Expanded Access Programs (EAPs), also referred to as Managed Access or Compassionate Use Programs, allow patients with no viable treatment options available to them to access investigational drugs before they are approved for market use. In the US, the FDA is charged with approving these programs before patients can receive drug, and many see this FDA involvement as a substantial roadblock to patient access.
However, there are others that argue that it is the pharmaceutical companies developing the drugs that are more likely to restrict patient access. Companies are never required to provide their investigational products to patients outside of the clinical trial, and often there are perceived barriers in place that prevent companies from doing so. Some companies do not have the resources (money, man-power, expertise, etc.) to manage an EAP. Others only have enough drug to supply patients in the clinical trial. Others do not want to take focus away from the clinical trial efforts, as this could lead to longer timelines and delayed drug approval.
With such limitations in mind, there are three important things patients should consider before reaching out to a pharmaceutical company to request access to a drug.
- A company can only provide expanded access use of its investigational products if the drugs are being developed to treat serious or life-threatening conditions. Expanded Access is intended to help patients who are desperate for treatment and who cannot wait for a new drug to receive approval and come to market. As such, companies developing drugs for common conditions, for which there is one of more treatment option available, will not agree to provide their products to patients outside the clinical trial.
- A company can only provide expanded access use of products that are in the late stages of clinical development. Usually, a drug will have to be in Phase II or Phase III clinical trials before a company can give it to patients outside of the established clinical studies. This is because expanded access use of a drug is provided in a minimally controlled outpatient setting, so there must be enough safety and efficacy data collected by the sponsor company before it can be permitted. There are scenarios in which a company can provide access during a Phase I trial, but that is only in extreme cases, like when a drug is being developed for an ultra-rare disease and it has shown promise in early studies.
- Although the access criteria are less stringent for an EAP than they are for a clinical trial, there are still reasons a sponsor company could deny a patient access to a drug through expanded access. Any adverse events reported during an EAP must be presented to the FDA. Because many sponsors worry that presenting adverse events will hurt their chances of receiving marketing approval, they may choose to withhold drug from patients that are likely to produce these events. This could be patients that are so far along in their disease that they are unlikely to respond to any form of treatment, or patients that are using concomitant medication that is likely to negatively affect how the patient responds to the investigational product.
It is important to remember that patients should always consult with their physician before reaching out to a pharmaceutical company. The expanded access process cannot proceed without a licensed physician willing to act on a patient’s behalf.
