Patients spend their last days pleading with reluctant drug companies and the FDA to get access to treatments that could save their lives.
By PAUL HOWARD – Wall Street Journal
Nov. 12, 2015 7:52 p.m. ET
In the late 1950s and early 1960s, thalidomide was used as an anti-nausea drug by tens of thousands of pregnant women in Europe and Japan, some of whose children developed severe birth defects. Although thalidomide was never approved by the FDA, millions of free tablets were given to doctors as part of company-sponsored clinical trials, and 40 American children were born gravely affected. Not surprisingly, this led to a backlash against the drug industry. In 1962, Congress passed the Kefauver-Harris Amendment, giving the FDA power to require drug makers to demonstrate not just that drugs were safe for human use, but that they effectively treat the symptoms they say they do.
The upshot of this tragic episode is that the current FDA can be excessively risk averse, slowing drug development by demanding copious amounts of testing from producers to rule out rare problems. There’s no doubt that clinical trials have become much more complex and expensive: From just the late 1990s to mid-2000s, researchers at Tufts University estimate that the average length of a trial increased by 70%. In 2014, Tufts estimated that it costs $2.6 billion to bring a single new medicine to market, up from just over $1 billion in 2003. The drugs that do eventually reach market take longer and cost more.
In her new book, “The Right to Try,” Darcy Olsen, president of the Goldwater Institute, illustrates the terrible cost of this predicament. She focuses on the plight of terminally ill patients navigating the FDA’s dense clinical trial regulations, pleading with reluctant drug companies and trying to convince paternalistic bureaucrats to grant access to medicines that could mean the difference between life and death. Ms. Olsen does an excellent job of interweaving the technical details of drug development and clinical trial design with deeply moving stories of patients dying from cancer, Lou Gehrig’s disease and other devastating ailments.
The FDA has a “compassionate use” policy, by which terminally ill patients may access experimental treatment. But a very wide gap persists between official policy and patient need. Ms. Olsen notes that “today, about 40 percent of cancer patients attempt to enroll in clinical trials, but only about 3 percent end up participating. That means that the vast majority don’t make the cut, whether because they fail to meet the strict criteria, or a trial is thousands of miles from their home.” Many of those who don’t get these experimental drugs are the sickest patients because they are deemed “too sick to be useful for the study.”
Ms. Olsen argues that terminally ill patients should be able to access such drugs—at their own risk and outside the context of FDA-required studies—if the companies are willing to provide them, and the book’s title alludes to her proposed remedy: the state-by-state campaign the Goldwater Institute is leading to pass “Right to Try” legislation. The bills would allow terminally ill patients who have “exhausted all conventional treatment options” to access an experimental treatment if their doctors believe it is “the best medical option to extend or save the patients’ life” and “the treatment has successfully completed basic safety testing and is part of the FDA’s ongoing evaluation and approval process.” Insurers, critically, would not be required to cover the treatment—a significant hurdle, largely unexplored here, since such costs could be significant.
The think tank’s campaign has been incredibly successful, with 24 states passing Right to Try laws to date. Still, Ms. Olsen doesn’t present such laws as a panacea. She doesn’t expect experimental treatments to always—or even often—work for terminally ill patients. But she believes that some chance is better than the alternative. “If you have the Right to Die, you have the Right to Try,” Ms. Olsen writes. “And you don’t have to wait on Washington to secure it.”
Yet therein lies the book’s main shortcoming. Washington, it turns out, has a fair bit of say here. Courts have found that the FDA’s powers to regulate drug development are extraordinarily broad. Many changes Ms. Olsen champions won’t be possible without congressional action to revamp the FDA’s drug development process and find new ways of paying for experimental drugs that would make widespread access sustainable for patients, companies and insurers. These issues, though touched on, are not grappled with in detail.
The FDA has taken some positive steps. The Breakthrough Therapy designation, for example, created by Congress in 2012, has allowed the agency to approve promising drugs for life-threatening ailments based on small, early-stage trials. But this doesn’t let the agency off the hook. Scientists are increasingly recognizing that even common diseases like cancer are made up of hundreds of distinct genetic variants. The challenge ahead will be to match many more medicines to these targeted populations—a strategy called precision medicine—while sharply reducing the time and cost needed to bring them to patients.
I would go even further than Ms. Olsen does here. By focusing only on terminally ill patients, she’s overlooked that at some point everyone will become a patient. Rapid advances in inexpensive whole-genome sequencing tests, like 23andMe, are already allowing individuals to peer into their own medical futures and, even more powerfully, those of their children. We may not be far from a world where medical problems—from Alzheimer’s to cancer—will be identified while patients are still young and healthy enough to demand dramatic reforms to how medicines are researched and tested. The right to know our own medical futures may become even more important than the right to try.
Mr. Howard is a senior fellow at the Manhattan Institute.