Introduction

In the U.S., patient access to investigational cancer drugs—outside of clinical trials licensed by the U.S. Food and Drug Administration (FDA)—has historically proven insurmountable for most patients for at least the following reasons:

1. Pharmaceutical companies are not legally obligated to provide access to investigational drugs to cancer patients who are not otherwise enrolled in a clinical trial that has been authorized by the FDA;
2. Investigational drug supply is often limited to what is needed to complete a clinical trial development program;
3. Adverse events that occur outside of the controlled clinical trial must be reported to regulatory authorities and have the potential to delay or completely derail FDA approval or licensing;
4. Product liability concerns have historically been a significant factor for pharmaceutical companies when deciding whether to provide access to experimental drugs outside of an authorized clinical study. Whether insurance will cover a patient who is injured, or who dies after receiving the drug, and the adverse publicity that may arise as a result of an associated adverse event or death, are additional significant factors considered when evaluating whether to provide access to an experimental drug;
5. Finally, gaining access to an experimental drug requires engaging in a complicated, expensive, and most importantly, time-consuming process that can take numerous days to complete and involve: physicians, pharmaceutical company lawyers, extensive legal documentation, an Investigation New Drug Application filed by the treating physician and approval by the FDA, preparation of a patient informed consent, and review and approval by an institutional review board (IRB). Many patients are not able to surmount these hurdles. Some patients die while attempting to do so. The FDA provides a webpage that outlines the steps required to gain access to an experimental drug outside of a licensed clinical trial.

Project Facilitate

Previously, we summarized the provisions of the newly passed Right to Try Act, a law which removes or attenuates some of the traditional barriers for patients to receive eligible investigational drugs. In the wake of the Right to Try Act, the FDA is implementing a new, cancer drug candidate specific program—Project Facilitate—to help ease the above burdens on patients who are seeking access to experimental cancer drugs outside of clinical trials (that is, to facilitate expanded access to these experimental cancer drugs).

The FDA defines expanded access as "a pathway for a patient with an immediately life-threatening or serious disease or condition to gain access to an investigational medical product (drug, biologic or medical device) for treatment outside of clinical trials when there are no comparable or satisfactory alternative therapy options available."

Under Project Facilitate, the FDA intends to be the first point of contact between pharmaceutical companies and cancer patients seeking outside-of-clinical-trial access to experimental cancer drugs. As part of Project Facilitate, the FDA will operate a call center for expanded access requests. The call center would help patients and their healthcare providers:

• Access relevant contacts at drug companies;
• Complete the expanded access request form; and
• Identify Institutional Review Board (IRB) resources.

In this way, when a pharmaceutical company receives an expanded access request, the company will know the FDA has already reviewed and approved the request. And while the FDA does not have the authority to compel a pharmaceutical company to give an expanded access drug to a patient, at least some advocateshope that the FDA's involvement will make the requests more compelling.

Healthcare providers seeking assistance with expanded access requests can contact the FDA's call center at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov. Healthcare professionals may call during regular business hours, 9 a.m. to 5 p.m., Eastern Daylight Time, Monday through Friday. Patients and families with questions can call 301-796-3400 or email druginfo@fda.hhs.gov.

As part of Project Facilitate, the FDA intends to gather information about whether a pharmaceutical company provides an experimental drug or not. In cases where the pharmaceutical company declines to provide the experimental drug, the FDA will try and document the company's reason(s) for declining. These expanded access requests may help pharmaceutical companies calibrate possible additional indications for their experimental drugs, and to understand more about potential off-label uses upon approval or licensing. Pharmaceutical companies should consider planning to devote additional resources for addressing expanded access requests.

Conclusion

With the launch of Project Facilitate, and the recent issuances of five guidance documents aimed at broadening cancer clinical trial eligibility, the FDA is attempting to facilitate broader patient access to experimental cancer drugs. For questions regarding Project Facilitate, expanded access, or any FDA-related question, please contact Vern Norviel, Lou Lieto, Maya Skubatch, David Hoffmeister, Georgia Ravitz, James Ravitz, or any member of WSGR's FDA/life sciences group.

Charles Andres contributed to the preparation of this WSGR alert.