Miracle Drug
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SICKENING: FDA BUREAUCRACY BLOCKS COMMON “MIRACLE DRUG” By Mark Flatten National Investigative Journalist for the Goldwater Institute ow do you tell a 7-year-old child she are facing the inevitable reality that we will not must go back on a feeding tube? be able to get it soon. C assie Le is facing that terrible “I fear she will need to get a feeding tube question. placed back in,” Le wrote. “How do I tell her H Her daughter spent the first that? How would you as a parent tell your child seven months of her life unable to eat without ... ‘sorry the medicine that has helped you to eat vomiting. The doctors ran the usual tests and and enjoy eating is no longer available so now tried the usual medications. you can’t eat ... you will get fed to your intestine Nothing worked. by a feeding tube.’ She is a little girl who has The girl was put on a feeding tube when worked extremely hard to overcome her fears she was 7 months old. It helped some, but she of eating and now her joy of being a normal continued vomiting regularly. little girl will be taken away.” “Feeding became something she feared,” Le Le said she is comfortable with the of Texas said in a May 2016 email describing her Goldwater Institute quoting from her email. daughter’s condition. However, she did not want to be interviewed Finally, doctors tried domperidone, a because of concerns for her daughter’s privacy. medication widely used throughout the world The child’s name is not being used. to treat gastric conditions like those afflicting “She has been able Le’s daughter. It worked. The feeding tube was removed about four years ago, and since then ‘SERIOUS OR LIFE to eat because of the girl has been able to eat normally. That is coming to an end because of new THREATENING’ Domperidone. Now we rules restricting the availability of domperidone imposed by the U.S. Food and Drug illions of people worldwide have are facing the inevitable Administration. regularly taken domperidone since it Domperidone is not approved to treat any M was first developed and marketed in reality that we will not condition in the United States. Despite that, 1978. It is a common treatment for a variety of it was relatively easy to obtain until recently gastric disorders, nausea and vomiting. through compounding pharmacies, which were The most serious condition treated with be able to get it soon.” able to legally get it from international suppliers domperidone is gastroparesis, a potentially and blend it into compounds to fill prescriptions deadly condition in which food basically sits – Cassie Le – from a doctor. in the stomach rather than being digested and But in the past couple of years, the FDA passed normally. Patients with gastroparesis has clamped down, restricting access and suffer from a variety of conditions, including importation of the drug. Doctors and pharmacies severe and uncontrolled vomiting, bloating and risk punishment from the FDA if they prescribe abdominal pain. It can lead to other conditions or provide it to patients. such as malnutrition and dehydration. So people like Le’s daughter can no longer The FDA classifies gastroparesis as a legally get it, except through an FDA program “serious or life-threatening” condition. It is the known as an Expanded Access Investigational condition Le’s daughter suffers from. New Drug (IND) protocol. The agency refuses to The agency’s concern about domperidone say how many people are receiving treatment is that it may cause a condition called QT through that program. No one younger than 12 prolongation. This is a pause in the electrical years old can qualify, so Le’s daughter would not buildup in the heart between beats. be eligible. There are conflicting studies about how “She has been able to eat because of much, if any, risk of QT prolongation can be Domperidone,” Le wrote in an email to the FDA linked to domperidone in patients who are as part of a public comment process. “Now we properly screened and monitored for heart 2 • DOMPERIDONE • RIGHT TO TRY ACT RIGHT TO TRY ACT • DOMPERIDONE • 3 complications. Even the FDA acknowledges the process by which a pharmacist mixes drugs, get out of bed. I’m so sick I can’t do anything. connection between domperidone use and heart chemicals or other products to tailor a medica- But when I take it, I have days that I can go complications is “not well characterized.” tion to an individual patient. watch my grandkids swim or play soccer or There are about 150 FDA-approved drugs Since only small quantities of compounded whatever. As far as I am concerned, that’s the on the market that have been linked to QT medicines are produced, they were treated risk-reward balance that I have to take.” prolongation, including cardiac drugs. differently than new drugs because it would Beener was a software consultant living in Only one drug is FDA-approved for treatment be unfeasible to go through the rigorous FDA Florida when the symptoms of gastroparesis of gastroparesis — metoclopramide, which is testing regimen for each unique compound. first surfaced about 2001. Her job required sold under the brand name Reglan. However, The FDA left the regulation of compounding extensive travel. One day she started to Reglan is only effective in about 40 percent of pharmacies to the states. experience uncontrolled vomiting. It got so the patients with gastroparesis, and it can cause For domperidone users, that meant bad that she routinely carried plastic bags and a serious and permanent disorder characterized compounding pharmacies still were able to paper towels on business flights, knowing she by uncontrollable muscle movement in the lips, obtain the drug through international sources would likely vomit while in the air. tongue, eyes, face, arms and legs. and compound it to fill prescriptions for “I threw up all across this country,” Beener Domperidone is available in more than 100 individual patients. said. “I was in hospitals all over the country.” countries, including those in the European Union, But Congress changed the law in 2013, Doctors tried Reglan, which worked for a Canada and Mexico. In many of those countries, clarifying the FDA’s power to fully regulate all while. But within about five months she suffered it is available over the counter. The prescription products made through compounding. The severe depression and was suicidal. It got so requirement was imposed only recently in the FDA responded by aggressively enforcing bad that her husband hid her pills. United Kingdom and some other European its import ban on domperidone, effectively When Beener stopped taking Reglan, the nations. In Canada, about 2 million prescriptions closing down the domestic market. The agency severe vomiting returned. for domperidone are written annually. initially attempted to halt importation of the A doctor in Georgia told her that he might In the United States, domperidone has drug in 2004, citing concerns it might be linked be able to get access to domperidone and never been approved by the FDA to treat any to heart problems and was being improperly warned her of possible heart problems. condition. prescribed to increase milk production in “I said I will take anything. I don’t care. I Before any new drug can be prescribed or lactating women. However, that ban was largely can’t live like this,” Beener said. sold in the U.S., it must be approved by the FDA unenforced until Congress clarified the FDA’s At first, domperidone was easy to get. after a series of clinical trials and agency reviews regulatory power over drug compounding in Doctors were willing to prescribe it and to determine whether it is safe for use in humans the 2013 law. compounding pharmacies would dispense it, and effective at treating the targeted condition. despite the FDA’s 2004 import alert. That process of getting FDA approval to market But by 2014, the FDA was warning a single new drug normally takes more than 10 WHATEVER IT TAKES pharmacies they could be shut down for years and costs drug companies in excess of $1 dispensing domperidone and doctors could billion. olleen Beener knows about the safety face sanctions if they prescribed it, said Beener, “I am choosing quality of life Janssen Pharmaceutical conducted clinical concerns associated with domperidone. who helped form a gastroparesis patient over quantity of life. I don’t trials and in 1985 submitted its new drug applica- C It is a risk she is willing to take because support group soon after she was diagnosed. tion to the FDA for approval. Those tests showed she cannot lead anything resembling a normal Beener still gets domperidone through want to spend my days in bed. domperidone was both safe and effective. How- life without it. other sources, but doesn’t want to say what ever, an FDA review panel had concerns the clini- Beener, 62, of Florida, said she has taken they are out of fear the FDA will close that And if I get 10 years less because cal testing was not robust enough and repeated- domperidone for 15 years, and she has never had supplier down. She has effectively been forced ly asked for additional studies. any heart problems associated with the drug. onto the black market because of the FDA’s of that, so be it. I want to enjoy Janssen withdrew its application instead. The “I am choosing quality of life over quantity actions, she said. exact date and reasons for the withdrawal are of life,” Beener said.