The FDA’s Expanded Access Program

In response to complaints about the slow development and approval process and the fact that patients cannot get new medicines until they are approved, the FDA had made a push to make certain medicines available to some patients in advance of approval.  Expanded access allows patients with  “immediately life-threatening disease or condition” to get medicine if certain criteria are met.

To qualify, the patient must not have access to alternatives that doctors think will work for them.  A licensed physician must agree to oversee treatment and to work with the FDA, drug maker, and Institutional Review Board.  The expanded access program applies to both small molecule drugs and biologics such as monoclonal antibodies.

The FDA prefers that patients receive these medications while they are enrolled in a clinical trial.  The reasons for this are (1) the patient’s health will be monitored while they are in the trial, (2) the people who run the trials will dole out the exact dosages and regimens recommended to the best of their knowledge, and (3) data from the patient’s response to the medication can form part of the clinical trial’s findings.

However, if the patient is unable to get into a trial for the medicine of interest, the FDA has other paths, but they may require more work for the patient, physician, and drug company than a clinical trial does.  

There is a webpage for you to submit your request:

The FDA does not allow individual patients to initiate requests.  A doctor must be on board, and manufacturers may also initiate expanded access requests.  The requests can be for individual patients or for groups.  The FDA says it approves the large majority of requests.

Right to Try

The federal Right to Try Act was passed in 2018; it followed on similar laws enacted in some states.  These laws allow seriously ill patients to use pharmaceuticals that have not been formally approved by the FDA.  Because it takes a long time for new medicines to get through the approval process, news of a new compound may reach patients who are interested in trying them years before they will be approved.  For years patient advocacy groups petitioned politicians and FDA officials and anyone who would listen with the plea that people with terminal diseases should be able to try new medicines.

The Right to Try Act allows drug makers to sell or give their products to patients.  There are strict criteria on which patients are eligible and which medicines are eligible.  Medicines must have completed the first phase of clinical trials.  Phase I is where safety is established.  The efficacy (whether it works) and side effects are established in Phases II and III before the medicine is approved for general use.

The pharmaceutical company that owns the rights to the investigational medicine (which is almost always the company sponsoring the clinical trials) has the option of making their drug available under this program.