Pre-Approval Information Exchange Can Help Improve Population Health Decision-Making

It can be challenging for population health care decision-makers—especially those responsible for planning, budgeting and forecasting programs associated with health benefit design—to readily have available information to aid in those choices and encourage value-based decisions. Yet current laws and Food and Drug Administration (FDA) regulations make it difficult, if not impossible, to have that information at hand or available in a timely manner.

These laws and regulations significantly restrict what information biopharmaceutical companies can share with population health decision-makers, such as insurers, about emerging therapies prior to FDA approval. Under the Affordable Care Act and state mandates, however, health plans are required to evaluate their plan designs, formularies and rates 12-18 months in advance to meet submission deadlines 6-9 months before the start of the intended plan year. In many cases, these population health decision-makers need to take into account therapies that have yet to be approved by the FDA but could come to market in the near term. Restricting the information that can be shared could result in unpleasant scenarios such as a lack access and reimbursement for newly approved treatments for patients, unexpected budgetary pressures for health plans and inability to engage in value-based payment models among health systems.

Health care stakeholders, including the National Pharmaceutical Council (NPC), gathered in September for a meeting hosted by the Academy of Managed Care Pharmacy to discuss potential ways to improve the exchange of information and address these challenges.

The recommendations that stemmed from this discussion were published online in the Journal of Managed Care and Specialty Pharmacy and include the following:

• The group agreed on a term, “pre-approval information exchange,” (PIE) to reflect a proposed communications process for emerging treatment 12-18 months prior to anticipated FDA approval.  
• PIE should be truthful and non-misleading, and may include a broad range of information (such as phase II clinical trial protocols, pharmacoeconomic data, patient outcomes, potential indications, place in therapy, etc.) to meet the needs of population health decision-makers.
• Recipients of this information should be limited to population health decision-makers who need the information for financial forecasting and planning purposes.
• The information should be distributed in a format that allows for a bidirectional exchange between manufacturers and decision-makers until FDA approval.
• Stakeholders suggested that new legislative language is needed because this represents a new category of scientific exchange of information, and therefore establishing a safe harbor should allow sharing of information.

These recommendations also were highlighted by NPC and other stakeholders during the FDA’s Nov. 9-10 public meeting on “Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products.” During the FDA meeting, NPC noted that greater efficiency and better care requires more information exchange—not less—about which treatments work best for whom and under which care settings. Additionally, examples were shared to demonstrate why the exchange of information about treatment benefits, risks and costs is central to guiding population health decision-makers as they budget, make coverage decisions and incentivize more efficient and high-quality care.

As NPC has stated, it’s important to ensure that communication is done in a way that promotes public health and supports ongoing developments in science and technology, medicine and health care delivery. Meeting these goals requires transparent, reproducible and ongoing evidence generation and communication. Improved communication includes sharing what is known about the benefits and risks of a treatment, along with the limitations of that evidence.