Compassionate Drug Use: A walk Through

Compassionate drug use, sometimes referred to simply as “compassionate use,” refers to the use of new and unapproved drugs to treat seriously ill patients when no other treatments are available. These drugs are usually in clinical trials, but using them outside of trials is considered compassionate use.

Patients must meet strict criteria to receive approval for the compassionate use of a drug. The treating physician must show that the patient has tried all other treatments, that their condition is serious or life-threatening, and that the drug is safe for the specific patient population. The physician must then submit a request and receive approval from both the drug company and the FDA. The physician must also agree to monitor the patient and report any adverse effects to the FDA. The compassionate use process safeguards public health and safety by ensuring patients receive effective and safe treatment in accordance with regulations and laws.

Criteria for Approval

1. To receive approval for compassionate use of a drug, patients must meet strict criteria, including:

• The disease is serious or life-threatening.
• No treatments are available or approved treatments have failed.
• The patient is not eligible for clinical trials.
• The doctor believes the experimental treatment may help and the benefits outweigh the risks.
• The drug company agrees to provide the drug.

Considerations for Compassionate Drug Use

When considering compassionate drug use, the following must be into account:

1. Patient eligibility: The patient must have a serious or life-threatening condition and have exhausted all other treatments.
2. Drug safety: The drug must be in clinical trials or have been approved by the FDA, and deemed safe for use in the specific patient population.
3. Physician request: A treating physician must submit a request and receive approval from both the drug company and the FDA.
4. Monitoring: The physician must agree to monitor the patient and report any adverse effects to the FDA.
5. Regulations: The use of the drug must be in accordance with any applicable regulations and laws.
6. Public health: The compassionate use process must protect public health and safety.
7. Effective treatment: The goal of compassionate drug use is to provide patients with safe and effective treatment.
8. The patient must have a medical need that cannot be met by existing treatments.
9. The criteria for compassionate drug use are stringent and must be complete before receiving approval.
10. Legal authorization: The use of compassionate drugs requires legal authorization.

Expanded Access Programs and “Right to Try”

In addition to compassionate drug use, patients may be able to access drugs through Expanded Access Programs (EAPs) or the “Right to Try” law. EAPs come in three forms: widespread, intermediate, and individual use. The “Right to Try” law allows patients to access unapproved drugs without involving the FDA, but it does not guarantee approval and the drug company may decline the request.

1. E. Access Programs: These programs are in approval of FDA and allow patients with serious or life-threatening conditions to receive experimental treatments when no other options are available.
2. “Right to Try”: This law provides a pathway for terminally ill patients to access experimental treatments without FDA approval.
3. Drug company agreement: Both programs require the drug company’s agreement for patients to receive the experimental treatment.
4. Monitoring and reporting: Patients participating in these programs must be in continuous monitoring simultaneously noting any adverse effects.
5. Patient eligibility: Patients must have exhausted all other treatments and have a serious or life-threatening condition.
6. Physician agreement: A treating physician must agree to participate in the program and monitor the patient’s health.
7. No FDA approval: The treatments received through these programs have not been approved by the FDA and may carry risks.
8. No guarantee of success: There is no guarantee that the experimental treatment will be effective.

Expanded Access Programs and “Right to Try” provide hope for patients with serious or life-threatening conditions. However, it is important to carefully consider the potential risks and benefits before participating in these programs. Patients must work closely with their treating physician to make the best decision for their health.

Criteria for “Right to Try” and Compassionate Drug Use

To be eligible for compassionate use through “Right to Try,” patients must have a life-threatening disease. Have tried all approved treatments, be unable to participate in a clinical trial, and provide written informed consent. The drug must have already gone through Phase I clinical trials and patients must be certified by a doctor. Both “Right to Try” and EAPs require specific criteria, a doctor’s request, and may have limitations regarding cost and drug safety oversight. Compassionate use pathways offer terminally ill patients a chance to receive life-prolonging or life-saving drugs.