Expanded access, also known as compassionate use, is a federal program facilitated by the FDA. It provides a pathway for patients to gain access to “investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials” (Jarow). In order for it to succeed, the program depends on the collaboration of the sick patient, treating physician, pharmaceutical company, IRB, and FDA. Specifically, the procedure is as follows: A physician caring for a terminally-ill patient “who has exhausted all other treatment options and is not eligible for a clinical trial appeals to a pharmaceutical company to provide an investigational drug that has undergone at least a Phase 1 trial, which studies the safety of a drug” (Rangarajan). If the pharmaceutical company agrees, the physician must acquire IRB approval and apply to the FDA for a green light to access the investigational drug through expanded access. In recent years, the FDA has modified its policy to simplify the application process. As a result, it now only takes approximately 45 minutes to complete the application form (Form FDA 3926), and “4 days to proceed for non-emergency expanded access requests and less than 1 day for emergency requests” (Jarow, 2017). The IRB has also simplified its approval procedure by requiring only one member of a facility’s IRB to sign off on the petition. 

However, regardless of such support, efforts to acquire investigational drugs are often unsuccessful. This is because many pharmaceutical companies deny expanded access requests. From the company’s perspective, granting access to investigational drugs produces two negative consequences. First, pharmaceutical companies worry that if an investigational drug is provided to one patient, it must also be provided to all patients who request it. This scenario will delay the approval and introduction of the drug to the market which not only negatively impacts the company's financial performance, but also reduces the drug’s possibility of helping other patients with the same disease. Provision of the investigational drug to all requesting patients can cause such delay by reducing potential participants in the upcoming clinical trial. For instance, in the case of rare diseases such as metachromatic leukodystrophy, only about 60 children develop the late infantile form annually in the United States. Therefore, if some of these children are provided with the investigational drug, the pharmaceutical company would have trouble recruiting patients when it finally opens a clinical trial. The second negative consequence is the increased possibility of the pharmaceutical company to become liable if an adverse event occurs. Clearly, such a case will “derail the company’s ability to push the drug forward for FDA approval, something they argue would ultimately undermine efforts to develop drugs that can help other families” (Rangarajan). 

One way to solve this issue is by creating an advisory committee to review and make decisions on a case by case basis. A pilot experiment conducted by Janssen Research & Development and NYU School of Medicine found that an interdisciplinary expert committee which includes professionals in medicine, bioethics and patient advocacy is useful to ensure a “transparent, fair, beneficent, evidence-based, and patient-focused compassionate access to investigational medicines, while not conflicting with the need to supply investigational drugs to the clinical trials used to evaluate safety and efficacy and leading to regulatory approval” (Caplan). For instance, in the case of an anti-cancer drug called daratumumab, the expert committee known as CompAC eliminated 144 expanded access requests out of 324 based on their pre-established criteria. CompAC’s success in eliminating some of the requests demonstrates the ability of an interdisciplinary expert committee to reach an agreement on guiding principles, and use these principles to determine a fair allocation decision for individual cases. This way, expanded access program and right-to-try law can still provide hope to terminally-ill patients as the decision to receive or not to receive a potential life-saving drug is determined comprehensively.

References:

Caplan, Arthur, et al. “A Pilot Experiment in Responding to Individual Patient Requests for  Compassionate Use of an Unapproved Drug: The Compassionate Use Advisory    Committee (CompAC) - Arthur Caplan, Alison Bateman-House, Joanne Waldstreicher,     Lisa Fedor, Ramana Sonty, Tito Roccia, Jon Ukropec, Rick Jansson, 2019.” SAGE  Journals, Therapeutic Innovation & Regulatory Science, 12 Jan. 2018,  journals.sagepub.com/doi/pdf/10.1177/2168479018759659?casa_token=6ETTtRky_OoAAAAA:ubRckoC3-f-g1hNTUSFfkR6b-A-AsQn2h66_sTjHssHosaUFlahe3RopDEhs4Kq5TeR8ZJWEn_aF9g.

Jarow, Jonathan P, et al. “Overview of FDA's Expanded Access Program for Investigational Drugs.” Therapeutic Innovation & Regulatory Science, U.S. National Library of Medicine, 1 Mar. 2017, www.ncbi.nlm.nih.gov/pmc/articles/PMC5443564/.       

Rangarajan, Vibhav. “Compassionate Use's 'Cruel Joke': Pharma Companies Don't Have to    Comply.” STAT, 5 June 2018, www.statnews.com/2018/06/05/right-to-try-compassionate-use-pharma-compliance/.