CAMBRIDGE — Food and Drug Administration Commissioner Margaret Hamburg told biotechnology executives Friday that her agency is working to speed up reviews of new drugs. But she said federal budget cuts could make that more difficult.
Hamburg said it was too soon to gauge the full impact of the mandatory US budget cuts under the sequestration process, which took effect March 1. But the FDA, like other federal agencies, is certain to be “significantly under the budget knife,” she said in a keynote address to an audience of more than 300 at the annual meeting of the Massachusetts Biotechnology Council.
“We are in a period of restrictive budgets, and we need to think in that context and prioritize,” Hamburg said. She said complicated federal rules under the required budget cuts prevent the agency from tapping “user fees” from companies that help underwrite the costs of reviewing new drugs.
“Sequestration has added an unexpected burden to us at a critical time” when many promising new drugs and medical devices are coming up for consideration, she said.
As Hamburg spoke inside the Royal Sonesta Hotel, about 20 people marched outside to protest what they said was slow FDA action in getting new drugs to patients. They wore T-shirts reading “Fix the FDA” and carried signs reading “Slow FDA Costs Lives.”
In particular, they said they wanted to call attention to the fact that the long-delayed breast cancer drug Kadcyla, approved by the FDA last month, still can’t be used by some patients with later-stage cancer in combination with other medicines or before they first try other breast cancer treatments.
“The drug has been in trials for seven years, and they’re still only giving it partial approval,” said one of the demonstrators, Lorraine Heidke-McCartin, a Hanson breast cancer patient who has been taking Kadcyla since 2010 under a “compassionate use” program that gives some people access to experimental drugs. “When are they going to fully approve it for all patients?”
Hamburg didn’t mention the protest during her speech, but in an interview afterward she said she was sympathetic. She said the FDA was developing new approval processes for “breakthrough” therapies, but stressed drug makers must first run thorough clinical trials that examine such complicated issues as how an experimental treatment works in combination with other medicines. Only then can the FDA review data and determine whether new drugs are safe and effective enough to be allowed on the market, she said.
“Obviously, I share their sense of urgency,” Hamburg said. But noting that the agency is also under pressure from safety advocates to fully evaluate potential side effects of drugs, she said, “Some people say we have only two speeds: too fast and too slow.”
In her talk, Hamburg said the FDA last year approved more than three dozen new drugs for sale in the United States, the most in a decade. She said an increasing number of treatments are being approved under the agency’s fast-track, priority review, and accelerated approval programs — all of which are designed to get critical and innovative medicines to market more rapidly.
“We have heard loud and clear that you want increased clarity, certainty, and predictability from the FDA,” she told the Massachusetts biotech executives. She also cited “early interactions” between companies seeking to design clinical trials and the regulators who coordinate reviews of the drugs based on data from those studies.
Among the newly launched medicines benefiting from these FDA efforts during the past year, Hamburg said, were four developed by Cambridge companies: the leukemia drug Iclusig from Ariad Pharmaceuticals, the cystic fibrosis drug Kalydeco from Vertex Pharmaceuticals, the irritable bowel drug Linzess from Ironwood Pharmaceuticals, and the cholesterol-lowering treatment Juxtapid from Aegerion Pharmaceuticals. All were approved in 2012.
Hamburg applauded the Massachusetts “biotech supercluster” as well as state legislation to expand health care access and control costs. Referring to the federal gridlock on reducing the deficit, she said, “I wish our governing system could be quite as efficient and congenial as yours.”
Earlier, discussing the state of the industry, MassBio president Robert K. Coughlin told executives that highlights of the past year included the cost containment law and national conventions in Boston by the largest biotech, pharmaceutical, and medical device trade groups.
“All eyes have been on Massachusetts,” said Coughlin.