For some battling cancer, especially those with more advanced or aggressive cancers, approved or available treatment options may eventually become limited. When this happens, some may turn to clinical trials, in hopes of receiving a promising, unapproved treatment that could be lifesaving. However, in some cases, a clinical trial with an encouraging experimental treatment may be too far away, or the individual may not qualify. Eventually, treatment options may run out, despite the fact that there are unapproved treatments in clinical trials somewhere else in the country that could benefit these individuals.
The Expanded Use pathway
In recent years, when treatment options have run low or clinical trials have not been feasible, individuals have sometimes still been able to receive these unapproved medications through the Expanded Use pathway. This pathway is facilitated by the United States Food and Drug Administration (FDA), and is also referred to as “compassionate use.” In certain cases, where an individual is battling a life-threatening condition and has no other treatment options, it may be possible for them to receive an experimental and unapproved drug, medical device, or other treatment option.1 However, using this pathway does not always mean the individual in need will receive the treatment, and the process through the pathway can be difficult to navigate or take significant time.
Project Facilitate and the Oncology Center of Excellence
In June 2019, the United States FDA announced its plans to begin a new pilot program designed to help those with limited options receive experimental or unapproved treatments in a more efficient and timely manner. The program will be run by the Oncology Center of Excellence within the FDA and is called Project Facilitate. The program will also use a searchable database created by the Reagan-Udall Foundation called the Expanded Access Navigator to help individuals with cancer, and their providers, find information and unapproved treatment options more efficiently.
Once a provider or patient finds an experimental or unapproved therapy in the database that they think might be helpful, a physician can reach out to the Project Facilitate call center for guidance on how to apply for Expanded Access and get the therapy to the individual in need as quickly as possible. Project Facilitate will also provide guidance on how to report on treatment outcomes. Physicians will be required to contact the pharmaceutical companies that make these treatments to get their approval, and a large majority of these are authorized by the FDA. The entire process will be streamlined through Project Facilitate, as opposed to being navigated across several different departments within the FDA.
Since these treatment options are unapproved, and not yet deemed safe and effective, there may be some risk in taking them. When determining if an experimental treatment option is appropriate, potential risks and benefits must be weighed, and in some cases, the unapproved treatment may be denied.
Accessing Project Facilitate
Patients and families with questions about Project Facilitate or an unapproved therapy can contact the Project Facilitate call center at 301-796-3400, Monday through Friday, 9am to 5pm, or email email@example.com,3
Jarow JP, Lurie P, Ikenberry SC, Lemery S. Overview of FDA’s Expanded Access Program for Investigational Drugs. Ther Innov Regul Sci. 1 Mar 2017; 51(2), 177-9. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5443564/. Accessed June 26, 2019.
FDA Announces Project Facilitate to Assist Physicians Seeking Access to Unapproved Therapies for Patients with Cancer. US Food and Drug Administration. https://www.fda.gov/news-events/press-announcements/fda-announces-project-facilitate-assist-physicians-seeking-access-unapproved-therapies-patients?utm_campaign=060319_PR_FDA%20announces%20Project%20Facilitate&utm_medium=email&utm_source=Eloqua. Published June 3, 2019. Accessed June 26, 2019.
FDA’s Project Facilitate will Assist Requested Access to Unapproved Cancer Therapies. Cure: Cancer Updates, Research, and Education. https://www.curetoday.com/conferences/asco-2019/fdas-project-facilitate-will-assist-requested-access-to-unapproved-cancer-therapies. Published June 4, 2019. Accessed June 26, 2019.