PHOTOGRAPHY: GETTY IMAGES

Global regulations are in place by which a patient may gain access to investigational medicines via their physician. To ensure patient demands are met, companies may choose to institute a prelaunch access program. Companies must carefully orchestrate use of these programs in conjunction with ongoing clinical trials in order to maximize their effectiveness while remaining within strict regulatory guidelines.

This article will describe the differences among early access programs, highlight how regulations can vary from country to country, and describe how programs can operate successfully in parallel with clinical trials.

Patients can potentially gain access to drugs in a preapproval stage by a variety of mechanisms including clinical trials, expanded access programs (EAPs), compassionate use programs, and named patient programs.

In 1987, the U.S. FDA formally put in place regulations to improve patient access to investigational drugs. These regulations focused on drugs that would address existing therapeutic gaps, including cases in which therapies were not available and those in which patients were unresponsive or not adequately treated with currently marketed drugs.

Nearly two decades later, proposed changes to these rules¹ are intended to clarify the procedures for allowing early access and give patients more latitude in accessing investigational drugs when there are no other therapeutic options. The 90-day window for public comment on the proposed regulation closed in March 2007. While no date has been specified, the FDA is expected to issue a final ruling on these amendments shortly.

An appropriate balance of possibly competing interests continues to be sought: facilitating access to unapproved drugs and minimizing risk to patients while not impeding the progress of clinical trials and data development needed for marketing approval.

FDA-recognized EAPs include treatment investigational new drugs (treatment INDs), treatment protocols, and single-patient INDs. EAPs allow patients who are too ill or do not otherwise qualify for clinical trials to access potentially lifesaving medicines that are still in development when no other medical alternatives exist. While these programs are often grouped under the umbrella of "compassionate use programs," the FDA does not have a regulation or policy defining a compassionate use program, instead preferring to consider "compassion" an element in all aspects of drug development.²

Treatment INDs and treatment protocols are similar in that they both allow large numbers of patients, who would not otherwise qualify for clinical trials, to gain access to an investigational drug. A treatment protocol is a formal addendum to an active clinical trial, one that sets criteria for allowing large numbers of patients to access the same treatment regimen that is under investigation and is submitted to the FDA by the drug company. In contrast, a treatment IND is initiated by a physician seeking early drug access on behalf of a patient or group of patients who do not qualify for a clinical trial but who may benefit from preapproval access to a new drug.

A single-patient IND is a request from a physician to the FDA that an individual patient be allowed access to an investigational drug on an emergency or compassionate basis—that is, when all other options have failed. The FDA makes it clear that the drug company must agree to the request first and that the FDA does not have the power to compel a company to release a drug for an unapproved use.³