February 24, 2015

FDA OKs Faster, Simpler Access to Unproven Drugs for Terminally Inn Patients

Earlier this month, Dr. Peter Lurie – the Food and Drug Administration’s associate commissioner for public health safety and analysis – announced a much simpler and faster process for doctors to get unapproved, experimental drugs for their patients who have serious or life-threatening diseases and no other alternative.

While a 60-day interval for comments must pass before the revolutionary process officially becomes effective, Lurie made it clear the FDA will allow doctors to submit requests according with the new policy now.

In the past, doctors needed about 100 hours to assemble applications for experimental drugs for a qualifying patients. They were required to supply 26 different kinds of information and seven separate attachments.

Now, the process will take less than one hour, requiring doctors to give only eight pieces of information, with only one attachment. They must provide the reason they want to use the experimental drug, along with the patient’s clinical history. 

In sharp contrast to a gridlocked Congress mired in partisan politics, the FDA slices away layers of red tape for sick and dying patients who have no other options. It seems a triumph of good sense, offering hope for terminally-ill people.

Conditions Still Apply
The process, while much simpler, still carries certain requirements:
  • There must be no other product that can diagnose, monitor, or treat the patient’s condition,
  • The patient may not be already involved in some clinical study testing the requested drug,
  • Doctors must show that the risk of using the unapproved drug doesn’t exceed the risk posed by the disease it is designed to treat,
  • Doctors must guarantee that the drug’s manufacturer is willing to provide it.

Even when all conditions are met, the FDA can’t force a manufacturer to supply the requested drug. It simply provides guidance on how to do it.

The trend toward simplification began in the early years of the AIDS crisis, when the FDA began considering “compassionate use” of experimental drugs whose effectiveness and safety had not been shown. But as that new approach broadened, it became much more complicated.

States’ “Right to Try” Laws
The greatly simplified FDA process seems to follow a campaign by the Goldwater Institute, a small-government group that considered the FDA overprotective of dying patients with little to lose by trying unproven drugs. The institute encourages states to pass “right to try” laws that greatly simplify the process for doctors to obtain experimental meds for dying patients.

These laws make an experimental drug available if it has passed only one of the FDA’s three testing phases – for safety. If it has, these state laws then remove the FDA from the process completely, and doctors can request experimental drugs directly from manufacturers. Still, these laws do not compel drug companies to supply their products or insurers to pick up the tab.

So far, “Right to Try” is now law in five states: Colorado, Louisiana, Michigan, Missouri, and Arizona. At least 26 other states have legislation pending. These laws have not revolutionized the process; to date,
they have apparently not yet helped any patients gain access to unapproved drugs, although the laws’ boosters seem confident that dying people will benefit eventually.

The Right to Try movement doesn’t have universal support. The FDA has not taken a position, and encourages doctors to use its new, simplified guidelines to obtain unapproved drugs for patients who qualify.

Some drug companies would prefer that the FDA remain part of the process. Critics of the new state laws argue that the FDA will know a lot more about the possible risks and benefits of unapproved drugs. 

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Among the many sources that covered this story:


Betty Owen said...

This comment is in reference to your article about the 3 supplements you take, but I couldn't find a place to leave a comment on that article. You mentioned that the PureEncapsulations Ashwagandha contained 12.5%, but according to the labels it's 2.5%. Are you going by what Consumer Labs reported or where are you getting your information?

John Schappi said...

I've reread the Pure Encapsulation label, the Consumer Labs report and my blog post. All say the same thing but it is confusing. I don't knows if this will make it any clearer, but here's what it boils down to:
Each pill contains 500 mg of Ashwagandha.
25 mg of that -- or 12.5% -- is withanolides.

Knowing nothing about withanolides, I read what Wikipedia had to say about it. I still don't know much about it.

Betty Owen said...

Thanks for your response. I was reading the label from the photo of the product online, so maybe it's not accurate. I'm sure you're looking at the label on an actual bottle of the product.