Clinical Researcher—May 2020 (Volume 34, Issue 5)

RECRUITMENT & RETENTION

Marjorie A. Speers, PhD

As a clinical research professional, most of your interactions with patients are related to their involvement in clinical trials: recruiting, assessing, and enrolling participants; obtaining informed consent; administering trial procedures; and monitoring patient responses to the investigational drug, biologic, or medical device. In some cases, your site might only conduct clinical trials and not offer treatment to patients who are not enrolled in a trial. Even so, eventually you might be approached by a patient who is interested in the U.S. Food and Drug Administration’s (FDA’s) Expanded Access Program, which permits an investigational product to be used outside a clinical trial for treatment in certain circumstances.

Perhaps the patient doesn’t qualify for a trial and has exhausted all other treatment alternatives. Maybe he or she or lives too far from a study site to participate. Regardless of the reason, the patient is out of standard options and not willing to give up hope for a suitable treatment.

How can you help? Prepare for this possibility ahead of time. Then you’ll be ready with information and, ideally, a policy on whether, how, and when your site will participate in an expanded access use.

What are the Options?

Today’s patients have two different pathways to access investigational treatments. The first, FDA Expanded Access, has long made investigational products available as a compassionate response to very ill patients who don’t meet clinical trial criteria and have no other treatment options. The second pathway, known as Right to Try, was established via federal legislation in 2018.

In both cases, if the manufacturer does not agree to make the investigational product available, there is little you can do to convince them otherwise. Also in both cases, permission to use the investigational product must be requested by a treating physician to treat a single patient. Patients cannot make a request on their own.

FDA Expanded Access typically is the preferred pathway because it offers access to investigational drugs, biologics, and medical devices. Equally important, this process provides a prompt response without sacrificing patient safety. Informed consent and review by an institutional review board (IRB) are required to help protect patients, but these requirements do not impede the process. In fact, FDA generally approves 99% of expanded access requests and usually completes its review in a few days. In addition, most IRBs have streamlined procedures to review requests for expanded access uses quickly.FDA has taken steps in recent years to streamline its process and improve access to information. IRB review can now be done by an IRB chair or designated IRB member, instead of by the convened board, when investigational drugs or biologics are to be considered. The Reagan Udall Foundation for the FDA offers an online Expanded Access Navigator. A pilot program, the Project Facilitate call center, connects FDA oncology staff with physicians seeking expanded access for individual patients with cancer.

Right to Try gives patients a second option, but only for access to investigational drugs that have already completed Phase I clinical trials. Right to Try does not apply to medical devices. Perhaps the biggest distinction between Right to Try and FDA Expanded Access is that Right to Try does not require FDA or IRB review. As a result, many physicians and patient advocacy groups have expressed concerns that patients have fewer protections under Right to Try. Because of these concerns, and because guidance on how to best use this pathway is still being developed, there have been very few Right to Try applications.

What is Your Policy?

For patients interested in expanded access, a key question will be whether your site provides investigational drugs, biologics, or medical devices for treatment uses outside clinical trials. That’s why it’s important to make this decision long before your first expanded access discussion with a patient or prospective trial participant.

If your organization decides not to offer expanded access, your message will primarily be an educational one. Provide some background on expanded access and refer the patient to his or her treating physician. If possible, be prepared to provide a list of resources, such as Reagan Udall Foundation Navigator and Clinical Research Pathways’ “Points for Patients to Consider.” You can print this document ahead of time to hand to patients during your discussion.

If you have a physician who decides to serve as a sponsor of expanded access uses, you will need to work with colleagues to develop a policy that covers a variety of details, such as:

Which patients are eligible for expanded access?
What procedure will the physician at your site follow?
Will you offer expanded access through the FDA pathway, Right to Try, or both?
How will you manage or defray expanded access-related expenses that your site is likely to incur?

Consider Potential Costs and Reimbursements

Drug and medical device manufacturers usually provide the investigational products for expanded access free of charge. However, as the sponsor of an expanded access treatment, your site will incur other expenses. This is also true for treating physicians involved in expanded access.

Expenses related to oversight, recordkeeping, reporting requirements, and other routine administrative tasks typically are not covered by insurance. Your expanded access policy should take this into account and specify how your site will handle these additional expenses.

Costs that are more directly related to treatment, however, might qualify for reimbursement. Examples include such medical procedures as infusions, blood tests, and imaging that are associated with administering and monitoring the effects of the investigational treatment. Reimbursement might also be available in cases where an investigational drug, biologic, or medical device is being administered as standard care.

Conclusion

Decisions about whether to offer expanded access can be complicated and must balance the interests of your site with your responsibilities to product manufacturers and patients. If your site has chosen not to permit expanded access treatments or does not yet have an expanded access policy in place, you still have an important role to play—and the time to prepare for it is now.

Familiarize yourself with expanded access and the alternatives available to patients. Gather and share information, and encourage patients to talk with their treating physician, since he or she must make the expanded access request.

If you believe your site should consider offering expanded access, continue to advocate for that internally. Even if that doesn’t come to pass, by offering information and resources, you will be performing a valuable service for patients who have exhausted all other treatment options.

Marjorie A. Speers, PhD, is the Executive Director of Clinical Research Pathways, a 501(c)(3) organization that develops programs to increase diversity in clinical research and expand access to experimental drugs.