The process by which access to medicines is provided prior to approval and launch can be confusing. Adding to the confusion is the fact that these programs are known by many different names around the world. In this overview, we clear away the confusion and describe what these programs are and how they work.

The Need for Managed Access

Around the world, many patients with serious illnesses are unable to access the medicines they need. They may have exhausted all available commercial therapies for their disease, they may not have access to a clinical trial, or they may live in a geography where a much needed drug will not be made available through commercial routes. For these patients, access to a medicine outside the clinical trial or commercial setting can represent a new, and in many cases, lifesaving treatment option.

“Managed Access” is a term we use at Idis to encompass a variety of regulatory approaches used to provide access to medicines outside the clinical trial or commercial setting for a variety of reasons including those drugs that:

• Are still in clinical development and have yet to be approved
• May never be approved, but still offer value for a very small population (for example, some orphan drugs for rare diseases)
• Are approved in one country, but not another
• Are approved for one indication but may have benefits for patients with a different indication

Managed Access Programs can also be used to provide access to a medicine when it has been discontinued in a particular market or to source an alternative to a drug discontinued globally.

The type of access enabled by these programs can offer significant benefits to patients and their healthcare providers as they can deliver a new therapeutic option when all alternatives have been exhausted.

For Patients in the United States

Since 1987, the US Food and Drug Administration (FDA) has had rules in place that have enabled patients (either individually or as part of a group), under specific circumstances, to access drugs or biologics that are still in development for treatment purposes. Access is typically considered once a medicine has shown positive results in later stage clinical trials and has been shown to be safe.

These expanded access program (EAP) rules were amended in 2009 to ensure “broad and equitable access to investigational drugs for treatment.” This type of access was previously referred to as “compassionate use”, but today the FDA refers to this as “expanded access”.

The regulations include the following:

• Criteria that must be met to authorize the expanded access use
• Requirements for expanded access submissions
• Safeguards to protect patients and the clinical trials process

The regulations also define the different types of mechanisms that are available including:

• Access for individual patients under a Single-patient IND (investigational new drug; also known as an Emergency IND)
• Access for small to intermediate groups of patients
• Access for larger groups of patients via a Treatment IND or Treatment Protocol

Single-patient INDs
A single-patient IND is a request from a physician to the FDA that an individual patient be allowed access to an investigational drug. Provisions are also in place to obtain medicines on an emergency use basis, allowing patient treatment prior to the completion of the requirements for a single patient IND. The term “compassionate use” is sometimes used in place of single-patient IND, but the term is not officially recognized by the FDA.

Intermediate-Size Patient Populations
The FDA established specific criteria when this type of IND may be appropriate. One example is that the drug is not being developed because the disease is so rare, impeding a company’s ability to recruit patients for clinical trials. The FDA may also ask the company to consolidate a significant number of single patient INDs for the same use under an intermediate-size patient population IND.

Treatment INDs and Treatment Protocols
Treatment INDs and treatment protocols provide large numbers of patients, who would not otherwise qualify for clinical trials, access to investigational drugs.

For Patients outside the United States

Outside the US, these access programs are known by a variety of names including “named patient programs” (NPPs; in which physicians can request access to drugs on behalf of individual or “named” patients), “named patient supply”, “compassionate use”, and “Autorisations Temporaires d’Utilisation (ATU)”.

Regulations differ widely among countries due to differences in national medical practices, resources available, product funding, hospital structures and national insurance systems.

These programs are similar to US-based EAPs in that they facilitate access to investigational drugs for individual patients or groups of patients with unmet medical needs. These programs also allow access, in specific circumstances, to drugs that are approved in other countries but not yet approved in a patient’s home country. Additionally, when there is a small patient population spread across a large number of countries (as is often case with a rare disease), a company may be able to offer access to their drug in lieu of seeking approval and commercial launch in dozens of geographies which might not be practical or financially feasible.

The thirty countries comprising the European Union (EU) and European Economic Area (EEA)— which fall under the jurisdiction of the European Medicines Agency (EMEA)—each have their own nationalized regulations regarding the access to unlicensed medications for individual patients and groups of patients. In Europe, named patient programs also allow patients to access drugs in the time period between centralized EMEA approval and launch in their home countries which can range from one year to eighteen months.

Countries outside the U.S. and Europe also offer these programs. Canada’s “Special Access Program” provides access to non-marketed drugs to practitioners treating patients with serious or life-threatening illnesses when conventional therapies have failed, are unsuitable, or are unavailable. Similarly, in Australia, patients can access experimental drugs via the “Special Access Scheme;” in Japan, government sanctioned “Named Patient Access” allows access to drugs with an expectation that the drug be approved in the exporting country.

Getting Started

If you think getting access to a medicine via a Managed Access Program might be an option for you as a patient, the first step is to contact your physician. In partnership with your doctor, you can determine if a medicine available via a Managed Access Program is appropriate for your specific situation.

Keep in mind that the pharmaceutical or biotechnology company that has developed the drug may choose not to offer it via a Managed Access Program. Companies are not required by regulatory authorities to provide this type of access – it is at their discretion.

To initiate the process, please have your physician contact Idis directly. A list of our offices and phone numbers can be found at www.idispharma.com/contact-us.