Posted 16 April 2014
By Alexander Gaffney, RAC
New legislation introduced in the US House of Representatives this week would allow for the import, manufacture and sale of investigational drugs and devices meant to treat terminally ill patients.
Overview and Background
The bill, H.R. 4475, the Compassionate Freedom of Choice Actof 2014, comes in the midst of a long-running debate over whether current regulatory structures-and in particular FDA's Compassionate Use exemption process-is sufficient to treat terminally ill patients who have exhausted all other options.
As written, the bill would amend the Federal Food, Drug and Cosmetic Act (FD&C Act), which ordinarily bans the import, distribution or sale of any product that has either not been approved or cleared by FDA, or is not the subject of an ongoing approved clinical trial.
The new amendment, to be found in 21 USC 360bbb, would prevent FDA from preventing or restricting the "manufacture, importation, distribution, or sale of an investigational drug or device intended for use by a terminally ill patient."
But what-or who-defines an illness as being terminal? The law explains that any licensed physician can diagnose a patient as having a "terminal illness," and so long as no other drug or device is "likely to cure the illness" (in the physician's opinion) and the patient provides written consent to the "known and potential risks and benefits" of the investigational medical product.
FDA would be subject to several restrictions under the law. For example, it could not require disclosure, collection or reporting of clinical outcomes, or information about the drug's administration or use. That might, for example, restrict FDA from collecting adverse event information.
While that might restrict FDA's ability to track if an investigational drug is safe, it might also placate pharmaceutical companies, which generally dislike providing expanded access to their drugs for fear that FDA might weigh any adverse events against its chances for approval.
The law would also institute a broad level of liability protection against all manufacturers, importers, distributors, prescribers, dispensers and distributors of investigational drugs.
Upsides Exist, but are the Loopholes too Great?
The law as written will likely come as a relief to some terminally ill patients, but is also likely to come under fire for its potential loopholes.
For example, FDA has long been entangled in a regulatory struggle with a Texas doctor, Stanislaw Burzynski, who critics contend abuses existing clinical trial regulations to sell patients expensive unproven drugs. In late 2013, FDA went after Burzynski with two warning letters (1, 2), chiding him for failing to adhere to clinical trial regulations, including the investigational plan.
But under the new legislation, physicians like Bursynski would-so long as they remained licensed and adhered to informed consent provisions-be afforded a wide latitude to manufacture, sell and administer experimental drugs, all without needing to report anything to FDA.
Even beyond this example, there are other loopholes, as well. For companies seeking to bring drugs to market for terminally ill patients, the legislation might offer a loophole to immediately begin manufacturing, distributing and selling a drug to patients.
And for patients, the appeal to convince their doctors to declare them "terminally ill" might afford them access to unapproved drugs. Take, for example, the case of patients with Duchene muscular dystrophy (DMD). Many of those patients have been advocating for FDA to approve a drug known as eteplirsen. While FDA turned the drug down last year citing a lack of conclusive data, under the Compassionate Freedom of Choice Act, doctors could declare a patient "terminally ill" (and many DMD patients die by the age of 25), meaning the drug could be manufactured and sold even without FDA approval.
In other words, FDA's decision not to approve a drug would likely just reduce a drug's ability to be marketed and the size of its market, and not prohibit it from the market.
The law might also prove tempting to foreign manufacturers of drugs, who could skirt FDA regulatory requirements and sell their drugs legally so long as they can convince a physician to approve their use and convince a patient of their benefits.
So far the bill, sponsored by House Energy and Commerce Committee member Morgan Griffith (R-VA), has at least one co-sponsor in Rep. Richard Hanna (R-NY).
Compassionate Freedom of Choice Act