Monday, May 24, 2010

Off-Protocol New Experimental Treatments

Frequently asked by a clinical investigator and/or a patient is how to get information pertaining to "Off-Protocol" experimental, new treatments outside of clinical trial? There are a number of procedures to follow that will allow consent for clinical investigators and patients to obtain new off-protocol experimental treatments. While it is not true that you may always or even eventually get off-protocol treatments, the possibility if not limited to treatments which involve only FDA approved drugs. Off-protocol treatments may involve promising new drugs which have not yet been approved by FDA or other regulatory agencies worldwide.

Off-protocol, out of clinical trial treatments are not recommended. Even if you can get a treatment outside a clinical trial, that does not mean you should follow this type of practice. Indeed for many reasons, reasons pertaining to patient safety and quality control, off-protocol treatments are not advised. If you can find a treatment on clinical trial, I strongly recommend this type of practical participation for the patient. Clinical trial protocols are governed with regulations, guidance and FDA GCP, Good Clinical Practice to ensure patient safety and data quality management.

If an off-protocol treatment is pursued, there are usually considerable reasons for selecting treatment outside of clinical trial.

  • There might not be an open clinical trial available.
  • You might not qualify for the clinical trial.
  • The clinical trial is randomized and "blinded" and you need active drug vs the possibility of being randomized to a placebo treatment arm.
  • Travel to the clinical trial site is difficult, although usually worked out with the clinical site coordinator whenever possible.
  • Insurance may cover the off-protocol treatment, and sometimes the reverse is true. This seems to be case-dependent.
  • Life threatening situations.
  • Terminal illness.

Let me stress again that it is not my recommendation to seek off-protocol treatments. It is strongly recommenced to seek well controlled clinical trials that adhere to GCP and are governed by FDA guidance to ensure patient safety.

In few instances, off-protocol, outside of clinical trial treatment may make sense. Risk and benefit considerations must be fully investigated before any decisions are made by the patient, by the physician, clinic or otherwise. Quality and GCP must always be followed with patients in general, even with off-protocol treatments, however, it is difficult to monitor, manage and enforce.

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