Named Patient Medicine

Named Patient Programs, also referred to as compassionate use programs or expanded access programs, play a pivotal role in providing access to potentially life-saving medications for patients who are facing serious or life-threatening conditions. These programs are designed to bridge the gap between the development of new medications and their regulatory approval, ensuring that patients in need have access to innovative treatments before they are widely available on the market.

Operated by pharmaceutical companies, Named Patient Programs operate on a case-by-case basis, allowing eligible patients to receive investigational drugs outside of the confines of traditional clinical trials. This can be particularly beneficial for patients who may not meet the criteria for participation in clinical trials or who have exhausted all approved treatment options.

Through Named Patient Programs, patients can gain access to promising new therapies that have not yet received regulatory approval in their country. This can provide a ray of hope for patients who are facing limited treatment options and may offer a chance for improved outcomes and quality of life.

In addition to providing access to investigational drugs, Named Patient Programs also facilitate the provision of unlicensed medicines and unregistered medicines, which have not yet undergone formal regulatory approval processes but may still hold potential benefits for patients in need. These programs serve as a lifeline for individuals who require access to treatments that are not available through conventional channels, offering hope and the possibility of improved health outcomes.

How Named Patient Program Work?

In every case, the request has to be unsolicited, and there are specific things that need to be true:

• The patient/s are afflicted by an illness that poses a significant threat to their life, persists for an extended duration, or severely impairs their quality of life.
• At present, there are no approved treatments available that effectively address the disease or condition in a satisfactory manner.
• The patient is ineligible to participate in a clinical trial that is currently underway.
• The medication or therapy should either be currently undergoing clinical trials or have progressed to the stage of entering the marketing authorization application process.
• For any Managed Access Program to proceed, a thorough assessment of the product's benefit-risk profile must be conducted, ensuring it aligns favorably with the needs of eligible patients. This evaluation relies on the latest data and necessitates that the intended use of the product corresponds to its targeted indication.

While numerous avenues exist for ensuring patient access to a product, it will only be accessible through the Named Patient basis.

Under this program, treating physicians have the opportunity to submit individual requests for specific patients, thereby facilitating access to the program for those in need of specialized treatment. These may be therapeutic drugs that are:

• Approved for use but not yet accessible for prescription in the patient’s country.
• Approved for use and readily accessible within one nation, yet it lacks approval and availability within the patient's own country.
• No longer available in the patient's country but still accessible elsewhere.
• In limited supply within the patient's country, while it remains readily available in another jurisdiction.