Introduction

Patients with life-threatening conditions who have not responded to the standard of care for their condition may have the option of choosing an Early Access Program.

Background

The Food and Drug Administration began the Early Access Program in 1987. This program allows patients access to investigational drugs that have had very positive results in clinical trials but have not yet been approved for public use. Since these drugs are still under development, their safety and efficacy remain questionable. However, because of the early positive results, these drugs may represent hope to the very ill patient.

Issue

How can patients qualify for an early access program?

Rule

The Code of Federal Regulations (21CFR312.305) requires patients to meet the following criteria in order to participate in an early access program:

  1. No satisfactory approved medication for the patient’s disease or condition is available.
  2. The potential benefits offered by the experimental drug compensate for potential risks it might bring to the patient.
  3. Making the experimental drug available to patients through expanded access will not interfere with the drug’s regulatory development.

Analysis

The first criterion can be met in several ways including:

  • The patient suffers from a rare disease that does not have an approved treatment
  • The patient has a disease with available treatment but it has failed to improve the patient’s condition
  • The patient is allergic to the available treatment

The second criterion is one that needs to be decided upon by the patient with input from his or her doctor and close family members. They review all possible known risks of the developmental drug and all possible benefits before deciding.  If the experimental drug has had positive results in earlier trials, and all other treatments have failed to help the patient, then the experimental drug may be a good option.

The third criterion is one directed to the drug manufacturer. Making the drug available to patients through an access program should not slow down clinical trials.  The manufacturer needs to continue to collect efficacy and safety data of the drug in question and not interrupt the drug development process.

Conclusion

If a patient suffers from a condition with no available treatment or all treatments available have failed to help him or her, an Early Access Program is a good option. The benefits from using the investigational drug must be greater than the risks, and the availability of the drug must not affect the development of the drug in question. If these requirements are met, the patient qualifies for the program and should participate.