Dead On Arrival: Federal “Compassionate Use” Leaves Little Hope for Dying Patients
To watch the full hearing on Connecting Patients to New and Potential Life Saving Treatments click here via C-SPAN
YOU ARE DYING AND HAVE NO HOPE.
Your disease is 100 percent fatal. It’s only a short time before it kills you.
There are no treatments that have been approved by the federal Food and Drug Administration.
There is a new therapy that could save your life. But it is still being tested in people who have the same disease in rigidly controlled studies called clinical trials that you are too sick to qualify for.
It will be a decade or more before the new drug is available to your doctor. You will be long dead by then.
What are you willing to do, and how much risk are you prepared to take to try to save your life?
Those are questions thousands of Americans face every year after being diagnosed with a deadly disease for which there is no cure, at least none that has been approved by the FDA.
For them, their only chance at survival will be to get access to an innovative new drug before it’s too late.
It may be a faint hope, or even a false one. But it is their only hope.
The FDA’s compassionate use program is supposed to be that one last chance.
Formally known as expanded access, compassionate use is meant as a way to treat dying patients with medications that are still being tested in clinical trials and are therefore not otherwise available. Compassionate use must be requested by the patient’s doctor, endorsed by the company that makes the drug, and approved by officials at the FDA.
But an investigation by the Goldwater Institute shows that the entire system for gaining access to an unapproved medication is so rigged with bureaucracy and disincentives that it is bound to fail in most cases. Critics say it was designed that way, ensuring that only a tiny number of patients are able to navigate the complex, costly, and time-consuming maze that must be cleared just to file a compassionate use application for the FDA to consider.
The problem with the current system is not just that it takes doctors 100 hours or more to complete the application process for FDA approval.
Or that clinical trials take too long and cost too much.
Or that new cures for deadly diseases like cancer are typically being developed by cash-strapped small companies that risk financial ruin if they grant early access to their products to save the lives of dying patients.
It’s the way all of those things interconnect into an unworkable system that strips dying patients of their final option to save their own lives.
It is a system of all risks and no rewards.
And the lynchpin that binds it all together is the regulatory scheme created by the FDA.