Greater Transparency at FDA Would Bring More Biosimilars to Market, Experts Say

The FDA is responsible for oversight of all drugs, biologics, biosimilars, and medical devices marketed in the United States, but it is also dedicated to advancing public health. This includes an obligation to advance the development of safe and effective medical products for the greatest number of patients. Many patients need to take very expensive life-saving biologics but cannot afford access to them—a problem whose solution could lie with the development and marketing of lower-priced biosimilars that can be interchangeable with approved biologics. Despite promises made by the FDA to improve the agency’s transparency by making more regulatory decisions and analyses available to the public, there has not been enough progress, according to a recent article by Joshua Sharfstein, MD, of Johns Hopkins Bloomberg School of Public Health in Baltimore, and Michael Stebbins, PhD, of the Laura and John Arnold Foundation in Houston, in the journal JAMA.

The value of increased FDA transparency is gaining greater recognition because the European Medicines Agency (EMA), Europe’s counterpart to the FDA, has adopted policies to release large amounts of scientific information and analysis about regulated products, write Sharfstein and Stebbins. Several pharma and device companies now release data files in partnership with academic centers to allow independent analysis. FDA researchers have also shown that companies often fail to disclose the agency’s reasons for not approving product applications, and the authors believe that disclosing this information would help patients, physicians, and researchers, who could learn from such failures in drug development.

The FDA typically does not offer details about how many biosimilars are under review or comment on recent Complete Response Letters (CRLs), which explain why products cannot be approved. The FDA could, for example, propose a rule allowing at least portions of CRLs to be released by the FDA and for the agency to disclose biosimilar application numbers if it wished to. Jim Miller, JD, MPH, of the Department of Health Policy and Management at Johns Hopkins Bloomberg School of Public Health, said in an interview that concerns about releasing trade secrets are overstated, because rules governing the FDA have a narrow definition of what constitutes trade secrets.

Miller and other researchers from Johns Hopkins, Yale, and Harvard Medical and Law Schools recently created “Blueprint for Transparency at the FDA,” which will be published later this year. The report makes 18 recommendations in 5 key areas, all of which can be implemented by the FDA without additional Congressional action. The report calls on the FDA to:

• Disclose more information about key milestones of the application process
• Disclose more of its own analyses and decision making
• Disclose more about the application and review processes for generic drugs and biosimilars
• Correct misleading information in the market
• Further disclose data from research studies.

“The agency might consider adopting a standard based on whether the information has the potential to cause significant confusion in the medical community and among patients about the safety or efficacy of a medical product for approved or unapproved uses,” the report notes. FDA should retain the authority to release information under circumstances vital to the public health, it concludes.