Expanded Access with Dr. Jacqueline Corrigan-Curay
Q&A with FDA Podcast | Transcript
Dr. Williams: Welcome to the “Q&A with FDA”, from FDA’s Division of Drug Information. In this podcast series we answer some of the most frequently asked questions that we’ve received.
My name is Dr. Celia Williams, and today we are discussing expanded access, a topic about which we continually receive questions. In fact, in the past year, our Division has responded to over 750 expanded access related inquiries.
Dr. Jacqueline Corrigan-Curay, Principal Deputy Director of FDA’s Center for Drug Evaluation and Research, who is an expert on this topic, is with me today. Welcome Dr. Corrigan-Curay, it’s a pleasure having you with us to talk about expanded access.
Dr. Corrigan-Curay: Thank you. I’m, I’m really pleased to be here to speak about what’s such an important topic.
Dr. Williams: The expanded access pathway is a way to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative treatment options are available. Can you expand upon this?
Dr. Corrigan-Curay: Sure, let me start, and explain who is eligible for expanded access. And you gave a little bit of that information. It is a potential pathway for a patient who has a serious or life-threatening disease or condition to gain access to an investigational drug - that’s a drug that is being tested to see if it’s safe and effective for a, a new condition or disease. And that patient does not have other available therapy that’s on the market and they’re unable to enter a clinical trial for that agent. And that may mean that, you know, they either don’t meet the eligibility requirements for that clinical trial or the trial is just not accessible. And in that case, if a company is able and willing to provide their investigational agent to the patient outside the clinical trial, they may be eligible for expanded access.
There are different types of expanded access. So there are what we call individual expanded access, which as the name implies might be when a physician is seeking an investigational drug for a single patient. There is also what we refer to as intermediate expanded access programs, and that may be when a company uh has decided they they’ll be willing to give the drug out for a number of uh different patients who could receive it. And in, in that case, it might be done under protocol that more than one uh patient, could receive the drug. Finally, we even have what we call much larger expanded access programs. It might occur when a sponsor has finished their clinical trials, they’re waiting, perhaps approval, there’s sufficient data on the drug such that more widespread access, uh, through expanded access is appropriate. What I would say is the overwhelming majority of submissions for expanded access are for those for those individual patient expanded access.
In all cases, it’s important to understand that expanded access refers to the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient’s disease or condition - rather than obtain the kind of information about the drug that’s generally derived from clinical trials. And that’s an important distinction, and we need to underscore that while the intention is to diagnose, monitor or treat, until we have the data to approve a drug – FDA approve a drug - we do not know whether that drug is really safe and effective for its use. And indeed the best way to obtain that information is through a clinical trial. And so another criteria for expanded access is that it does not interfere with the clinical development program that’s designed to answer those important questions regarding safety and effectiveness. You know, ultimately the broadest access is through an approved product.
Dr. Williams: You’ve already alluded to this in defining expanded access, but can you expand on why this pathway is sometimes referred to as “compassionate use”?
Dr. Corrigan-Curay: Sure, compassionate use is not a term used in FDA regulations but is occasionally used informally to describe the use of an investigational drug to treat a patient. As I said, you know, a criterion for obtaining a medication through expanded access is that there are not available alternatives. Therefore, patients’ seeking a drug through expanded access often have life-threatening or severely debilitating disease and have exhausted all other available therapies. So of the over a thousand individual expanded access requests received by the Center for Drugs each year, for individual access, many are for patients with, you know, very serious cancers or infectious diseases. And we recognize this need, and so we have long history of facilitating expanded access to investigational drugs for this treatment use. In fact, on average, the Center for Drugs determines that over 99% of all expanded access requests may proceed.
Dr. Williams: Thank you for sharing that statistic with our listeners. We are most frequently contacted by physicians and other healthcare professionals with questions about their role in the expanded access process. Can you briefly describe the role of the physician in making an expanded access request?
Dr. Corrigan-Curay: Yes, the, the physician a, a number of very important roles in expanded access. You know, first they are responsible for confirming that the patient’s current disease or condition is serious or life-threatening; there are no available alternative approved treatments; and there is not a clinical trial that that patient can enroll in. They are also responsible for doing the risk benefit analysis of proceeding with treating that patient with an investigational drug. So if all these criteria are met, then they can submit an application, an IND, to find out if the patient does qualify for expanded access.
Now the physician is also responsible in this individual expanded access for confirming that the company that makes or owns the drug, the investigational drug is willing to provide the product for expanded access. They are also responsible for fulfilling the requirements for expanded access use, evaluating and discussing the potential benefits and risks with the patient, including obtaining uh informed consent and IRB review, and they have responsibilities to submit reports and records to us under our regulations within the required time frame. Now I know that can sound like a lot to a physician who could be hearing about this for the first time and interested in this.
We have really, we realize that that physicians are, are practicing physicians and we've tried to simplify the process for an individual physician. We developed a special form. It's a Form 3926 that is really designed to streamline the information that a physician needs to submit to us when applying for an individual expanded access. And there’s a helpful resource for physicians in getting more familiar with this process on the Reagan-Udall Foundation’s Expanded Access Navigator website.
Dr. Williams: Agreed. The Expanded Access Navigator is an excellent resource for physicians and patients and one we frequently recommend. A link to the Navigator is available on our FDA Expanded Access website at fda.gov/expandedaccess or it can be visited directly at navigator.reaganudall.org.
Dr. Corrigan-Curay: The Navigator is also a great resource for physicians to identify an institutional review board, or the IRB. It’s the IRB that reviews and approves the expanded access plan and informed consent, or, in emergency situations, it’s informed of an expanded access use within five days of uh the drug being administered. Now one change that we implemented for individual non-emergency expanded access is that the IRB review can be done by an IRB chairperson or another designated IRB member rather than a full IRB meeting. And this can also facilitate timely review.
Dr. Williams: When we talk with physicians, other healthcare professionals and patients, frequently they are looking for more information about how to get started with the process, and we often refer them to FDA’s expanded access website at fda.gov/expandedaccess. What parts of the expanded access website do you think are the most useful for physicians and patients?
Dr. Corrigan-Curay: Yes, the FDA expanded access website is really a great resource. The resources are organized by audience type so there is a section that specifically speaks to physicians and what they need to know separately from, you know, the section for patients, which speaks directly to what they need to know. The Expanded Access Navigator that we just spoke about is also set up the same way and broken down with information for physicians and information for patients or caregivers.
So both the FDA website and the Navigator explain the two options for a single patient expanded access request, which as I said are the majority of the expanded access requests we receive. These two options are for emergency and non-emergency treatment. I want to note that emergency and non-emergency requests follow different timelines and have different processes, and there are step-by-step guides for physicians on our FDA website that describe in detail how each option works and what must be completed and when. Emergency expanded accesses is when you really need to administer that drug within a very short time window, perhaps hours, and this may be a patient who, you know, is quite ill and these are often reviewed within hours.
We also offer a 4-part video series for physicians that reviews the single patient expanded access process from start to finish, as well as home study continuing education courses focused on expanded access. I could talk about our web resources for a long time because there is just so much helpful information available. So to get started, visiting the appropriate audience tab is what I recommend doing first.
Finally, for expanded access requests for patients with cancer, Project Facilitate offers oncologists assistance in applying for expanded access for their patients. There is someone available to speak during the day and really assist with the application process.
Dr. Williams: I’ll add that for emergency requests, physicians and their staff can call us at FDA 24 hours a day / 7 days a week. The agency has its own team of healthcare professionals available around-the-clock to assist with emergency expanded access requests.
Dr. Corrigan-Curay: That’s right, and, and we really appreciate your Division’s efforts and all the other groups involved that make it possible to process these requests and process them in such a timely manner. It’s truly a team effort.
Dr. Williams: Absolutely. Before we let you go, is there anything you didn’t get a chance to discuss that you would like to add?
Dr. Corrigan-Curay: Yes. I’d like to mention is that there is a new electronic submission option made available in the last few years, and we want to make sure physicians know about it. It is the EA eRequest app on the Reagan Udall Foundation Expanded Access Navigator webpage. The EA eRequest allows physicians to submit expanded access requests online as well as submit follow-up documentation. And the app reaches multiple product Centers at FDA for drugs and biologics. I would encourage physicians thinking about individual expanded access use to use this application because it walks you through the process and provides an electronic platform to track your submissions. You can create drafts and it can even remind you if you have unsubmitted drafts. So I would encourage you to go to that website and consider using the eRequest app.
Dr. Williams: Very helpful! Thank you again Dr. Corrigan-Curay for being with us today.
To get started with the resources discussed today, visit fda.gov/expandedaccess.
Thanks for listening to “Q&A with FDA”. The full podcast and transcript of this recording is available at fda.gov/QAwithFDA. If you have questions about this episode, or anything drug-related, email us at druginfo@fda.hhs.gov.