Specialty Drugs Research Articles

Prescription rebate guarantees: employerinsights.

To describe (1) rebate arrangements for specialty drugs, (2) the use and influence of benefits brokers and consultants, and (3) the importance of rebate-related factors when selecting a pharmacy benefits manager (PBM) among a sample of employers with self-funded pharmacybenefits. A national survey of employer drug benefit decision makers (N = 110) for organizations with self-insured pharmacy benefits. We summarized respondents' current rebate agreements for specialty drugs and their perspectives on the importance of rebates and rebate guarantees overall as well as by type of rebate agreement and by the person or entity identified as most influential in rebate strategy. Nearly two-thirds of employers reported having rebate agreements with a rebate guarantee for specialty drugs (n = 69; 62.7%). The person or entity most influential to rebate strategy decisions was often a benefits consultant (37.3%), a human resources/benefits leader (29.1%), or a benefits broker (21.8%). Employers with rebate guarantees ascribed a higher level of importance to guarantees when selecting a PBM (median [IQR], 9 [7-10]) than employers receiving rebates without a guarantee (7 [6-8]) and those who do not receive rebates (7.5 [4-9]) (P = .001). These findings shed light on the importance of rebate guarantees and the role of employer benefits consultants and brokers in PBM selection. Asthe public discourse on PBMs and drug rebates continues, it is important to recognize the role employer benefits consultants may play in perpetuating employer reliance on guaranteed rebate arrangements.

A descriptive survey of patient experiences and access to specialty medicines with alternative funding programs.

Alternative funding programs (AFPs) seek to reduce health plan sponsor costs, for example by excluding specialty drugs from a beneficiary's plan coverage and requiring patients to obtain medications through alternative sources (typically, the manufacturer's patient assistance programs) via an AFP vendor as a third-party. To describe patients' experiences and specialty medication access with AFPs. A survey method consisting of 26 optional single-choice and multiple-choice questions with branching logic divided across 5 sections (related to patient challenges with AFPs) was administered to patients recruited from an experienced AFP online patient panel and a patient advocacy group. The survey assessed patients' awareness of AFPs from their employers, experience with the patient assistance program application process via the AFP vendor, timeliness of medication access (if granted), and/or the health impact of delay in access. All descriptive and exploratory subgroup analyses were conducted by disease area and reported income levels; statistical analyses were carried out for the exploratory analyses. The final sample included 227 patients. Most patients (61% [136/223]) first heard of the AFP as part of their health benefit when trying to obtain their medication. Of 198 patients, 88% reported being stressed because of the medication coverage denial and the uncertainty of obtaining their medication. More than half of patients (54% [115/213]) reported being uncomfortable with the benefits manager from the AFP vendor. On average, patients reported waiting to receive their medication for 68.2 days (approximately 2 months); 24% (51/215) reported the wait for the medication worsened their condition and 64% (138/215) reported the wait led to stress and/or anxiety. Patients who indicated the wait time negatively affected them had considered a job change or left their job at a 3-5-fold higher rate than those who reported no impact from wait time. A significantly higher proportion of patients with hemophilia and other bleeding disorders reported receiving their prescribed medication less often than patients with other conditions (63% [19/30] vs 81% [52/64]; P = 0.022), whereas more patients with lower incomes (<$50,000 vs >$50,000) reported not receiving any medication (12% [7/57] vs 5% [7/129]; P = 0.657), although these differences were not significant. Most patients who obtain their specialty medicines via AFPs reported being uncomfortable with the process and experiencing treatment delays, which may have been linked to disease progression, worsened mental well-being, and consideration of a job change. Employers should be aware of the potential downstream impacts on employee health, retention, and the employee-employer relationship when considering implementing an AFP into their health plan.

Medical Professionals and Pharmacological Intervention for the Treatment of Insomnia: A Cross-Sectional Study

Abstract Objective To explore the preferences of medical practitioners concerning various medications and other remedies to manage insomnia, and to ascertain whether these preferences are associated with their respective medical specialties. Materials and Methods Employing the snowball sampling technique, we administered two versions of a questionnaire to an international group of medical professionals, including trainees and specialists from diverse medical backgrounds. Results Zopiclone, zolpidem, and mirtazapine were evaluated as the most effective treatments for insomnia, while physicians would typically avoid using other tricyclic antidepressants, dual orexin receptor antagonists, and tryptophan for insomnia treatment. Noteworthy statistical correlations between physicians' specialty and preferred drug therapy, were observed in three out of five cases: 1) first-line drug treatment for short-term intervention against insomnia; (2) second-line treatment for long-term intervention; and 3) cases involving the elderly. Discussion Psychiatrists demonstrated a greater preference for antipsychotics and antidepressants for the treatment of insomnia compared with other physicians. Conversely, other medical professionals exhibited a preference for benzodiazepines and Z-drugs (zopiclone and zolpidem). Although Z-drugs were evaluated as the most effective in the treatment of insomnia, in the clinical practice, physicians administer or would administer antidepressant or antipsychotic drugs more often (mirtazapine and quetiapine respectively). Regarding Dual Orexin Receptor Antagonists (DORAs), the high prevalence of “Do not know/No opinion” answers implies that our sample was not familiar with this innovative treatment.

Client and program-level factors associated with planned use of medications for opioid use disorder in specialty substance use treatment programs: Evidence from linked administrative data and survey data

BackgroundMost patients in specialty drug treatment programs that are not federally licensed Opioid Treatment Programs (OTPs) programs do not receive medications for opioid use disorder (MOUD). MethodsWe linked results from a survey of non-OTP treatment program directors in New Jersey (n = 81) to statewide administrative records of admissions for opioid use to those programs between July 2021–June 2022. Using multi-level regression, we examined the association of three types of factors with planned MOUD use: program survey responses, client-level factors, and program-level client characteristic mix. ResultsOf 9583 opioid treatment admissions in non-OTP settings, 41 % included treatment plans involving MOUD. Programs where directors reported staff concerns about buprenorphine's efficacy or diversion had a lower proportion of clients with planned MOUD, as did programs reporting too little physical space to prescribe. Being self-referred to treatment, unemployed and not looking for work, aged 30–49, heroin use (vs. prescription opioid use), and stimulant use in addition to opioids, were positively associated with planned MOUD; while non-Medicaid insurance, and Black and Hispanic race/ethnicity, were negatively associated with planned MOUD. Clients were more likely to have planned MOUD if their programs had a higher proportion of clients aged 30 or older, heroin as primary “drug of abuse,” stimulant use, and not working but actively looking for work. ConclusionFindings suggest addressing program staff attitudes toward buprenorphine could help increase planned MOUD. There is also a need to improve access for clients with non-Medicaid insurance, address within-program race and ethnic disparities, and address employment-related barriers to medication.

Agreement of Medication Information Derived From EHR Data Compared to Medicare Insurance Claims: An Analysis of Biologic Disease-Modifying Antirheumatic Drugs.

Few studies have reported the agreement between medication information derived from ambulatory EHR data compared to administrative claims for high-cost specialty drugs. We used data from a national EHR-enabled registry, the Rheumatology Informatics System for Effectiveness (RISE), with linked Medicare claims in a population of patients with rheumatoid arthritis (RA) to investigate variations in agreement for different biologic disease-modifying agents (bDMARDs) between two data sources (RISE EHR data vs. Medicare claims), categorized by drug, route of administration, and patient insurance factors (dual eligibility). Patients ≥ 65 years old, with ≥ 2 visits in RISE with RA ICD codes ≥ 30 days apart, and continuous enrollment in Medicare Parts B and D in 2017-2018 were included. We classified patients as bDMARD users or nonusers in Medicare claims or EHR data in 2018, and we calculated sensitivity, specificity, positive predicted value (PPV), and negative predicted value (NPV) of EHR data for identifying bDMARD users, using Medicare as the reference standard. We also calculated these metrics after stratifying by clinic-administered (Part B) versus. pharmacy-dispensed (Part D) bDMARDs and by patient dual-eligibility. A total of 26 097 patients were included in the study. Using Medicare claims as the reference standard, EHR data had a sensitivity of 75.0%-90.8% for identifying patients with the same medication and route. PPV for Part B bDMARDs was higher compared with Part D bDMARDs (range 94.3%-97.3% vs. 51.0%-69.6%). We observed higher PPVs for Part D bDMARDs among patients who were dual-eligible (range 82.4%-95.1%). The risk of misclassification of drug exposure based on EHR data sources alone is small for Medicare Part B bDMARDs but could be as high as 50% for Part D bDMARDs, in particular for patients who are not dually eligible for Medicare and Medicaid.

Impact of 340B drug pricing program on specialty medication access: A policy analysis and future directions

The 340B Drug Pricing Program, initially designed to assist healthcare providers in offering affordable medications to vulnerable populations, has increasingly come under scrutiny, particularly concerning its impact on specialty medication access. This review critically examines the program’s effectiveness in improving access to high-cost specialty drugs, highlighting significant challenges such as program misalignment, economic pressures, and criticisms from the pharmaceutical industry. It further explores the regulatory landscape, stakeholder perspectives, and ethical considerations that shape the program’s implementation. The paper proposes reforms to refine eligibility criteria, enhance transparency, and adjust pricing structures to serve better patients needing specialty medications. Additionally, it discusses innovative strategies like digital health integration and industry partnerships that could enhance the program’s efficacy. The long-term implications of these proposed changes on healthcare systems, patients, and the pharmaceutical industry are considered, emphasizing the need for a balanced approach to sustain the 340B program’s mission in the evolving healthcare environment.

Assessing the feasibility and likelihood of policy options to lower specialty drug costs.

Specialty drugs are high-cost medications often used to treat complex chronic conditions. Even with insurance coverage, patients may face very high out-of-pocket costs, which in turn may restrict access. While the Inflation Reduction Act of 2022 included policies designed to reduce specialty drug costs, relatively few policies have been enacted during the past decade. In 2022-2023, we conducted a scoping literature review to identify a range of policy options and selected a set of 9 that have been regularly discussed or recently considered to present to an expert stakeholder panel to seek consensus on (1) the feasibility of implementing each policy and (2) its likely impact on drug costs. Experts rated only 1 policy highly on both feasibility and impact: grouping originator biologics and biosimilars under the same Medicare Part B reimbursement code. They rated 3 policies focused on setting payment limits as likely to have positive (downward) impact on costs but of uncertain feasibility. They considered 4 policies as uncertain on both criteria. Experts rated capping monthly out-of-pocket costs as feasible but unlikely to reduce specialty drug costs. Based on these results, we offer 4 recommendations to policymakers considering ways to reduce specialty drug costs.

Clinical Considerations for HealthcareProvider-Administered Lipid-Lowering Medications.

Atherosclerotic cardiovascular disease (ASCVD), a leading cause of mortality and morbidity, is associated with a substantial healthcare and economic burden. Reduction of low-density lipoprotein cholesterol (LDL-C) to guideline-recommended goals is crucial in the prevention or management of ASCVD, particularly in those at high risk. Despite the availability of several effective lipid-lowering therapies (LLTs), up to 80% of patients with ASCVD do not reach evidence-based LDL-C goals. This nonattainment may be due to poor adherence to, and lack oftimely utilization of, LLTs driven by a range of variables, including polypharmacy, side effects, clinical inertia, costs, and access issues. Inclisiran was approved by the US Food and Drug Administration in 2021 as a novel, twice-yearly, healthcare provider (HCP)-administered LLT. In-office administration allows HCPs more control of drug acquisition, administration, and reimbursement, and may allow for more timely care and increased patient monitoring. In the USA, in-office administered drugs are considered a Medical Benefit and can be acquired and reimbursed using the "buy-and-bill" process. Buy-and-bill is a standard system for medication administration already established in multiple therapeutic areas, including oncology, vaccines, and allergy/immunology. Initiating in-office administration will involve new considerations for clinicians in the cardiovascular specialty, such as the implementation of new infrastructure and processes; however, it could ultimately increase treatment adherence and improve cardiovascular outcomes for patients with ASCVD. This article discusses the potential implications of buy-and-bill for the cardiology specialty and provides a practical guide to implementing HCP-administered specialty drugs in US clinical practice.

US commercial health plan coverage dynamics in pulmonary arterial hypertension therapies.

Health plan coverage is central to patient access to care, especially for rare, chronic diseases. For specialty drugs, coverage varies, resulting in barriers to access. Pulmonary arterial hypertension (PAH) is a rare, progressive, and fatal disease. Guidelines suggest starting or rapidly escalating to combination therapy with drugs of differing classes (phosphodiesterase 5 inhibitors [PDE5is], soluble guanylate cyclase stimulators [sGC stimulators], endothelin receptor antagonists [ERAs], and prostacyclin pathway agents [PPAs]). To assess the variation in commercial health plan coverage for PAH treatments and how coverage has evolved. To examine the frequency of coverage updates and evidence cited in plan policies. We used the Tufts Medical Center Specialty Drug Evidence and Coverage database, which includes publicly available specialty drug coverage policies. Overall, and at the drug and treatment class level, we identified plan-imposed coverage restrictions beyond the drug's US Food and Drug Administration label, including step therapy protocols, clinical restrictions (eg, disease severity), and prescriber specialty requirements. We analyzed variation in coverage restrictiveness and how coverage has changed over time. We determined how often plans update their policies. Finally, we categorized the cited evidence into 6 different types. Results reflected plan coverage policies for 13 PAH drugs active between August 2017 and August 2022 and issued by 17 large US commercial health plans, representing 70% of covered lives. Coverage restrictions varied mainly by step therapy protocols and prescriber restrictions. Seven plans had step therapy protocols for most drugs, 9 for at least one drug, and 1 had none. Ten plans required specialist (cardiologist or pulmonologist) prescribing for at least one drug, and 7 did not. Coverage restrictions increased over time: the proportion of policies with at least 1 restriction increased from 38% to 73%, and the proportion with step therapy protocols increased from 29% to 46%, with generics as the most common step. The proportion of policies with step therapy protocols increased for every therapy class with generic availability: 18% to 59% for ERAs, 33% to 77% for PDE5is, and 33% to 43% for PPAs. The proportion of policies with prescriber requirements increased from 24% to 48%. Plans updated their policies 58% of the time annually and most often cited the 2019 CHEST clinical guidelines, followed by randomized controlled trials. Plan use of coverage restrictions for PAH therapies increased over time and varied across both drugs and plans. Inconsistency among health plans may complicate patient access and reduce the proportion who can persist on PAH treatments.

Assessment of the utilization of real-time prescription benefits for patient cost savings within an outpatient setting

BackgroundThis study evaluates the impact of Real-Time Prescription Benefits (RTPB), a tool integrated into electronic health records (EHRs), on patient out-of-pocket costs in an academic institution. RTPB provides prescribers with alternative, less expensive medications based on insurance plans. The primary measure was cost-savings, defined as the difference between the out-of-pocket cost of the prescribed medication and its alternative. MethodsA retrospective analysis of prescriptions from outpatient clinics in a university-based health system was conducted between May 2020 and July 2021. Prescriptions were analyzed at the 2nd level of the Anatomical Therapeutic Chemical (ATC) classification system. Costs were standardized to a 30-day supply. Standardized cost and total cost per prescription, and overall savings for the top 20 medication classes at the 2nd ATC level were calculated. The overall impact of RTPB was estimated based on selecting the least expensive alternative suggested by RTPB. ResultsThe study found that RTPB information was provided for 22% of prescriptions, with suggested alternatives for 1.26%. Among prescriptions with an alternative selected, the standardized average cost saving was $38.83. The study realized $15,416 in patient total cost savings. If the least expensive RTPB-suggested alternative were chosen for all prescriptions, an estimated $276,386 could have been saved. Psychoanaleptic and psycholeptic medications were the most prescribed with an alternative, with most savings in specialty drugs like anthelmintic and immunostimulant medications. ConclusionThe study highlights the importance of RTPB in reducing patient costs. It reports patient cost-savings with RTPB in prescribing decisions. Future research could explore the impact of RTPB on medication adherence using pharmacy claims data.

Oral cancer drug repositories: Challenges and solutions.

11063 Background: Frequent drug shortages and high out-of-pocket costs due to inadequate and unaffordable insurance coverage hinder access to oral chemotherapy. Amidst this, oral cancer drugs estimated a mean of $4290/patient are wasted due to dose modification, discontinuation or death[Lam M. et al, JAMA Oncol. 2023;9(9):1238-1244]. To combat this discrepancy, multiple states have passed laws establishing drug repositories that collect unused oral cancer medications and redistribute them to patients in need. ASCO has also endorsed these programs, even advocating for open distribution systems allowing donations from individuals. Methods: We analysed statewise cancer drug repositories(source:National Conference of State Legislatures) and communicated with representatives from programs including SIRUM, RemediChain and I-DROP Coalition. We used a mixed methods approach to examine the breadth and functioning of these programs. Results: 28 out of the 44 states with laws establishing prescription drug repositories operate state-run drug recycling programs (Updated September 2023). However, only 14 states (California, Florida, Iowa, Michigan, Minnesota, Montana, Nebraska, Nevada, Ohio, Pennsylvania, Tennessee, Utah, Washington and Wisconsin) have provisions for donation of cancer medications, or for creation of separate cancer drug repositories. Other nonprofits like RemediChain and SIRUM function at a national level, covering 47 states and Washington D.C. between them. Conversations with 3 such programs revealed multiple obstacles that limit the effectiveness of drug recycling. All 3 programs reported inadequate donations, ranging from 2-3 donations/month in a state level program (I-DROP) to 1-10 donations/day in a larger national level program (RemediChain). 3/3 programs received more donations of unused pills from patients or their families than from pharmacies/health facilities. All 3 surveyed programs reported limited and inconsistent supply of donated drugs; expiration of drugs before redistribution; lack of demand for specialty drugs and unavailability of common oncology drugs that patients request since these drugs are less likely to go unused. All surveyed programs mentioned struggling with funding and staffing. Notably, all 3 repositories stressed that informing patients about recycling options right at the beginning of treatment was crucial to improve donation rates; and that expanding repository donor and recipient coverage could increase the chances of successfully redistributing drugs. Conclusions: Improving awareness amongst providers and patients, campaigning for federal funding and increasing the scale of coverage by expanding repository programs to all states and allowing interstate transfer of drugs could address current challenges to drug recycling. This would limit wastage of expensive medication while reducing financial burden on patients and improving access to life saving treatment.

Projected environmental and public health benefits of extended-interval dosing: an analysis of pembrolizumab use in a US national health system

Health care is a major source of greenhouse gas emissions, leading to climate change and public health harms. Changes are needed to improve the environmental sustainability of health-care practices, but such changes should not sacrifice patient outcomes or financial sustainability. Alternative dosing strategies that reduce the frequency with which specialty drugs are administered, without sacrificing patient outcomes, are an attractive possibility for improving environmental sustainability. We sought to inform environmentally sustainable cancer care by estimating and comparing the environmental and financial effects of alternative, clinically equivalent strategies for pembrolizumab administration. We conducted a retrospective analysis using a cohort of patients from the Veterans Health Administration (VHA) in the USA who received one or more pembrolizumab doses between May 1, 2020, and Sept 30, 2022. Using baseline, real-world administration of pembrolizumab, we generated simulated pembrolizumab use data under three near-equivalent counterfactual pembrolizumab administration strategies defined by combinations of weight-based dosing, pharmacy-level vial sharing and dose rounding, and extended-interval dosing (ie, every 6 weeks). For each counterfactual dosing strategy, we estimated greenhouse gas emissions related to pembrolizumab use across the VHA cohort using a deterministic environmental impact model that estimated greenhouse gas emissions due to patient travel, drug manufacture, and medical waste as the primary outcome measure. We identified 7813 veterans who received at least one dose of pembrolizumab-containing therapy in the VHA during the study period. 59 140 pembrolizumab administrations occurred in the study period, of which 46 255 (78·2%) were dosed at 200 mg every 3 weeks, 12 885 (21·8%) at 400 mg every 6 weeks, and 14 955 (25·3%) were coadministered with infusional chemotherapies. Adoption of weight-based, extended-interval pembrolizumab dosing (4 mg/kg every 6 weeks) and pharmacy-level stewardship strategies (ie, dose rounding and vial sharing) for all pembrolizumab infusions would have resulted in 24·7% fewer administration events than baseline dosing (44 533 events vs 59 140 events) and an estimated 200 metric tons less CO2 emitted per year as a result of pembrolizumab use within the VHA (650 tons vs 850 tons of CO2, a relative reduction of 24%), largely due to reductions in distance travelled by patients to receive treatment. Similar results were observed when weight-based and extended-interval dosing were applied only to pembrolizumab monotherapy and pembrolizumab in combination with oral therapies. Alternative pembrolizumab administration strategies might have environmental advantages over the current dosing and compounding paradigms. Specialty medication dosing can be optimised for health-care spending and environmental sustainability without sacrificing clinical outcomes. None.

Addressing financial toxicity in cancer treatment-An opportunity for the 340B drug pricing program.

Cancer treatment has become increasingly expensive, partially due to the use of specialty drugs. The costs of these drugs are often passed down to patients, who may face the consequences of paying for more than they can afford, leading to financial toxicity. The 340B drug pricing program is a health care policy that may provide an opportunity to mitigate the financial consequences of cancer care. The 340B program requires manufacturers to sell outpatient drugs at a discount to hospitals caring for a significant number of socioeconomically disadvantaged individuals. The program intended for hospitals to use savings from discounted purchases to expand their safety net to vulnerable patients. Some studies have shown that participating hospitals do this by offering more charity and discounted care, whereas others have demonstrated that hospitals fail to sufficiently expand their safety net. A potential flaw of the program is the lack of guidance from governing bodies on how hospitals should use savings from discounted purchases. There has been growing discussion among stakeholders to reform the 340B program given the mixed findings of its effectiveness. With the rising costs of specialty drugs and associated prevalence of financial toxicity in patients with cancer, there is an opportunity to address these issues through reform that improves the program. Directing hospitals to offer specific safety net opportunities, such as passing along discounted drug prices to vulnerable populations, could help the growing number of patients who are financially burdened by medications at the core of the 340B program.

PHARMACEUTICAL INDUSTRY ANALYSIS: A COMPREHENSIVE REVIEW OF MARKET TRENDS, COMPETITIVE LANDSCAPE, AND FUTURE OUTLOOK

The pharmaceutical industry, a cornerstone of modern healthcare, undergoes constant evolution to meet the dynamic demands of the healthcare landscape. This comprehensive review delves into the multifaceted dimensions of the industry, encompassing market trends, competitive dynamics, and future projections. Market trends reveal a shift towards personalized medicine, leveraging advancements in genomics and biotechnology, alongside a growing focus on specialty drugs catering to niche patient populations. The prevalence of chronic diseases and an aging populace accentuate the necessity for innovative therapies, while the digital revolution introduces new paradigms in patient care and drug development, propelled by telemedicine, digital therapeutics, and artificial intelligence. Simultaneously, regulatory pressures and pricing constraints exert significant influence, necessitating adaptability among industry players. In the competitive landscape, established pharmaceutical giants vie for market dominance amidst patent expirations and generic competition, while biotech startups and niche players disrupt traditional models. Mergers and acquisitions serve as strategic imperatives for pipeline fortification and market expansion. Looking forward, the industry must navigate a terrain characterized by rapid technological advancements, evolving patient needs, and regulatory complexities, all while striving for innovation, accessibility, and sustainability in the delivery of healthcare solutions. Keywords: Pharmaceutical industry, healthcare landscape, market trends, personalized medicine, specialty drugs, chronic diseases.

Auditing the prescription drug consumer price index in a changing marketplace.

Changes in the dynamics of prescription drug markets have raised issues regarding whether the United States Bureau of Labor Statistics' (BLS') Prescription Drug Consumer Price Index (CPI-Rx) has adequately kept up with the evolving marketplace. The CPI-Rx limits its sampling frame to retail outpatient outlets and excludes prescription pharmaceuticals dispensed in non-retail settings such as hospitals, physician/clinic outpatient facilities, and nursing homes. Thus, the CPI-Rx overlooks the increasingly important specialty pharmaceuticals dispensed in non-retail settings, whose transactions are instead captured in the overall hospital and professional services component of the medical care CPI. Specialty drugs now account for about 55% of all U.S. drug spending, double the share of a decade earlier. To the extent specialty drug price growth differs from that of traditional pharmaceuticals, the CPI-Rx could provide an inaccurate measure of overall drug price inflation. We calculate a chained Laspeyres CPI using data from the Merative™ MarketScan® Databases for the years 2010-2019 and IQVIA-designated specialty drugs and offer evidence showing that by not sampling specialty drugs in non-retail settings, the CPI-Rx has understated overall U.S. prescription drug inflation by just under 75 basis points annually. We discuss implications for health care policy and suggest the BLS examine the feasibility of publishing an overall pharmaceutical price index incorporating both traditional and specialty pharmaceuticals dispensed in retail and non-retail settings.

National trends in prescription drug expenditures and projections for 2024.

To report historical patterns of pharmaceutical expenditures, to identify factors that may influence future spending, and to predict growth in drug spending in 2024 in the United States, with a focus on the nonfederal hospital and clinic sectors. Historical patterns were assessed by examining data on drug purchases from manufacturers using the IQVIA National Sales Perspectives database. Factors that may influence drug spending in hospitals and clinics in 2024 were reviewed-including new drug approvals, patent expirations, and potential new policies or legislation. Focused analyses were conducted for biosimilars, cancer drugs, endocrine drugs, generics, and specialty drugs. For nonfederal hospitals, clinics, and overall (all sectors), estimates of growth of pharmaceutical expenditures in 2024 were based on a combination of quantitative analyses and expert opinion. In 2023, overall pharmaceutical expenditures in the US grew 13.6% compared to 2022, for a total of $722.5 billion. Utilization (a 6.5% increase), new drugs (a 4.2% increase) and price (a 2.9% increase) drove this increase. Semaglutide was the top drug in 2023, followed by adalimumab and apixaban. Drug expenditures were $37.1 billion (a 1.1% decrease) and $135.7 billion (a 15.0% increase) in nonfederal hospitals and clinics, respectively. In clinics, increased utilization drove growth, with a small impact from price and new products. In nonfederal hospitals, a drop in utilization led the decrease in expenditures, with price and new drugs modestly contributing to growth in spending. Several new drugs that will influence spending are expected to be approved in 2024. Specialty, endocrine, and cancer drugs will continue to drive expenditures. For 2024, we expect overall prescription drug spending to rise by 10.0% to 12.0%, whereas in clinics and hospitals we anticipate an 11.0% to 13.0% increase and a 0% to 2.0% increase, respectively, compared to 2023. These national estimates of future pharmaceutical expenditure growth may not be representative of any health system because of the myriad of local factors that influence actual spending.

The increasing costs of medicines and their implications for patients, physicians and the health system.

Most new medicines entering the market are high-cost speciality drugs. These drugs can cost tens to hundreds of thousands of dollars per course of treatment and in some cases millions of dollars per dose. Approximately half of all spending on medicines is projected to target only 2-3% of patients, raising important questions about resource allocation. While there is no doubt that breakthrough innovations have transformed clinical care in some disciplines, it is also true that cost is becoming one of the primary barriers to treatment access and that many new medicines do not provide value commensurate with their prices. This article examines pricing trends, the reasons for high prices and their implications for access and clinical practice.

Specialty drug use for autoimmune conditions varies by race and wage among employees with employer-sponsored health insurance.

The relationship between race and ethnicity, wage status, and specialty medication (SpRx) use among employees with autoimmune conditions (AICs) is poorly understood. Insight into sociodemographic variations in use of these medications can inform health equity improvement efforts. To assess the association of race and ethnicity and wage status on SpRx use and adherence patterns among employees with AICs enrolled in employer-sponsored health insurance. In this observational, retrospective cohort analysis, data were obtained from the IBM Watson MarketScan database for calendar year 2018. Employees were separated into race and ethnicity subgroups based on employer-provided data. Midyear employee wage data were used to allocate employees into the following annual income quartiles: $47,000 or less, $47,001-$71,000, $71,001-$106,000, and $106,001 or more. The lowest quartile was further divided into 2 groups ($35,000 or less and $35,001-$47,000) to better evaluate subgroup differences. Outcomes included monthly days SpRx-AIC supply, proportion of days covered (PDC), and medication discontinuation rates. Generalized linear regressions were used to assess differences while adjusting for patient and other characteristics. From a sample of more than 2,000,000 enrollees, race and ethnicity data were available for 617,117 (29.8%). Of those, 47,839 (7.8%) were identified as having an AIC of interest, with prevalence rates of AICs differing by race within wage categories. Among those with AICs, 5,358 (11.2%) had filled at least 1 SpRx-AIC prescription. Following adjustment, except for the highest wage category, prevalence of SpRx-AIC use was significantly less among Black and Hispanic subpopulations. Black patients had significantly lower SpRx-AIC use rates than White patients (≤$35,000: 4.9 vs 9.4%, >$35,000-$47,000: 5.5 vs 10.6%, >$47,000-$71,000: 8.5 vs 11.1%, and >$71,000-$106,000: 9.1 vs 12.7%; P <0.001 for all). For Hispanic patients, prevalence rates were significantly lower than White patients in 3 different wage categories (≤$35,000: 4.5 vs 9.4%, >$35,000-$47,000: 6.1 vs 10.6%, and >$71,000-$106,000: 8.6 vs 12.7%; P < 0.001). PDC and 90-day discontinuation rates did not differ among race and ethnicity groups within the respective wage bands. Race and ethnicity and wage-related disparities exist in SpRx use, but not PDC or discontinuation rates for treatment of AICs among non-White and low-income populations with employer-sponsored insurance, and may adversely impact clinical outcomes.

The 340B Program and oral specialty drugs for advanced prostate cancer.

Expensive oral specialty drugs for advanced prostate cancer can be associated with treatment disparities. The 340B program allows hospitals to purchase medications at discounts, generating savings that can improve care of the socioeconomically disadvantaged. This study assessed the effect of hospital 340B participation on advanced prostate cancer. The authors performed a retrospective cohort study of Medicare beneficiaries with advanced prostate cancer from 2012 to 2019. The primary outcome was use of an oral specialty drug. Secondary outcomes included monthly out-of-pocket costs and treatment adherence. We evaluated the effects of 1) hospital 340B participation, 2) a regional measure vulnerability, the social vulnerability index (SVI), and 3) the interaction between hospital 340B participation and SVI on outcomes. There were 2237 and 1100 men who received care at 340B and non-340B hospitals. There was no difference in specialty drug use between 340B and non-340B hospitals, whereas specialty drug use decreased with increased SVI (odds ratio, 0.95, p=.038). However, the interaction between hospital 340B participation and SVI on specialty drug use was not significant. Neither 340B participation, SVI, or their interaction were associated with out-of-pocket costs. Although hospital 340B participation and SVI were not associated with treatment adherence, their interaction was significant (p=.020). This demonstrated that 340B was associated with better adherence among socially vulnerable men. The 340B program was not associated with specialty drug use in men with advanced prostate cancer. However, among those who were started on therapy, 340B was associated with increased treatment adherence in more socially vulnerable men.

The 340B Drug Pricing Program and oral specialty drugs for advanced prostate cancer.

26 Background: Oral specialty drugs like abiraterone and enzalutamide are increasingly used in the management of men with advanced prostate cancer. However, the costs of these drugs can pose barriers to access and continued use, particularly in the socioeconomically disadvantaged population. The 340B program allows participating hospitals to purchase these medications at steep discounts, generating millions of dollars in savings per hospital. These savings afford them the opportunity to invest in programs (e.g., drug discounts) aimed at expanding their safety net. We assessed the effect of 340B participation on the care of men with advanced prostate cancer. Methods: A 20% sample of national Medicare claims data was used to perform a retrospective cohort study of men with advanced prostate cancer from 2012 through 2019 who received their care at a hospital. The primary outcome was the initial use of an oral specialty drug for prostate cancer and secondary outcomes included associated monthly out-of-pocket costs and adherence to treatment. To account for heterogeneity, we evaluated the effects of 1) hospital 340B participation, 2) socioeconomic status, using the social vulnerability index (SVI), and 3) the interaction between hospital 340B participation and SVI on each outcome. Results: Of the men with advanced prostate cancer, 2,237 (67%) and 1,100 (33%) received care at 340B participating and non-participating hospitals. There was no difference in the use of an oral specialty drug in men who received care at a hospital participating and not participating in the 340B program (22% vs 23% p=0.63). Conversely, when assessing variation across SVI, use of an oral specialty drug decreased with increased SVI (i.e., increased vulnerability; OR 0.95, p=0.038). However, the interaction between hospital 340B participation and SVI did not affect oral specialty drug use. Neither 340B participation, SVI, nor their interaction affected patient out-of-pocket costs. Additionally, although neither hospital 340B participation or SVI affected adherence to treatment, the interaction between 340B participation and SVI was significant (OR 1.2, p=0.040). This demonstrated that 340B was associated with better adherence to treatment among more socially vulnerable men. Conclusions: The 340B program did not affect the frequency of men with advanced prostate cancer who were started on an oral specialty drug. However, among those who were already started on therapy, 340B was associated with increased adherence to treatment in more socially vulnerable men. The underlying mechanism by which this occurred was unclear. In light of growing calls from policymakers to reform the 340B program, due to concerns regarding its effectiveness, policymakers could initially shift focus towards providing hospitals with guidance on how to effectively use savings to benefit those most in need.