Washington Editor

WASHINGTON – The FDA is counting on a fistful of strategies to prepare it for the regulatory and public health challenges of the future.

In enumerating the priorities laid out in the agency's new five-year plan last week, Margaret Hamburg started with the strategy she has been pushing ever since she became FDA commissioner – regulatory science, or in other words, the tools needed to evaluate new technologies.

"Science underlies everything we do at this agency," Hamburg said, "and to serve the public health, we must have the capacity to effectively oversee the translation of breakthrough discoveries in science into innovative, safe and effective products and life-saving therapies."

For the most part, the FDA's plan to advance regulatory science and innovation is vague and relies on the creation of . . . more plans. The one concrete example it gives is the need to modernize tools used to assess toxicology safety.

In filling this "critical gap in regulatory science," the agency could better predict how novel compounds will behave in humans, the five-year plan said, thereby reducing drug development costs and the time to market for new technologies, while helping prevent adverse health events.

A second regulatory shortcoming addressed in the strategic plan is the need for a more flexible approval process for medical countermeasures (MCM).

"The complex regulatory pathway required for approval of these types of medical products is one major contributing factor to the limited availability of MCMs," according to the plan.

As the result of a Department of Health and Human Services' (HHS) review last year that identified the FDA as one of the most critical components of the nation's public health MCM program, the agency developed an action plan.

As part of its MCM program, the FDA will assemble teams to tackle a range of regulatory, scientific and policy issues that may hinder the development and approval of drugs designed to respond to public health emergencies and threats.

It also is reviewing existing legal and policy approaches to MCM development, distribution, post-use surveillance and data collection. Where needed, it will propose new approaches. Other steps the agency plans to take include:

• developing a comprehensive program to support the federal government's efforts to establish a "nimble, flexible capacity to produce MCMs rapidly in the face of any attack or threat;"

• updating guidance documents and assessing new endpoints for clinical trials of antibacterial drugs (see BioWorld Today, June 17, 2010);

• working with the Office of the Assistant Secretary for Planning and Evaluation at HHS to develop innovative strategies that promote the development of antibiotics to treat resistant organisms.

The other three strategic priorities outlined in the five-year plan – strengthening the global supply chain, strengthening compliance and enforcement efforts and expanding efforts to meet the needs of special populations – all add to programs already under way at the FDA.

However, to protect the drug supply chain in the face of growing globalization challenges that outstrip the FDA's resources, the agency is looking at a paradigm shift that focuses on prevention rather than interception.

Such a shift will require more, and better, information about product supply chains, which would necessitate a "greater global safety net that includes a global alliance of regulators; coordination of activities with international capacity-building and standards-setting organizations; new adequate funding to allow for inspections, examinations and sample collections and analysis; and updated systems, including IT support, to assist with the increased workload," according to the plan.

Much of the FDA's work on expanding programs for special populations will center on pediatric therapies. For instance, the plan calls for developing validated endpoints for neonates, exploring why therapies that work in adults don't work in children and working with other regulators on ethical, scientifically sound, global pediatric trials for rare diseases.

As for enforcement, the FDA said it will continue to pursue criminal prosecution to force compliance with its regulations. As a result, more CEOs could face prison sentences, fines, restitution and forfeiture over the next five years as the agency joins forces with HHS and the Department of Justice to hold corporate officials responsible for the actions of their companies. (See BioWorld Today, April 15, 2011.)

Three Rivers Snagged by Bad Ad Program

Three Rivers Pharmaceuticals is in hot water with the FDA over a "STATgram" distributed to health care providers, touting the benefits of its Infergen injection.

Three Rivers recently received a warning letter from the agency's Division of Drug Marketing, Advertising and Communications (DDMAC) that claims the STATgram broadens the approved indication for Infergen (interferon alfacon-1), overstates its efficacy, makes an unsubstantiated claim, and omits and minimizes risk information.

For instance, the STATgram refers providers to a product website to get information on the biologic's safety profile and the "box warning for all interferon alphas regarding neuropsychiatric, autoimmune, ischemic and infectious disorders," according to the letter posted on the agency website last week.

"This statement represents the sole presentation in the piece of the significant and potentially fatal risks associated with Infergen," DDMAC said. The STATgram doesn't mention common adverse events, other boxed warnings related to combination use with ribavirin, contraindications to the use of Infergen and ribavirin, and most of the biologic's warnings and precautions.

The STATgram was developed and approved by Three Rivers prior to the company's October acquisition by Kadmon Pharmaceuticals LLC, of New York. "We are in the process of reviewing all promotional materials to confirm compliance with applicable law and are working with the agency to complete this process," Kadmon spokesman David Pitts told BioWorld Today.

The STATgram was called to DDMAC's attention through the FDA's Bad Ad program, which was launched nearly a year ago to encourage health care providers to report drug promotional pieces and practices that violate agency regulations.

The FDA is peddling the program at medical conferences and will host a webinar on it Thursday for providers.

Committee Wants PhRMA White House Records

Trying to peer behind closed doors at the White House, the leadership of the House Energy and Commerce Committee sent the Pharmaceutical Research and Manufacturers of America (PhRMA) a request last week for information about its private negotiations with the Obama administration over the health care reform legislation passed last year.

The April 18 letter requests details about all meetings and correspondence PhRMA had with the administration since March 2, 2009, that dealt with health care reform. The committee also wants estimates or analyses of the impact the legislation has had on the organization and its members. Similar letters were sent to 11 other groups.

PhRMA said it is cooperating with the committee.

FDA Beefs up Labeling for Tysabri

The FDA updated the Tysabri label last week to strengthen the warning about the risk of progressive multifocal leukoencephalopathy (PML).

The revised label for Tysabri (natalizumab), a Biogen Idec Inc. drug used to treat multiple sclerosis and Crohn's disease, gives new information about the size of the risk and includes new safety information about patients who have taken other drugs that suppress the immune system and may be at a higher risk for PML. Despite the risks, the Weston, Mass.-based company saw sales of Tysabri increase 15 percent in the first quarter. (See BioWorld Today, April 22, 2011.)