by Jane Sprague Zones
The Food and Drug Administration (FDA) will soon be issuing new rules and guidelines to allow seriously ill patients access to as yet unapproved (experimental) drugs that are still in clinical trials. The agency has been permitting such access on a case-by-case basis for individual patients since the 1970s, but the new rulings will formalize the procedures and make them somewhat more efficient.
In order for a physician to prescribe a drug, the treatment must have received FDA approval as safe and effective for a particular condition. Approval requires a drug to pass through three phases of clinical trials. About 90 percent of new drugs entering clinical trials never gain FDA approval due to unforeseen safety problems or lack of effectiveness. Although the approved treatment options for women with advanced breast cancer have been growing, experimental drugs have continued to be in demand for women with metastatic breast cancer for whom available treatments have not been effective.
Current FDA regulations allow for compassionate use of an experimental drug in the case of a serious disease during Phase III of a clinical trial and for “immediately life-threatening” disease earlier than Phase III but generally not before Phase II. In recent years, the FDA has been under pressure by some cancer activists to allow access to experimental drugs before Phase II of clinical trials, when the effects of the drugs are not fully understood and may be too risky for compassionate use.
The FDA is mandated to regulate the safety and effectiveness of new drugs and must carefully balance this mandate with the needs of terminally ill patients for whom all approved drugs have been ineffective. In order to clarify its position on expanded access, the FDA issued proposed rules in December 2006 to codify the process for access to experimental drugs outside of clinical trials.1
BCA and many other concerned groups and individuals sent comments about the proposed rules (available online at www.bcaction.org/FDAadvocacy). The FDA is now considering modifications prior to announcing the final expanded access policy. In general, BCA endorsed the FDA’s rules for the following reasons: They protect the clinical trial process by requiring that those eligible for expanded access are not eligible for entry into a trial; provide some protection for patients by limiting access to experimental drugs to those in later phases of testing; and make clear how costs can be recovered by companies that wish to charge for expanded access to experimental drugs. The FDA states that the rules “clarify that allowing charging for treatment use of an experimental drug is intended to facilitate and encourage access to drugs that might not be made available for treatment use unless a manufacturer is able to recover its costs.”2
The proposed rules will also expedite patient access to particular experimental treatments by grouping similar applicants. Decisions have previously been made on a case-by-case basis, which has slowed the availability of treatment.
BCA supports the attempts of seriously ill patients to seek new treatments when their approved options have run out. At the same time, BCA is a staunch supporter of the clinical trial process, which, though slow, is the best source of information about the safety and efficacy of a treatment prior to going into widespread use.
1 Food and Drug Administration press release, “FDA Proposes Rules Overhaul to Expand Availability of Experimental Drugs: The Agency Also Clarifies Permissible Charges to Patients,” December 11, 2006.