The currently proposed 21st Century Cures legislation includes language that is intended to provide greater clarity around the evidence manufacturers can communicate with payers and other key decision-makers about the value of products. Avalere’s survey results, suggest that if approved, this could result in better evidence on cost-effectiveness, comparative benefit, and real-world outcomes for payers determining patient access to these medications.

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A number of U.S. laws and U.S. Food and Drug Administration (FDA) regulations govern pharmaceutical company communications about drugs, biologics, and devices to physicians and consumers. These rules require that companies’ clinical claims are substantiated by two well-controlled trials,¹ are consistent with the FDA-approved labeling, and provide fair balance. Meanwhile, Section 114 of the FDA Modernization Act (FDAMA 114) sets different parameters for how health economic evidence may presented to payers.² However, in the absence of FDA guidance, interpretation of the statute, particularly around permissible evidence and possible audiences for communication, has varied dramatically across industry.

Given the current emphasis on cost-containment within healthcare, demands for high-quality, accurate, and evidence-based information on the value of treatments has never been higher. Pharmaceutical companies are being asked to provide more evidence about product value, including information about effectiveness, costs, and risks of new or existing therapies – sometimes in comparison to other treatment options. Despite this demand for evidence, uncertainty remains about what type of data manufacturers can share, a result of numerous evidentiary standards maintained by governmental agencies and language that leaves significant room for interpretation.

“As our system continues to shift from fee-for-service to value-based decisions, it’s important that payers and providers have better access to clinical economic evidence,” said Dan Mendelson, CEO of Avalere. “Our research finds that one of the main challenges to this outcome is that the evidence desired by the marketplace is in an environment that limits sharing that type of information.”

When addressing potential solutions, survey respondents believe that additional guidance to interpret current regulations would be helpful and that it would spur greater investment in evidence generation. Manufacturers also believe that further clarity is needed on study endpoints in economic studies, specifically, use of endpoints that could affect the value of a therapy to a patient, including patient satisfaction and quality of life, but fall outside the typical clinical and monetary endpoints.

Avalere’s research also finds that approximately 86 percent of the manufacturers believe existing rules restrict the generation and communication of economic information. Additionally, 93 percent find similar issues with comparative effectiveness research (see Figure 2). In these instances, manufacturers are concerned about FDA enforcement action, including warning letters, and potential misuse of FDAMA 114’s “safe harbor” provisions.

Approach

Between January 17 and February 14, 2014, Avalere Health surveyed 14 representatives of small, medium, and large global biopharmaceutical companies to determine the extent and nature of barriers they have encountered or perceived when considering or attempting to proactively communicate evidence to payers or healthcare professionals. We also aimed to assess whether potential hurdles vary across different types of evidence. To more specifically explore the impact of FDAMA 114 on the ability of manufacturers to develop and share information about the economic value of their products, Avalere conducted follow-up semi-structured interviews with representatives of five of the companies.

Funding support for this research came from the Pharmaceutical Research and Manufacturers of America. Avalere maintained full editorial control over the content in this issue brief.

Notes

1. 21 USC § 355(d). This refers to the “substantial evidence” standard, which usually but not always requires two trials.
2. Allows healthcare economic information that “directly relates” to an approved indication and is based on “competent and reliable scientific evidence” to be proactively provided to formulary committees or “similar entities.”