FDA encourages new drug investigations following approvals slump in 2005

Following the drop in number of New Drug Application (NDA) approvals from 36 in 2004 to 20 in 2005, the US Food and Drug Administration (FDA) has released new guidance documents for investigational new drug (IND) studies.

'Exploratory IND StudiesApproaches to Complying with CGMP During Phase 1' will 'help more researchers conduct earlier, more-informed studies of promising treatments so patients have more rapid access to safer and more effective drugs', according to Mike Leavitt, secretary at the US Department of Health and Human Services (HSS).

Part of the FDA's Critical Path Initiative, launched in a March 2004 to reduce the time and resources expended on candidate products that are unlikely to succeed by distinguishing promising candidates earlier in the process, the documents lay out specific approaches for researchers who are planning to conduct very early clinical studies in people and offers approaches for performing appropriate safety testing and producing small amounts of drugs safely. According to Leavitt, nine out of ten experimental drugs fail in clinical studies.

'Existing regulations allow a great deal of flexibility in the amount of data that needs to be submitted with an IND application, depending on the goals of the proposed investigation, the specific human testing proposed, and the expected risks,' states the guidance. 'The Agency believes that sponsors have not taken full advantage of that flexibility and often provide more supporting information in INDs than is required by regulations.'

The guidance covers safety testing, manufacturing, and clinical approaches that can be used in exploratory INDs, which, because of their use of smaller amounts of drugs than later studies, represent fewer potential risks to humans.

'One of the biggest barriers research and academic institutions face is the ability to get discoveries made in the lab into clinical testing,' said Andrew von Eschenbach MD, acting FDA commissioner of food and drugs.

'The problem is that researchers conducting very early studies were required to follow the same manufacturing procedures as those companies that mass produce products for broad scale distribution,' added Janet Woodcock MD, FDA deputy commissioner for operations. 'These requirements are so burdensome for early phase 1 studies that many leading medical research institutions have not been able to conduct these studies of discoveries made in their laboratories.'

Pfizer, Eli Lilly and Johnson & Johnson all failed to win approval for a new drug invented in their own labs in 2005, despite spending on research by the industry reaching a new high of around $38bn last year.

According to the New York Times: 'The paucity of new products is a big reason that the stock prices of large drug makers have tended to fare poorly in recent years'. It cites Pfizer as an example, whose shares peaked at $49 in July 2000 but fell to $24.44 on 10 January, before going on to note that 'the biotechnology industry is still coming into its own and the medical payoffs from genomics still lie mainly in the future'.