Compassion [kuhm–pash-uhn]
noun
A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering.
Compassion Corner is a weekly series from Patient Worthy that will focus on the subject of compassion in the healthcare and rare disease space. In this series, we explore the role of compassion in this field and what it means for caregivers, patients, and others.
If a patient runs out of treatment options for a serious condition or disease then finds that they are not eligible for clinical trial enrollment, that patient has an unmet need. Yet there are still opportunities to access drugs that are in development.
As noted an the article published in myTomorrows, if a patient understands the constantly changing, often confusing, and inconsistent terminology, the patient will be a more active participant in treatment decisions.
International Terminology for the Pre-Approval Process
Patients may want to learn the various terms used in other parts of the world describing access to drugs that have not been approved by regulatory authorities.
“Pre-approval access” is a term that applies to drugs available by participating in clinical trials and other programs allowing for temporary (exceptional) use of a drug.
The terminology to describe mechanisms for pre-approval vary not only between countries but also between manufacturers. Sample terminologies are Expanded Access, Managed Access, Compassionate Use, and Named Patient Supply.
The approval process varies among countries and may be confusing even to physicians and pharmaceutical companies. However, the purpose is very similar. The general purpose involves getting access to the investigational drug when patients may not survive if they must wait for the completion of the lengthy process in their own country.
One regulation is unique to certain countries. These countries have regulations mandating that the drug has been approved in one or more other countries before considering pre-approval for the new drug.
The United States is responsible for a large portion of new drugs that have been approved and developed. It is for that reason other countries would benefit by having a regulatory system that would facilitate approval in their own country. Otherwise, drugs that have been newly approved will continue to be termed “investigational drugs” in these countries.
If a drug has been approved in one or more countries, a patient who has an unmet need will often be allowed to receive the drug if the following conditions are met:
· The patient must comply with the country’s pre-approval regulations
· The patient must fulfill the country’s eligibility criteria
· The patient must have a physician who is willing to prescribe the drug
“Managed Access Program” (MAP) is an umbrella term adopted by the pharmaceutical industry describing a worldwide program that involves various regulatory pathways in many different countries.
The MAP program offers access to patients who have no means of obtaining medicinal drugs that are not available or that have not yet been approved. A distinction is made between calling one patient a “named patient” versus calling a group a “cohort.”
About the U.S. FDA Approval Process
In the United States, the pre-approval process is termed “Expanded Access.” The physician requesting the drug has the responsibility of treating the patient. The physician is also responsible for fulfilling program and regulatory requirements. With respect to larger programs, the developer takes a greater program and regulatory responsibility.
About the European Process
In France, the French Temporary Authorization (ATU) program is used while the UK refers to the Early Access to Medicines Scheme (ATU). These are two well-structured programs used for purposes of pre-approval.
In addition, rather than depending on overseas approval, mainly from the U.S., both aforementioned programs make provisions for their own data collection. Other countries rely on the ATU and EAMS programs for guidance.
From Compassionate Use to Expanded Access to Right to Try
The fact that several of these terms have been used interchangeably has caused considerable confusion. Currently, FDA regulations refer to Expanded Access. Yet Compassionate Use is frequently mentioned in the media when referring to patients requesting the use of investigational drugs that are not within the confines of a Clinical Trial.
Over time the FDA began to receive complaints about the Expanded Access Program being burdensome and lacking progress. This resulted in federal law that is called the Right to Try Act. Although it gave U.S. patients another path to receive investigational drugs it also added to the confusion. People refer to the Right to Try Act when they are looking for pre-approval from the FDA’s Expanded Access program.
In 2017, a team from Langone Group at NYU suggested using a global term such as “pre-approval access.” Again there was a chorus of objections arguing that the term insinuates definite future approval of the drug. The term Expanded Access is an umbrella term that is preferred by the pharmaceutical industry, especially when referring to the United States.
It is understandable that the inconsistency has caused confusion among physicians, patients, and even the pharmaceutical industry. Understanding the global and historical context will aid in using the correct terms while meeting the goal of helping patients who are not eligible for trials but clearly have unmet needs.
Continued Next Week in Part Two
