Should age matter when requests for compassionate use are considered? Are children less likely or more likely to be granted access to a drug? Should age be excluded from consideration, especially in circumstances when there is not enough drug supply and tough decisions about allocating drug must be made? Experts on compassionate use ponder these questions to ensure equity in access to investigational drugs.
For kids with cancer, age does matter, and it should be taken into consideration in reviewing applications for compassionate use. It matters not only because when a child dies, she or he loses over 70 years of life. Age matters not because cancer, which is the #1 killer of children, is somehow more important than other childhood diseases – it is not. Many other diseases are painful, debilitating, and deadly. Age matters because while huge strides are being made in cancer drug development, children are being left behind. There are almost no clinical trials of new drugs for kids with cancer. Nearly all new cancer medicines are studied and approved first in adults. It takes many years after a cancer drug is approved for studies to be conducted in children, so the drugs to treat kids are dozens of years old. This is an eternity in a field like cancer which is being rapidly transformed by new drugs. For kids with cancer, a clinical trial is not just a research vehicle or a step along the path to approval. Trials are the most important way to get treatment for kids with cancer who have run out of options. When a child applies for compassionate use, it means this child cannot participate in a trial. The drug manufacturer needs to be made aware of the patient’s age when considering a compassionate use request.
The patient’s age is also important for keeping statistics, which are currently missing, on how many kids need compassionate use drugs. If we know how many children are applying for compassionate use access, FDA will know if there is a lot of demand for a particular drug and should suggest that a company open an expanded access trial for children.
In the past, physicians used FDA Form 1571 to request compassionate use, and many failed to list the applicant’s age, making it difficult for FDA to know how many pediatric requests there were for a particular drug or overall. This year, a new FDA form (Form 3926) was created to simplify the process of submitting compassionate use requests. However, the new form asks for a patient’s age, weight, gender, diagnosis, and clinical history all on one line, which makes it easy to again miss the question on age.
To ensure that this data is collected in the future, Form 3926 should be changed to make the patient’s age a separate question. Age should not be overlooked.