Should the FDA Make Exceptions in Terminal Cancer Cases?

CRegal Editor
  • Andrea Sloan, the executive director of the Texas Advocacy Project and a respected Austin attorney, is fighting for her life.  She was diagnosed with a rare form of ovarian cancer associated with the gene BRCA 1, which limits the number of treatment options available to her. 


    However, her doctors think that she is the perfect candidate for one specific medication called BMN 673.  There is just one problem: the drug is not yet approved by the FDA.


    The FDA drug approval process is long and arduous, though it ensures that the safety and efficacy of the drugs that do hit the market meet very high standards.  It can take more than a decade and cost hundreds of millions of dollars for a drug to be approved, and many drugs are rejected during initial testing, clinical trials or in the actual application process.

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    Sloan would appear to be out of luck.  If the medication has not yet been approved, she doesn’t have access to it, one would think.  But it isn’t quite that simple. 


    The FDA can grant a waiver giving permission for a drug company to provide access to a medication in development outside of a clinical trial setting.  This exception is often referred to as “compassionate use,” where an investigational drug can be used to treat a serious or immediately life-threatening disease or condition, according to the FDA website.  The patient is only eligible if there is no comparable or satisfactory alternative treatment option, as appears to be the case with Sloan.


    But Sloan isn’t being treated with BMN 673, even though the FDA has said that it would issue a waiver on her behalf, because the manufacturer of the drug – BioMarin Pharmaceutical – is not willing to provide it to her.  It notes that the medication has not yet been proven to be safe or effective, and that there could be serious side effects.  Giving the drug to Sloan could be extremely dangerous--not to mention that investigational drugs are expensive to manufacture and that insurance will rarely (if ever) cover the costs of the medication.  Administrative costs and the expenses related to monitoring the use of the drug by doctors can also be expensive, leaving either the manufacturer or the patient with the bill.


    Sloan’s supporters have set up a petition to BioMarin to encourage the company to provide her access to the medication. The story has received significant media coverage both in her hometown of Austin and on national platforms, including Former Speaker of the House Newt Gingrich’s blog and the Huffington Post.


    Despite the emotional story associated with this specific application, there remain questions about the potential “compassionate use” of a drug that has not yet been approved by the FDA. Does a patient deserve access to a potentially life-saving medication, even if it comes with significant risks?   Or is a drug manufacturer justified in denying use of an expensive drug that’s not fully tested and potentially dangerous?


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    Keep your eye on this story – it could help change the way that the FDA, drug manufacturers and patients are able to apply the compassionate use exception. 

Published On: September 24, 2013