Stakeholders Recommend Manufacturers Share Off-Label Information on Medications Awaiting FDA Approval with Decision Makers

Alexandria, Va., Sept. 16, 2016 — Biopharmaceutical companies and health care decision makers would be able to more easily communicate information on products awaiting Food and Drug Administration (FDA) approval, under recommendations developed at an Academy of Managed Care Pharmacy (AMCP) partnership forum this week.

Currently the sharing of clinical and health care economic information (HCEI) on products yet to be approved is significantly restricted by federal laws and FDA regulations. Organizations that make coverage decisions (i.e., setting copayments, premiums and formulary placement) have expressed a need for receiving this information sooner, as long as safeguards are in place to prevent preapproval information from reaching unintended entities.

“Early dissemination would allow decision makers to build this information into their financial forecasting for the plan year ahead,” said AMCP CEO Susan A. Cantrell, RPh, CAE. “Waiting until after the FDA actually approves a product often comes too late for this critical planning to occur. The recent introduction of high-cost medications has added urgency to the need for better evaluation and preparation.”

To address the issue, AMCP convened a partnership forum on Sept. 13-14 that included a diverse group of stakeholders representing managed care, the pharmaceutical industry, academia, providers, patients and others.

The group adopted the term “Preapproval Information Exchange” (PIE) to describe this proposed new communication process, and to reflect the fact that some of the information will not always include typical clinical or economic data. Rather, it may include more basic information such as anticipated indications and routes of administration. The proposed communication process also is designed to allow for greater flexibility by appropriate stakeholders in determining what information should be shared.

Another key recommendation was to ensure that any sharing of preapproval information would be limited to manufacturers and population health decision makers who need this information for financial forecasting and planning purposes. Other topics for discussion, included:

  • Clinical and scientific standards that preapproval information should meet. 
  • Value that each entity would gain by disseminating and receiving preapproval information.
  • Safeguards to prevent preapproval information from reaching unintended entities.

The forum was moderated by noted health policy expert Susan Dentzer, President and CEO of the Network for Excellence in Health Innovation. The event follows a March 2016 AMCP partnership forum in which participants developed recommendations ( for clarification of Section 114 of the FDA Modernization Act, which provides a safe harbor for companies to disseminate HCEI on their marketed products. This provision has been underutilized since its inception two decades ago due to a lack of FDA guidance.

AMCP will share recommendations from both forums during the FDA’s recently announced public hearing and request for comments on “Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products.” AMCP also will share the recommendations with Congress as it considers legislative solutions to improve communication of HCEI.

The recommendations will be published in the December 2016 issue of AMCP’s Journal of Managed Care & Specialty Pharmacy (

The AMCP partnership forum was supported by AbbVie, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Intarcia Therapeutics, Eli Lilly and Company, GlaxoSmithKline, Merck, National Pharmaceutical Council, Novo Nordisk, Pfizer, Pharmaceutical Research and Manufacturers of America, Precision for Value, Sanofi, Takeda and Xcenda.