldwater Institute contends:
These bureaucratic impediments violate an individual’s fundamental right to try to save his own life. Unfortunately, the federal government has shown little interest in reforming the FDA as bills to reform the process for terminal patients have been introduced, but have never received a vote in Congress.
State legislators, however, have the opportunity to protect their citizens’ right to try investigational medications by enacting Right to Try measures. These measures would ensure the right to protect one’s life by returning medical decisions where they belong – to patients and doctors.
The FDA strictly controls which medications are available in the United States. Before a drug can be made available to the general public, it must undergo a lengthy and expensive clinical trial process to determine its safety and efficacy, which takes on average 10 to 15 years and over $800 million to complete.
Terminally ill patients can request exemptions, but the exemption process can take several months and requires doctors to complete paperwork that the FDA itself notes will require more than 100 hours to complete. Ultimately, the decision still rests with the FDA.
“Terminally ill people don’t have time to wait for new drugs to make their way through the decade-long approval process. Prop 303 lets patients work directly with their doctors to access promising investigational medicines now,” said Darcy Olsen, president of the Goldwater Institute.
The FDA has a process that allows people to seek permission to access investigational medicines. This “Compassionate Use” process takes hundreds of hours of paperwork and time to navigate. While many people ultimately receive FDA permission, there are dozens of documented cases of people dying while waiting on their approval.
“Americans shouldn’t have to ask the government for permission to try to save their own lives,” said Olsen. “They should be able to work with their doctors directly to decide what potential treatments they are willing to try.”
Right To Try has been featured on CNN, Fox News, Good Morning America, NPR’s Diane Rehm show, and endorsed by several newspapers around the country, including USA Today.