Congress Approves Increase in Cancer Funding.

The federal spending bill enacted by the U.S. Congress in December for fiscal year 2021 totals $1.4 trillion, plus another $900 billion in emergency COVID-19 relief funding. The $1.4 trillion includes budget increases for the NIH, NCI, and FDA that help the agencies keep pace with inflation. Research advocates say more than $10 billion in emergency supplemental funds for the NIH is urgently needed to support medical research affected by the COVID-19 pandemic.

Include Payers in Your Product Development and Clinical Use Processes.

Wound/ulcer management scientists, researchers, manufacturers, professionals, and providers cannot assume that clearance or approval by the Food and Drug Administration (FDA) will guarantee reimbursement for medical devices they develop or wish to use in their practices. Even if a relative code and a published payment rate for the code exist, if the payers do not provide coverage for the technology, the devices may not be able to be sold and used in all settings throughout the continuum of care. Unfortunately, reimbursement (particularly coverage) is often an after-thought once FDA clearance or approval is achieved. This article describes two new Medicare coverage processes that should encourage all stakeholders to educate payers early and often why important medical devices should be covered for their patients with wounds/ulcers.

Cost-Effectiveness of the US Food and Drug Administration Added Sugar Labeling Policy for Improving Diet and Health.

BACKGROUND: Excess added sugars, particularly from sugar-sweetened beverages, are a major risk factor for cardiometabolic diseases including cardiovascular disease and type 2 diabetes mellitus. In 2016, the US Food and Drug Administration mandated the labeling of added sugar content on all packaged foods and beverages. Yet, the potential health impacts and cost-effectiveness of this policy remain unclear. METHODS: A validated microsimulation model (US IMPACT Food Policy model) was used to estimate cardiovascular disease and type 2 diabetes mellitus cases averted, quality-adjusted life-years, policy costs, health care, informal care, and lost productivity (health-related) savings and cost-effectiveness of 2 policy scenarios: (1) implementation of the US Food and Drug Administration added sugar labeling policy (sugar label), and (2) further accounting for corresponding industry reformulation (sugar label+reformulation). The model used nationally representative demographic and dietary intake data from the National Health and Nutrition Examination Survey, disease data from the Centers for Disease Control and Prevention Wonder Database, policy effects and diet-disease effects from meta-analyses, and policy and health-related costs from established sources. Probabilistic sensitivity analysis accounted for model parameter uncertainties and population heterogeneity. RESULTS: Between 2018 and 2037, the sugar label would prevent 354 400 cardiovascular disease (95% uncertainty interval, 167 000-673 500) and 599 300 (302 400-957 400) diabetes mellitus cases, gain 727 000 (401 300-1 138 000) quality-adjusted life-years, and save $31 billion (15.7-54.5) in net healthcare costs or $61.9 billion (33.1-103.3) societal costs (incorporating reduced lost productivity and informal care costs). For the sugar label+reformulation scenario, corresponding gains were 708 800 (369 200-1 252 000) cardiovascular disease cases, 1.2 million (0.7-1.7) diabetes mellitus cases, 1.3 million (0.8-1.9) quality-adjusted life-years, and $57.6 billion (31.9-92.4) and $113.2 billion (67.3-175.2), respectively. Both scenarios were estimated with >80% probability to be cost saving by 2023. CONCLUSIONS: Implementing the US Food and Drug Administration added sugar labeling policy could generate substantial health gains and cost savings for the US population.

U.S. Food and Drug Administration-Approved Poly (ADP-Ribose) Polymerase Inhibitor Maintenance Therapy for Recurrent Ovarian Cancer: A Cost-Effectiveness Analysis.

OBJECTIVE: We sought to determine whether use of a poly (ADP-ribose) polymerase inhibitor is cost effective for maintenance treatment of platinum-sensitive recurrent ovarian cancer. METHODS: A decision analysis model compared four maintenance strategies: 1) observation, 2) BRCA germline mutation testing and selective treatment of carriers (gBRCA only), 3) BRCA germline and tumor homologous recombination deficiency testing and selective treatment of either BRCA carriers or those with tumor HRD (gBRCA and HRD only), and 4) treat all with niraparib to progression (treat all). Costs were estimated in 2016 U.S. dollars. Incremental cost-effectiveness ratios were in dollars per progression-free quality-adjusted life-year (QALY). One-way sensitivity analyses tested multiple assumptions. RESULTS: Maintenance poly (ADP-ribose) polymerase inhibitor was costlier and more effective than observation. Mean costs and progression-free QALYs were $827 and 3.4 months for observation, $46,157 and 5.7 for a BRCA-only strategy, $109,368 and 8.5 for a gBRCA and homologous recombination deficiency-only strategy, and $169,127 and 8.8 for a treat-all strategy. gBRCA-only had an incremental cost-effectiveness ratio of $243,092/progression-free QALY compared with observation; other strategies did not approach cost effectiveness. Using the current U.S. Food and Drug Administration label for maintenance poly (ADP-ribose) polymerase inhibitor regardless of biomarker status, the third-party payer cost per month (28-day supply) would need to be reduced from approximately $14,700 to $3,600 to be considered cost effective compared with observation using a willingness to pay threshold of $100,000/progression-free QALY. CONCLUSION: Maintenance poly (ADP-ribose) polymerase inhibitor therapy for platinum-sensitive recurrent ovarian cancer is not cost effective. Treatment of patients with BRCA mutation alone or with homologous recombination deficiency-positive tumors are preferred strategies compared with a treat-all strategy. Lowering the cost may make selective niraparib maintenance therapy cost effective compared with observation.

Regulation and Innovation: Role of Regulatory Science in Facilitating Pharmaceutical Innovation.

Regulatory science is defined as science and research intended to inform decision making in a regulatory framework. This issue of Clinical Pharmacology & Therapeutics and the papers herein deal with the expansive topic of regulatory science and its role in fostering innovation and accelerating access to medicines. Regulatory health authorities, industry, and multiple stakeholders have a shared objective in advancing regulatory science to keep up with the ever-increasing pace of biomedical science.

Association Between Market Competition and Prices of Generic Topical Dermatology Drugs.

Importance: During the last decade, increases in drug prices for commonly prescribed dermatologic medications have outpaced the rate of inflation, national health care growth, and reimbursements. Among nondermatologic medications, studies have shown a role for robust generic market competition in reducing drug prices. The association between competition and the costs of topical dermatologic generic drugs has not been evaluated. Objective: To characterize the association between changes in drug price and the number of US Food and Drug Administration (FDA)-approved manufacturers among the most commonly used topical dermatologic generic products. Design, Setting, and Participants: This retrospective cost analysis of the most commonly prescribed topical dermatologic generic drugs used cumulative annual claims data from the Medicare Part D Prescriber Public User File to identify 597 dermatologist-prescribed drugs with more than 10 claims. The number of manufacturers and the price per unit were identified from the FDA Orange Book and the National Average Drug Acquisition Cost (NADAC) database, respectively, for 2013 through 2016. Drugs that were nondermatologic, were not topically administered, were missing NADAC data, were lacking a generic formulation, or had fewer than 400 claims were excluded. Main Outcomes and Measures: Primary outcomes included per-unit drug price and number of FDA-approved manufacturers. Pricing measures were adjusted for inflation and are reported in 2016 dollars. Results: The present analysis included 116 topical dermatologic generic formulations, representing 70.5% of the total Medicare Part D dermatologist-coded claims from 2015. Drug formulations with 1 to 2 manufacturers during the study period sustained a median percentage increase in price of 12.7%, whereas those with more than 6 manufacturers had a median percentage decrease in price of 20.5%. Formulations with 1 to 2 manufacturers had a 20.6%, 19.5%, and 33.2% higher percentage increase in price than those with 3 to 4 manufacturers, 5 to 6 manufacturers, and more than 6 manufacturers, respectively. There was a statistically significant inverse association between the percentage change in drug price and median number of manufacturers (Spearman correlation coefficient, -0.26; P = .005). Conclusions and Relevance: The negative association between the change in drug price and the median number of manufacturers of generic topical dermatologic drugs indicates a role for market competition in controlling the costs of generic drug prices within dermatology. These findings support policies that facilitate robust market competition among topical dermatologic generic drugs produced by a limited number of manufacturers.

The Partnership for Accelerating Cancer Therapies.

As a part of the Cancer Moonshot, the National Cancer Institute, part of the National Institutes of Health, the Foundation for National Institutes of Health, the US Food and Drug Administration, and 12 pharmaceutical companies have formed a 5-year, $220 million precompetitive public-private research collaboration called the Partnership for Accelerating Cancer Therapies. A systematic cross-sector effort to identify and develop robust, standardized biomarkers and related clinical data, Partnership for Accelerating Cancer Therapies will support the selection and testing of promising immunotherapies for the treatment of cancer, with the goal of bringing effective therapy to more patients.

US Adult Cigar Smoking Patterns, Purchasing Behaviors, and Reasons for Use According to Cigar Type: Findings From the Population Assessment of Tobacco and Health (PATH) Study, 2013-2014.

Introduction: The US cigar market is diverse, yet until recently most research studies and tobacco surveillance systems have not reported behavioral and related outcomes by cigar type. Methods: The 2013-2014 Population Assessment of Tobacco and Health Study collected data separately for filtered cigars (FCs), cigarillos, and traditional cigars, which were further distinguished as premium or nonpremium. Descriptive statistics for adult established current smokers of each cigar type and cigarettes were calculated for demographic characteristics, tobacco use patterns, purchasing behaviors and reasons for use. Adjusted prevalence ratios (APRs) using a marginal predictions approach with logistic regression assessed correlates of dual cigar and cigarette smoking. Results: Age, sex, race/ethnicity, education level, and poverty status of smokers varied according to cigar type. Daily cigar smoking prevalence and number of cigars smoked per day were higher for FCs (37.3%; median: 1.6 cigars/day, respectively), than all other cigar types (6.7%-25.3%, all p < .01; 0.1-0.4 cigars/day, all p < .01, respectively); daily smoking and cigars per day were similar for nonpremium cigars and cigarillos (p = .11; p = .33, respectively). Cigarette smoking was twice as common among smokers of nonpremium cigars, cigarillos, and FCs (58.0%-66.0%) than among premium cigars (29.9%). Among current cigar smokers, FC smokers (APR = 1.23, 95% confidence interval [CI] = 1.09-1.39), other tobacco product users (APR = 1.27, 95% CI = 1.15-1.41), and those with a GED/high school diploma or less (APR = 1.20, 95% CI = 1.09-1.33) were more likely to also smoke cigarettes. Conclusion: User characteristics, cigar smoking patterns, and dual smoking with cigarettes varied by cigar type highlighting the importance of adequately describing the cigar type studied and, where appropriate, differentiating results by cigar type. Implications: Despite the diversity of the cigar market place, historically many research studies and tobacco surveillance systems have treated cigars as a single product type. This study describes similarities and differences in the user characteristics, tobacco use patterns, and purchasing behaviors of premium, nonpremium, cigarillo, and filtered cigar smokers. To enhance tobacco regulatory science, sufficient descriptions of the cigar type(s) studied and, where appropriate, differentiation of the particular cigar type(s) studied should be undertaken to improve the interpretation of study findings, understanding of cigar use patterns and related behaviors and future approaches to reducing cigar-attributable morbidity and mortality.

Comparison of Sampling Strategies for Tobacco Retailer Inspections to Maximize Coverage in Vulnerable Areas and Minimize Cost.

Introduction: In the United States, tens of thousands of inspections of tobacco retailers are conducted each year. Various sampling choices can reduce travel costs, emphasize enforcement in areas with greater noncompliance, and allow for comparability between states and over time. We sought to develop a model sampling strategy for state tobacco retailer inspections. Methods: Using a 2014 list of 10,161 North Carolina tobacco retailers, we compared results from simple random sampling; stratified, clustered at the ZIP code sampling; and, stratified, clustered at the census tract sampling. We conducted a simulation of repeated sampling and compared approaches for their comparative level of precision, coverage, and retailer dispersion. Results: While maintaining an adequate design effect and statistical precision appropriate for a public health enforcement program, both stratified, clustered ZIP- and tract-based approaches were feasible. Both ZIP and tract strategies yielded improvements over simple random sampling, with relative improvements, respectively, of average distance between retailers (reduced 5.0% and 1.9%), percent Black residents in sampled neighborhoods (increased 17.2% and 32.6%), percent Hispanic residents in sampled neighborhoods (reduced 2.2% and increased 18.3%), percentage of sampled retailers located near schools (increased 61.3% and 37.5%), and poverty rate in sampled neighborhoods (increased 14.0% and 38.2%). Conclusions: States can make retailer inspections more efficient and targeted with stratified, clustered sampling. Use of statistically appropriate sampling strategies like these should be considered by states, researchers, and the Food and Drug Administration to improve program impact and allow for comparisons over time and across states. Implications: The authors present a model tobacco retailer sampling strategy for promoting compliance and reducing costs that could be used by US states and the Food and Drug Administration (FDA). The design is feasible to implement in North Carolina. Use of the sampling design would help document the impact of FDA's compliance and enforcement program, save money, and emphasize inspections in areas where they are needed most. FDA should consider requiring probability-based sampling in their inspections contracts with states and private contractors.