Gaining Access

Desperately ill patients who have run out of treatment options can face an agonising wait for experimental therapies to be approved. While unlicensed drugs often can hold hope for those patients, the traditional method of obtaining these therapies – that is, the clinical trial – can be a daunting process. Many patients may not meet any eligibility criteria, may not be located close to a trial centre, or may find that the trial is already fully enrolled.

An increasingly used alternative for patients lacking an effective treatment option who cannot enrol in a trial may be a compassionate use or expanded access programme. These are similar to a clinical trial in that the patient must meet specific criteria; however, the requirements are typically less stringent. Patients often also can gain access to unlicensed drugs through a single-patient compassionate use programme, frequently referred to as a named patient programme.

While compassionate use schemes are relatively similar throughout the developed world, they can differ in detail. In the US and the EU significant changes have either taken place or have been proposed that may have the effect of harmonising disparate schemes and improving access to investigational drugs, with benefits to both patients and manufacturers alike.

TREATMENT INDs AND COMPASSIONATE USE OF INVESTIGATIONAL DRUGS IN THE US

The historical basis for current US guidelines on the compassionate use of drugs lies largely rooted in the thalidomide tragedy of the late 1950s to early 1960s. This resulted in the birth of more than 10,000 congenitally deformed infants in Europe. US public attention was riveted on drug regulation, when in the summer of 1962, newspaper stories disclosed links between the experimental drug and severe birth defects. Three months later the US Congress unanimously passed the first major amendments to the Federal Food, Drug and Cosmetics Act of 1938 (1).