One year after becoming law, a “right-to-try” measure to make experimental drugs more accessible to terminally ill patients has some physicians optimistic about the potential for more patients to try experimental therapies but others concerned it may give patients false hope and delay important conversations about advanced care planning. Meanwhile, it’s unknown how many patients have taken advantage of this new law the Texas Legislature passed last session, according to the July issue of Texas Medicine magazine, the Texas Medical Association’s (TMA’s) official publication.
Reps. Kyle Kacal (R-College Station) and Ken King (R-Canadian) sponsored the legislation, House Bill 21, which Gov. Greg Abbott signed into law on June 16, 2015. Both sponsors’ mothers died of ovarian cancer within 40 days of each other in 2013. That same year, a beloved lobbyist at the Texas Capitol, Andrea Sloan, publicized her own fight to obtain an experimental drug for her ovarian cancer. Bureaucracy prevented Ms. Sloan from beginning an experimental treatment right away. She died on Jan. 1, 2014, two months after finally getting access to the investigational drug. The death of these three women were the collective impetus behind the legislation, said Representative Kacal.
“Terminal patients, they don’t have weeks, months,” he said. “They’ve got a short window of opportunity, and if the drug is out there that has good science and passed that first part of U.S. Food and Drug Administration [FDA] approval, let’s go [try it].”
HB 21 provides expedited access to investigational drugs to cut through the bureaucracy patients like Ms. Sloan have faced when pursuing them through the FDA’s compassionate-use exemption. The bill allows a patient with a terminal illness to access an investigational drug or treatment if the patient’s physician has considered all other options and determined they are “unavailable or unlikely to prolong the patient’s life.” The patient must sign an informed consent form before receiving an investigational drug. HB 21 defines an investigational drug as one that successfully completed Phase I of a clinical trial (a first stage of FDA approval), meaning it has been evaluated for safety and proper dosing but is still under investigation in the trial and isn’t yet fully FDA-approved.
Many physicians see promise in the bill but caution experimental treatments are not for every patient and novel therapies need to be carefully considered by patients and their doctors.
Austin oncologist Debra Patt, MD, past chair of TMA’s Committee on Cancer, says HB 21 effectively gives cancer patients options when standard treatments are no longer available, and the patients can’t participate in a clinical trial. But Dr. Patt says patients should use that option infrequently because a clinical trial, if available, is the best setting in which to try experimental drugs.
“For most patients who receive treatment, it really should be under evidence-based care that we believe is more likely to be effective and less likely to have substantial harm,” Dr. Patt said. “I do think that this is important legislation, and it’s really important to allow doctors and patients to make the right decisions with regard to their care. But I think it is in the best interest of patients if it’s used extremely infrequently.”
Echoing Dr. Patt’s apprehension was Sen. Charles Schwertner, MD (R-Georgetown), who raised “concerns that vulnerable individuals could be hurt by the law.” Senator Schwertner said at the bill’s hearing that he worried physicians and drug companies might “sell hope” using unproven medications.
“History is replete with snake oil salesmen in the medical field,” he said.
Houston gynecologic oncologist Lois Ramondetta, MD, chair of TMA’s Committee on Cancer, says she’s concerned that it’s “always easier to give another drug than really sit and talk with someone about advance care planning, for both the patients and the physicians, to avoid the elephant in the room and the fact that the end of life is coming.”
“I would say it’s very important to recognize that more drugs [are] not always better and can in fact sometimes even shorten life,” she said. “Really, with the speed at which new drugs are discovered, they need to be evaluated carefully before being used.”
Charles Levenback, MD, a Houston gynecologic oncologist who treated Ms. Sloan at MD Anderson Cancer Center for seven years, was more optimistic about the law, citing its ability to draw attention to experimental therapies and the difficulties patients can face while pursuing them.
Anything that shines a light on the complexity of the health care system is a good thing, Dr. Levenback said. Thus far none of the physician’s other patients have applied for compassionate-use access under HB 21.
But he asked why it’s so difficult for a patient like Ms. Sloan to gain access to potentially life-extending therapy when good science provided evidence the drug could help.
“Andrea paid her taxes, she was insured, she was a good servant of society, she was responsible, and now she'’ asking back for a drug [that’s] not going to cure her but maybe prolong her survival,” he said. “And it felt like everything was conspiring to keep her away from it.”