An expanded access program (EAP) provides patients and healthcare professionals with access to experimental pharmaceuticals that have been pre-approved for usage outside of clinical trials.
Patients with a serious or life-threatening condition who do not have access to any viable treatment options are eligible to participate in these support schemes.
EAPs allow sponsors to give access to pre-approved medications outside of clinical trials. These initiatives are unique to each country and need differing degrees of regulatory approval.
How Expanded Access Works
The decision-making process for requests for expended access is notably challenging. Taking into account the patient's situation, the current state of medical and scientific knowledge about experimental medicine(s), the likelihood of receiving regulatory approval, and the timeliness of such approval, healthcare professionals strive to make these decisions most ethically and acceptably possible. They prioritize implementing expanded access to minimize the risk of harm to individuals.
How Is an Expanded Access Program Different from Clinical Trials?
A clinical trial and an expanded access program are two techniques for providing investigative new drugs (INDs) to patients who are in serious condition and have exhausted all other therapeutic options available. However, these two concepts are not synonymous. As clinical trials become more common in the global healthcare system, healthcare practitioners, patients, and advocacy organizations representing patient interests will need to understand the difference between an EAP and a clinical trial. This is because the distinction between the two will become more important.
The fundamental difference between an EAP and a clinical study is the aim they seek. A pharmaceutical business conducts clinical research to get approval for its investigational new drug (IND) and a license to use it. To get approval for the medication, the manufacturer must show the national health regulatory body that the medicine is safe for use and that it is comparable to, if not superior to, the pharmaceutical that is currently on the market. Clinical trials allow a corporation to use its product to treat patients in a controlled and regulated setting. This enables the company to gather critical data on the drug's efficacy and safety.
An EAP is a distinct option. While an IND producer may still gather data on a medicine's efficacy and safety from EAP participants, these programs do not aim to facilitate the successful entry of a new pharmaceutical into the market. Conversely, these projects primarily aim to provide medication to areas with unfulfilled medical needs. Because it is the patient's final opportunity, the drug company will often provide the medicine via an EAP.
Why Are EAPs Important?
The goal of clinical trials and the FDA approval procedure in the United States is to protect patients from harm and save them from wasting valuable time and money on medicines that will not affect their condition. The FDA performs evaluations to ensure that the information acquired from clinical research is reliable and trustworthy.
Participating in a clinical trial is one of the few opportunities for a considerable number of people suffering from terminal and/or severe illnesses to get a potential medication. However, this option is not available to everyone; for example, some people may be ineligible for a trial owing to enrollment criteria, while others may not have a trial site close by. Certain people may not be able to participate in a study because there are insufficient spaces available. Amyotrophic lateral sclerosis (ALS) typically lasts three to five years after diagnosis, but the clinical trial process and its three phases can take up to ten years to complete. This is especially difficult for individuals who suffer from a sickness that develops quickly and eventually kills them.
Individual Patient Access to Investigational Drugs
In recent years, both the general public and policymakers have shown an interest in expanded access. This attention also extends to the broader population. Since 2009, the FDA's expanded access regulations have amplified campaigns and online petitions calling for specific individuals to have access to experimental drugs through social media platforms. Furthermore, state lawmakers persist in their pursuit and approval of right-to-try legislation, enabling firms to distribute experimental drugs without the Food and Drug Administration's oversight.
A great deal of information and cooperation from many stakeholders is required to offer expanded access programs for specific patients. Physicians, medication manufacturers, institutional review boards (IRBs), and the Food and Drug Administration (FDA) must work together for the expanded access approach to be a success. The patient's doctor must first contact the pharmaceutical company to find out whether they are prepared to provide the medicine. As soon as the company gives its approval, the doctor must apply to the FDA and get approval from the Institutional Review Board (IRB). If the FDA cannot complete a formal request, it may permit the administration of the emergency drug through electronic means, such as telephone. Despite the need to make a formal request later, the doctor must obtain the patient's informed consent before administering the experimental medication.
