Side Effects

Right-to-try laws could give patients access to experimental drugs, but the risks are extreme

Back Longreads Sep 8, 2015 By Rich Ehisen

Many of us are familiar with Woodroof’s plight — it was the subject of the critically acclaimed movie “The Dallas Buyers Club.” But while Hollywood took many liberties in telling his story, Woodroof’s real-life dilemma is one still being shared by many terminally ill people today. That struggle is also at the heart of a movement to allow those patients access to drugs the FDA has not authorized.

According to the National Conference of State Legislatures, since January 2014 at least 36 states and the District of Columbia have introduced so-called right-to-try bills, legislation that would allow terminally ill patients to access experimental medications still in clinical trials. At least 22 of those states have adopted such proposals into law. Many others remain pending, including a pair of bills here in California.

The measures are based on model legislation crafted by the libertarian Goldwater Institute in Arizona. The aim is to give terminally ill patients who may be out of treatment options access to medications they otherwise could only obtain through a clinical drug trial or the FDA’s compassionate-use program. To Goldwater Institute President Darcy Olsen, providing that option should come without question.

“If you know there’s a treatment that is helping people survive, who is anyone else to say,  ‘No, you don’t have the right to try to save your own life or to save your child’s life?’” she says in a statement on the group’s website.

But right-to-try laws are not without serious questions and complications. For one, the FDA still holds primacy over pharmaceutical drugs — experimental or otherwise — and none of the new state laws can compel a drug maker to provide a medication that is in clinical trials. Nor can they require doctors to recommend them or health insurers to pay for them.

“These laws only guarantee a right to try experimental drugs; they do not guarantee a right to access them,” says law professor Lisa Ikemoto, who teaches health care law at UC Davis.

For health professionals like Dr. David Gorski, a surgical oncologist and the managing editor of the blog Science-Based Medicine, right-to-try laws are also bad medicine. “These bills are simply a horrible idea,” he says. “They are presenting a fake premise that there is this whole range of miracle drugs out there that could help terminally ill people. That simply isn’t true.”

Gorski notes that right-to-try laws only require a drug to have passed through phase one of the 3-part clinical trial process. That stage is conducted on only a very small sample of generally healthy volunteer subjects, sometimes as few as 20 people.

“Phase-one trials are only designed to test for the worst toxicities and to determine a drug’s maximum tolerated dose,” he says.

“[Right-to-try] bills are simply a horrible idea. They are presenting a fake premise that there is this whole range of miracle drugs out there that could help terminally ill people. That simply isn’t true.” Dr. David Gorski, surgical oncologist, and managing editor, ScienceBasedMedicine.org

The real safety testing for a new drug comes in stages two and three, which is where a huge percentage of phase-one-approved drugs wash out. According to data from the American Medical Association, only 30 percent of drugs that make it to the second trial phase are approved for phase three. The FDA says that, in fact, only about 7 percent of drugs that enter clinical trials make it through all three stages and get to market.  

In that regard, Gorski questions the ethics behind giving an already-sick person a medication that very well could make them worse or even kill them. The Goldwater template also offers doctors wide civil immunity, which could unintentionally encourage an unethical or irresponsible physician to suggest harmful drugs, with no legal or civil repercussions.

“These bills really should be called Freedom to Practice Quackery,” Gorski says.

Cost is also a significant factor. Since insurance coverage for such medications is not mandated — and is highly unlikely to be offered any other way — a patient receiving such drugs would almost certainly be on the hook for paying for them out of pocket. That can be prohibitively expensive even for people of means. And because of the civil-immunity aspect of the laws, patients have no legal recourse if things go really wrong.

Tom Ikelman, owner and proprietor of Fantastic Produce, a wholesaler in Natomas, sees both sides of the coin. In 2008 he was diagnosed with advanced non-Hodgkin’s lymphoma, a cancer that attacks a person’s lymphatic system. Because his illness was discovered so late in the game, he was originally given a very poor chance to survive. But he was fortunate — his body was able to successfully fight off his cancer with traditional chemotherapy. Today, seven years into remission, he says he is still acutely familiar with the fear he felt in facing his own mortality. He also knows that anyone with a life-threatening illness will try almost anything to survive, even at the highest of costs.

“On the one hand, I would absolutely spend every penny I had if it means I would live. What good are assets if you are dead?” he says. “But then I also think of quality of life. If an experimental drug lets me live three months longer but also makes me a lot sicker during that time, what is the point?”

Ironically, although none of his medications were experimental, he has not escaped the impact of their side effects. During his treatment, he developed a condition called cold urticaria, a severe allergic reaction to cold temperatures. Even sticking his hand into his pool for a few minutes was enough to make his skin turn bright red, develop welts and swell all the way to his shoulder. The condition lasted for years, only recently beginning to fade. Nobody knows for sure what causes it, but he believes the damage to his immune system, brought on by both the disease and the treatments, is responsible.

The U.S. Food and Drug Administration has not taken an official position on any state’s right-to-try law but has said it works “with companies to provide patients access to experimental therapies through enrollment in clinical trials or through the expanded access provisions described in the FDA’s statute and regulations.” But both options are challenging at best. Clinical trials are controlled by the drug makers and have been historically difficult for doctors to get their patients into. The expanded access program, meanwhile, provides patients with another avenue through which to pursue experimental medications. And in theory it works exceptionally well. Acceptance has been almost universal: The FDA has, on average, rejected only about six of the approximately 1,040 annual applications it has received since 2009. But getting a patient into the program has long been an incredibly time-consuming, labor-intensive proposition, with doctors facing up to 100 hours of work to complete an application.  

In that regard, observers like Dr. Gorski and Professor Ikemoto say pressuring or even weakening the FDA is what these bills are really all about. If that’s the case, it might be working. In February the FDA announced a new compassionate-use application process that promises to take doctors just 45 minutes to complete. But as of this writing that process is still only under consideration and nothing has actually changed. Even so, in another statement released this spring the agency says it “is strongly supportive of appropriate use of expanded access protocols and stands ready to work with companies that are interested in providing access to experimental drugs.”

“On the one hand, I would absolutely spend every penny I had if it means I would live. What good are assets if you are dead? But then I also think of quality of life. If an experimental drug lets me live three months longer but also makes me a lot sicker during that time, what is the point?” Tom Ikeman, owner, Fantastic Produce, and non-Hodgkin’s lymphoma survivor

Still, Gorski doubts the FDA will ever concede much. “Why should they?” he asks. “Drug approval is in their purview, not the states.’ If you were the FDA, would you surrender your authority in this area for no practical reason?”

It is in many ways a moot argument. To date, not a single person has obtained an experimental drug via a state right-to-try law, and it is unclear if anyone ever will. Ikemoto notes it is illegal for pharmaceutical companies to ship non-FDA approved drugs across state lines, which makes supplying them problematic. And a drug company risking the possible long-term financial success of a product it may have already poured millions of dollars into by allowing people to access it before it has been fully vetted seems unlikely.

Sascha Haverfield, vice president of scientific and regulatory affairs for the Pharmaceutical Research and Manufacturers of America, the drug maker’s main lobbying group, said as much in a statement in March, noting PhRMA has “serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration and clinical trial process, which is not in the best interest of patients and public health.”

Still, Kurt Altman of the Goldwater Institute believes that could change. “Sure, a company won’t risk huge amounts of money to let one person try a drug in development,” he says. “But they might for a large number of people.” Which, he says, is exactly what will happen if states open the doors to accessing those medications.

“Is this going to fix millions of people tomorrow? No,” he says. “But it is a first step.”

Dr. Gorski says he understands that taking a position against giving the terminally ill a last gasp at life “is like opposing mom, apple pie and the American flag.” But the picture, he says, is so much bigger than that.

“In this situation, everything should be about the patient and only the patient,” he says. “The bottom line is that there is actually something worse than living with a terminal illness: cutting your life even shorter and maybe making it a lot more painful even than it would have been.”  

Comments

Mike Hackard (not verified)September 8, 2015 - 11:05am

This is an excellent article. The concept of a right-to-try law will be welcomed by many who are personally (or having a family member) facing a terminal illness. The article is well balanced and informative. The competing policy and moral considerations identified by the Goldwater Institute on the one hand and the surgical oncologist provide a broad framework for thought. Thank you for posting this article.

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