A Political Activist Challenges the Drug Approval Process and the U.S. Government

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Title: The Right to Try: How the Federal Government Prevents Americans From Getting the Lifesaving Treatments They Need

Author: Darcy Olsen

Publisher: Harper Collins

Publication date: November 2015

Price: $26.99; hardcover, 320 pages

The U.S. Food and Drug Administration’s (FDA’s) primary concern in the drug approval process is to ensure that the drug is safe and effective. For the past several decades, the advocacy groups have vociferously painted the agency as a stodgy bureaucracy that prevents desperate patients access to experimental therapies. Policy advocate Darcy Olsen takes this thorny issue head on in her new book, The Right to Try: How the Federal Government Prevents Americans From Getting the Lifesaving Treatments They Need.

Numerous health-care wonks have tackled this theme, but Ms. Olsen puts a face on the issue by introducing most of the book’s chapters with compelling stories of patients facing life-threatening diseases who—as presented by the author—have run into a brick wall called the FDA in their pursuit of experimental therapies. The FDA’s response to this ongoing complaint is that even in the case of someone facing a terminal illness, approving a drug that doesn’t work is simply giving that patient a toxic placebo. In fact, the FDA currently has a Compassionate Use policy that allows patients with dire diagnoses access to drugs that have passed phase I clinical trials and have been judged safe. It is however, an onerous process burdened by massive amounts of paperwork.

Compelling Patient Stories

In her introduction, Ms. Olsen describes a young man, Ted, who is diagnosed with amyotrophic lateral sclerosis (ALS); his doctor tells him there is no cure for this disease. Ted is a fighter and pursues an experimental phase I trial, in which stem cells are implanted into the spinal column. The difficult and dangerous procedure went off without a hitch, and to the wonderment of his surgical team, Ted’s symptoms began to disappear. And for about 10 months, Ted was essentially asymptomatic. His doctors were astounded by his recovery.

However, Ted’s symptoms returned, and he underwent another stem cell transplant, which once again worked. But according to the author, Ted was now barred from further stem cell treatments because the trial had moved to phase II testing, disallowing participation by those who had been in the phase I trial. Understandably, Ted was angry and crestfallen. In his mind, the system’s rules, ostensibly devised for patient safety, were sentencing him to slow death.

This and other similar cases caught Ms. Olsen’s attention, and she set out to challenge the FDA and the federal government. Ms. Olsen, a woman of considerable moxy, is the President of the Goldwater Institute, a national policy and litigation organization. “I am a Goldwater conservative and a firm believer in federalism, the constitutional authority and responsibility of the states to check and balance Washington’s overreach,” writes the author.

A Tale of Political Action

And so, gripping patient case histories aside, this book is first and foremost a tale of advocacy and political action, which is a storyline woven into the fabric of the oncology community. On the surface it seems to be a no-brainer. Patients facing a deadly disease should have the option to undergo an experimental therapy. But there are two conflicts involved: usurping the federal government and putting vulnerable patients at risk of doing more harm than good by allowing them access to toxic drugs that confer no benefit.

Undaunted by the uphill battle, Ms. Olsen launched into a difficult state-by-state campaign to have Right to Try (RTT) laws passed. The law bars the federal government from prohibiting or restricting the production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biologic product, or device that is (1) intended to treat a patient who has been diagnosed with a terminal illness; and (2) authorized by, and in accordance with, state law.

“The first Right to Try law passed in May 2014. In little more than a year, RTT laws have passed in 24 states and counting…that is a legislative record,” Ms. Olsen writes. Whether or not one agrees with the law, Ms. Olsen has tapped into the frustration of the lay public and many of its elected officials. And her messaging is crafted to grab attention and sharpen the issue down to its core component: The FDA and sluggish federal government are preventing Americans from receiving the lifesaving treatments they need. Well, if only it were that simple.

Ms. Olsen writes: “Do you remember what your cell phone looked like 15 years ago? If you showed that big hunk of plastic with a large stub antenna to your kids today, they would laugh at how out-of-date it was…. Well, if you wouldn’t use your 15-year-old cell phone technology to make a call, why should you be forced to depend on 15-year-old technology to save your life? Because that’s exactly what you are getting when you put the latest FDA-approved drug in your body.”

A Reductionist by Nature

While reading her book, it becomes obvious that Ms. Olsen is a reductionist by nature. For instance: “Today, doctors and scientists routinely come up with novel ways to treat disease only to run into delays at the FDA.” But they are complex issues that are not well served by making the FDA the bad guy. To her credit, she lists six anticancer drugs that took an exorbitant amount of time to reach approval status, and she backs it up with data. Moreover, many well-known physicians in the oncology community, such as Vincent DeVita, MD, have publically criticized our clinical trial and Darcy Olsonal system.

New Territory for Change

In her 10-chapter book, a bit too much is devoted to personal case histories. By chapter seven, Ms. Olsen devotes her energy to giving a full account of our flawed approval system and strikes some new territory for change. That said, she fails to acknowledge the real concerns of those who oppose RTT laws and those, like Ms. Olsen who have politicized this into a state’s right issue.

Today, doctors and scientists routinely come up with novel ways to treat disease only to run into delays at the FDA.
— Darcy Olson

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Opponents of these laws feel they undermine the clinical trial infrastructure regulated by the FDA, which was developed to ensure that medical products are tested on a sufficiently diverse population in a scientifically sound manner to bring safe and effective products to market. By allowing anyone to obtain an experimental treatment outside the confines of a regulated clinical trial, RTT laws reduce incentives to participate in clinical trials, increasing the complexity of recruiting sufficient study participants.

Ms. Olsen has laid the gauntlet down. This is not a Blue State, Red State issue. California Governor Jerry Brown recently vetoed a RTT law passed by the state legislature. Interestingly, he has signed bills for the legalization of marijuana and the Right to Die Act. Ms. Olsen says, “If people have a right to die should they not have a right to try.”

It is only a matter of time before the courts attempt to strike RTT laws down, making the case that these laws offer vulnerable patients misplaced hope and do not attempt to minimize the serious health and safety risks inherent in treatment with unproven treatments. There’s a lot at stake here for the oncology community, which depends on the drug pipeline more than any other specialty. Despite some grandstanding and polemics, The Right to Try, especially the last three chapters, is recommended for readers of The ASCO Post. ■