Bioethics Blogs

Group-Level Expanded Access to Unapproved Medicine: The realistic solution for patients

Guest Blog Author: Jess Rabourn*, Managing Director, ALS – Emergency Treatment Fund

Since 1962, Federal law prohibits open sales of any new pharmaceutical product until that product goes through years of clinical research and is granted approval by the FDA.  

Yet unapproved new drugs are provided to thousands of patients daily.  They’re not openly sold, but instead made available by drug companies to study safety and effectiveness in humans.    FDA allows such managed uses of unapproved drugs through authorized Investigational New Drug (IND) exemptions, which are filed by drug companies, physicians, or other program sponsors.

INDs are filed for clinical trials that are intended to gather data and to support marketing approval.  Additionally, INDs for Expanded Access may be filed when seriously ill patients lack access to traditional clinical trials and there is no other effective treatment available.  Expanded Access programs were used extensively in the late 1980s and early 1990s to provide exploratory treatment to persons living with AIDS. As new antiretroviral drugs were studied in research trials they were simultaneously made available in much larger Expanded Access programs for the many patients who were too sick to take part in the research trials.

Between 2002 and 2012, a new class of lifesaving TK-1 inhibitors was made available to tens of thousands of cancer patients through several Expanded Access programs.

FDA understands the need for treatment options and has a track record of liberally authorizing Expanded Access through group-level “Intermediate Size INDs” and larger group “Treatment INDs”.  A third kind of Expanded Access IND exists for exceptional single-patient cases, but this channel is inappropriate for most diseases, provides no useful data, and is almost never agreed to by the drug companies.  True Expanded Access programs enroll on a group level just like regular clinical trials and require no patient-level FDA filings.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.