Turning to drugs and treatments before they are “ready for prime time”

Amy Ship, MD

Contributing Editor

It’s not a situation any of us would wish for. What if you had a terminal illness like cancer or ALS (Lou Gehrig’s disease), or a rare, debilitating disease, and knew there was treatment that might help you, but was not yet approved by the FDA? Fortunately, there is a way to gain access to experimental treatments or drugs. Your doctor can request their use through the FDA’s “expanded access” or “compassionate use” programs.

But some patients and doctors seeking treatment through these programs have felt the process was just too long. And when time is short, delays of any kind are intolerable. Since 2014, 21 states have enacted legislation to help speed up this process. These laws, called “right-to-try” laws, enable patients to bypass the cumbersome FDA process and allow doctors to request certain medications (which have already been FDA-tested for safety, but are not yet on the market) directly from the drug companies that manufacture them.

This may sound good in theory, but getting medications before they are available to everyone is risky — even for those with “nothing to lose.” Drugs that haven’t been thoroughly tested may cause side effects that obliterate any potential benefits, making the precious time left to these people far more miserable than it need be. And doctors who want to weigh the risks and benefits of such treatments are effectively in the dark; they have no way to access the information that would help them counsel patients well.

These laws also raise broad ethical issues. Asking your doctor to ask to prescribe a drug that’s still under development requires that you know this is even possible. It is likely that these requests will perpetuate already significant inequalities in healthcare and favor those with access, resources, and money.

There are also concerns about the unintended consequences of bypassing the usual FDA process. If providing a drug to a very small number of people interferes with the usual testing of a promising medication, then the benefits for all are trumped by the needs of the very few.

In an effort to tackle some of these issues, one pharmaceutical company is working with New York University School of Medicine’s Division of Medical Ethics to address patients’ requests for its medications. A committee that includes medical experts, bioethicists, and patient representatives meets to consider each medication request. The goal is to consider each request in a thoughtful, fair, and consistent way.

The rapid emergence of right-to-try legislation opens the door to broader choices for patients, but they are no guarantee that patients’ requests will actually be granted. These laws do not force pharmaceutical companies to provide experimental drugs, or health insurance companies to pay for them. In fact, for example, Colorado right-to-try laws explicitly allow insurance companies to deny coverage altogether — not just for the experimental medication — to patients who use investigational drugs. So right-to-try laws may, in reality, do little to improve access.

The shortcomings of right-to-try laws are disheartening. However, in February of this year, the FDA proposed a revised and “faster” process for expanded access to investigational treatments. This may be a way to address two powerful competing needs: getting help swiftly to those whose time is short and making sure that the medications we offer are distributed in an equitable and safe way.

Related Information: Harvard Health Letter


  1. svfx

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  3. L Clark

    Expanded access (EAP) / compassionate use has many barriers: Only a few people with significant education, wealth, and medical resources are likely to realize any benefit from EAP. Furthermore, drug developers are often unwilling to provide the treatment for a host of reasons:
    • risks to future FDA approval,
    • inadequate resources to manufacture and distribute the drug free of charge, or
    • a desire to shelter the true manufacturing cost to avoid jeopardizing future market value.
    For imminently fatal diseases like ALS, the Accelerated Approval Program (AAP) should be applied – a conditional approval of promising therapies. AAP requires post-marketing studies in lieu of the Phase 3 trial which take years and millions of dollars. FDASIA, passed in 2012 urged the FDA to utilize AAP for fatal diseases like ALS, yet the FDA is still rejecting it. They told us directly in our meetings with them, Hope Now for ALS.org

  4. Megean Bagarozza

    There is absolutely no sense to be made of the sad fact that after decades there is no viable treatment for ALS and other neuro muscular diseases. Let’s be real please. This is not a “MC- medically correct” disease, this is a horrific way to wither away as your spouses, CHILDREN and friends watch in agony. I am all in for safety and benefits outweighing risks. But when you are living the disease, those are the words that frankly, deflate any and all hope a PALS has!
    PS. Perhaps people would stop having all of their dental work removed and drinking tree bark juice from some remote island if their were an actual and realistic shred of hope here, in their own countries, by their own Neurolologists! Better yet, spend a five hour day at “clinic” with a loved one as they are poked, prodded and Inundated with questions…to only leave exhausted with –NO- not a new medication silly- but a smile from the receptionist and an appointment for three months later. Absurdity at its best
    Thanks, that felt great.. 🙂

  5. M C Collet

    Having been around the fight against ALS for a long time, I truly believe that EAPs are not the answer. They can’t possibly be attractive to drug developers whose substantial investments are needed to address these fatal, unmet-need diseases.

    There are paths for accelerated drug approvals that make much more sense to me. Find a drug with a decent safety profile, be more nimble and creative with trial designs, and get those drugs to market to dying patients much, much sooner. That is a more constructive and realistic approach in my opinion.

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