For some advanced cancer patients, the best course of action may lie beyond standard treatment options. Some of these patients can enroll in clinical trials that investigate promising new treatments. But others may find their best option is to apply for individual “expanded access” to an investigational treatment.
Here, our Curious Dr. George asks two leaders from the Reagan-Udall Foundation for the Food and Drug Administration (FDA) how their organization supports the FDA by striving to facilitate patient access to investigational products:
Curious Dr. George: The goals and process of “Right to Try” legislation have blended with existing policies and practices of expanded access to investigational treatments for patients beyond standard of curative care—especially for those with cancer. How does the Reagan-Udall Foundation for the FDA help physicians better serve their patients’ needs by more easily navigating expanded access, including use of institutional review boards (IRBs) for Investigational New Drugs (IND) for individual patients?
Susan C. Winckler, RPh, Esq. and Lea Ann Browning-McNee, MS, of the Reagan-Udall Foundation for the FDA: Expanded access, sometimes called compassionate use, provides a pathway to investigational treatments not approved by the FDA for patients who have serious or life-threatening illnesses and cannot participate in clinical trials. Single-patient expanded access allows a treating physician to request an investigational product from a pharmaceutical company on behalf of an individual patient. Typically, FDA approves close to 99% of single-patient expanded access requests.
FDA and its Foundation, the Reagan-Udall Foundation for the FDA, are working to make it easier for physicians to navigate the process and better serve their patients by creating new tools to simplify and de-mystify the process.
One such tool is Project Facilitate, created by FDA’s Oncology Center of Excellence as a single point of contact for physicians (and their healthcare teams) who have questions or simply want help submitting a request for their patient. Project Facilitate staff answer questions via phone or email and walk the physician through every step of the process.
Another new tool, Expanded Access eRequest, launched last year by the FDA Foundation and experts at FDA, moves the request from paper or fax to an online app. eRequest takes physicians screen-by-screen through the expanded access application process — from determining if expanded access is appropriate for their patient to submitting the request to FDA. It even sends reminders to let physicians know when follow up reports should be submitted. And the app’s home on the FDA Foundation’s Expanded Access Navigator website means physicians can identify potential investigational therapies; access sponsor (pharmaceutical) information; complete, sign, and submit the request form (FDA form 3926); and upload supporting documentation all in one place.
There are important considerations to bear in mind before pursuing any investigational treatment—whether through a clinical trial or expanded access. In addition to practical considerations like travel and treatment costs, there are medical considerations, including potential risks and unknowns of the investigational treatment, the severity or stage of illness, comorbid conditions, and the likelihood that the treatment will be effective. That’s where the Institutional Review Board (IRB) Curious Dr. George references comes in. The IRB is a committee of medical and ethics experts along with community or legal representatives who review and approve biomedical research involving patients. Even though expanded access is considered a treatment and not formal research, the IRB wants to be sure all patient safeguards are adhered to because an investigational product is involved. One such safeguard is informed consent, which helps ensure patients have details about the treatment protocol that clearly defines expectations, foreseeable outcomes and risks, and patient rights.
The patient, physician, IRB, FDA, and sponsor all need to agree that expanded access to a particular product is right for the patient. Each of these voices and perspectives is important. The company, for example, may not approve a request for a variety of reasons, such as simply not having enough of the investigational product available. A patient might, after considering all the information, choose not to pursue it. The overall expanded access process is intended to help each party understand the situation, and then decide about going forward.
FDA continues to simplify the expanded access journey for physicians and, ultimately, their patients. You can learn more on the Expanded Access Navigator.
- Encouraging and Paying for Clinical Trials, Right to Try, and Expanded Access: Part One
- A Proposed New FDA Drug Approval Pathway: “Conditional”
- Proposed FDA “Conditional Approval”- More Details
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