The Pharmaceutical research and Manufacturers of America has welcomed moves by the US Food and Drug Administration to advance the earliest phases of clinical research in the development of innovative medicines.
New FDA guidance documents set out specific approaches for researchers planning very early clinical studies in people and offer approaches for performing appropriate safety testing and producing small amounts of drugs safely. These changes will enable US researchers to evaluate much more efficiently the promise of scientific advances discovered in their laboratories, says the agency.
Announcing the initiative, Health and Human Services Secretary Mike Leavitt noted that, currently, nine out of ten experimental drugs fail in clinical studies because how they will behave in people cannot be predicted, based on laboratory and animal studies. Andrew von Eschenbach, Acting FDA Commissioner of Food and Drugs, added: “as we enter the era of personalised medicine, these exploratory approaches enable scientists to take full advantage of new technologies to target the development of more individualised therapies.”
The Exploratory IND Studies guidance will facilitate very early exploratory scientific studies in people before Phase I studies begin. Because only small amounts of drugs are used in these early studies, they represent fewer potential risks for people in these trials, and the document explains how researchers can take full advantage of the flexibility built into existing regulations in the amount of data needed when seeking FDA permission to proceed with such a study.
Related draft guidance outlines a suggested approach to complying with current Good Manufacturing Practice requirements for drugs intended for use solely in Phase I studies.
“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,” said Janet Woodcock, FDA Deputy Commissioner for Operations, adding: “these requirements are so burdensome for early Phase I studies that many leading medical research institutions have not been able to conduct these studies of discoveries made in their laboratories.”
PhRMA chief executive Billy Tauzin called this an important move not only for researchers but also for patients who rely on new life-saving drugs “as their only hope of surviving a deadly disease. “Too many times, promising treatments fall through the cracks because the process involved with experimental clinical studies casn be arduous and full of red tape,” he said.
