Postmarket Surveillance: Ethics Cited as Critical Weakness

According to a commentary by former Institute of Medicine (IOM) committee members, published this week in the New England Journal of Medicine, increased “fast-tracking” of drug approvals (i.e., for medical conditions with no effective treatment)...

Off-label Drug Use: Fact vs. Fiction

The authors – CM Wittich, CM Burkle, and WL Lanier – offer a concise review of the topic of off-label drug use including its definition, prevalence, and implications for drug safety.   The article format addresses 10 common questions and their answers...

Potential Academic Contributions to Drug Development

Dr. Janet Woodcock (CDER, FDA) stated that for every 10 drugs that enter Phase I clinical trials, only 1 drug is approved.  The cost of bringing an innovative drug to market often requires a decade and a billion dollars of investment.  The paradigm where...

Orphan Drug Development: Midsize to Large Company Perspectives

Rare diseases can be described in terms of incidence, etiology, morbidity, and survival.  Incidence can vary, however, from rare (e.g., Gaucher disease, cystic fibrosis) to epidemic proportions (e.g., HIV, malaria, cholera).  The incidence of less frequently occurring...

FDA Encourages Applications for Orphan-Drug Status

Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including 7 years’ marketing exclusivity and tax breaks.  Last year, just 250 requests for orphan-drug designation were filed, and 160 were...