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June 9, 2014 at 9:43 pm #22026RJoeyBParticipant
I’ve seen several posts here and elsewhere about “Expanded Access Programs” and “Compassionate Use”, including comments that they are not the same thing and along with suggestions that there needs to be changes to how patients with advanced disease (and out of treatment options) get access to experimental medications and treatments — I use the term “experimental” loosely to simply encompass any therapy not approved by the FDA (in the U.S.) I’m bringing this up as a separate thread not to start a debate, but to understand the issue better myself, and perhaps educate others as well. I mentioned the topic in another thread here not long ago, but am still not clear.
So, if they are not the same thing, what is the difference? If I search “fda compassionate use”, the first link at:
http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm ” class=”bbcode_url”> http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm
takes me to the FDA site, where they state in the first sentence:
Expanded access, sometimes called “compassionate use,” is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options.
According to the FDA, they’re the same thing. Other sources all state something similar. In my recent post, I wondered if perhaps there was a subtle difference wherein an “expanded access program” was a formalized way, authorized by the FDA, for a pharmaceutical company to offer larger scale access to an experimental treatment prior to FDA approval to patients in “no other option” scenarios, while “compassionate use” was the same situation, but on an individual, one-off basis, where the doctor and patient work with the pharmaceutical company and FDA to obtain individual approval. If that’s the difference, is the issue that the individual compassionate use scenario is happening less frequently, primarily due to resistance from the pharmaceutical companies to make their medications available on an individual basis?
I also noted that “expanded access” may be a less frightening term than “compassionate use”, sounding less like a last ditch effort for someone who is out of FDA-approved options. Using the current anti-PD-1 EAPs as an example, to me “expanded access” sounds less harsh to me, but the fact is that to qualify for the program, it is a “compassionate use” scenario insofar as the requirement is that other options (ipilimumab and, if appropriate, anti-BRAF) must have all been tried and failed. Is the issue with the anti-PD-1 EAPs that by having certain criteria for brain metastases that they are not truly “compassionate use”?
I’d like to understand the difference and issue(s) better, both so I can make my voice heard on the issue(s) and so I know what I’m up against if I should find myself in the situation where this suddenly becomes much more personal. Catherine, any insight you (or anyone else reading this) can provide is appreciated.
[Personal note: I’m not currently participating in or considering an anti-PD-1 trial or EAP. I was diagnosed at Stage IV in the summer of 2010 and have never been NED (close a few times). I’ve had TIL, IL-2, ipilimumab, 7 surgeries, and 6 rounds of radiation. I’m BRAF-positive, but we’ve been able to keep the BRAF/MEK medications in our “back pocket”. The same goes for the anti-PD-1 medications. Nothing at all against them; every new metastasis has been a decision point where it hasn’t been the right time, and if we reach that time, hopefully we’ll be at a point where at least one is approved and we don’t have to deal with the complications of an EAP or trial, i.e. “we’ll cross that bridge when we come to it.”]
JoeJune 9, 2014 at 9:56 pm #64664Catherine PooleKeymaster
From my conversations with the FDA and pharma they are two entirely different things. Compassionate use is usually a patient by patient basis whereas they appeal to the FDA and they work with Pharma to provide. The Expanded Access opens up the therapy to all in a trial like setting with restrictions and usually prior to the drug being approved.June 10, 2014 at 2:23 pm #64665RJoeyBParticipant Thanks for responding Catherine, it sounds like what I was thinking is the difference. It’s subtle but huge for someone when an EAP isn’t available or when the EAP requirements, e.g. about brain mets, keep someone from participating in the EAP. Do you have any insight into why the FDA seems to publicly communicate that they are the same thing?
There certainly aren’t any easy answers. I’ve monitored the ever-growing debate about the “right to try”, with laws passed or pending in three states (Colorado, Louisiana, and Missouri) attempting to give patients greater access to experimental medications. One recent article is here:
http://www.washingtonpost.com/national/health-science/right-to-try-laws-spur-debate-over-dying-patients-access-to-experimental-drugs/2014/05/16/820e08c8-dcfa-11e3-b745-87d39690c5c0_story.html ” class=”bbcode_url”> http://www.washingtonpost.com/national/health-science/right-to-try-laws-spur-debate-over-dying-patients-access-to-experimental-drugs/2014/05/16/820e08c8-dcfa-11e3-b745-87d39690c5c0_story.html
The biggest issue seems to be that there is nothing in any of the new legislation that compels or requires a pharmaceutical company to offer a medication on a compassionate use basis. Perhaps by reducing the red tape and requirements currently in place by the FDA would bring more pharmaceutical companies to the table, but I think the problem is larger than that.
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