Responding to the continued lack of guidance concerning the sharing by pharmaceutical manufacturers of off-label drug information with payers and providers, the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) weighed in with a set of principles released on July 27 that govern the sharing of data and information outside of FDA-approved labeling.

Under the policy of the Food and Drug Administration (FDA), companies can be subject to criminal prosecution and civil liability for promotion of products for indications not specifically approved by the FDA. However, the outcomes of various lawsuits over the past few years have increased uncertainty regarding the enforceability of FDA regulations that biopharmaceutical companies have long critiqued as being overly burdensome and unclear.

The FDA policy has been dealt multiple setbacks in federal lawsuits questioning its constitutionality under the First Amendment. Most recently, Amarin Pharma, Inc., in March reached a settlement with the FDA in a lawsuit challenging FDA off-label promotion regulations on constitutional grounds. Despite the agency’s commitment to issue revised guidance by the end of 2014, industry stakeholders continue to await action by the FDA.

The document centers around three key concepts – a commitment to science-based communication,a commitment to providing appropriate context about data anda commitment to accurate representation of data – and nine principles, including the following: 

  • Companies should provide scientific substantiation if shared information is not contained in FDAapproved labeling;
  • Additional science-based information from sources other than FDA-approved labeling helps health care professionals and payers make informed decisions for patients;
  • Communications should be tailored to the sophistication of the intended audience;
  • Communicating with payers about new medicines and new uses of approved medicines facilitates patient access upon approval; and
  • Real-world evidence based on patient experience and pharmacoeconomic information can improve understanding of health outcomes and costs.

PhRMA and BIO released its off-label principles in part to offer perspective on what a modified FDA framework could look like.