Biopharmaceutical pipeline funded by venture capital firms, 2014 to 2024.

Venture capital (VC) firms fund biopharmaceutical research and development (R&D) while incurring substantial financial risk. VC firms seek to invest in clinical areas with the greatest potential for financial return. Using a combination of data for clinical trials and VC investment deals between January 2014 and March 2024, we found that approximately 75% of VC investments were allocated to clinical trials studying small-molecule drugs compared to biologics or gene therapies, without substantial changes over the study period. Most of VC firms' investment in biopharmaceutical R&D was concentrated in phase 1 and phase 2 clinical trials. This trend has increased in recent years, with phase 1 trials accounting for nearly half of total deals and capital investments in 2023. VC investments were concentrated in several therapeutic areas, including cancer.

Manufacturer-sponsored drug coupon use and drug-switching behavior among patients with type 2 diabetes.

BACKGROUND: Patients often use manufacturer-sponsored coupons to reduce their out-of-pocket spending. However, little is known whether coupon use is associated with medication-switching behaviors. OBJECTIVE: To examine if using a manufacturer-sponsored coupon to initiate a medication is associated with patterns of medication-switching behaviors among patients with type 2 diabetes. METHODS: Using IQVIA's retail pharmacy claims data from October 2017 to September 2019, we analyzed commercially insured patients with type 2 diabetes who had newly started taking the following noninsulin diabetes drugs: generic metformin (nearly no coupon use), Sodium-glucose Cotransporter-2 (SGLT2) Inhibitors (SGLT2, high coupon use), and dipeptidyl peptidase IV inhibitors (DPP-IV inhibitors, moderate coupon use). We assessed if drug-switching behaviors, defined as no switching, switching to a same-class drug, or switching to a drug in a different class, differed among patients who did and did not use coupons to initiate treatments. We performed multinomial logistic regression to estimate the probability of each switching type associated with patients' initial coupon use. RESULTS: Among 9,781 patients in our sample, 83.7% of them initiated treatments with metformin, 8.2% with SGLT2, and 8.1% with DPP-IV inhibitors. The overall switching rate was the lowest for generic metformin (40%) than brand-name drugs (56%-57%). Among the brand-name drug users, patients who used a coupon to initiate these drugs were less likely to switch to any drug compared with patients without coupon use (SGLT2 = -18% [95% CI = -24% to -13%]; DPP-IV inhibitors = -9% [-16% to -2%]). These patients were also less likely to switch to drugs in other competing classes (SGLT2 = -16% [95% CI = -22% to -10%]; DPP-IV inhibitors = -9% [-16% to -2%]). CONCLUSIONS: Patients who started their treatment with generic metformin had the lowest rate of drug switching. Using coupons to initiate brand-name drugs in classes with prevalent coupons was associated with reduced medication switching to other class drugs.

Association of Hospitals' Experience with Bundled Payment for Care Improvement Model with the Diffusion of Acute Hospital Care at Home.

OBJECTIVE: To examine whether hospitals' experience in a prior payment model incentivizing care coordination is associated with their decision to adopt a new payment program for a care delivery innovation. DATA SOURCES: Data were sourced from Medicare fee-for-service claims in 2017, the list of participants in Bundled Payment for Care Improvement initiatives (BPCI and BPCI-Advanced), the list of hospitals approved for Acute Hospital Care at Home (AHCaH) between November 2020 and August 2022, and the American Hospital Association Survey. STUDY DESIGN: Retrospective cohort study. Hospitals' adoption of AHCaH was measured as a function of hospitals' BPCI experiences. Hospitals' BPCI experiences were categorized into five mutually exclusive groups: (1) direct BPCI participation, (2) indirect participation through physician group practices (PGPs) after dropout, (3) indirect participation through PGPs only, (4) dropout only, and (5) no BPCI exposure. DATA COLLECTION/EXTRACTION METHODS: All data are derived from pre-existing sources. General acute hospitals eligible for both BPCI initiatives and AHCaH are included. PRINCIPAL FINDINGS: Of 3248 hospitals included in the sample, 7% adopted AHCaH as of August 2022. Hospitals with direct BPCI experience had the highest adoption rate (17.7%), followed by those with indirect participation through BPCI physicians after dropout (11.8%), while those with no exposure to BPCI were least likely to participate (3.2%). Hospitals that adopted AHCaH were more likely to be located in communities where more peer hospitals participated in the program (median 10.8% vs. 0%). After controlling for covariates, the association of the adoption of AHCaH with indirect participation through physicians after dropout was as strong as with early BPCI adopter hospitals (average marginal effect: 5.9 vs. 6.2 pp, p < 0.05), but the other categories were not. CONCLUSIONS: Hospitals that participated in the bundled payment model either directly or indirectly PGPs were more likely to adopt a care delivery innovation requiring similar competence in the next period.

Hospital and Physician Group Practice Participation in Prior and Next-Generation Value-Based Payment Programs.

This cohort study examines whether prior direct or indirect participation in the Centers for Medicare & Medicaid Innovation Bundled Payments for Care Improvement (BCPI) Initiative was associated with their participation in the next generation of the program.

Characteristics of Prescription Drug Fills Using Pharmacy-Pharmacy Benefit Manager Discount Programs: The "GoodRx" Model.

OBJECTIVES: This study aimed to characterize products using pharmacy-pharmacy benefit manager (PBM) discounts and to estimate the association among such discounts, prescription utilization, and out-of-pocket costs. METHODS: This is a retrospective cohort study using IQVIA's Formulary Impact Analyzer, which contains anonymized, individual-level pharmacy claims representing US retail pharmacy transactions. We focused on 20 products with the greatest number of transactions using a pharmacy-PBM discount. Our unit of analysis was a treatment episode, defined as the length of time from an incident fill to no continuous use for 60 consecutive days after allowing for indefinite stockpiling. Outcome measures included products with greatest pharmacy-PBM discount use, characteristics of treatment episodes, and out-of-pocket costs with and without pharmacy-PBM discount. RESULTS: Across all products, 3.82% of transactions and 7.69% of treatment episodes were accompanied by a pharmacy-PBM discount. Commonly discounted products included generic treatments for chronic disease (lisinopril, levothyroxine, metformin) and neuropsychiatric conditions (alprazolam, amphetamine, buprenorphine, hydrocodone). The median postdiscount out-of-pocket cost was >2.5-fold higher during treatment episodes with a discount than those without ($15.15, interquartile range [IQR] $8.53-32.00, vs $5.88, IQR $1.40-15.00). Median treatment episode duration was 249 days (IQR 132-418) with discount use compared with 236 days (IQR 121-396) without discount use, although treatment episodes that began with a discount had fewer transactions per treatment episode and were shorter (median 212 days, IQR 114-360) than those that did not (313 days, IQR 178-500). CONCLUSIONS: Pharmacy-PBM discounts may foster market competition and improve access for under- and uninsured individuals; however, these programs may not generate savings for many insured individuals.

Comparison of Out-of-Pocket Spending on Ultra-Expensive Drugs in Medicare Part D vs Commercial Insurance.

Importance: Little is known about how out-of-pocket burden differs between Medicare and commercial insurance for ultra-expensive drugs. Objective: To investigate out-of-pocket spending for ultra-expensive drugs in the Medicare Part D program vs commercial insurance. Design, Setting, and Participants: This was a retrospective, population-based cohort study of individuals using ultra-expensive drugs included in a 20% nationally random sample of prescription drug claims from Medicare Part D and individuals aged 45 to 64 years using ultra-expensive drugs included in a large national convenience sample of outpatient pharmaceutical claims from commercial insurance plans. Claims data from 2013 through 2019 were used, and data were analyzed in February 2023. Main Outcomes and Measures: Claims-weighted mean out-of-pocket spending per beneficiary per drug by insurance type, plan, and age. Results: In 2019, 37 324 and 24 159 individuals using ultra-expensive drugs were identified in the 20% Part D and commercial samples, respectively (mean [SD] age, 66.2 [11.7] years; 54.9% female). A statistically significant higher share of commercial enrollees vs Part D beneficiaries were female (61.0% vs 51.0%; P < .001), and a statistically significantly lower share were using 3 or more branded medications (28.7% vs 42.6%; P < .001). Mean out-of-pocket spending per beneficiary per drug in 2019 was $4478 in Part D (median [IQR], $4169 [$3369-$5947]) compared with $1821 for commercial (median [IQR], $1272 [$703-$1924]); these differences were statistically significant every year. Differences in out-of-pocket spending comparing commercial enrollees aged 60 to 64 years and Part D beneficiaries aged 65 to 69 years exhibited similar magnitudes and trends. By plan, mean out-of-pocket spending per beneficiary per drug in 2019 was $4301 (median [IQR], $4131 [$3000-$6048]) in Medicare Advantage prescription drug (MAPD) plans, $4575 (median [IQR], $4190 [$3305-$5799]) in stand-alone prescription drug plans (PDPs), $1208 (median [IQR], $752 [$317-$1240]) in health maintenance organization plans, $1569 (median [IQR], $838 [$481-$1472]) in preferred provider organization plans, and $4077 (median [IQR], $2882 [$1075-$4226]) in high-deductible health plans. There were no statistically significant differences between MAPD plans and stand-alone PDPs in any study year. Mean out-of-pocket spending was statistically significantly higher in MAPD plans compared with health maintenance organization plans and in stand-alone PDPs compared with preferred provider organization plans in each study year. Conclusions and Relevance: This cohort study demonstrated that the $2000 out-of-pocket cap included in the Inflation Reduction Act may substantially moderate the potential increase in spending faced by individuals who use ultra-expensive drugs when moving from commercial insurance to Part D coverage.

Patterns of Manufacturer Coupon Use for Prescription Drugs in the US, 2017-2019.

Importance: Although manufacturer-sponsored coupons are commonly used, little is known about how patients use them within a treatment episode. Objectives: To examine when and how frequently patients use manufacturer coupons during a treatment episode for a chronic condition, and to characterize factors associated with more frequent use. Design, Setting, and Participants: This is a retrospective cohort study of a 5% nationally representative sample of anonymized longitudinal retail pharmacy claims data from October 1, 2017, to September 30, 2019, obtained from IQVIA's Formulary Impact Analyzer. The data were analyzed from September to December 2022. Patients with new treatment episodes using at least 1 manufacturer coupon over a 12-month period were identified. This study focused on patients with 3 or more fills for a given drug and characterized the association of the outcomes of interest with patient, drug, and drug class characteristics. Main Outcomes and Measures: The primary outcomes were (1) the frequency of coupon use, measured as the proportion of prescription fills accompanied by manufacturer coupon within the treatment episode, and (2) the timing of first coupon use relative to the first prescription fill within the treatment episode. Results: A total of 36 951 treatment episodes accounted for 238 474 drug claims and 35 352 unique patients (mean [SD] age, 48.1 [18.2] years; 17 676 women [50.0%]). Among these episodes, nearly all instances (35 103 episodes [95.0%]) of first coupon use occurred within the first 4 prescription fills. Approximately two-thirds of treatment episodes (24 351 episodes [65.9%]) used a coupon for the incident fill. Coupons were used for a median (IQR) of 3 (2-6) fills. The median (IQR) proportion of fills with a coupon was 70.0% (33.3%-100.0%), and many patients discontinued the drug after the last coupon. After adjustment for covariates, there was no significant association between an individual's out-of-pocket costs or neighborhood-level income and the frequency of coupon use. The estimated proportion of fills with a coupon was greater for products in competitive (19.5% increase; 95% CI, 2.1%-36.9%) or oligopolistic (14.5% increase; 95% CI, 3.5%-25.6%) markets than monopoly markets when there is only 1 drug in the therapeutic class. Conclusions and Relevance: In this retrospective cohort analysis of individuals receiving pharmaceutical treatment for chronic diseases, the frequency of manufacturer-sponsored drug coupon use was associated with the degree of market competition, rather than patients' out-of-pocket costs.

Ultra-Expensive Drugs And Medicare Part D: Spending And Beneficiary Use Up Sharply.

The proliferation of "ultra-expensive" drugs has sparked debate on their sustainability and affordability. Medicare Part D's share of annual spending on these drugs increased by 1,170 percent between 2012 and 2018, largely because the number of beneficiaries receiving them increased during this period.

Characteristics of Copayment Offsets for Prescription Drugs in the United States.

Importance: Despite ongoing debate regarding the high prices that patients pay for prescription drugs, to our knowledge, little is known regarding the use of coupons, vouchers, and other types of copayment "offsets" that reduce patients' out-of-pocket drug spending. Although offsets reduce patients' immediate cost burden, they may encourage the use of higher-cost products and diminish health insurers' ability to optimize pharmaceutical value. Objective: To examine the drugs most commonly covered by offsets, the percentage of out-of-pocket costs covered by offsets, and the characteristics of patients using offsets for retail pharmacy transactions in the United States in 2017 through 2019. Design, Setting, and Participants: A retrospective cohort analysis was conducted of a 5% nationally random sample of anonymized pharmacy claims from IQVIA's Formulary Impact Analyzer, which captures more than 60% of all US pharmacy transactions. This analysis focused on 631 249 individuals who used at least 1 offset between October 1, 2017, and September 30, 2019. Main Outcomes and Measures: Offset source, types of drugs covered by offsets, offset dollar value and percentage of out-of-pocket payment covered, and county characteristics of offset recipients. Results: The 631 249 individuals in the study (361 855 female participants [57.3%]; mean [SD] age, 45.7 [18.6] years) had approximately 33 million prescription fills, of which 12.8% had an offset used. Of these, 50.2% originated from a pharmaceutical manufacturer, 47.2% originated from a pharmacy or pharmacy benefit manager (PBM), and 2.6% originated from a state assistance program. A total of 80.0% of manufacturer-sponsored offsets were concentrated among 6.2% of unique products, and 79.9% of pharmacy-PBM offsets were concentrated among 4.9% of unique products. Most manufacturer offsets (88.2%) were for branded products, while most pharmacy-PBM offsets were for generic products (90.5%). The median manufacturer offset was $51.00, covering 87.1% of out-of-pocket costs; the median pharmacy-PBM offset was $16.30, covering 39.3% of out-of-pocket costs. There was no meaningful association between offset magnitude and county-level income, health insurance coverage, or race/ethnicity. Conclusions and Relevance: In this analysis of patient-level pharmacy claims from 2017 to 2019, approximately half of all offsets involved pharmacy-PBM contractual arrangements, and half were offered by manufacturers. All offsets were associated with a significant reduction in patients' out-of-pocket costs, were highly concentrated among a few drugs, and were generally not more generous among individuals in counties with lower income or larger Black or uninsured populations.

Assessing the Added Therapeutic Benefit of Ultra-Expensive Drugs.

OBJECTIVES: While the United States does not have a method for assessing the added therapeutic benefit of drugs, France, Canada, and Germany do. We examined the added therapeutic benefit of the most expensive drugs prescribed to Medicare Part D beneficiaries in the United States. METHODS: We identified ultra-expensive drugs with annual Medicare spending that exceeded $62 794 (United States GDP per capita in 2018) using Medicare Part D Prescription Drug Spending and Utilization Data. We used added therapeutic benefit ratings assessed by health technology assessment agencies in France, Canada, and Germany. RESULTS: We identified 122 ultra-expensive drugs in 2018. Sixty-five percent of these drugs (n = 79) were assessed by at least one of the countries. Based on these assessments, approximately 75% received a low added therapeutic benefit rating. CONCLUSIONS: Most ultra-expensive drugs prescribed in the United States and assessed by France, Canada, and Germany provide low added therapeutic benefit. Policy reforms in the United States could use added therapeutic benefit to inform coverage and pricing decisions for ultra-expensive drugs. Similar to Germany, one approach would be to allow the company to set a market price for a limited period of time before requiring a price reduction if the added therapeutic benefit is below a certain threshold. Another approach would be to identify when drug prices are substantially more expensive in the United States and conduct an added therapeutic benefit assessment and price review on these drugs.

Factors Associated With Manufacturer Drug Coupon Use at US Pharmacies.

Importance: Drug companies offer coupons to lower the out-of-pocket costs for prescription drugs, yet little is known about why they do so for some drugs but not for others. Objective: To examine whether the following factors are associated with manufacturer drug coupon use: (1) patient-cost characteristics (mean per-patient cost per drug, mean patient copay); (2) drug characteristics (generics availability or "later-in-class-entrant" drugs); (3) drug-class characteristics (in-class coupon use among competitors; in-class generic competition; in-class mean cost and copay). Design Setting and Participants: This was a retrospective cohort analysis of anonymized transactional pharmacy claims sourced from retail US pharmacies from October 2017 to September 2019, supplemented with information derived from Medi-Span, Red Book, and FDA.gov. Data were analyzed from September 2020 to February 2021. Main Outcomes and Measures: The primary outcome was availability of a manufacturer's coupon. The secondary outcome was the mean proportion of transactions in which a coupon was used for each product. Results: The sample of 2501 unique brand-name prescription drugs accounted for a total of 8 995 141 claims. Manufacturers offered a coupon for 1267 (50.7%) of these drugs. When the manufacturer offered a coupon, it was used in a mean (SD) 16.3% (20.3%) of the transactions. Within a drug class, higher mean total cost per patient was positively associated with the likelihood of coupon use (odds ratio [OR], 1.03 per 10% increase; 95% CI, 1.01-1.04), but higher mean patient copay was inversely associated (OR, 0.98; 95% CI, 0.97-0.99). For drug characteristics, single-source later-in-class-entrant products were associated with a greater likelihood of coupon use compared with first entrants and multisource brands (OR, 1.44; 95% CI, 1.09-1.89). The intensity of coupon use was associated with later-in-class-entrant products and the class mean per-patient cost (4.16-percentage-point increase; 95% CI, 1.20-7.13; 0.27 per 10% increase; 95% CI, 0.09-0.44). Drugs with a new in-class brand-name competitor had greater mean coupon use compared with drugs without a new competitor (10.2% of claims with a coupon vs 5.9%). Conclusions and Relevance: In this cohort study of transactional pharmacy claims, higher mean per-patient total cost within a class was significantly associated with the likelihood of coupon use, but not patient out-of-pocket cost. Manufacturers' coupons were more likely to be used for expensive later-in-class-entrant products facing within-class competition where coupon use was prevalent.

Critical Care Beds With Continuous Lateral Rotation Therapy to Prevent Ventilator-Associated Pneumonia and Hospital-Acquired Pressure Injury: A Cost-effectiveness Analysis.

OBJECTIVES: Mechanical ventilation increases the risk of hospital-acquired conditions (HACs) such as ventilator-associated pneumonia (VAP) and pressure injury (PrI). Beds with continuous lateral rotation therapy (CLRT) are shown to reduce HAC incidence, but the value of switching to CLRT beds is presently unknown. We compared the cost-effectiveness of CLRT beds with standard care in intensive care units. METHODS: A cost-effectiveness analysis from the healthcare sector and societal perspectives was conducted. A Markov model was constructed to predict health state transitions from time of ventilation through 28 days for the healthcare sector perspective and 1 year for the U.S. societal perspective. Value of information was calculated to determine whether parameter uncertainty warranted further research. RESULTS: Our analysis suggested that CLRT beds dominate standard care from both perspectives. From the healthcare sector perspective, expected cost for CLRT was U.S. $47,165/patient compared with a higher cost of U.S. $49,258/patient for standard care. The expected effectiveness of CLRT is 0.0418 quality-adjusted life years/patient compared with 0.0416 quality-adjusted life years/patient for standard care. Continuous lateral rotation therapy dominated standard care in approximately 93% of Monte Carlo simulations from both perspectives. Value of information analysis suggests that additional research is potentially cost-effective. CONCLUSIONS: Continuous lateral rotation therapy is highly cost-effective compared with standard care by preventing HACs that seriously harm patients in the intensive care unit.

Adverse Effects of the Medicare PSI-90 Hospital Penalty System on Revenue-Neutral Hospital-Acquired Conditions.

OBJECTIVE: Health systems are grappling with improving the quality and safety of health care. By setting clear expectations, there is an opportunity to configure care models to decrease the risk of adverse events and promote the quality of care. The US Centers for Medicare and Medicaid Services have used Patient Safety Indicator 90 (PSI90), a composite rate of hospital-acquired conditions (HACs), to adjust payments and score hospitals on quality since 2015. However, PSI90 may be associated with adverse prioritization for preventing some conditions over others.Our objective was to evaluate the time-dependent rates of HACs between 2013 and 2016 to assess the association of funding models on adverse events, particularly pressure injury. METHODS: We analyzed a retrospective observational cohort of patients hospitalized in US Academic Medical Centers observed by the Vizient CDB/RM pre-post PSI90 implementation. Changes in HAC component rates of PSI90 between 2013 and 2016 were measured longitudinally using mixed-effects negative binomial regression modeling. RESULTS: Regardless of whether the composite measure of patient outcomes was PSI90 or all HACs, in general, there was significant decrease after PSI90 was implemented, reflecting an association between PSI90 and CMS reimbursement policy. However, pressure injury rates increased by 29.4% (SE = 0.08; P < 0.05) during this time frame, the only HAC observed to increase related to PSI90. CONCLUSIONS: Patient safety in hospitals will only thoroughly improve when hospitals are fully incentivized to practice prevention of all HACs rather than work around the harms that result from failed prevention efforts.

Comparative Approaches to Drug Pricing.

The United States relies primarily on market forces to determine prices for drugs, whereas most other industrialized countries use a variety of approaches to determine drug prices. Branded drug companies have patents and market exclusivity periods in most industrialized countries. During this period, pharmaceutical companies are allowed to set their list price as high as they prefer in the United States owing to the absence of government price control mechanisms that exist in other countries. Insured patients often pay a percentage of the list price, and cost sharing creates some pressure to lower the list price. Pharmacy benefit managers negotiate with drug companies for lower prices by offering the drug company favorable formulary placement and fewer utilization controls. However, these approaches appear to be less effective, compared with other countries' approaches to containing branded drug prices, because prices are substantially higher in the United States. Other industrialized countries employ various forms of rate setting and price regulation, such as external reference pricing, therapeutic valuation, and health technology assessment to determine the appropriate price.

Financial Eligibility Criteria and Medication Coverage for Independent Charity Patient Assistance Programs.

Importance: Although independent charity patient assistance programs improve patient access to costly prescription drugs, recent federal investigations have raised questions about their potential to increase pharmaceutical spending and to violate the federal Anti-Kickback Statute. Little is known about the design of the programs, patient eligibility, or drug coverage. Objective: To examine the eligibility criteria of the independent charity patient assistance programs and the drugs covered by them. Design, Setting, and Participants: Descriptive cross-sectional study of the 6 largest independent charities offering patient assistance programs for patients including, but not limited to, Medicare beneficiaries in 2018. These charities offered 274 different disease-specific patient assistance programs. Drugs were identified for subgroup analysis that had any use reported on the Medicare Part D spending dashboard and any off-patent brand-name drugs that incurred more than $10 000 in Medicare spending per beneficiary in 2016. Exposures: Support by independent charity patient assistance programs. Main Outcomes and Measures: The primary outcomes were the characteristics of patient assistance programs, including assistance type, insurance coverage (vs uninsured), and income eligibility. The secondary outcomes were the cost of the drugs covered by the patient assistance programs and the coverage of expensive off-patent brand-name drugs vs substitutable generic drugs. Results: Among the 6 independent charity foundations included in the analysis, their total revenue in 2017 ranged from $24 million to $532 million, and expenditures on patient assistance programs ranged from $24 million to $353 million, representing on average, 86% of their revenue. Of the 274 patient assistance programs offered by these organizations, 168 (61%) provided only co-payment assistance, and the most common therapeutic area covered was cancer or cancer treatment-related symptoms (113 patient assistance programs; 41%). A total of 267 programs (97%) required insurance coverage as an eligibility criterion (ie, excluded uninsured patients). The most common income eligibility limit was 500% of the federal poverty level. The median annual cost of the drugs per beneficiary covered by the programs was $1157 (interquartile range, $247-$5609) compared with $367 (interquartile range, $100-$1500) for the noncovered drugs. Off-patent brand-name drugs (cost: >$10 000) were covered by a mean of 3.1 (SD, 2.0) patient assistance programs, whereas their generic equivalents were covered by a mean of 1.2 (SD, 1.0) patient assistance programs. Conclusions and Relevance: In 2018, among 274 patient assistance programs operated by the 6 independent charity foundations, the majority did not provide coverage for uninsured patients. Medications that were covered by the patient assistance programs were generally more expensive than those that were not covered.

Using External Reference Pricing In Medicare Part D To Reduce Drug Price Differentials With Other Countries.

Many countries use external reference pricing to help determine drug prices. However, external reference pricing has received little attention in the US-perhaps because the US is often the first adopter of drugs. External reference pricing could be used to set prices for drugs that were already established in the market. We compared the price differentials between the US and the UK, Japan, and Ontario (Canada) for single-source brand-name drugs that had been on the market for at least three years. We found that the prices averaged 3.2-4.1 times higher in the US after rebates were considered. The price differential for individual drugs varied from 1.3 to 70.1. The longer a drug remained on the market, the greater the differential. The estimated savings to Medicare Part D of adopting the average price of drugs in the reference countries was $72.9 billion in 2018. Medicare could use external reference pricing in Part D to improve affordability for patients.

Pharmaceutical Industry Support of US Patient Advocacy Organizations: An International Context.

OBJECTIVES: To examine whether the share of pharmaceutical industry funds allocated to patient advocacy organizations (PAOs) is disproportionately large in the United States relative to other industrialized countries and to compare pharmaceutical companies' disclosure practices across industrialized countries. METHODS: We examined funding of PAOs among the 10 largest pharmaceutical companies in 2016. We compared funding allocated to organizations across 8 large industrialized countries and pharmaceutical companies' disclosure practices in each country. RESULTS: Only 6 of the 10 largest pharmaceutical companies disclosed their financial transactions with PAOs in the United States. All 10 companies disclosed transactions in France, Germany, and the United Kingdom, with varying levels of disclosure in other countries. In 2016, the 6 companies that disclosed transactions in the United States allocated 74% of their patient advocacy funding ($88 million) in the United States. CONCLUSIONS: The disproportionate funding of US PAOs in the absence of any disclosure requirements suggests that the United States should consider adoption of regulatory actions to enhance the transparency of relationships between the pharmaceutical industry and PAOs, and to ensure the integrity of public health decision-making.

Development of a Model for the Prediction of Treatment Response of Uterine Leiomyomas after Uterine Artery Embolization.

Background: Uterine artery embolization (UAE) is one of the minimally-invasive alternatives to hysterectomy for treatment of uterine leiomyomas. There are various factors affecting the outcomes of UAE, but these have only been sporadically studied. Study Objective: To identify factors associated with the efficacy of UAE for the treatment of uterine leiomyoma, and to develop a model for the prediction of treatment response of uterine leiomyomas to UAE. Study design: A retrospective cohort study (Canadian Task Force Classification II-2) Patients: One hundred ninety-eight patients with symptomatic uterine leiomyomas. Intervention: UAE Measurements and Main Results: Among 198 leiomyoma patients who were treated with UAE, 104 who underwent pelvic magnetic resonance imaging (MRI) with diffusion-weighted imaging were selected for developing prediction model. Variables that were statistically significant from the univariate analysis were: location of leiomyoma, total number of lesions, sum of leiomyomas diameters, T2 signal intensity of largest leiomyoma, and T2 leiomyoma:muscle ratio. After a logistic regression analysis, leiomyoma location and T2 signal intensity of the largest leiomyoma were found to be statistically significant variables. Using intramural myomas defined as controls, submucosal leiomyomas showed a greater response to UAE with an odds ratio of 7.6904. The odds ratio of T2 signal intensity with an increase in signal intensity of 10 was 1.093. Using these two variables, we developed a prediction model. The AUC in the prediction model was 0.833, and the AUC in the validation set was 0.791. Conclusion: We identified that submucosal leiomyomas and those leiomyomas that show high signal intensity on T2-weighted imaging will exhibit a greater response to UAE. Prediction models are clinically helpful in selecting UAE as an appropriate treatment option for managing uterine leiomyoma.