A blog post by Clinical Research Pathways Executive Director Marjorie A. Speers, Ph.D.

A streamlined Food and Drug Administration (FDA) expanded access process has made it easier for desperately ill patients to try experimental drugs, biologics, and medical devices. Despite this progress, costs—to patients, healthcare providers, and product developers—present significant barriers to participation.

As a result, many desperately ill patients can’t afford to try treatments of last resort. Equally important, many product development and health care professionals are discouraged from providing, delivering, and administering investigational products because of non-reimbursable costs.

How do we address these challenges?

That was the focus of “Who Should Pay for Pre-Market Treatment Access Programs?” a discussion that Clinical Research Pathways board member David Farber, J.D., and I moderated, last week at the third annual Expanded Access Summit in Washington, D.C. About 150 policy makers, patient advocates, representatives of pharmaceutical companies, and others interested in expanded access participated.

Our goal was to identify systematic, sustainable solutions that have the potential to benefit the greatest number of expanded access patients. Some of the most promising options involve pursuing reimbursement through existing avenues, such as insurers including Medicare and Medicaid. Patient assistance programs also were discussed.

What are the costs?

Patients and their families bear some of the most significant and least recoverable expenses. Because the products involved are investigational, they typically are not covered by insurance. Many patients also must travel to major medical centers to receive the investigational product. That adds expenses for travel, overnight stays, meals, child care, lost wages, and other costs of being far from home.

Pharmaceutical and biotechnology companies face costs related to administering an investigational product in addition to the direct costs of manufacturing the product for expanded access use. Companies are not paid for the time spent providing and overseeing the use of experimental medications. Furthermore, that time otherwise could be devoted to developing products that, because they serve larger groups of patients, have the potential to provide greater public good.

Health care providers (individuals and institutions) devote considerable time and resources to treating the patient through expanded access. They complete and submit the application for the investigational product, obtain informed consent from the patient, administer the experimental product to the patient, and monitor, record, and report its effects, including any adverse effects. Providers also must track the use and storage of the experimental product and provide a summary report about the treatment. Most of these activities result in unreimbursed expenses. Larger institutions sometimes budget for these costs. In such cases, like the product development companies, health care institutions face the difficult question of whether time devoted to individual, desperately ill patients could otherwise be used for the public good.

Solutions worth pursuing

Most of our expanded access session focused on attendees’ firsthand experiences with investigational products—as patients, developers, clinician investigators, and regulators. Many have had success using the expanded access process to provide treatment, and their experiences proved invaluable in evaluating hurdles, identifying some immediate solutions, and charting a course for the future.

For example, some who administer investigational drugs pointed out that, although insurers will not cover the cost of experimental medications, expenses for related medical procedures often qualify for reimbursement. Therefore, in some cases the cost of blood work to monitor patient response might be covered by insurance. If an investigational product is delivered via infusion, the drug itself might not be covered, but the infusion procedure could be.

Reimbursement might be possible in cases where an investigational drug is being used as part of an FDA-approved treatment. Since federal regulations permit sponsors to recover costs, that option might be available to academic or health care institutions that serve as sponsors for treatments under expanded access.

Above all, our discussion highlighted the need and potential to work more closely with insurers to identify opportunities for patients and providers to recoup their costs. If investigational drugs, biologics, and medical devices were more affordable, expanded access would be an option for more desperately ill patients and the providers who can give patients and their families another chance for hope.