FDA Increases Public’s Access to Investigational Drugs

The Food and Drug Administration today is making it easier for seriously ill patients to gain access to unapproved, investigational drugs when they are not eligible to participate in clinical trials of the drugs and do not have other viable treatment options.

The FDA has just published two new rules designed to make it easier for patients and their healthcare providers to understand the procedures and standards for qualifying to receive investigational drugs outside of participating in clinical studies of the proposed medications.

“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.”

One of the new FDA rules, called “Expanded Access to Investigational Drugs for Treatment Use,” clarifies and explains how people can qualify to receive investigational drugs, which have not yet been reviewed or approved by the FDA and are not available in pharmacies. The other rule, known as “Charging for Investigational Drugs Under an Investigational New Drug Application,” further explains in greater detail the specific circumstances and costs drug companies can charge patients for investigational drugs when the patient is not involved in a clinical trial.

In a clinical trial, drug companies test proposed new drugs or medical devices on patients to determine whether the products are safe and effective at treating a variety of diseases, disorders, and conditions. People can enroll in the clinical study to receive the drugs, but for patients who for one reason or another do not qualify to participate in the study, their access to the drugs is very limited.

In some cases, the FDA grants drug companies limited permission to market investigational drugs for patients with specific medical problems for which there are no other viable, approved therapies. Since the 1970s, thousands of patients with cancer, HIV/AIDS, and other conditions have been given emerging treatments that had not yet been approved by the FDA.

However, today’s new rules further expand access to these unapproved, cutting-edge medical treatments for patients who are seriously ill but cannot receive the drugs through clinical studies, the FDA said.

“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.”

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