Med Check

FDA to Issue Rules About Promotion of Off-Label Drug Use

The Food and Drug Administration (FDA) has proposed development of guidelines for drug manufacturers on distributing information about unapproved uses of their medications, a practice that the agency currently bans.

The agency’s focus on developing new guidelines comes in response to recent lawsuits and court decisions, including a 2012 federal appeals court ruling that overturned the conviction of a pharmaceutical sales representative who was prosecuted for encouraging physicians to prescribe a drug for uses that the FDA had not approved. His conversations with physicians had been recorded. The court ruled that his First Amendment rights were violated, since the federal government failed to prove that his remarks were false or misleading. The government did not appeal the federal court’s decision. Pharmaceutical companies have paid billions of dollars in fines over the last several years for violating the FDA ban on promoting off-label uses of their products.

In the newly proposed guidelines, the FDA will address how drug companies should respond when receiving requests from health care providers about off-label use of medication and procedures for distributing reprints from medical journals, which may contain information on off-label use.

In a letter to the lawyers representing the pharmaceutical companies that filed a pair of citizen petitions against the FDA, Leslie Kux, an FDA assistant commissioner for policy, wrote, “we recognize the evolving legal landscape in the area of the First Amendment, and we are reviewing and analyzing the agency’s policies, guidance, and regulations in this area more broadly.” Guidelines are expected to be completed by the end of this year.

Companies Collaborate on Alzheimer’s Drug Development

Bionomics Limited announced that it has entered a research collaboration and licensing agreement with Merck for itsBNC375 research development program targeting cognitive dysfunction associated with Alzheimer’s disease and other central nervous system conditions.

Bionomics CEO and managing director Deborah Rathjen, Ph.D., said, “We believe that the combination of Bionomics’ innovative approach and technologies, within its ionX platform, has the potential to rapidly advance new treatments.”

Under the agreement, Merck will provide funding for all research and development activities and will be responsible for marketing any products generated from the research efforts. Bionomics will receive upfront payments totaling $20 million and is eligible to receive up to $506 million for achievement of certain research developmental milestones and undisclosed royalties on any product sales.

The compound of interest, BNC375, and related compounds have displayed potent efficacy in animal cognitive impairment models.

FDA Gives Public Access to More of Its Data

In June, the FDA launched openFDA,a new initiative designed to make public-health datasets collected by the agency more accessible to Web developers, researchers, and the public.

“The openFDA initiative leverages new technologies and methods to unlock the tremendous public data and resources available from the FDA in a user-friendly way,” said Walter Harris, M.B.A., the FDA’s chief operating officer and acting chief information officer. “OpenFDA . . . will help those in the private and public sectors use FDA public data to spur innovation, advance academic research, educate the public, and protect public health.”

The initiative began after the FDA’s Internet officials and external developers conducted research to identify datasets that were in recurrent demand and difficult to access or use. The agency plans to phase in openFDA beginning with a pilot program that will allow access to millions of reports of drug-related adverse events and medication errors that were submitted to the agency from 2004 to 2013. Previously, the data were available only through reports that were difficult to access or from requests made through the Freedom of Information Act.

Alzheimer’s Research Characterized by Disappointing Results

Researchers from the Cleveland Clinic Lou Ruvo Center for Brain Health conducted the first meta-analysis of prevalence and success rates of trials testing potential new treatments for Alzheimer’s disease (AD).

“With an estimated 44 million people living worldwide with the condition, the study shows that the AD drug development ecosystem needs more support given the magnitude of the problem,” said lead author Jeffrey Cummings, M.D., Sc.D., director of the center.

Using information gathered from ClinicalTrials.gov of studies conducted from 2002 to 2012, the researchers found that of the 244 tested drugs, only one was a success—resulting in a failure rate of 99.6 percent for AD pharmacotherapy development. Analysis also showed that the number of medications being developed for AD treatment has declined since 2009.

As diagnosis rates and economic burdens for AD increase, the study authors emphasized that AD research must be supported and coordinated to improve success and development of new therapies—starting with “repositioning studies, which involve studying an already-approved drug in a new use or condition.” ■