In April of 2016, the Academy of Managed Care Pharmacy (AMCP) released a new Format for Formulary Submissions with revised requirements for manufacturers seeking coverage, reimbursement, and/or formulary placement of new drugs, tests, or devices.
The last major update to the Format made in December of 2012 (version 3.1) added new information about comparative effectiveness research and specified evidence requirements for specialty pharmaceuticals and companion diagnostic tests. The updated Format (version 4.0) now includes numerous
changes that reflect the increasing attention paid to value for money of new health technologies and greater rigor in coverage and reimbursement determinations in the U.S.
The new Format provides guidance on evidence that a manufacturer may provide to payers prior to FDA approval. Pre-approval dossiers may include published clinical studies, status of ongoing clinical trials, product data (product description and mechanism of action), and information about disease, treatment, and unmet needs (epidemiology, burden of disease, treatment options, clinical guidelines, etc.). This pre-approval evidence is seen as part of a continuing dialogue between manufacturers and payers to support payers’ planning needs with information about technologies on the
horizon that have the potential to impact medical care and health plan budgets.
The new Format establishes standards for reporting evidence about the value of diagnostic tests and medical devices, particularly those related to the use of a drug, including companion diagnostics, implantable drug delivery devices, blood glucose measuring
devices, inhalation devices, and devices or diagnostics to assess health status, diagnosis, prognosis, etc. The new standards include evidence most relevant for coverage and reimbursement determination that are designed to support assessments that span multiple related health technologies and evaluate overall
health system impact.
The new Format includes numerous additions that effectively raise the bar for evidence review by U.S. health plans, payers, pharmacy benefit managers, and other decision makers. Examples of new evidence requirements include information about how the new technology affects quality measures (such
as HEDIS scores, CMS Star ratings, etc.) related to the new technology and inclusion of published ex-U.S. clinical guidelines and health technology assessments (HTA). Overall, the AMCP Format is evolving to more closely resemble the more stringent reimbursement submission
requirements for HTA conducted by national health systems outside the U.S.
What happened: A new version of the AMCP Format was released
Who is affected: Manufacturers of pharmaceuticals, medical devices, and value-priced diagnostic tests
Why are the changes important: Standards for evidence review are a key part of the continuing debate about the value of new health technologies
Medical professional societies have recently developed a number of frameworks or scoring algorithms to assess the value of new health technologies, including methods described by the American College of Cardiology and American Heart Association (ACC-AHA), the Memorial Sloan Kettering Cancer
Center (MSKCC), the National Comprehensive Cancer Network (NCCN), and the American Society of Clinical Oncology (ASCO).3 Future dossier formats may specifically recognize particular value-scoring methods, or otherwise provide guidance on how manufacturers might prepare and communicate evidence in anticipation of such value assessments.
While cost-effectiveness analysis has been a central part of HTA by national health authorities outside the U.S., managed care decision makers have not typically requested evidence of cost-effectiveness or used cost-effectiveness criteria as a major component in coverage and reimbursement determinations. The
U.S. Institute for Clinical and Economic Review (ICER) has recently published assessments of several emerging therapies, in which they applied new methods for determining economic value, including a value-priced benchmark that considers both budget impact and cost-effectiveness. Future dossier formats may provide guidance for
preparation of pharmacoeconomic analyses using standard approaches in order to support direct comparison with ICER or other third-party assessments.
Changes to the new Format include new guidance on objective criteria for inclusion and exclusion of clinical studies to be summarized or otherwise listed in the dossier. While this requirement does address challenges in selecting evidence relevant for
consideration by payers without the appearance of bias, it falls short of requirements of HTA authorities for SLRs to identify clinical or economic evidence relevant to coverage and reimbursement determinations.
Read the AMCP Format Version 4.0
Dave Nellesen is a vice president in Analysis Group’s Menlo Park office who specializes in developing dossiers.