Posted: December 11, 2019
In June 2019, the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence launched Project Facilitate, a call center that assists oncology healthcare professionals with the submission process involved in obtaining unapproved therapies for individual patients with cancer via the Expanded Access program.1 Richard Pazdur, MD, director of the FDA Oncology Center of Excellence fielded questions from the IASLC Lung Cancer News on the impetus behind this pilot project as well as its potential benefits to patients and the broader cancer care community.
Q: What is Expanded Access?
A: Expanded Access is a potential pathway for a patient with an immediately life-threatening or serious disease or condition to gain access to investigational therapies for treatment outside of clinical trials when there are no comparable or satisfactory alternative therapy options available. In those cases in which patients do not fit the trial requirements or live too far from a trial site, healthcare professionals can request permission from the FDA to treat a patient with an investigational medical product through Expanded Access.
Q: What is Project Facilitate, and what gap is it intended to address? How will the program’s success be determined?
A: Navigating the Expanded Access process can be complex, particularly for oncologists who don’t have experience working with clinical trials or these types of requests. Project Facilitate is a call center that is a single point of contact where FDA oncology staff help oncology healthcare providers through the process to submit an Expanded Access request for an individual patient. Experienced FDA oncology staff support oncologists and other healthcare professionals with their questions, assist in filling out the appropriate paperwork, and act as a facilitator for the process. As with all Expanded Access requests, the drug manufacturer has the right to approve or disapprove the physician’s request. We will also need to determine if oncology healthcare providers are using Project Facilitate. There are several factors that will be used to evaluate the program, including use of the call center as represented by the number of calls to Project Facilitate.
Q: If Project Facilitate is deemed successful, are there plans to expand the program?
A: The FDA has been working to improve the Expanded Access framework, including the development of an updated and more streamlined application form. Project Facilitate is part of our continued commitment to Expanded Access, and we hope that the pilot program will simplify the process for oncology healthcare providers and will ultimately benefit patients. As Project Facilitate is a pilot program, it is too early to determine if the program will be expanded to areas outside of oncology.
Q: The FDA has also published new guidance on broadening cancer trial eligibility. How many patients are expected to be affected by that guidance, and will it reduce the need for Expanded Access?
A: The first option for patients for whom available treatments have been exhausted is to enroll in a clinical trial. However, in clinical trials testing treatments for cancer, some eligibility criteria have become commonly accepted over time or used as a template across trials without a clear scientific or clinical rationale or justification. In other cases, eligibility criteria can be deliberately restrictive, even though it is not clinically merited. In March 2019, the FDA published four draft guidances and one final guidance regarding cancer trial eligibility criteria.2 These guidances provide recommendations on how sponsors could safely and effectively broaden the criteria for the inclusion of certain patient populations in clinical trials, when appropriate, for pediatric patients and patients with HIV, Hepatitis B and C Virus Infections, brain metastases, prior or concurrent malignancies, or organ dysfunction. It is too early to tell how many patients will be affected, but we hope that our recommendations will help to shift the design of oncology clinical trials to be more representative of the patients who may ultimately benefit from novel treatments. In cases in which patients do not fit the trial requirements or live too far from a trial site, healthcare professionals can request permission from the FDA to treat a patient with an investigational medical product through Expanded Access.3
Q: Are there benefits to the FDA and the greater research community to having a program like Project Facilitate, apart from improving access for individual patients?
A: The pilot program includes a central office for oncology requests so that the FDA can follow up on individual requests and gather data, such as how many patients received the investigational medical products and if not, why the requests were denied. The FDA will use these data to determine how the process is benefiting patients and healthcare professionals. In addition, the data could assist in encouraging sponsors to open clinical trials to study drugs for additional indications. ✦
1. FDA announces Project Facilitate to assist physicians seeking access to unapproved therapies for patients with cancer. U.S. Food and Drug Administration. 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 September 21, 2019.
2. FDA In Brief: FDA takes new steps to broaden patient participation in cancer clinical trials, advancing policies to promote inclusion of pediatric patients and patients with medical conditions that can occur alongside cancer. U.S. Food and Drug Administration. www.fda.gov/news-events/fda-brief/fda-brief-fda-takes-new-steps-broaden-patient-participation-cancer-clinical-trials-advancing. Published March 12, 2019. Accessed September 21, 2019.
3. Expanded Access. U.S. Food and Drug Administration. www.fda.gov/news-events/public-health-focus/expanded-access. Updated May 6, 2019. Accessed September 21, 2019.