The US Food and Drug Administration has published new rules to make it easier for seriously-ill patients to receive investigational drugs and biologics, and inform drugmakers when, and how much, they can charge in these cases to recoup their costs.

The new rules, which apply to patients who are not eligible to participate in a clinical trial and have no other satisfactory treatment options, are backed up by a new web site where patients and their health care professionals can learn about options for investigational drugs. In general, these options include being treated with a drug that has been approved by FDA, being given an investigational drug as part of a clinical trial or obtaining access to an investigational drug outside of a clinical trial.

“With these initiatives, patients will have the information they need to help them decide whether to seek investigational products,” said FDA Commissioner Margaret Hamburg. The aim of the new rules is to achieve “a balance between the need to gather information to demonstrate whether drugs work and the fact that critically ill patients urgently want access," she added.

The initiatives will not create any changes to the FDA’s drug approval processes, agency officials point out.
They also note that the agency has in fact allowed expanded access to experimental drugs and biologics since the 1970s, thus allowing tens of thousands of patients with HIV/AIDS, cancer and other conditions to receive promising therapies when no approved alternative is available. The new rules do not change this but establish a clearer pathway, and are expected to increase applications to receive such unapproved drugs, made by and for patients individually or in groups, by 50%.

“The final rules balance access to promising new therapies against the need to protect patient safety and seek to ensure that expanded access does not discourage participation in clinical trials or otherwise interfere with the drug development process,” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research (CDER). She added that making such drugs available outside of a clinical trial can be costly for manufacturers, and that by allowing them to charge for expanded access, the agency hopes to encourage more firms to provide investigational drugs, although they will have to satisfy officials that the drug would not be developed unless the company was able to charge for this use.

- In January 2008, the US Supreme Court turned down a request for it to review earlier court rejections in the long-running cases brought by the Abigail Alliance for Better Access to Developmental Drugs. The Alliance had claimed that “a terminally-ill patient with no approved treatment options has a right to decide for himself, in consultation with his own doctor, whether to take a drug that the FDA concedes is safe and promising enough to be tested in substantial numbers of human subjects.”