Vanguard Neurologist

From Bloomberg.com:

Seriously ill patients will be allowed greater access to experimental drugs under regulations approved by the U.S. Food and Drug Administration.

The rules let drugmakers give or sell experimental medicines to patients who have no other options, including in emergencies, the agency said today on its Web site. Patients and doctors can see a new Web site to learn about the use of experimental treatments as part of a clinical trial or outside of one, the FDA said in a statement.

The FDA has allowed tens of thousands of patients access to unapproved therapies since the 1970s, the agency said in its statement. The regulations clarify existing programs for providing novel medicines through clinical trials and expand drug access to additional groups of patients. The rule changes adopted today were proposed in December 2006 and may provide experimental drugs to as many as 3,095 more patients each year, the agency said.

“The final rules balance access to promising new therapies against the need to protect patient safety,” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, in the statement. “Clinical trials are the most important part of the drug development process in determining whether new drugs are safe and effective.”

Protecting Trials

Some drugmakers and their trade organizations told the FDA that expanded access outside of clinical trials might impede the development of new drugs, since there would be less reason to participate in those studies, according to comments on the agency’s Web site. Clinical trials are used to test safety, dosing and effectiveness of new drugs and support applications for drug approvals. The trials attract participants by offering access to treatments that otherwise may not be available.

The Standard & Poor’s 500 Pharmaceuticals Index of 13 companies rose less than 1 percent to 268.73 at 4 p.m. New York time.

Under the rules, the company and the FDA must agree to allow pre-approval use of a drug, Woodcock said today during a conference call. Companies will have the option of charging patients for the cost of producing and administering drugs when not given as part of a trial. The regulation is meant to encourage companies that may not be able to afford to provide a drug for free, the FDA said.

“On the surface, it doesn’t look like this is going to have a great impact or be a new revenue boon,” said David Heupel, a portfolio manager at Thrivent Financial for Lutherans in Minneapolis, in an interview. “The intentions here are good. They want to make it easier for somebody who is really out of treatment options to try something that might have some benefit.”

Unintended Consequences

The expanded access could have the unintended consequence of making drug approval more difficult, Heupel said. Whenever a patient gets sick or dies while taking an experimental medicine, that must be reported to the FDA. When the therapies are given to very sick patients outside the close monitoring of a clinical trial, such reports could make regulatory evaluations of a drug’s safety “a little more cloudy,” Heupel said.

“Three thousand patients is just a drop in the bucket,” said Frank Burroughs, a founder of the Abigail Alliance, an advocacy group that supports allowing more patients to use experimental drugs. “These regulations do very little to increase expanded access.”

The FDA should do more to protect drugmakers from lawsuits over experimental drugs and allow patients to receive treatments at early stages of clinical development, Burroughs said.

Choice to Charge

“We support the FDA’s efforts to improve access to investigational drugs for patients with serious or immediately life-threatening diseases,” said Ken Johnson, senior vice president of the industry group, Pharmaceutical Research and Manufacturers of America, or PhRMA. “As to charging for investigational drugs, these are decisions that will have to be made individually by research sponsors.”

If a drugmaker wants to charge for a therapy as part of a clinical trial, the company must provide financial information to the FDA and persuade the agency it couldn’t otherwise offer the drug, Woodcock said. Testing and administration expenses can’t be charged in trials.

About 162 to 3,095 additional patients may gain access to experimental drugs each year because of the rule changes, the FDA estimated in its documents.

Woodcock said it’s too early to know the exact numbers and the agency will track the program, which is scheduled to take effect in two months.

“I don’t think this will radically expand access, but we believe a large number of clinicians, investigators, patients and patient groups who weren’t totally aware of these programs will now become aware of them and will have access,” she said.

To contact the reporter on this story: Tom Randall in New York at trandall6@bloomberg.net.