The Potential Effect of the 21st Century Cures Act on Drug Development.

Enacted in December 2016, the 21st Century Cures Act is a pivotal piece of legislation that will influence the FDA drug and device approval process for the foreseeable future. Although this legislation received overwhelming support by members of Congress for much needed budgetary increases for the FDA and other national health organizations, there is much controversy over certain provisions that potentially diminish the robustness of the evidence base required for drug and medical device approvals. This article discusses the key provisions from "Title III - Development" under "Division A - 21st Century Cures" related to drug development and, specifically, explores those aspects that address patient-focused drug development, use of surrogate endpoints in clinical trials, modernization of trial design, and use of real-world evidence for decision making and health care economic information. In addition, specific legislation on regulatory changes is discussed pertaining to antimicrobial stewardship. Essentially, summarized interpretations are presented here of the provisions covering the aforementioned topics, along with insights into potential intended and unintended consequences for U.S. health care payers, health care providers, government entities, and product manufacturers. DISCLOSURES: No outside funding supported this writing. Goble is completing fellowship training sponsored by Novartis Pharmaceuticals.

Evaluating Oncology Value-Based Frameworks in the U.S. Marketplace and Challenges in Real-World Application: A Multiple Myeloma Test Case.

BACKGROUND: With the continuous rise in costs for oncology drugs, the American Society of Clinical Oncology (ASCO), the Institute for Clinical and Economic Review (ICER), the Memorial Sloan Kettering Cancer Center's Drug Abacus (DrugAbacus), and the National Comprehensive Cancer Network (NCCN) have developed value-based frameworks (VBFs) to assist stakeholders in formulary and treatment decision-making processes. Since emerging VBFs have the potential to affect available treatment options for patients, it is important to understand the differences associated with these VBFs within various therapeutic areas. OBJECTIVES: To (a) compare VBFs across 3 therapeutic options for relapsed or refractory multiple myeloma (RRMM) and (b) identify challenges and limitations associated with real-world decision making using VBFs in the U.S. marketplace. METHODS: The values of regimens carfilzomib (CFZ), elotuzumab (ELO), and ixazomib (IX) were generated using the ASCO, NCCN, ICER, and DrugAbacus VBFs. These regimens, used for second- or third-line treatment of RRMM, shared a common comparator in clinical trials: lenalidomide + dexamethasone (LEN + DEX). ASCO's 2016 VBF, which incorporated clinical benefit, toxicity, and bonus points, was used to generate a net health benefit score, along with the drug wholesale acquisition cost, for each regimen compared with LEN + DEX. Results of the 2016 NCCN Evidence Blocks for multiple myeloma and the ICER 2016 report of treatment options for RRMM were extracted to generate the value of CFZ, ELO, and IX. No output was generated from DrugAbacus because of the lack of regimens included in the test case. Shortcomings associated with running the test case in RRMM for each VBF were also identified. RESULTS: Among the 3 therapeutic agents, CFZ, in combination with LEN + DEX, was the most valued. ASCO and ICER VBFs suggested that CFZ + LEN + DEX may be the most valued, followed by ELO + LEN + DEX and IX + LEN + DEX. NCCN suggested that LEN + DEX may be the most valued followed by CFZ + LEN + DEX, IX + LEN + DEX, and ELO + LEN + DEX. A number of shortcomings were noted across each VBF, such as complexities of drug evidence evaluation with the ASCO VBF, the inability to adjust the ICER and NCCN VBFs to specific populations, and subjectivity associated with the NCCN VBF and DrugAbacus. CONCLUSIONS: Although the test case provided some consensus on treatment decisions, there is much nuance and limitations with the VBFs available for RRMM. Clearer objectivity and better adaptability to specific treatment decisions are warranted. DISCLOSURES: No outside funding supported this study. The authors have nothing to disclose. All authors contributed to study concept and design, as well data collection and interpretation. Djatche and Goble wrote and revised the manuscript, along with Chun and Varga. Portions of this work have previously been presented at the AMCP Managed Care and Specialty Pharmacy Annual Meeting 2017 in Denver, Colorado, March 27-30, 2017, and at the ISPOR 22nd Annual International Meeting in Boston, Massachusetts, May 20-24, 2017.

Performance-Based Risk-Sharing Arrangements: U.S. Payer Experience.

BACKGROUND: As a result of global concern about rising drug costs, many U.S. payers and European agencies such as the National Health Service have partnered with pharmaceutical companies in performance-based risk-sharing arrangements (PBRSAs) by which manufacturers share financial risk with health care purchasing entities and authorities. However, PBRSAs present many administrative and legal challenges that have minimized successful contract experiences in the United States. OBJECTIVE: To (a) identify drug and disease characteristics and contract components that contribute to successful PBRSA experiences and the primary barriers to PBRSA execution and (b) explore solutions to facilitate contract negotiation and execution. METHODS: A 37-item, web-based survey instrument (Qualtrics), approximately 20 minutes in duration, was open during July and August 2016. The survey was emailed to 90 pharmacy and medical directors of various health care organizations. Statistical analysis included the Kruskal-Wallis test and chi-square tests to examine differences among payer responses. Survey responses were anonymized and data were aggregated. RESULTS: Twenty-seven individuals completed the survey (30% completion rate). The majority of respondents worked for regional health plans (52%, n = 14), covering at least 1 million lives (63%, n = 17), with at least 7 years of managed care experience (81%, n = 22). A total of 51 PBRSAs were active among respondents at the time of the survey. Easily obtainable and evaluable drug data and medical data were the most important drug and disease attributes for successful PBRSAs, respectively. Pharmacy claims and patient demographic data were assessed as "very easy and inexpensive" to collect. Type and amount of manufacturer payment for drug outcome performance failure, endpoint measurement, and necessary clinical data for drug performance measurement were all critical factors for successful PBRSAs. Standardized contract templates and transparent contract financial risk evaluation and modeling ranked highest among methods of manufacturer facilitation of PBRSAs. This study was limited by sample size and survey questions were limited to explanation of PBRSAs at the disease state level. CONCLUSIONS: On the basis of PBRSA experiences, respondents noted that drug use in chronic medical conditions and objective drug outcome performance measurements were favorable drug characteristics and serve as the primary source of satisfaction for these types of contracts. Third parties and manufacturers can facilitate the uptake and success of PBRSAs by developing standardized contracting templates in addition to other methods that increase their stake in the arrangement. Looking forward, mounting perceptions of success in this realm of contracting for pharmaceuticals may contribute in the quest for value-based payments in the U.S. health care system. DISCLOSURES: The construction of the survey and payment for survey respondents were supported by Charles River Associates. Parece is an employee of Charles River Associates. Goble and Ung are completing fellowship training sponsored by Novartis and Celgene, respectively, but do not have any conflicts of interest and did not receive any funding related to this study. Navarro reports consulting fees from Analysis Group, TEVA, and Amgen, unrelated to this study. Van Boemmel-Wegmann declares no conflict of interest. Study concept and design were contributed by Navarro, Goble, Ung, and Parece. Navarro took the lead in data collection, along with Goble and Ung, and data interpretation was performed by van Boemmel-Wegmann, Goble, and Ung. The manuscript was written by Goble, Ung, Navarro, and van Boemmel-Wegmann and revised by all of the authors.

Point-of-Care Testing.

This article provides an overview of the current use of point-of-care testing (POCT) and its utility for patients' self-management of chronic disease states. Pharmacists utilize POCT to provide rapid laboratory diagnostic results as a monitoring tool in the management of their patients and in order to improve medication outcomes. Considerations for the transition to use of POCT in the home to further improve disease management and improve health care cost-effectiveness are discussed. Devices available for home use include those suitable for management of diabetes mellitus, hypertension, congestive heart failure, and anticoagulation. Many of these devices include software capabilities enabling patients to share important health information with health care providers using a computer. Limitations and challenges surrounding implementation of home POCT for patients include reliability of instrumentation, ability to coordinate data collection, necessary training requirements, and cost-effectiveness. Looking forward, the successful integration of POCT into the homes of patients is contingent on a concerted effort made by all members of the health care team.