Biotech execs: FDA can be good, or “horrifying”

From Reuters Health: By Lisa Richwine BOSTON, Feb 24 (Reuters) - Biotechnology companies trying to get new drugs to the market have had experiences with U.S. regulators that range from productive to "horrifying" as they craft early development plans, senior executives told Reuters this week. Interactions can vary vastly between Food and Drug Administration divisions that review products and provide guidance to companies long before they seek approval to sell a drug, company officials said. "We have some very good experiences and we have had some horrifying experiences, where you wonder, 'Where did that come from?'" Genzyme Chief Executive Henri Termeer said at the Reuters Biotechnology Summit in Boston. "What is reasonable and what is not reasonable differs by individual. ... It's quite frustrating," he added. FDA officials have acknowledged inconsistencies, or a lack of clarity, from some reviewers during early stages, when companies are seeking input on how to design clinical studies of experimental medicines. They have pledged improvements and launched major efforts to work with manufacturers earlier in the process to help more drugs make it to the market. The agency holds more meetings than ever with drugmakers, more than 2,000 annually. "We believe more and better interactions will lead to better outcomes and have invested a lot of resources in these efforts," Scott Gottlieb, FDA deputy commissioner for medical and scientific affairs, said via e-mail. The FDA also is providing guidelines on how to develop better applications, he said. Officials will announce other measures to improve the review process soon. Biotechs can face more challenges than traditional pharmaceutical companies. Many biotechs are small, inexperienced and testing their first product. Their technology may be new and unfamiliar to regulators. Alnylam Pharmaceuticals Inc. CEO John Maraganore said his company has had "remarkably good" dealings with the FDA on efforts to develop treatments based on a new "gene silencing" technology. "You can engage them in a science-based discussion ... People that run into troubles don't engage them in that type of dialogue," he told the Reuters Summit. The staff at the FDA is very responsive on potential breakthrough drugs, said Ariad Pharmaceuticals Inc. CEO Harvey Berger. Having "fast-track" status for promising medicines helps insure regular communication, he said. His company's employees talk often with the agency in person and on the phone and get answers to questions within days. "I think the key is you have got to have a drug that they see a real opportunity for," Berger said. Cell Therapeutics Inc. CEO Jim Bianco agreed FDA staff will move quickly for drugs that are "sexy," but said less exciting, though still important, medicines can get left behind. His company has been frustrated in its development of a cancer drug, Xyotax, which he described as a traditional chemotherapy medicine with fewer side effects. European regulators are open to clearing the drug based on evidence it is equivalent to other chemotherapy, rather than superior, because it has less toxicity, but U.S. regulators are not, Bianco said. The FDA staff is "not equipped to handle that," he said. To the agency, "standard chemo is not what moves the field forward," he said. Genzyme's Termeer said it has become easier to deal with European regulators, who routinely approve his company's products before the FDA does. "The Europeans are willing to delegate much more of the process ... to expert opinions. Much more of the work is done outside the bureaucracy," he said. Copyright © 2005 Reuters Limited.