FDA proposes new rules for investigational drugs


The Food & Drug Administration recently proposed regulatory changes to make investigational drugs more widely and easily available to seriously ill patients with no other treatment options. The proposal also clarifies the circumstances and the costs for which a pharmaceutical company can charge for developmental drugs. The proposed regulations were published in the Dec. 14 issue of the Federal Register. They are described in detail at http://www.fda.gov/cder/regulatory/applications/IND_PR.htm and are open for comment for 90 days.

The most significant proposals would update applicable regulations to clarify all circumstances under which access to developmental drugs is permitted-including single patients, small groups of patients, and larger clinical trial populations. For expanded access uses, the FDA must be satisfied that no satisfactory or alternative therapy is available, that the potential benefit to the patient(s) outweighs the risk, and that expanded access will not interfere with the standard drug approval process.

He went on to note that "other regulatory issues must also be addressed, including keeping track of adverse reactions among patients taking experimental drugs."

Greg Simon, president of FasterCures, an advocacy group, agreed that overextension of compassionate use-or use of an investigational drug by patients not enrolled in clinical trials because they have run out of treatment options-may limit the number of patients willing to participate in clinical trials. He said patients may be unwilling to participate in a clinical trial if they think they may gain access to the drug without study participation. Simon also said pharmaceutical companies may be discouraged from conducting clinical trials for fear that adverse effects among a relatively small number of patients taking the drug for compassionate use may reduce the likelihood of its approval.

Pharmacists can play a significant role in educating patients regarding compassionate use, Simon noted. They can help people understand why clinical trials are the best long-term solutions to curing diseases, and explain that compassionate use is an extraordinary measure that may help certain individuals. If overused, however, it might slow the development of cures for serious or life-threatening illnesses.

The question of whether dying patients should gain easier access to unapproved drugs is at the center of a lawsuit, Abigail Alliance v. von Eschenbach, now heading toward a possible showdown in the U.S. Supreme Court. FDA's new proposal to provide expanded access to experimental drugs was issued after the suit was filed.

According to Albert Wertheimer, Ph.D., MBA, director of the Center for Pharmaceutical Health Services Research, Temple University School of Pharmacy, Philadelphia, greater access does not mean that patients will abuse experimental drugs. "People will not abuse these drugs or try to get high. They are not cheap, and they would be used primarily in cases of terminal illness when nothing available seems to work. In addition, many of these drugs have horrible side effects, so they would normally be used only when really needed."

More information on expanded access is available through FasterCures (http:// http://www.fastercures.org/) and the Abigail Alliance for Better Access to Investigational Drugs (http:// http://www.abigail-alliance.org/).

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