The U.S. Food and Drug
Administration published two rules today that seek to clarify the
methods available to seriously ill patients interested in gaining access
to investigational drugs and biologics when they are not eligible to
participate in a clinical trial and don’t have other satisfactory
treatment options. The agency also launched a new support Website.
The agency’s new Website will be 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.
The new rule, “Expanded Access to Investigational
Drugs for Treatment Use,” makes investigational drugs more widely
available to patients by clarifying procedures and standards.
The other rule, “Charging for Investigational Drugs
Under an Investigational New Drug Application,” clarifies the specific
circumstances and the types of costs for which a manufacturer can charge
patients for an investigational drug when used as part of a clinical
trial or when used outside the scope of a clinical trial.
“With these initiatives, patients will have the
information they need to help them decide whether to seek
investigational products,” said Margaret A. Hamburg, M.D., Commissioner
of Food and Drugs. “For patients seeking expanded access to
investigational drugs and biologics, the new rules make the process
easier to understand.”
Clinical trials are studies of drugs and biologics
that are still in development and have not yet been approved by the FDA.
Many patients enroll in clinical trials to gain access to
investigational therapies and contribute to finding out how well an
investigational therapy works, and how safe it is for patients.
Obtaining a drug or biologic under an expanded
access program may be an option for some patients who are not able to
enroll in clinical trials.
The FDA has allowed expanded access to experimental
drugs and biologics since the 1970s. That access has allowed tens of
thousands of patients with HIV/AIDS, cancer, and other conditions to
receive promising therapies when no approved alternative is available.
“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, M.D., director of the FDA’s Center for Drug Evaluation
and Research.
“Clinical trials are the most important part of the
drug development process in determining whether new drugs are safe and
effective, and how to best use them.”