You may wish to check out the October 9, 2013 Sacramento Bee article by Anita Creamer, "Auburn woman’s quest for cancer treatment on hold as shutdown continues." In the Sacramento area, cancer patients are having their hope taken away by the government shutdown that strands cancer patients receiving experimental and other medicines from the NIH in similar clinical trials.
The article details how in the Sacramento area, an Auburn resident, a young woman and mother of a 19-month old toddler is fighting to get an experimental drug as a last resort to quell her rare cancer that is not responding to conventional treatment with chemo and radiation. Numerous cancer patients have been put on hold when the government shut down because they were in the middle of applying for participation in clinical trials with experimental drugs to halt their cancer. For them, it's the medicine of last resort, last hope. And now hope also has been put on hold. We live in an age when hope has been called the excuse for not taking action. But these patients were in the middle of taking action.
Cancer patients applying for help have to wait as government shutdown shuts them out of receiving acceptance into NIH experimental drugs being tested in clinical trials programs
Their medication didn't yet start, but they were in the process of applying or waiting to hear whether they qualified for experimental treatment in clinical trials for cancer drugs. These people had turned to the NIH clinical trials as their last resort. But now the NIH has shut down along with other parts of the federal government, except for those patients right in the middle of being treated with the new experimental cancer drugs being tested in the clinical trials. Most patients have turned to those clinical trials as their last resort.
The issue is the shutdown has caused some patients whose only hope is experimental drugs being used in clinical trails to be stopped cold. Many of those cancer patients are children. See the website, Change.org. Many cancer patients have turned to NIH clinical trials as their only chance of survival. Of that number, 30 are children, according to the NIH.
With the government on hold, those applying to the programs are put on hold and have to wait as hope runs out. Some have applied to the Compassionate Use programs. Check out the sites, Compassionate Drug Use - American Cancer Society and the site, Access to Investigational Drugs Outside of a Clinical Trial, or Compassionate Use Programs - The ALS Association.
Some turn to Compassionate Use programs allow desperately ill patients with no other options to be treated with medications not yet approved by the U.S. Food and Drug Administration. But compassionate use requires permission from the drug company – in this case, Bristol-Myers Squibb – as well as either the NIH or FDA.
As the government shutdown continues, the clinical trials needed to save many cancer patients' lives are also put on hold. Hope has to wait. But what happens if hope runs out before the government shutdown ends? The National Institues of Health has put new enrollment for its experimental drug programs such as clinical trials on hold until the government shutdown ends. Employees have been furloughed.
So cancer treatment gets put in hiatus, at least for those in the process of applying for treatment. For those already in the middle of their treatments, the treatments may continue. They're not stopped cold right in the middle of a treatment. But for those applying, hope has been put on hold. That's why patients will have to wait to find out whether they even qualify to be accepted into any government clinical trials program.
Cancer treatment programs are largely funded by federal government funding. As the NIH stopped, most patients already enrolled in experimental treatments are being allowed to continue in those studies. But for those in the middle of the application process, hope has bee placed on hold. Unfortunately, you can't tell a cancer that's spreading to shut down when the government shuts down. So hope again, is put on hold. For more information, check out the site, "Access to Investigational Drugs Outside of a Clinical Trial."
Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available.
Drugs that are being tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. These drugs are generally available only to people who are taking part in a clinical trial (a research study that is testing the drug). Being able to use one of these drugs when you are not in a clinical trial has many names, but is most commonly referred to as compassionate use. Also see the site, "What is compassionate use of experimental drugs? - MayoClinic.com."
Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions. The FDA first approved investigational drugs to be used in this way for critically ill patients in 1987. For people who aren’t in clinical trials, there are 2 ways a drug company can allow use of their unapproved drug: through expanded access programs (EAPs) or through single patient access.
A company sponsoring a drug in the late stages of drug development, such as Phase III clinical trials, can offer expanded access programs for patients who are not able to enroll in a clinical trial. (See Clinical Trials: What You Need to Know to learn more about the phases of clinical trials.)
The FDA generally approves these EAPs if the drug has shown that it works at least somewhat to treat cancer in the clinical trials that are being done. This can allow a lot of people access to the unapproved drug, as long as they meet the requirements of the EAP.
Single patient access
Patients who don’t qualify for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access, says the Compassionate Use website. In this case, the patient’s doctor must first ask the drug company if the drug can be used for the patient and see if the drug company will supply it. If the company agrees, the patient’s doctor works with the drug company to ask the FDA to approve the drug for use by this one patient.
The FDA requires the doctor to send information about the patient, why the request is being made, the proposed treatment plan, and a signed informed consent from the patient (see the site's document Informed Consent for more on this). The length of time it takes to get single patient access varies. But if it’s an emergency, the FDA can complete the paperwork in 24 hours.