Change Compassionate Use guidelines to expand access for Omegaven.
This petition had 1,666 supporters
I am the mother of a 4 year old son who has a disorder known as short bowel syndrome. Short bowel syndrome is a condition in which nutrients are not properly absorbed (malabsorption) because a large part of the small intestine is missing or has been surgically removed. In my sons case, 90% of his small bowel was removed after an infection (necrotizing enterocolitis) destroyed most of the organ. Most of his nutrients are delivered via a central IV catheter because he does not have enough bowel to absorb what his body needs from food. The problem is that IV nutrition, TPN, is hard on the liver and can cause liver failure. My son only weighs 26 lbs at age 4 because his doctors cannot administer enough TPN to his body to keep it properly growing without causing further damage to his already compromised liver.
Doctors at Boston Children's Hospital have experimented with a drug called Omegaven, an Omega-3 form of IV fat derived from fish oil. Omegaven has been shown in some cases to reverse the liver damage caused by TPN and help achieve proper growth and development in these children. Because of the FDA's policy on "Compassionate use" for Omegaven, my son will not even be eligible for the drug until his liver is further compromised, putting him at risk for progressive liver disease, liver failure, or much worse. Currently doctors will not perform the clinical trials mandated by the FDA for approval of Omegaven. They are unwilling to risk the lives of the control group that would receive a placebo instead of Omegaven, and I applaud them for their choice. However I would like to see drugs like Omegaven made more available to patients like my son without their lives being placed at further risk.
IF the FDA would change their "Compassionate Use" guidelines to include patients with any degree of TPN related liver disease, this could change the lives and living conditions of many children and adults who suffer with this disorder. Then, after informed consent; the option to use Omegaven as a life saving treatment should be made available before there is further risk of of it being too late.
Please help me by signing my petition below and sharing it with others. We are asking the FDA to change their guidelines for "Compassionate Use," and to include patients with any degree of TPN related liver disease so that they might receive Omegaven. This will help change and SAVE the lives of so many who suffer needlessly. Thank you.
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