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Experimental Treatments? Unapproved But Not Always Unavailable

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As if these hurdles are not enough, there is the long-held, but incorrect, public perception that FDA erects regulatory barriers that block patients from getting investigational new drugs (INDs). These are drugs that pharmaceutical companies have in clinical trials to demonstrate their safety and effectiveness, but which have yet to be approved by FDA for marketing. For those with a serious illness, the agency rarely blocks access to unproven medications. But FDA does strive to protect all patients, even those who may be dying, from undue risks associated with investigational new drugs. At the same time, FDA believes that the best way to benefit all patients is to speed promising new therapies through the development and approval process so safety, effectiveness and proper use can be established.

"FDA has worked diligently to balance two compelling, and sometimes competing, factors," says FDA Commissioner Jane E. Henney, M.D. "On one hand, there is the need for the disciplined, systematic, scientifically controlled studies necessary to identify treatments that may improve patient health and that lead to the approval of new drugs. At the same time, there is the desire of seriously ill persons, with no effective options available, to have the earliest access to unapproved products that could be the best therapy for them."

Over the last decade, FDA's institutional philosophy has evolved to be more supportive of thoughtful risk-taking by patients who have run out of options. As a result, the agency has put in place a number of regulatory mechanisms and worked with manufacturers to ensure that seriously ill patients can get access to promising, but not fully evaluated, products. At the same time, FDA has protected the critical scientific studies that must be carried out so that patients, physicians and the agency can determine which drugs are truly safe and effective, and how they can best be used.

"We believe that the best means of providing access to useful medical treatments for all Americans is to continue to shorten the review times," Henney says, "and to continue to work with the industry to shorten development times for drugs, biologics and medical devices."

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