New Goldwater Institute Investigation Shows FDA ‘Compassionate Use’ Process Leaves Little Hope for Dying Patients

Contact: Starlee Coleman (602) 758-9162

Phoenix—The federal government has a process to allow terminally ill Americans who have exhausted all government-approved treatments to try treatments that are still under review by the Food and Drug Administration. But a new investigation by the Goldwater Institute shows the entire system for gaining access to unapproved medications is so rigged with bureaucracy and disincentives that it is bound to fail in most cases.

The report, “Dead on Arrival: Federal “compassionate use” leaves little hope for dying patients,” shows the FDA’s process for applying for compassionate use is choked with cumbersome paperwork that is difficult for doctors to navigate, and is loaded with disincentives that prevent drug companies from making investigational treatments available to dying Americans. It is a system of all risk and no reward.

As a result, only about 1,200 Americans a year qualify for treatment through compassionate use, even though nearly 600,000 die annually of cancer alone.

FDA officials insist the current system works well and the agency is not an impediment to terminal patients getting the care they need, because the agency approves 99.5 percent of the applications it receives for compassionate use.

But critics say the FDA’s numbers are meaningless. No one knows how many requests for compassionate use drug companies receive and reject because of the risks the FDA has created for drug companies that participate. No agency is required to keep or report that information.

“Can you actually believe that only 1,200 dying Americans want to live badly enough to find a legitimately applicable, unapproved therapy and ask to get it?” said Carla Mann Woods, formerly a medical device industry executive, and now a board member of the Alfred E. Mann Institute for Biomedical Engineering at the University of Southern California.

“If you deliver the application at the top level of Mount Everest, they will approve it,” said Garo Armen, chief executive officer of Agenus Inc., a small biopharmaceutical company developing immunotherapies to help treat cancer and other diseases. “The FDA will do the approval process, but everything that needs to be put into place [first], which is an FDA requirement, makes the process very onerous.”

Twenty-four states have adopted a law, called Right To Try, that allows terminal patients and their doctors to work directly with a drug company that has a product in clinical trials to access that product without using the FDA’s compassionate use process. The law is under consideration in 20 additional states.

The bill has become law in almost every state where it has been voted on; California is the only exception. In 2015, the California Legislature passed Right To Try with bipartisan, near unanimous support. But California Governor Jerry Brown vetoed the bill, saying he wanted to give the FDA more time to reform the compassionate use process. In February 2015, the FDA proposed revisions to its application process, but those changes still have not been adopted.

The new Goldwater Institute report shows that even if the FDA streamlines its application, the rest of the process is set up to discourage pharmaceutical companies from participating in compassionate use requests.

“For drug companies, agreeing to compassionate use is all risk and no reward,” said Mark Flatten, national investigative reporter at the Goldwater Institute and author of the report.

Because of the way the FDA designs clinical trials, if a patient treated under compassionate use does well and begins to recover, it does nothing to help the drug’s approval chances. Bad news, however, does count. All major adverse events, even the death of an already dying patient, must be reported to the FDA if they occur in compassionate use cases. When this has happened in the past, the FDA has responded by shutting down clinical trials—even if the death wasn’t related to the drug.

This can be a major financial problem for small drug companies that are doing most of the research and development of new treatments. Adverse events, even in compassionate use cases, can wreck a company’s stock, especially if the FDA responds by suspending clinical trials. Because the FDA does not guarantee that adverse events stemming from compassionate use cases will not be held against the company providing the investigational drug, there is little incentive for the companies to do so.

Read “Dead on Arrival: Federal “compassionate use” leaves little hope for dying patients” here.

About the Goldwater Institute

The Goldwater Institute drives results by working daily in courts, legislatures and communities to defend and strengthen the freedom guaranteed to all Americans in the constitutions of the United States and all 50 states. With the blessing of its namesake, the Goldwater Institute opened in 1988. Its early years focused on defending liberty in Barry Goldwater’s home state of Arizona. Today, the Goldwater Institute is a national leader for constitutionally limited government respected by the left and right for its adherence to principle and real world impact. No less a liberal icon than the New York Times calls the Goldwater Institute a “watchdog” that plays an “outsize role” in American political life.