Patient access to investigational cancer drugs—those outside of clinical trials licensed by the FDA—has historically proven insurmountable for most patients for at least the following reasons:
Pharma is 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;
- 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;
- Investigational drug supply is often limited to what is needed to complete a clinical trial development program;
- 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;
- 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;
- 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 the licensed clinical trial.
On the link below, JD Supra shares more information on “The Right to Try Act” as well as Expanded Access programs. Under Project Facilitate, the FDA intends to be the first point of contact between the pharmaceutical companies and cancer patients seeking access to experimental drugs that may be outside of clinical trial programs. The FDA is setting up a call center for expanded access request. The call center will help patients and their healthcare providers to do the following:
- Access relevant contacts at drug companies
- Complete the expanded access request form
- Identify Institutional Review Board (IRB) resource
TrialSite News Comment
Academic medical centers and commercial biopharma sponsors will need to understand Project Facilitate and how to ensure that they are a participating in optimal ways.
JD Supra reports that 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 email@example.com.
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.
The FDA is seeking to facilitate broader patient access to investigational medicines with A) the launch of Project Facilitate and B) recent issuances of five guidance documents aimed at broadening cancer clinical trial eligibility.Source: JDSUPRA