NPC’s Dr. Jennifer Graff Testifies Before FDA on Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Devices

National Pharmaceutical Council (NPC) Vice President for Comparative Effectiveness Research Jennifer Graff, PharmD, delivered testimony during the Food and Drug Administration public hearing held Nov. 9-10 on manufacturer communications regarding unapproved uses of approved or cleared medical products. In her testimony, she highlighted two key issues: the need for broader communication to achieve public health goals and health care reform initiatives, and the existence of best practices and standards that can be relied on to ensure scientific integrity.

“To achieve our shared goal of a high-performing, value-based health care system, informed and evidence-based decisions are needed,” Dr. Graff testified. “Greater efficiency and better care requires more information exchange—not less—about which treatments work best for whom and under which care settings.”

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. This information is not typically included in labeling approved by the Food and Drug Administration (FDA). Although communication of this information was later permitted by laws and regulations like Section 114 of the Food and Drug Administration Modernization Act of 1997, these laws and regulations are vaguely written and have been implemented without further FDA guidance, resulting in more limited use of these provisions and lack of clarity for typical payer questions.[1]

Additionally, there has been a proliferation of best practices and standards for conducting observational or real-world studies. These include initiatives with which NPC has had an opportunity to be involved, such as:

1. The Good ReseArch for Comparative Effectiveness (GRACE) Principles[2] and GRACE checklist[3];
2. The CER Collaborative, a partnership with the Academy of Managed Care Pharmacy and the International Society for Pharmacoeconomics and Outcomes Research (professional societies of experts in their respective areas), which created online tools and checklists to help population health decision-makers evaluate the relevance and credibility of studies using different research designs.[4]

“This work provides insight on the level of scientific integrity required for credible, reliable, truthful and non-misleading evidence,” Dr. Graff explained.

Dr. Graff emphasized the need for and importance of all stakeholders to work collaboratively to understand how treatments work in the real world.

“NPC shares the goal of the FDA: 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,” Dr. Graff said. “Improved communication includes sharing what is known about the benefits and risks of a treatment, along with the limitations of that evidence.”

The FDA hearing will continue through November 10 and can be viewed online.

[1] Neumann PJ, Saret CJ. When Does FDAMA 114 Apply? Ten Case Studies. Value Health. 2015 Jul;18(5):682-9. doi: 10.1016/j.jval.2015.02.013. Epub 2015 Apr 16.

[2] Dreyer NA, Scheneeweiss S, McNeil BJ et al. GRACE Principles: Recognizing High-Quality Observational Studies of Comparative Effectiveness. Am J Manag Care. 2010;16(6):467-71.

[3] Dreyer NA, Velentgas P, Westrich K, Dubois R. The GRACE Checklist for Rating the Quality of Observational Studies of Comparative Effectiveness: A Tale of Hope and Caution. J Manag Care Phar. 2014;20(3):301-8.

[4] Perfetto EM, Anyanwu C, Pickering MK, Zaghab RW, Graff JS, Eichelberger B. Got CER? Educating Pharmacists for Practice in the Future: New Tools for New Challenges. J Manag Care Spec Pharm. 2016;22(6):609-16.