Drugs

FDA OKs 98% of Individual Expanded Access Requests

David McFarland

— 1 min read
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Four FDA officials say that in the period FY 2010-2014, FDA allowed 98% of individual patient expanded-access requests to proceed. Writing in The Journal of Clinical Pharmacology, the four say that for individual expended access requests, FDA must determine that: 

  •          the patient to be treated has a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
  •          the potential patient benefit justifies the potential risks of the treatment use, and those potential risks are not unreasonable in the context of the disease or condition to be treated;
  •         providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded-access use or otherwise compromise development of the expanded-access use; and
  •          the person cannot obtain the investigational drug under another IND or protocol.

The researchers also looked at how many of the expanded access drugs were later approved for marketing. They say the data show that doctors and patients should not assume that the drugs will later be approved for marketing. “During the study period,” they write, “for drugs not previously approved in any dosage form or for any indication, 24% of unique drugs and 20% of INDs received marketing approval by one year after initial submission; 43% and 33% respectively were approved by five years after initial submission. Expanded access provides just that: access. There is no guarantee that the product sought will be effective and/or safe, much less that it will be effective and/or safe for the particular patient, and these individual INDs are not purposed to collect data on the drug.”

The article concludes that FDA maintains a careful balance between permitting patients to obtain access to potentially beneficial drugs and protecting them from drugs that may have unknown risks. At the same time, the authors say, the agency wishes to maintain the integrity of the clinical trials process as ultimately the best way to get safe and effective drugs to patients.

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