May 13, 2022 - Written By: Maria Arini Lopez, PT, DPT
In 2019, the pharmaceutical industry spent upwards to $83 billion dollars on research and development of new treatments. A critical step in the research and development process are clinical trials to determine if the treatment is effective and safe for humans diagnosed with the condition which the drug is designed to treat. Participating in clinical trials is often the last option and hope for people who do not have any other viable treatment options.
What happens when you don't meet the criteria for inclusion in the trial? What happens when travel to trial centers is impossible? What happens when all existing or approved treatment options have failed?
This is where expanded access programs (EAPs), also known as Managed Access Programs (MAPs), Early Access Programs, or Compassionate Use Programs (CUPs), come into the picture.
The US Food and Drug Administration (FDA) developed EAPs so patients could access treatments that were still undergoing the process for FDA approval. EAPs differ from Right to Try programs supported by the Federal Right to Try Act of 2017. Right to Try programs do not have as many patient protections in place as EAPs.
The following EAP steps are designed to protect the patient, whose application goes through several layers of approval.
First, the patient's physician must identify a patient with an immediate life-threatening or serious condition as a potential candidate for the experimental treatment after all other treatment options have been exhausted OR alternative treatment options do not exist. The physician must weigh all options while simultaneously adhering to the oath to do no harm.
Next, the physician must submit an online application by filling out Form 3926 on the FDA's website on behalf of the patient. The Expanded Access Navigator streamlines the application process for the physician.
Additionally, the company conducting the clinical trials for the treatment in question must agree that the patient is a candidate. The company must determine that providing advanced access to the drug prior to FDA approval will not deter the drug's development process. The company must also have manufactured enough of the drug and have the financial resources to supply the EAP. Large research centers are more capable and open to participating in Expanded Access Programs.
Then, an Institutional Review Board (IRB) reviews and approves the application according to FDA regulations. Approximately 99% of EAP applications obtain IRB approval by the FDA. Denials occur because of significant safety issues that are identified or because of inadequate safety information for the review board to determine the risk-benefit of the treatment for the patient.
Lastly, the patient follows strict informed consent requirements based on risk-benefit assessments. These consent concerns are heightened in situations involving pediatric patients. To avoid therapeutic misconceptions, it is important that the patient (or the patient's parents or caregivers) recognizes that the drug is still experimental, so there is no guarantee of treatment success. Patients should be aware that only 10 to 15% of treatments that enter the research and development pipeline actually succeed in obtaining FDA approval.
The physician must handle the logistics of storing and administering the treatment properly whether it is administered orally, intravenously, or in other ways. The physician also is responsible for monitoring the patient for and reporting adverse side effects caused by the treatment.
Expanded Access Programs are one way to try experimental treatments as a last resort for serious, life-threatening conditions with FDA approval. The patient must collaborate with their doctor, the treatment manufacturer, and the FDA for this whole process to succeed.
While it is important to recognize that these investigational treatments offer no guarantee, what they do tend to offer is access to hope. After all, when all else has failed, hope makes anything possible.
References and Resources
An Introduction to Preapproval Access: Expanded Access, Compassionate Use, and Right to Try. Presented by NYU Grossman School of Medicine Division of Medical Ethics Working Group on Compassionate Use & Preapproval Access (CUPA). May 4, 2022.
Expanded Access Programs. Boehringer Ingelheim
Maria Arini Lopez, PT, DPT
Maria Arini Lopez, PT, DPT, CSCS, CIMT, CMTPT is a freelance medical writer and Doctor of Physical Therapy from Maryland. She has expertise in the therapeutic areas of orthopedics, neurology, chronic pain, gastrointestinal dysfunctions, and rare diseases, especially Ehlers Danlos Syndrome.