"Compassionate Use"... "expanded access studies"...etc.

[JLS1]

I've discovered a few drugs in clinical trials that sound like they might be the Hail Mary my husband needs now, so I asked our MO if he may be able to help us get into a clinical trial, or help us via "compassionate Use", etc..... Sadly, and no surprise, he basically told us no. (is this due to hospital protocol...liability concerns, etc?? - or just being unwilling?)

I thought this info might be helpful to others here. Note how it says your MO should be helping you with this.

Note also that Patrick brought this up 2 years ago: healthunlocked.com/advanced......

mayoclinic.org/healthy-life...

I've heard that compassionate use is a way to get access to experimental treatments. How does it work?

Answer From Timothy J. Moynihan, M.D.

In certain situations, the Food and Drug Administration (FDA) allows companies to provide their experimental drugs to people outside of clinical trials. This is referred to as compassionate use. But getting access to not-yet-approved drugs through a compassionate use request can be a long and challenging process.

If you're interested in trying an experimental treatment, talk to your doctor about your options. For you to receive an experimental drug through the compassionate use program, your doctor must contact the drug company and then submit an application to the FDA. For the FDA to consider your request, you must meet certain criteria:

Your disease is serious or immediately life-threatening.

No treatment is available or you haven't been helped by approved treatments for your disease.

You aren't eligible for clinical trials of the experimental drug.

Your doctor agrees that you have no other options and the experimental treatment may help you.

Your doctor feels the benefit justifies the potential risks of the treatment.

The company that makes the drug agrees to provide it to you.

To find out more about the rules regarding compassionate use, visit the FDA website and search for "access to investigational drugs."

Another way to get access to experimental treatments is through expanded access studies. In these studies, experimental drugs in the later stages of clinical trials are offered to people who don't qualify for the clinical trials. To find out if a drug is available this way, contact the drug's manufacturer. Or go to ClinicalTrials.gov and search for "expanded access studies."

As you consider whether to try to obtain an experimental treatment, it's important to keep a few things in mind:

You aren't guaranteed to benefit. Experimental drugs haven't been approved by the FDA, and their efficacy may not yet be proved.

The risks of the drug may be unknown. Experimental drugs may not have been fully tested, so the range of side effects may be unknown.

Some companies don't give access to experimental drugs. Drug companies aren't required to comply with your request for an experimental drug. The company you ask could refuse your request.

Your doctor may not agree with your request. Your doctor might be unwilling to pursue your request if he or she thinks an experimental drug is dangerous or ineffective for your condition. You can ask for a second opinion from another doctor or seek advice from groups that advocate for people with your disease.

You may pay out of pocket for experimental treatment. The drug company may charge you for the experimental drug. Also, your insurance company is unlikely to pay associated costs of your treatment, such as fees for your doctor to administer the experimental drug and monitor side effects.

Getting an answer may take time. Unless your situation is an emergency, the review process may take some time. Because each compassionate use application is decided on a case-by-case basis, there is no set timeline and no one can predict how long you'll wait for an answer.

[tallguy2]

Thank you for sharing this. I hope to never need the "compassionate use" pathway given how laborious the process appears to be. Of course, it must be difficult for the doctors and drug companies given our litigious society.

[AlanLawrenson]

I arranged for my brother to get access to the late stage PCa drug Veyonda (Noxopharm Ltd) on compassionate grounds. It has successfully completed Phase 1b trials in Australia (DAART1 trial). Its protocol is SBRT radiation of ONE of the tumours followed by the drug over eight days.

I contacted the company directly. (I had previously met their CEO who made a full (?) recovery previously using this protocol. A DAART 2 trial will start in the USA, UK and Australia in late 2020.

I know that the company has supplied a good few men with Veyonda on compassionate grounds (some overseas men). Sadly, my brother's MO team were not prepared to authorise his having the drug as it "did not meet the approved standard of care". The head MO was the chair of the treatment guidelines committee here. We found doctors who were prepared to authorise its use, but it meant moving him to another hospital. By that stage he had full leg paralysis and had PCa in his bone marrow. Sadly, he died on the 22nd January after a 20 year battle with the disease. He was one of the early Lutetium -177 patients in Australia, which kept the disease at bay for a further two years of quality time.

I discuss Veyonda in my latest PCa book available from Amazon.