Should Patients Have Access to Investigational Unapproved Drugs?

As a cancer physician, I often cared for patients who found themselves in very desperate medical situations. When all available approved treatment alternatives have been exhausted, many patients are willing to try investigational or restricted therapies- -but then learn they are not eligible for the protocols that govern their use. What are such desperate patients to do?

My take is that once approved treatment regimens have been exhausted, desperately ill patients should have appropriate access to any reasonable treatment that has the potential to be of benefit and yet doesn’t present unjustifiable risks. The best way to provide these drugs is through clinical trials, where the rigor and discipline of the trial enables patient response, side effects, and outcomes to be carefully measured to determine safety and effectiveness in a systematic way. When this is not possible, there can be an alternative pathway to receiving the investigational therapy.

The FDA has called this pathway “single patient access,” often referred to by the public as ” compassionate use.” We have a long history of helping patients gain access to potential treatments this way. For example, just in FDA’s oncology division, we review and approve hundreds of requests each year for single patient access to investigational drugs.

More recently, we have encountered circumstances in which a drug is approved and is commercially available, but can only be used under very strict rules – these are called an approved Risk Evaluation and Mitigation Strategy (REMS). But we have adapted “single patient expanded access” to this category of drugs as well in an effort to do everything appropriate to serve these unfortunate patients.

So, how can patients gain access to an investigational drug or a restricted drug through these mechanisms? To permit such treatment use of an investigational drug, the company must first agree to make the drug available. Then, the FDA, in conjunction with the patient’s treating physician, must determine, among other things that the potential benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or the condition to be treated; and that providing the investigational drug for the treatment use will not interfere with the initiation, conduct, or completion of clinical investigations that are required to support marketing approval of the investigational drug for the greatest number of patients who can benefit. A similar path is taken when considering use of restricted commercially available drugs.

Drugs under investigation, in either a clinical trial or an expanded access program, don’t offer a guarantee of success, but they do offer an option. Information about clinical trials of investigational drugs and expanded access options, including contacts and locations, is available through the web site. At the FDA, there is an Office of Special Health Issues which you can call and where they have trained personnel to listen to your story and assist you with the necessary information. The number to call is 301-827-4460.

At the end of the day, benefitting patients is the goal of the FDA. And how it is achieve requires a structured and disciplined process to facilitate getting the right drug or medical product to the right patient in the right way – to get the best possible outcome.

Andrew C. von Eschenbach, M.D.
Commissioner of Food and Drugs (FDA)


  1. quiact says

    If patients are terminally ill, they should have free access to yet to be approved drugs by the FDA. Such patients are without treatment options, and should be able to decide to acquire such drugs, if they choose to do so.

    The FDA, by the way, approves drugs with concerning frequency that prove to be toxic if not deadly. So a drug not approved by the FDA is not a valid issue.

  2. quiact says

    Yes, I think premature therapeutic agents should be available to certain patients- terminally ill ones specifically. They have no other options. With premature death known to them, they, as would I, would try anything that may alter their future.

  3. sharon says

    I believe whatever treatment is available should be utilized, what is the sense of making these patients suffer more than they are. They are probably more aware of the side effects than the average person anyway. What’s the worst that can happen to them? They lose their life? Isn’t in question anyway?

  4. says

    I believe patients should have access to whatever methods available to them, how many times have we seen commercials where death or stroke is a possibility of a new drug, and yet it passes but cancer patients who are on the road to death are not allowed to try new drugs. This is just plain wrong, why let some drugs pass that have all these crazy side effects and the ones that might help still have a rigorous road ahead of them? Who knows, maybe some of these unorthodox drugs might help, what’s the worst that can happen anyway? They get some side effects? America should wake up and smell the coffee, the FDA should also, after all it’s in their hands to help our cancer patients, and not a one patient at a time but many at the same time.

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