Regulatory Focus™ > News Articles > New Bill Would Expand Access to Investigational Drugs and Devices

New Bill Would Expand Access to Investigational Drugs and Devices

Posted 06 August 2012 | By Alexander Gaffney, RAC 

A proposed piece of legislation would allow terminally ill patients to access drugs and medical devices that have not yet received approval from the US Food and Drug Administration (FDA).

The bill, the Compassionate Freedom of Choice Act (CFCA) of 2012, was introduced in the US House of Representatives on 2 August by Rep. Ron Paul (R-TX).

In a statement on the House floor, Paul said the bill would address the needs of patients suffering greatly from an advanced diagnosis who are too ill to enroll in a clinical trial.

"Denying these patents a possible opportunity to cure their illness--or at least reduce their suffering--is nonsensical and cruel," Paul said. "So why should the FDA deny terminally ill patients access to drugs, devices, and treatments that the patient's physicians have determined represents the patent's only possible chance for survival?"

Patients wishing to access the otherwise-restricted medicines under the CFCA would be required to first have their physician certify that no other viable options for treatment are available and that the patient is terminally ill. In addition, the patient would be required to sign an informed consent document acknowledging the risks of receiving an untested product.

Potential Issues

While FDA is allowed to make exemptions under current law, it may choose not to do so if there are safety concerns about the drug, Robert Temple, then-associate director of the Center for Drug Evaluation and Research (CDER), testified in a 2001 hearing before the House Committee on Government Reform.

Patients wishing to access drugs may also face a non-governmental hurdle in their face to obtain investigational new drugs and devices: the companies themselves. Companies may be loath to release stock of the drug, either for fear of it being used in an uncontrolled way, competitors getting access to it, unknown safety signals coming to light or insufficient manufacturing capacity.

Perhaps the largest issue of all is that enrollment in clinical trials, the basis for eventual FDA approvals, would decrease, delaying access to an even greater number of patients, said Temple.

The bill has been referred to the House Committee on Energy and Commerce, which is set to take it up after the August congressional recess.


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