This document was prepared by Jeff Lee, PharmD, FCCP, on behalf of the AMCP Format Executive Committee. Committee members reviewed and provided feedback on the final draft. No conflicts of interest, financial or otherwise, were reported.
Copyright © 2016, Academy of Managed Care Pharmacy. All rights reserved.Managed care pharmacists are increasingly presented with complex considerations related to prescription drug formulary management. As prescription drug spending soars, and new effective, but expensive drugs rush to the market, pharmacists and other health care decision makers must evaluate a myriad of important clinical and economic considerations in determining the relative value and, subsequently, the appropriate placement of a product within a formulary. The AMCP Format for Formulary Submissions, Version 4.0, is the next iteration of the Format, which was first released in 2000. Version 4.0, developed by pharmacists from health plan, manufacturer, and academic perspectives, provides updated recommendations on acquiring and evaluating clinical and economic evidence to inform formulary and medical policy decisions. It also includes new guidance related to emerging special topic considerations such as biosimilars, specialty pharmacy products, and companion diagnostic tests. Version 4.0 has been modified to improve the usability of the Format, with clarifying guidance related to logistical considerations such as a recommended time frame for implementation of Version 4.0, as well as dossier updates and ongoing communication between manufacturers and health care decision makers. The Format should be used as a framework for ongoing evidence-based dialogue between manufacturers and payers. The evolving health care landscape will require new levels of collaboration and communication among key stakeholders to successfully navigate the challenges of this new environment. The Format provides a framework to support these critical interactions related to product value by facilitating an evidence-based, transparent approach.
The role of the pharmacist in managed care settings has evolved considerably in recent years. This is in part because of a flood of new, effective, and often expensive interventions that require careful analysis for developing and managing a viable pharmacy benefit. The financial impact of these new products is considerable, contributing in part to a 12.2% increase in national prescription drug spending in 2014—the largest increase since 2002. 1 The proliferation of specialty products poses a unique challenge, since it is estimated that these products will soon represent 50% of pharmacy costs. 2 While in many instances these products represent a significant step forward in advancing the treatment paradigm compared with existing options, they often come with a price tag that requires thorough consideration of the incremental treatment benefit relative to other alternatives, meticulous identification of the appropriate patient population, and a detailed accounting of the total cost of care. As such, traditional pharmacy roles have evolved to incorporate complex considerations related to evaluating and balancing important attributes such as treatment benefit and risks, cost-effectiveness, and affordability.
This enhanced focus on deciphering the inherent value of new products is reinforced by the recent proliferation of initiatives from a number of health care organizations to develop value frameworks with the objective of providing a more rigorous and comprehensive assessment of value when considering the adoption of new health technologies, including new pharmaceutical products. 3,4 These initiatives serve to provide guidance to health care decision makers (HCDMs) and patients regarding important considerations related to assessing the value of health technologies. While these initiatives represent a positive step to encourage a more focused dialogue regarding the value of health technologies, they also highlight, as noted by Neumann and Cohen (2015), that “value is an elusive target, and there’s no consensus about what dimensions should be taken into account.” 3
Since its initial release in 2000, the AMCP Format for Formulary Submissions has served as a benchmark to guide drug manufacturers regarding important payer evidence requirements for evaluating new technologies for formulary and medical policy consideration. The Format serves to improve the timeliness, scope, quality, and relevance of clinical and economic information provided by manufacturers to HCDMs, as well as to streamline the evidence acquisition and review process for decision makers. Given that value is in the eye of the beholder, the Format does not prescribe a specific formula to calculate the overall value of a health technology. Rather, the Format endeavors to provide guidance on the key clinical and economic evidence requirements that serve as the fundamental components of determining value. Thus, one of the intended benefits of the Format is to improve the credibility and transparency of the process, which is aligned with the core values of the Academy of Managed Care Pharmacy (AMCP).
Given the evolving health care landscape and enhanced focus on discussions of value, the Format Executive Committee, composed of AMCP members representing a mix of health plan, manufacturer, and academic perspectives, identified a full revision to the Format as a top priority in 2015. The Committee solicited input from AMCP members and revisited content considerations noted since the previous update (Version 3.1), which was published in 2012. 5 Based on this input and a detailed review of relevant developments in the payer evidence landscape, the Committee moved forward with a full revision, forming 3 work groups composed of committee members and external contributors to address updated considerations regarding clinical evidence, economic evidence, and key logistical considerations related to the Format. A draft of the Format, Version 4.0, was released for public comment in December 2015. The Committee carefully considered the input received and made revisions where appropriate. Subsequently, the final draft was reviewed and approved by the AMCP Board of Directors in February 2016.
With the release of the Format, Version 4.0, we have endeavored to address evolving considerations in the health care environment, including considerations related to biosimilars, medical devices, comparative effectiveness research, and companion diagnostic tests, to name a few. Guidance on logistical matters related to updating dossiers, the challenge of providing preapproval evidence, and ongoing communication between manufacturers and HCDMs is provided as well. Some critical updates to the Format include the following:
Detailed and clarified guidance for handling of contemporary content topics such as comparative effectiveness research; use of the Format for drugs, tests, and devices; biosimilars; and assessing heterogeneity of treatment effect.
Inclusion of guidance related to evidence requirements for companion diagnostic tests.Comprehensive guidance added for specialty pharmaceutical product information, including handling and distribution requirements and restrictions; appropriate settings; supportive care services; and medical benefit considerations, such as coding.
Specific guidance related to using the Format with biosimilar products, including comparative data requirements relative to reference products.
Restructured Section 3.0 (Clinical Evidence) and Section 5.0 (Additional Supporting Evidence), with additional guidance provided on recommended evidence components for each section.
Revision of the economic evidence section of the Format to more closely align with updated best practices published by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and Society for Medical Decision Making (SMDM) Modeling Good Research Practices Task Force for economic models. 6
Incorporating new guidance on budget impact models as unique deliverables in addition to cost-effectiveness analyses as a component of the overall economic evidence dataset.
Guidance on modeling considerations for biosimilars and specialty products.Updated guidance on updating dossiers and handling dossier requests before U.S. Food and Drug Administration approval.
Implementation time frame for adoption of the Format, Version 4.0.Revision of many references throughout the Format, with the provision of links to publications or PubMed where available.
In developing this new version of the Format, our primary objective was to provide updated guidance to HCDMs and manufacturers that enhanced the clarity, transparency, and usability of the Format, while reinforcing the role of the Format in facilitating ongoing scientific dialogue between payers and manufacturers. Additionally, we wanted to ensure that this new version addressed important new developments in the marketplace. One challenge in providing this guidance is the recognition that our audience is comprised primarily of 2 disparate groups: dossier developers, such as manufacturers, medical writers, and consulting groups, and dossier consumers, who are primarily HCDMs involved in evidence assessment and formulary management. We have attempted to target an appropriate middle ground in developing this updated version. By outlining and describing the key evidence components that are relevant to a formulary or medical policy decision, we hope to reinforce a multifaceted approach to evaluating new technologies, while leaving the final value determination to the ultimate decision maker. On the other end of the spectrum, we have developed specific guidance to dossier producers regarding the content and format of a dossier, while relying on existing standards related to the more technical considerations of value evidence generation and communication. With this approach, we hope that the Format will meet the needs of a broad range of perspectives, while providing clear direction to related resources that may offer additional relevant technical detail as required.
Ultimately, we want the Format to be viewed as a vehicle to enhance evidence-based communication between manufacturers and HCDMs. Substantial, ongoing communication throughout the evaluation process is a critical success factor in promoting credible, transparent discussions related to product value. We encourage HCDMs to use the unsolicited request process as a means to engage manufacturers in these important conversations, and we encourage manufacturers to provide clinical and economic evidence in a consistent, transparent framework as represented by the Format. We view the Format as offering a clear, shared vision of the requirements to facilitate the collaboration necessary between HCDMs and manufacturers to support appropriate and evidence-based product evaluation.
It is important to emphasize that the scope and context of communications between manufacturers and HCDMs should evolve over the product lifecycle as new evidence becomes available. While launch-timed dossiers may rely to a greater extent on modeled projections based on clinical trial evidence and reasonable assumptions related to market dynamics and product uptake, new evidence describing the actual use and effect of the product in a real-world setting should be developed and used to inform formulary management or medical policy criteria across the product lifecycle. Ongoing generation of real-world evidence serves the important purpose of further defining and validating claims related to product value. 7 HCDMs are encouraged to revisit and appropriately revise formulary decisions based on the availability of new and compelling evidence. As such, ongoing communication between manufacturers and HCDMs as the value evidence evolves is a critical component to the process.
In revising the Format, we appreciate the input provided by our committee members and external contributors and, particularly, the outstanding feedback we received during the open comment period by HCDMs, manufacturers, and other interested parties. This feedback provided useful confirmation of key issues and helped us identify areas that required additional work. We hope the result is an updated version of the Format that provides enhanced clarity, transparency, and usability to HCDMs and manufacturers alike. The Format should stimulate relevant, evidence-based, ongoing communication among the key stakeholders in the process and support the development of informed, rational formulary decision making.
JEFF LEE, PharmD, FCCP, Associate Professor of Pharmacy Practice, Lipscomb University College of Pharmacy (Committee Chair); PETE PENNA, PharmD, President, Formulary Resources (Chair for Version 4.0 Clinical Evidence Work Group); KIM SAVERNO, PhD, RPh, Research Lead, Comprehensive Health Insights, Humana (Chair for Version 4.0 Economic Evidence Work Group); IRIS TAM, PharmD, Vice President, Patient Access and Quality, Medical Affairs, Otonomy (Chair for Version 4.0 General Information Work Group); J. DANIEL ALLEN, PharmD, Regional Outcomes Liaison, Sanofi US; STEVEN G. AVEY, RPh, MS, FAMCP, Vice President, Specialty Pharmacy, MedImpact; DIANA BRIXNER, PhD, RPh, Professor, Department of Pharmacotherapy, Executive Director Outcomes Research Center, College of Pharmacy, University of Utah; VINCENT W. LIN, PharmD, MS, Manager, Amgen Global Health Economics; DANIEL C. MALONE, RPh, PhD, Professor, College of Pharmacy, University of Arizona; NEWELL MCELWEE, PharmD, MSPH, Associate Vice President, Center for Observational and Real World Evidence, Merck & Co.; ALAN PANNIER, PharmD, MBA, Clinical Services Manager, Veridicus Health; ELIZABETH R. SAMPSEL, PharmD, MBA, BCPS, System Director of Ambulatory Pharmacy Services, Ochsner Health System; HELEN SHERMAN, RPh, PharmD, Vice President, Solid Benefit Guidance; JOHN WATKINS, PharmD, MPH, BCPS, Pharmacy Manager, Formulary Development, Premera Blue Cross; JEFFREY WHITE, PharmD, MS, Staff Vice President, Clinical Pharmacy Services, Anthem; SUSAN OH, PharmD, MS, Assistant Director of Pharmacy Affairs, Academy of Managed Care Pharmacy (Staff Liaison); and LYNN NISHIDA, RPh, Assistant Vice President, Pharmacy Services, Solid Benefit Guidance (AMCP Board Liaison to the Format Executive Committee).
AUTHOR CORRESPONDENCE: Jeff Lee, PharmD, FCCP, FEC Chair, Associate Professor, Pharmacy Practice, Lipscomb University College of Pharmacy, One University Park Dr., Nashville, TN 37204. Tel.: 615.966.7012; E-mail: ude.bmocspil@eel.ffej.
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