Navigating the landscape of food allergies: Insights and perspectives from the AMCP Market Insights Program.

AMCP convened a panel of clinical and managed care experts to identify insights regarding the prevalence, clinical manifestations, and management approaches for immunoglobulin E-mediated food allergies. This article aims to summarize expert perspectives on health care system challenges and areas of agreement concerning the management of food allergies, and to advance payers' understanding of their role in supporting health care for patients with food allergies. Food allergy management requires dietary modification and is associated with significant patient and caregiver burdens. Emerging therapies provide hope for those living with food allergies but will likely lead to a rise in health plan pharmacy expenses. In considering the value of new treatments, it is important to consider the total cost of care and the value of preventing anaphylaxis and enhancing the patient's quality of life. Several challenges remain in identifying the appropriate patient population for treatment with newer agents and in optimizing treatment outcomes. Addressing health disparities will require standardized clinical protocols, better access to specialized allergy care, and management of comorbid conditions.

Optimizing the management of inherited blood disorders in a changing market: Findings from the AMCP Market Insights Program.

In this market insights program, AMCP brought together a panel of experts representing various stakeholders: national and regional health plans, integrated health care systems, employer benefits groups, clinical experts, the Centers for Disease Control and Prevention, and patient advocacy organizations. The objectives were to gain insights into the current and evolving treatments in hemophilia, sickle cell disease, and ß-thalassemia; measure the effects of recently approved therapies on clinicians, payers, and patients; recognize emerging trends within the stop-loss market; address potential issues and obstacles related to monitoring and reporting outcomes; and identify concerns associated with both existing and emerging contracting and reimbursement models. This article aims to summarize expert perspectives on health care system challenges and strategies concerning the management of inherited blood disorders and to advance managed care professionals' understanding of their role in supporting care for these patients. The experts emphasized that when shaping coverage policies, a patient-centered approach is crucial, focusing on preserving organ function to maintain eligibility for future gene therapies among individuals with inherited blood disorders. These strategies, including benefit design modifications, specialized provider networks, and centralized mechanisms like registries, are vital for evaluating effectiveness, facilitating decision-making, and managing costs and risks associated with new and emerging treatment options for inherited blood disorders.

Impact of mental health in persons living with rare disease: Findings from the AMCP Market Insights Program.

Within the framework of its Market Insights Program, AMCP convened a panel of experts representing diverse stakeholders to identify alterations to plan design and/or coverage options geared toward improving the diagnosis and treatment of mental health conditions among persons living with rare diseases (PLWRD). PLWRD face unique mental health challenges because of the misunderstood nature of their conditions, potential misdiagnosis, and limited treatment options. Economic burdens arise from increased medical needs, reliance on caregivers, and work disruptions. The interplay of these factors, along with health insurance coverage, creates a distinctive mental health landscape for PLWRD and a need to prioritize mental health support for this patient population. This article aims to (1) summarize expert perspectives on health care system challenges and areas of agreement concerning the management of mental health conditions and (2) advance payers' understanding of their role in supporting mental health care for patients with rare diseases. Addressing mental health needs of PLWRD presents multifaceted challenges. Managed care organizations play a pivotal role in supporting mental health care for PLWRD through their quality improvement initiatives and policies for coverage and reimbursement, which can impact both the rare disease treatment and mental health services PLWRD receive.

Cardiologists' Perspectives on BiDil and the Use of Race in Drug Prescribing.

OBJECTIVES: We explored cardiologists' attitudes and prescribing patterns specific to the use of generic isosorbide dinitrate and hydralazine hydrochloride, and the fixed-dose patented drug, BiDil. BACKGROUND: Since the Food and Drug Administration approved BiDil in 2005 with an indication for self-identified black patients, disagreement about the appropriateness of race-based drugs has intensified and led to calls for providers and researchers to abandon race-based delimitations. This paper reports empirical evidence of cardiologists' views on BiDil's race-based indication and their ongoing inertia with respect to the debate about BiDil. METHODS: We conducted a 2010 cross-sectional online survey of members of the Association of Black Cardiologists. RESULTS: Fifty-nine cardiologists responded to the survey. Most participants (62.7%) prescribed BiDil to their patients. More than 40% of respondents did not prescribe BiDil to any non-African Americans. When considering whether to prescribe BiDil, a patient's race determined by physician assessment was the third most important factor considered by participants. The majority of participants (72.7%) selected symptoms as the most important factor. Most participants (59.2%) perceived race as defining biologically distinct individuals. Respondents prescribed BiDil more often to African American patients than non-African American patients. However, they prescribed the generic components that makeup BiDil to African Americans and non-African American patients similarly. CONCLUSIONS: The survey provides useful findings that, when viewed within the context of ongoing debates about race-based medicine, show little progress toward appropriately utilizing BiDil to maximize health outcomes, yet, might inform the development of practical and effective guidelines concerning the use of race in medicine.

Meeting the affordability challenges posed by orphan drugs: a survey of payers, providers, and employers.

BACKGROUND: As an increasing number of orphan drugs are FDA approved, health care payers, employers, and providers are attempting to strike a balance between patient access to innovative treatments and overall affordability. Payers and employers are evaluating how traditional specialty pharmacy management strategies and innovative models can support continued coverage of orphan drugs. OBJECTIVE: To understand how health care stakeholders are meeting the financial challenges posed by the increasing number and cost of orphan drugs and how these strategies are affecting orphan drug acquisition for providers. METHODS: A survey was conducted with payer, provider, and employer decision makers recruited from both AMCP and a proprietary database of market-access decision makers in July and August 2020. Respondents were asked about their experiences and activities in the orphan disease space, including tactics to manage affordability of drugs to treat orphan diseases. RESULTS: Reinsurance was the most commonly utilized strategy to maintain affordability of the benefit for both payers (42%) and employers (55%). Although 31% of payers have adopted gene therapy carve-outs, no employers had done so. Approximately three quarters (76%) of payers believe that limited distribution networks impede their abilities to manage orphan drugs, compared with 4% who believe limited networks improve orphan drug management. For most payers (78%), the decision to cover orphan drugs on either the medical or pharmacy benefit depends on the specific drug. Medical benefit coverage was driven primarily by site-of-care policies (55%) and the lower drug cost of average sales price pricing (50%). Pharmacy benefit coverage was driven primarily by a greater ability to manage the orphan drug (71%) and by rebates (62%). One in 3 (33%) of providers with experience treating orphan diseases acquire orphan drugs exclusively through buy and bill, whereas 10% acquire them exclusively through a specialty pharmacy provider. Buy-and-bill acquisition by providers was driven primarily by improved patient affordability (47%) and 340b pricing (47%). Specialty pharmacy provider acquisition was driven primarily by payer requirements (64%) and reduced administrative burden (64%). CONCLUSIONS: Payers and employers are adopting innovative benefit designs and strategies to cover orphan drugs while maintaining plan affordability. Cost considerations are prominent factors in determining whether orphan drugs will be covered under the pharmacy or medical benefit and how providers will acquire orphan drugs. DISCLOSURES: This research was sponsored by AMCP and PRECISIONvalue. Lopata, Terrone, and Gopalan are employees of PRECISIONvalue. Ladikos and Richardson are employees of AMPC. The authors have nothing further to disclose. This research was presented during the AMCP Partnership Forum "Preparing for and Managing Rare Diseases" held virtually September 8-10, 2020.

Value of Investment as a Key Driver for Prioritization and Implementation of Healthcare Software.

Health systems across the nation are recovering from massive financial and resource investments in electronic health record applications. In the midst of these recovery efforts, implementations of new care models, including accountable care organizations and population health initiatives, are underway. The shift from fee-for-service to fee-for-outcomes and fee-for-value payment models calls for care providers to work in new ways. It also changes how physicians are compensated and reimbursed. These changes necessitate that healthcare systems further invest in information technology solutions. Selecting which information technology (IT) projects are of most value is vital, especially in light of recent expenditures. Return-on-investment analysis is a powerful tool used in various industries to select the most appropriate IT investments. It has proven vital in selecting, justifying, and implementing software projects. Other financial metrics, such as net present value, economic value added, and total economic impact, also quantify the success of expenditures on information systems. This paper extends the concept of quantifying project value to include clinical outcomes and nonfinancial value as investment returns, applying a systematic approach to healthcare software projects. We term this inclusive approach Value of Investment. It offers a necessary extension for application in clinical settings where a strictly financial view may fall short in providing a complete picture of important benefits. This paper outlines the Value of Investment process and its attributes, and uses illustrative examples to explore the efficacy of this methodology within a midsized health system.

GM-CSF regulates a PU.1-dependent transcriptional program determining the pulmonary response to LPS.

Alveolar macrophages (AMs) normally respond to lipopolysaccharide (LPS) by activating Toll-like receptor (TLR)-4 signaling, a mechanism critical to lung host defense against gram-negative bacteria such as Pseudomonas aeruginosa. Because granulocyte macrophage colony-stimulating factor (GM-CSF)-deficient (GM(-/-)) mice are hyporesponsive to LPS, we evaluated the role of GM-CSF in TLR-4 signaling in AMs. Pulmonary TNF-alpha levels and neutrophil recruitment 4 h after intratracheal administration of Pseudomonas LPS were reduced in GM(-/-) compared with wild-type (GM(+/+)) mice. Secretion of TNF-alpha by AMs exposed to LPS ex vivo was also reduced in GM(-/-) mice and restored in mice expressing GM-CSF specifically in the lungs (SPC-GM(+/+)/GM(-/-) mice). LPS-dependent NF-kappaB promoter activity, TNF-alpha secretion, and neutrophil chemokine release were reduced in AM cell lines derived from GM(-/-) mice (mAM) compared with GM(+/+) (MH-S). Retroviral expression of PU.1 in mAM cells, which normally lack PU.1, rescued all of these AM defects. To determine whether GM-CSF, via PU.1, regulated expression of TLR-4 pathway components, mRNA and protein levels for key components were evaluated in MH-S cells (GM(+/+), PU.1(Positive)), mAM cells (GM(-/-), PU.1(Negative)), and mAMPU.1+ cells (GM(-/-), PU.1(Positive)). Cluster of differentiation antigen-14, radioprotective 105, IL-1 receptor-associated kinase (IRAK)-M mRNA, and protein were dependent upon GM-CSF and restored by expression of PU.1. In contrast, expression of other TLR-4 pathway components (myeloid differentiation-2, TLR-4, IRAK-1, IRAK-2, Toll/IL-1 receptor domain containing adapter protein/MyD88 adaptor-like, myeloid differentiation primary-response protein 88, IRAK-4, TNF receptor-associated factor-6, NF-kappaB, inhibitor of NF-kappaB kinase) were not GM-CSF or PU.1-dependent. These results show that GM-CSF, via PU.1, enables AM responses to P. aeruginosa LPS by regulating expression of a specific subset of components of the TLR-4 signaling pathway.