Avastin Shouldn’t Make the FDA Give Up on ‘Accelerated Approval’ of Drugs

An important but obscure aspect of the Food and Drug Administration’s regulation of drugs has been in the news in recent months. Called “accelerated approval,” this “quick-on, quick-off” mechanism for medicines to reach the marketplace can work to the advantage of drug companies and needy patients alike.

Introduced almost two decades ago, accelerated approval permits the FDA to issue what amounts to a limited, or conditional, approval of a new drug that is intended for a “serious or life-threatening disease” and for which there is an “unmet medical need.”

Such an approval has two defining characteristics. First, it can be based on clinical trials that do not yet show an improvement on a definitive health endpoint such as increased longevity, reduction in the incidence of heart attacks or cancer cure, but merely on a “surrogate endpoint” that is thought to correlate with clinical benefit. Examples of surrogate endpoints are the shrinking of a tumor or improvement in a laboratory value such as “good” cholesterol.

Second, the drug sponsor (company) must perform confirmatory trials to prove that the medicine is effective in meeting a definitive endpoint, at which time the approval is converted to a standard, unconditional approval. If the studies fail to provide such confirmation, the FDA can pull the drug from the market.

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