Premarket Notifications and Patents for Breast Pumps Before and After the ACA.

This study investigates whether ACA policies to increase access to breast pumps and lactation care were associated with innovation in the market for breast pumps.

Key cost drivers of pharmaceutical clinical trials in the United States.

BACKGROUND: The increasing cost of clinical research has significant implications for public health, as it affects drug companies' willingness to undertake clinical trials, which in turn limits patient access to novel treatments. Thus, gaining a better understanding of the key cost drivers of clinical research in the United States is important. PURPOSE: The study which is based on a report prepared by Eastern Research Group, Inc., for the US Department of Health and Human Services, examined different factors, such as therapeutic area, patient recruitment, administrative staff, and clinical procedure expenditures, and their contribution to pharmaceutical clinical trial costs in the United States by clinical trial phase. METHODS: The study used aggregate data from three proprietary databases on clinical trial costs provided by Medidata Solutions. We evaluated per-study costs across therapeutic areas by aggregating detailed (per patient and per site) cost information. We also compared average expenditures on cost drivers with the use of weighted mean and standard deviation statistics. RESULTS: Therapeutic area was an important determinant of clinical trial costs by phase. The average cost of a Phase 1 study conducted at a US site ranged from US$1.4 million (pain and anesthesia) to US$6.6 million (immunomodulation), including estimated site overhead and monitoring costs of the sponsoring organization. A Phase 2 study cost from US$7.0 million (cardiovascular) to US$19.6 million (hematology), whereas a Phase 3 study cost ranged from US$11.5 million (dermatology) to US$52.9 (pain and anesthesia) on average. Across all study phases and excluding estimated site overhead costs and costs for sponsors to monitor the study, the top three cost drivers of clinical trial expenditures were clinical procedure costs (15%-22% of total), administrative staff costs (11%-29% of total), and site monitoring costs (9%-14% of total). LIMITATIONS: The data were from 2004 through 2012 and were not adjusted for inflation. Additionally, the databases used represented a convenience, that is, non-probability, sample and did not allow for statistically valid estimates of cost drivers. Finally, the data were from trials funded by the global pharmaceutical and biotechnology industry only. Hence, our study findings are limited to that segment. CONCLUSION: Therapeutic area being studied as well as number and types of clinical procedures involved were the key drivers of direct costs in Phase 1 through Phase 3 studies. Research shows that strategies exist for reducing the price tag of some of these major direct cost components. Therefore, to increase clinical trial efficiency and reduce costs, gaining a better understanding of the key direct cost drivers is an important step.

Costs of Drug Development and Research and Development Intensity in the US, 2000-2018.

Importance: Understanding the cost of drug development can help inform the development of policies to reduce costs, encourage innovation, and improve patient access to drugs. Objective: To estimate the cost of drug development by therapeutic class and trends in pharmaceutical research and development (R&D) intensity over time. Design, Setting, and Participants: In this economic evaluation study, an analytical model of drug development constructed using public and proprietary sources that collectively cover data from 2000 to 2018 was used to estimate the cost of bringing a drug to market, overall and for specific therapeutic classes. The analysis for the study was completed in October 2020. Main Outcomes and Measures: Three measures of development cost from nonclinical through postmarketing stages were estimated: mean out-of-pocket cost or cash outlay, mean expected cost, and mean expected capitalized cost. Pharmaceutical R&D intensity, defined as the ratio of R&D spending to total sales, from 2008 to 2019, based on the time frame for available data, was also analyzed. Results: The estimated mean cost of developing a new drug was approximately $172.7 million (2018 dollars) (range, $72.5 million for genitourinary to $297.2 million for pain and anesthesia), inclusive of postmarketing studies. The cost increased to $515.8 million when cost of failures was included. When the costs of failures and capital were included, the mean expected capitalized cost of drug development increased to $879.3 million (range, $378.7 million for anti-infectives to $1756.2 million for pain and anesthesia); results varied widely by therapeutic class. The pharmaceutical industry as a whole experienced a decline of 15.6% in sales but increased R&D intensity from 11.9% to 17.7% from 2008 to 2019. By contrast, R&D intensity of large pharmaceutical companies increased from 16.6% to 19.3%, whereas sales increased by 10.0% (from $380.0 to $418.0 billion) over the same 2008 to 2019 period, even though the cost of drug development remained relatively stable or may have even decreased. Conclusions and Relevance: In this economic evaluation of new drug development costs, even though the cost of drug development appears to have remained stable, R&D intensity of large pharmaceutical companies remained relatively unchanged, despite substantial growth in revenues during this period. These findings can inform the design of drug-related policies and their potential impacts on innovation and competition.

Assessing the state of antibacterial drug discovery through patent analysis.

Patent filings suggest increasing intensity of antibacterial drug discovery in recent years, but the share of patents published by commercial companies has declined.

Benefit-Cost Analysis of the HHS COVID-19 Campaign: April 2021-March 2022.

INTRODUCTION: This study estimated the benefits and costs of the U.S. Department of Health and Human Services' We Can Do This COVID-19 public education campaign (the Campaign) and associated vaccination-related impacts. METHODS: Weekly media market and national Campaign expenditures were used to estimate weekly first-dose vaccinations that would not have occurred absent the Campaign, weekly Campaign-attributed complete vaccinations, and corresponding COVID-19 cases, hospitalizations, and deaths averted. Benefits were valued using estimated morbidity and mortality reductions and associated values of a statistical life and a statistical case. Costs were estimated using Campaign paid media expenditures and corresponding vaccination costs. The net Campaign and vaccination benefit and return on investment were calculated. Analyses were conducted from 2022 to 2024. RESULTS: Between April 2021 and March 2022, an estimated 55.9 million doses of COVID-19 vaccines would not have been administered absent the Campaign. Campaign-attributed vaccinations resulted in 2,576,133 fewer mild COVID-19 cases, 243,979 fewer nonfatal COVID-19 hospitalizations, and 51,675 lives saved from COVID-19. The total Campaign benefit was $740.2 billion, and Campaign and vaccination costs totaled $8.3 billion, with net benefits of approximately $732.0 billion. For every $1 spent, the Campaign and corresponding vaccination costs resulted in benefits of approximately $89.54. CONCLUSIONS: The We Can Do This COVID-19 public education campaign saved more than 50,000 lives and prevented hundreds of thousands of hospitalizations and millions of COVID-19 cases, representing hundreds of billions of dollars in benefits in less than one year. Findings suggest that public education campaigns are a cost-effective approach to reducing COVID-19 morbidity and mortality.

Estimated Cost of Developing a Therapeutic Complex Medical Device in the US.

Importance: The US medical device market is the world's largest, but estimates of the cost to bring a medical device to market are not available to help inform policy making and regulatory efforts to enhance device safety and innovation. Objective: To estimate the mean expected capitalized cost of developing a novel therapeutic complex medical device. Design, Setting, and Participants: In this economic evaluation, an analytical model of novel therapeutic complex medical device development using data from public and proprietary sources with coverage from 2000 through 2018 was used to estimate the cost, duration, and phase transition success probability associated with each stage of development. Data analysis was completed in September 2021. Exposures: Conduct of nonclinical and clinical studies; payment of FDA user fees for novel therapeutic complex medical devices. Main Outcomes and Measures: Mean development cost (in 2018 US dollars) incurred by developers for an FDA-approved novel therapeutic complex medical device, accounting for failures and cost of capital. Results: In this economic analysis, the mean development cost for a novel therapeutic complex medical device was $54 million (95% CI, $25 million-$200 million) excluding any postapproval studies that might be required. After accounting for the cost of failed studies and cost of capital, the mean capitalized cost of bringing a novel therapeutic complex medical device to the US market was $522 million (95% CI, $205 million-$3382 million). The key factors associated with this cost were the phase transition probabilities: 46.9% for nonclinical to feasibility study, 48.0% for feasibility to pivotal study, 75.7% pivotal study to FDA premarket approval submission, and 80.5% for FDA premarket approval submission to approval. The nonclinical development stage constituted the largest portion of overall cost at 85.0% with the FDA review stage with the highest phase transition probability accounting for only a small fraction at 0.5%. Conclusions and Relevance: In this economic evaluation study, the cost of therapeutic complex medical device development from proof of concept through postapproval stages was assessed accounting for the cost of failures and the cost of capital. Existing estimates did not account for all stages of development, capitalization, or failure costs, which this study suggests were substantial.

Characteristics Of Medical Device Shortages In The US, 2006-20.

Shortages of critical medical devices during the COVID-19 pandemic exposed vulnerabilities in device markets. From 2010 to 2019 there were approximately five shortages annually, but that number increased fourfold in the first half of 2020. Although most 2020 shortages were triggered by the pandemic, pre-2020 shortages stemmed from regulatory and enforcement actions related to product quality and manufacturing.

The impact of recent power morcellator risk information on inpatient surgery and patient outcomes.

Aim: We examine the impact of the new risk information about a surgical device on surgery and patient outcomes for hysterectomy in the inpatient setting. Methods: We utilize a difference-in-differences approach to assess the impact of new risk information on patient outcomes in the inpatient setting between 2009 and 2014. The inpatient data come from a nationally representative sample of hospitalizations in the USA. We use the likelihood of laparoscopic surgery, measures of resource use and surgical complications as outcome variables. Results: We estimate a three-percentage point decrease in the likelihood of receiving laparoscopic hysterectomy, a one-percentage point increase in the likelihood of experiencing a surgical complication and no impact on resource use, relative to pre-existing means. Conclusion: Our findings show that there was movement away from laparoscopic surgery in the months following the dissemination of new risk information. These changes had limited effect on patient outcomes.

Fracking and risky behaviors: Evidence from Pennsylvania.

In the past decade, the technological developments in the oil and natural gas extraction industry made the extraction of shale gas economically feasible and prompted local economic booms across the US. Anecdotal evidence suggests that areas with unconventional gas development experience a disproportionate increase in the young male population who are more likely to be involved in risk-taking behavior. Moreover, the sudden income gains or demographic shifts might increase the demand for various goods and services, including entertainment and illegal activities provided by the adult entertainment industry. We investigate the relationship between unconventional gas development and a variety of risk-taking outcomes such as sexually transmitted infections, and prostitution-related arrests. Our identification strategy exploits the variation in shale gas or unconventional well drilling across time and counties in conjecture with a number of datasets that allow us to investigate the potential mechanisms. Our findings indicate that Pennsylvania counties with fracking activities have higher rates of gonorrhea and chlamydia infections (7.8% and 2.6%, respectively), as well as higher prostitution related arrests (19.7%). We posit that changes in the labor market and associated impacts to income or composition of workers may play a role in the estimated effects, but we do not find evidence in support of these hypotheses.

Health shocks and their long-lasting impact on health behaviors: Evidence from the 2009 H1N1 pandemic in Mexico.

Worldwide, the leading causes of death could be avoided with health behaviors that are low-cost but also difficult to adopt. We show that exogenous health shocks could facilitate the adoption of these behaviors and provide long-lasting effects on health outcomes. Specifically, we exploit the spatial and temporal variation of the 2009 H1N1 influenza pandemic in Mexico and show that areas with a higher incidence of H1N1 experienced larger reductions in diarrhea-related cases among young children. These reductions continue even three years after the shock ended. Health improvements and evidence of information seeking via Google searches were consistent with changes in hand washing behaviors. Several robustness checks validate our findings and mechanism.

Current and Projected Characteristics and Unique Health Care Needs of the Patient Population Served by the Department of Veterans Affairs.

The Veterans Access, Choice, and Accountability Act of 2014 addressed the need for access to timely, high-quality health care for veterans. Section 201 of the legislation called for an independent assessment of various aspects of veterans' health care. The RAND Corporation was tasked with an assessment of the current and projected demographics and health care needs of patients served by the Department of Veterans Affairs (VA). The number of U.S. veterans will continue to decline over the next decade, and the demographic mix and geographic locations of these veterans will change. While the number of veterans using VA health care has increased over time, demand will level off in the coming years. Veterans have more favorable economic circumstances than non-veterans, but they are also older and more likely to be diagnosed with many health conditions. Not all veterans are eligible for or use VA health care. Whether and to what extent an eligible veteran uses VA health care depends on a number of factors, including access to other sources of health care. Veterans who rely on VA health care are older and less healthy than veterans who do not, and the prevalence of costly conditions in this population is projected to increase. Potential changes to VA policy and the context for VA health care, including effects of the Affordable Care Act, could affect demand. Analysis of a range of data sources provided insight into how the veteran population is likely to change in the next decade.