EXOME

all the information, none of the junk | biotech • healthcare • life sciences

FDA’s Hamburg Stepping Down With Biopharma Turnaround as Her Legacy

Xconomy National — 

[Corrected 2/6/15, 2:40 pm. See below.] FDA Commissioner Margaret “Peggy” Hamburg will step down in March after nearly six eventful years, transforming the agency from one of the life science industry’s biggest bete noirs into, arguably, a fairly reliable arbiter for innovators.

She announced her decision this morning in an open letter to FDA employees.

Speculation about a transition gained momentum in late January when the FDA named Duke University cardiologist Rob Califf a top deputy.

Hamburg (pictured above) won the post in 2009. She previously ran New York City’s public health department.

Her legacy could end up being a profound one for the life sciences industry, particularly for biopharma.

Before Hamburg arrived, approvals of new and novel drugs languished—18 were approved in 2007, and 24 the following year—and the agency’s drug chief gave his reviewers leeway to miss their deadlines.

Stung by criticism over the approval—and subsequent withdrawal—of the painkiller Vioxx, the agency had expanded the criteria for drugs meant for wide patient populations such as cardiovascular and metabolic disease.

Under Hamburg, the agency has streamlined reviews, in part with faster timelines for drugs that promise to treat unmet medical needs. Those initiatives have helped boost approval numbers to near-record levels; 41 new and novel drugs were approved in 2014, the highest total since a record 53 in 1996.

“These programs have proven invaluable in bringing groundbreaking new therapies to patients more efficiently, while maintaining the FDA’s high standards for safety and efficacy,” said BIO chairman Jim Greenwood in a statement.

In a survey of investors last year conducted by BIO, 65 percent said regulatory changes had “some impact” in spurring more biotech investment in recent years; another 13 percent said those changes had had “major impact.”

When respondents were asked this question—“Has FDA decision-making become more balanced with respect to risk/benefit analysis over the last few years?”—63 percent cited a “minor shift” to a more balanced approach, while 13 percent cited a “major shift.”

In particular the “breakthrough” designation, created and put into practice four years ago, has sped to market drugs for hepatitis C, meningitis, and various cancers. Most recently, the cancer immunotherapy blinatumumab (Blincyto) got the green light five months faster than expected, based on a Phase 2 trial in a subset of patients with B-cell acute lymphoblastic leukemia.

The agency can also take away: Earlier this week, it rescinded the designation for Merck’s hepatitis C combination treatment (grazoprevir/elbasvir), citing the availability of other treatments for genotype-1 patients. That marked the first time FDA has rescinded a breakthrough designation, according to FDA data. (Hat tip: Fortune Magazine.)

It hasn’t hurt that the FDA budget under Hamburg has risen from $2.7 billion to $4.5 billion, although about $500 million of that increase is due to the addition of a tobacco oversight group.

The agency’s mandate to protect public safety hasn’t always meshed with industry goals, but that tension has abated under Hamburg.

[An earlier version of this story had the wrong location for 23andMe and mischaracterized the FDA’s order. We regret the error.] One high profile exception was the case of 23andMe, the Mountain View, CA-based personal genetics company.

Part of 23andMe’s business used to be “spit kits” that gave consumers their genetic profiles. The company can still offer ancestry reports and genetic information. But the FDA put a stop to 23andMe’s health-related reports, which the agency said threatened to stray into the realm of medical advice that the firm had no authority to provide. It has since expanded its genomic research business, partnering with Genentech and Pfizer to help those companies with their drug discovery programs.

The agency is also working on new regulations for laboratory tests, a key factor in the development of more targeted therapies, which President Obama and the National Institutes of Health highlighted in the administration’s precision medicine initiative last week.

FDA chief scientist Stephen Ostroff will be acting commissioner when Hamburg steps down on March 31.