Texas poised to pass right-to-try legislation

Jimmy Clark figures he has only so much time left, maybe five years in a best-case scenario, less than two if things go south quickly. He's trying to stay positive, knocking things off his bucket list, but he knows the deterioration that comes with ALS portends a gradual loss of the ability to speak, move, eat and breathe.

Clark, a 59-year-old Texarkana retailer, wants to take an experimental drug its manufacturer touts produced "dramatic" results in a recent trial with a dozen patients afflicted with the fatal neuro-degenerative disorder known as Lou Gehrig's disease. But because federal regulators require larger-scale clinical trials before deeming new drugs safe and effective, it's likely to be years before the potential lifeline is available, if ever.

"Shouldn't we be able to make the decision to try an experimental drug that has some promise for improving or prolonging our lives?" asked Clark, who's experiencing some muscle and balance loss but is doing relatively well a year after his diagnosis. "I could never understand why a terminally ill patient with nothing to lose should be denied a drug because everything isn't known about it yet. We're intelligent adults, able to think for ourselves, aware of the risks. Why not let us decide?"

Texas lawmakers are striving to facilitate just such a sentiment. In the coming weeks, the Legislature will take a final vote on a "right to try" bill, a bold attempt to remove the Food and Drug Administration from the equation and make experimental drugs more accessible to terminal patients who have exhausted other options. Under the legislation, versions of which have been unanimously passed by both Houses, patients could directly seek from the manufacturer a drug that has passed at least the first of three FDA-required investigatory trials and that their doctor thinks might provide a benefit.

The legislation is part of a growing national movement based on the perception that a cumbersome drug regulatory process keeps people from potentially life-saving treatment. In all, the movement now numbers 32 states - six that already have put into effect right-to-try laws; 11 that have passed but not implemented measures; and 15 that have bills somewhere in current legislative sessions. All the measures are based on a template provided by the libertarian Goldwater Institute.

Critics say trials threatened

But the movement has its critics. They charge that the right-to-try measures could undermine the federal process that ensures patients are protected from receiving a potentially harmful or ineffective treatment and could threaten clinical trials essential to new drug development if enough people get access to drugs through such a law. After all, they ask, why risk getting a placebo in a trial when you can get the actual drug under the law?

Still, the greater criticism involves whether right-to-try laws are likely to get experimental drugs to patients. Sometimes derided as "feel-good" measures that don't address the real reasons that patients can't get experimental therapy, they have no authority to require a manufacturer to provide a drug. In the year that the first five states had laws on the books, no patient apparently has yet used one to obtain such a drug.

"I like to call them 'right-to-beg' laws," said Art Caplan, director of medical ethics at New York University. "They provide no funding, impose no pressure. If they haven't resulted in a single patient getting a drug in a year, that's not much of a law."

But that seems almost beside the point to movement proponents, who say that in their own quieter way the right-to-try measures mark a popular revolt against the system not seen since the 1980s, when AIDS activists demanded the FDA make new treatments available more quickly. They argue it's setting the stage for a national debate about who should decide if a drug is too risky for a terminal patient.

If it's not yet had any success getting unapproved drugs to patients in states where laws are in effect, there are signs the right-to-try movement may be having an indirect national effect. Johnson & Johnson last week announced the creation of an outside bioethics panel to give it advice on patient requests for experimental treatment. The FDA is streamlining its "compassionate-use" program, the time-consuming, unwieldy mechanism by which it facilitates such access to unapproved drugs; and the 21st Century Cures Act in Congress promises sprawling reform of FDA drug approval processes.

Texas' right-to-try legislation could go to conference committee as early as next week. The only difference in the two bills is that the House version would allow manufacturers to provide experimental drugs to patients at cost, whereas the Senate version would require that companies that provide such drugs donate them. Sen. Charles Schwertner, R-Georgetown, had expressed concern a law could embolden some pharmaceutical companies to sell "snake oil" to patients facing death.

Inspired in part by lobbyist

The legislation was partly inspired by the late Andrea Sloan, an Austin lobbyist who sought an experimental drug for her ovarian cancer, which was treated at M.D. Anderson Cancer Center. She ultimately got a drug through the FDA's compassionate use program but only after much delay. After a manufacturer denied her request, another stepped forward to provide a similar drug, which doctors said appeared to be helping fight the cancer, but by that time Sloan's health had weakened and she succumbed to a complication, pneumonia, on Jan. 1, 2014.

"For patients with terminal illness, time is the great predator," said Sen. Paul Bettencourt, R-Houston, a sponsor of one of the bills. "The key is to get government out of the way and make every possible tool available so doctor and patient can take quick action. It's a no-brainer."

Citing its bipartisan support, Bettencourt predicts easy passage of the legislation and optimism that Gov. Greg Abbott will sign it. A spokeswoman in Abbott's office declined comment.

Sloan's case attracted much attention in 2013. More than 230,000 people signed an online petition supporting her effort to obtain the drug from the California company BioMarin; former House Speaker Newt Gingrich and U.S. Sen. Ted Cruz, R-Texas, took up her cause in Washington; and more than 80 Texas legislators signed a letter asking the California State Assembly to put pressure on BioMarin.

BioMarin never budged, but Bettencourt notes that another company did. He said that once some companies start granting right-to-try drug requests, "good news will travel fast and the idea will spread like wildfire."

Others aren't so sure, noting that drug companies have little motivation to supply unapproved medications outside the supervision of the FDA. FDA patient liaison director Richard Klein said that "there's a certain integrity to the regulatory system that it's hard to see why a company would want to circumvent." And the Pharmaceutical Research and Manufacturers of America, the industry trade organization, said in a statement that it has "serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the FDA and clinical trial process."

Beyond Klein's comment, the FDA has no official position on the right-to-try laws, which two legal experts told the Chronicle likely wouldn't prevail if they were challenged in court. They said they'd expect the FDA to mount a challenge if the laws result in companies frequently giving out drugs.

Dr. Mike Ewer, a semi-retired M.D. Anderson professor of medicine and University of Houston Health Law and Policy Institute guest professor of law, said the problem is that it all comes down to one's point of view.

"As a researcher, I have to be in favor of concluding the clinical trial, of determining those patients a new drug will benefit and those it will not," Ewer said. "As a doctor, a zealous advocate for each and every one of my patients, I have to fight tooth and nail if I truly believe a new drug might be able to help them. The question is, which hat do I put on?"

Like 'a really drunk old man'

Of course, the other key hat belongs to the patient, people such as Jay Smith, 37, an ALS patient in Austin who was diagnosed last year. Though his disease is considered late-stage - he responded to Chronicle questions by e-mail rather than orally because his speech is so affected - Smith said he's great, mentally sharper, not sick, just physically like "a really drunk old man." He accused the FDA of "playing God in these scenarios," given that the patients not able to access drugs until all clinical hurdles are finished will be dead by then.

But Smith said that there needs to be a bigger conversation about how to implement any Texas right-to-try law that's passed, noting that the FDA's decades-old program for fast-tracking promising new drugs has never been used for ALS patients. He said he is concerned "right to try won't be any different."

Still, between patients skeptical and hopeful, ethicists concerned about experimental drugs giving false hopes and lawmakers eager to help desperate constituents, one consensus may emerge from the right-to-try movement: that the current system is broken.

"The spread of these laws shows the level of public dissatisfaction with this part of the medical economy," said Dr. Charles Levenback, an M.D. Anderson professor of gynecologic oncology and reproductive medicine and Sloan's doctor. "It just seems fundamentally not right to put so much time and money into drug development, to brag about this great health-care system that produces such great medical innovations and then for it to prove so difficult for deserving patients to get new drugs. We deserve better."