How a Simplified ‘Compassionate Use’ Form Could Save Your Life

Compassionate UseHow a Simplified ‘Compassionate Use’ Form
Could Save Your Life


Five years ago Mark, a 67-year-old epidemiologist, came into my lung fibrosis clinic dragging an oxygen tank behind him. He was seeking my guidance about pirfenidone, a medication approved for “compassionate use” in some European countries and Japan but not the U.S.

A week prior to his last visit he brought a Ziploc bag full of a white powder, claiming that he was able purchase pirfenidone online. He wanted to know if I thought it was safe but I was only able to offer an ear, love and compassion. I discouraged him from taking this unknown “white powder” but he was determined to use his $10,000 investment. It was, he said, his last resort.

Mark died a week later, a mere 18 months before pirfenidone was approved by the U.S. Food and Drug Administration

In my practice, I am sometimes faced with a very sick patient like Mark who has run out of options. There is no medication, procedure or cure on the market approved by the FDA that can save them. In trying to help them I sometimes identify a potential treatment that might work but because it’s still in the experimental stages, we aren’t allowed to try it. Even when it is made available, it is often financially out of reach.

Last month I saw a glimmer of hope for such patients when the FDA announced that it is simplifying its compassionate use form that physicians of terminally ill patients must complete when requesting unapproved drugs and other medical treatments.

The compassionate use program – sometimes referred to as expanded access — was implemented in the 1980s at the dawn of the AIDS epidemic. It allows the physicians of patients in dire situations to petition for drugs not yet cleared for use by the FDA which may be nonetheless safe and effective for their condition. In many cases, it is a patient’s last resort.

I don’t believe every patient should seek a miracle cure. But some would truly benefit from trying a drug that may otherwise be years away from availability.

The newly streamlined process can literally make the difference between life or death. The old application could take up to 100 precious hours to complete and required up to eight exhibits. The new form will only take about 45 minutes to fill out and requires a single attachment, the agency promises.

There is one other piece of good news. Under the new compassionate use guidelines, pharmaceutical companies will have limitations placed on what they may charge for experimental drugs. For example, they will be able to pass along manufacturing costs but they won’t be allowed to tack on heavy administration fees. Private and government insurance programs aren’t obligated to subsidize compassionate use but the revised rules should remove at least some of the obstacles that have led to rejected claims in the past.

The shorter form is just the start however. The compassionate use program still has a long way to go. Challenges remain for the the vast majority of patients with terminal illness trying to get their hands on promising investigational treatments.

Until the government overhauls the archaic and at times inhumane “right to try” laws, patients are still forced to beg for their own lives. And their physicians have little power to help them.

Dr. Ferrer has been my colleague for many years and his wealth of knowledge continues to impress me. He always strives to learn more and stay on the cutting edge in his field.”

Dr. Jeremy Robertson

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