Feel Like Experimenting?

Over the past weeks, we have talked quite a bit about clinical trials and complementary and alternative medicine and the like.  Another thing to consider as we navigate the complexities of health care and health insurance, chronic illness and palliative approaches to managing life with challenging medical conditions is this: who has access to experimental drugs?  More precisely, what is entailed with the compassionate use of experimental drugs and sorting out who qualifies?

The Mayo Clinic‘s Dr. Timothy J. Moynihan has some specific questions and answers about this very special field.

Question: do pharmaceutical companies allow access to their experimental drugs and treatments?

Answer: Yes. However…

Getting this access involves a long and arduous process.  But in certain cases, the Food and Drug Administration (FDA) allows drug companies to provide their experimental drugs for compassionate use.


The compassionate use program under the FDA’s guidelines essentially reserves the okay for only those patients with no other treatment options.  To receive experimental drugs through the compassionate use program, it may be required that:

  • The disease is fatal
  • The disease is rare and has no treatment
  • Approved treatments for the disease have not worked
  • The patient is not eligible for any clinical trials currently studying the experimental drug that may be helpful
  • The patient’s doctor agrees that there are no other options and that the patient may benefit from the experimental treatment
  • The drug company agrees to make its experimental drug available to the patient
How does someone get experimental drugs?
First, talk with your doctor about your options.  Then keep in mind the following:
  • An individual applies for compassionate use of an experimental drug.  A patient’s doctor can submit an application on behalf of his patient.  To find out more, check out the FDA’s website and search for ‘investigational drugs‘.
  • There are expanded access studies that offer experimental drugs.  Expanded access studies are programs in which experimental drugs that have reached the later stages of clinical trials are occasionally offered to patients who do not qualify for the trials.  If there is an experimental drug that is of interest, check  with the drug company to see if such a program is available.  Or go to and search ‘expanded access studies’.
What are the risks?
The compassionate use application process is difficult and often very frustrating.  The hurdles and challenges include any or all of the following:
  • There are no guarantees that a patient will benefit.  Remember that experimental drugs are not FDA approved.  They may be approved at a later date, or may never be approved.  The efficacy of the treatment may not be proven yet.
  • The risks and side effects of the drug may be unknown or misunderstood.  Again, experimental drugs are not FDA approved and may not have been fully tested.  The whole range and nature of side effects may still unknown.
  • A patient’s physician may not agree with the request.  A doctor may believe an experimental drug is too dangerous to use, or is ineffective for the person’s condition or illness.  He or she may be unwilling to pursue applying for compassionate use.  A patient can always seek another doctor’s opinion, or could seek advice and guidance from groups that advocate for those who suffer from a particular condition or disease.
  • There are some drug companies that do not grant access to their experimental drugs.  The drug companies are not required to grant access to their experimental drugs; the company could refuse a patient’s request.
  • A patient may have to pay out-of-pocket for any experimental treatment.  The drug company may well charge a patient for its experimental drug.  And it is very unlikely that a health insurance plan covers the costs associated with this treatment, including fees for a physician to administer the experimental drugs and monitor side effects and reactions.
  • The approval process is slow.  Being approved for compassionate use is a long, slow process, unless a situation is a genuine emergency.  Compassionate use applications are reviewed and decided on a case-by-case basis; there is no time limit or timeline and no one can accurately predict how long a decision, yes or no, will take.

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