Potential policy reforms to strengthen the accelerated approval pathway

Author(s):  
Anna Kaltenboeck ◽  
Amanda Mehlman ◽  
Steven D Pearson

The accelerated approval pathway for new drugs in the United States is often praised but faces growing criticism of whether it is finding the appropriate balance between uncertainty, access and cost. To support efforts to strengthen the pathway, this paper provides an analysis of key concerns and the advantages and disadvantages of ten potential policy reforms – those achievable through the US FDA action alone, and those that would require a combination of government, payer and life science industry actions. Accelerated approval sits at the heart of many of the controversies regarding drug approvals and pricing, and this analysis provides perspectives on how best to strengthen the pathway within the broader landscape of an innovative US healthcare system.

Author(s):  
Elina Reponen ◽  
Thomas G Rundall ◽  
Stephen M Shortell ◽  
Janet C Blodgett ◽  
Ritva Jokela ◽  
...  

Abstract Background Healthcare organizations around the world are striving to achieve transformational performance improvement, often through adopting process improvement methodologies such as Lean management. Indeed, Lean management has been implemented in hospitals in many countries. But despite a shared methodology and the potential benefit of benchmarking lean implementation and its effects on hospital performance, cross-national Lean benchmarking is rare. Healthcare organisations in different countries operate in very different contexts, including different healthcare system models, and these differences may be perceived as limiting the ability of improvers to benchmark Lean implementation and related organisational performance. However, there is no empirical research available on the international relevance and applicability of Lean implementation and hospital performance measures. To begin to understand the opportunities and limitations related to cross-national benchmarking of Lean in hospitals, we conducted a cross-national case study of the relevance and applicability of measures of Lean implementation in hospitals and hospital performance. Methods We report an exploratory case study of the relevance of Lean implementation measures and the applicability of hospital performance measures using quantitative comparisons of data from Hospital District of XX XX University Hospital in Finland and a sample of 75 large academic hospitals in the United States. Results The relevance of Lean-related measures was high across the two countries: almost 90% of the items developed for a US survey were relevant and available from XX. A majority of the US-based measures for financial performance (66.7%), service provision/utilisation (100.0%), and service provision/care processes (60.0%) were available from XX. Differences in patient satisfaction measures prevented comparisons between XX and the US. Of 18 clinical outcome measures, only four (22%) were not comparable. Clinical outcome measures were less affected by the differences in healthcare system models than measures related to service provision and financial performance. Conclusions Lean implementation measures are highly relevant in healthcare organisations operating in the United States and Finland, as is the applicability of a variety of performance improvement measures. Cross-national benchmarking in Lean healthcare is feasible, but a careful assessment of contextual factors, including the healthcare system model, and their impact on the applicability and relevance of chosen benchmarking measures is necessary. The differences between the US and Finnish healthcare system models is most clearly reflected in financial performance measures and care process measures.


2012 ◽  
Vol 108 (08) ◽  
pp. 291-302 ◽  
Author(s):  
Matthew E. Borrego ◽  
Alex L. Woersching ◽  
Robert Federici ◽  
Ross Downey ◽  
Jay Tiongson ◽  
...  

SummaryHealthcare reform is upon the United States (US) healthcare system. Prioritisation of preventative efforts will guide necessary transitions within the US healthcare system. While annual deep-vein thrombosis (DVT) costs have recently been defined at the US national level, annual pulmonary embolism (PE) and venous thromboembolism (VTE) costs have not yet been defined. A decision tree and cost model were developed to estimate US health care costs for total PE, total hospital-acquired PE, and total hospital-acquired “preventable” PE. The previously published DVT cost model was modified, updated and combined with the PE cost model to elucidate the same three categories of costs for VTE. Direct and indirect costs were also delineated. For VTE in the base model, annual cost ranges in 2011 US dollars for total, hospital-acquired, and hospital-acquired “preventable” costs and were $13.5-$27.2, $9.0-$18.2, and $4.5-$14.2 billion, respectively. The first sensitivity analysis, with higher incidence rates and costs, demonstrated annual US total, hospital-acquired, and hospital-acquired “preventable” VTE costs ranging from $32.1-$69.3, $23.7-$51.5, and $11.9-$39.3 billion, respectively. The second sensitivity analysis with long-term attack rates (LTAR) for recurrent events and post-thrombotic syndrome and chronic pulmonary thromboembolic hypertension demonstrated annual US total, hospital-acquired, and hospital-acquired “preventable” VTE costs ranging from $15.4-$34.4, $10.3-$25.4, and $5.1-$19.1 billion, respectively. PE costs comprised a majority of the VTE costs. Prioritisation of effective VTE preventative strategies will reduce significant costs, morbidity and mortality within the US healthcare system. The cost models may be utilised to estimate other countries’ costs or VTE-specific disease states.


2020 ◽  
Vol 9 (6) ◽  
pp. 12
Author(s):  
Kim D. ◽  
O’Connor S.J. ◽  
Williams J.H. ◽  
Opoku-Agyeman W. ◽  
Chu D.I. ◽  
...  

Health literacy has become an important topic to discuss in the US healthcare system. Almost nine out of ten adults in the United States lack the knowledge and skills required to manage their health and prevent disease. While studies have shown the importance of health literacy, not may have explored its’ history and conceptual roots. Hence, the purpose of this study is to address the gap in the literature by reviewing studies related to the past, present, and the effect of health literacy. The results have shown that inadequate health literacy does affect patients’ general health and performance of the US healthcare system.


2021 ◽  
pp. 232020682110301
Author(s):  
Colleen Watson ◽  
Laura Rhein ◽  
Stephanie M. Fanelli

Aim: To compare following the Cuban Revolution, Cuba’s economy and civil society was transformed by the initiation of a program of nationalization and political consolidation. The Cuban government operates a national health system and assumes fiscal and administrative responsibility for the healthcare of its citizens. Other industrialized nations continue to surpass the US in health-related outcomes indicating areas of improvement in its healthcare system. Assessing the successes and failures as well as the advantages and disadvantages of other countries’ healthcare systems may be instrumental in the development of modifications to the organization and delivery system of healthcare in the US. This paper aims to report the information attained from previous literature as well as from first-hand observations from a public health trip to Cuba in order to compare the healthcare systems in Cuba and the United States. Materials and Methods: A group of New York University College of Dentistry faculty and students traveled to Cuba in April 2019 for professional research and professional meetings (CFR 515.564). While in Cuba, the researchers took written notes of the lecture-based material and conversations. Upon return to the United States, published literature was searched for the collection of any additional data and all qualitative data and quantitative data was compiled and organized. Since 1959, Cuba has made continuous adjustments and improvements to its universal, free and accessible healthcare system. Results: There have been notable improvements to the country’s public health status, such as the implementation of an immunization program and subsequent eradication of communicable diseases, such as polio and rubella. Additionally, the implementation of the National Program on Dentistry guarantees dental care to all Cuban children under the age of 19. Today, the Cuban National Health System (NHS) initiatives have evolved to combat the novel coronavirus (COVID-19) pandemic. Conclusion: Recognizing the advantages as well as the disadvantages of the Cuba’s National Health System (NHS) would be useful for future policymakers in the United States. Cuban approaches to health could be tailored to the United States environment to improve healthcare effectiveness and population health status in the future.


2020 ◽  
Vol 18 (3) ◽  
pp. 2160
Author(s):  
Teresa M. Salgado ◽  
Meagen M. Rosenthal ◽  
Antoinette B. Coe ◽  
Tana N. Kaefer ◽  
Dave L. Dixon ◽  
...  

The United States (US) has a complex healthcare system with a mix of public, private, nonprofit, and for-profit insurers, healthcare institutions and organizations, and providers. Unlike other developed countries, there is not a single payer healthcare system or a national pharmaceutical benefits scheme/plan. Despite spending over USD 10,000 per capita in healthcare, the US is among the worst performers compared to other developed countries in outcomes including life expectancy at birth, infant mortality, safety during childbirth, and unmanaged chronic conditions (e.g., asthma, diabetes). Primary care is delivered by physicians and advanced practice providers (i.e., nurse practitioners and physician assistants) in a variety of settings including large health systems, federally qualified health centers or free clinics that provide care to the underserved, or specific facilities for veterans or American Indian and Alaska native peoples. Since 2010, primary care delivery has shifted toward providing patient-centered, coordinated, comprehensive care focused on providing proactive, rather than reactive, population health management, and on the quality, versus volume, of care. Community pharmacy comprises a mix of independently owned, chain, supermarket and mass merchant pharmacies. Community pharmacies provide services such as immunizations, medication therapy management, medication packaging, medication synchronization, point-of-care testing and, in specific states where legislation has been passed, hormonal contraception, opioid reversal agents, and smoking cessation services. There has been criticism regarding the lack of standard terminology for services such as medication synchronization and medication therapy management, their components and how they should be provided, which hampers comparability across studies. One of the main challenges for pharmacists in the US is the lack of provider status at the federal level. This means that pharmacists are not allowed to use existing fee-for-service health insurance billing codes to receive reimbursement for non-dispensing services. In addition, despite there being regulatory infrastructure in multiple states, the extent of service implementation is either low or unknown. Research found that pharmacists face numerous barriers when providing some of these services. State fragmentation and the lack of a single pharmacy organization and vision for the profession are additional challenges.


1999 ◽  
Vol 07 (04) ◽  
pp. 313-330
Author(s):  
CAROL A. FORBES ◽  
NIGEL M. HEALEY

This paper explores the phenomenon of barter exchanges, which have become widespread in the United States and provide small retailers and service companies with a means of trading goods or services directly with each other. Using a case study approach in the United States, the papers examines the mechanics of barter exchanges and the advantages and disadvantages to its users in the small business sector. It then considers the spread of barter exchanges to Europe and, using a questionnaire survey, identifies a range of obstacles to the successful transfer of this US model.


2018 ◽  
Vol 14 (1) ◽  
pp. e34-e41 ◽  
Author(s):  
Atsushi Hyogo ◽  
Masayuki Kaneko ◽  
Mamoru Narukawa

Purpose: With the recent use of expedited drug development and approval programs for several oncology products in the United States, the importance of postmarketing plans to confirm clinical benefits and safety is increasing. To discuss postmarketing requirements (PMRs) and postmarketing commitments (PMCs) required for oncology products approved in the United States, we investigated the factors that influenced the US Food and Drug Administration (FDA) decisions for PMR/PMCs during FDA review. Methods: Characteristics of new drug approvals and PMR/PMCs for oncology products (new molecular entities and new therapeutic biologic products) in the United States between 2008 and 2015 were analyzed. Results: Of the 58 oncology products analyzed, PMR/PMCs were required for 54 products. The proportion of approvals that required confirmatory PMR/PMCs was 100% for accelerated approval (AA) and was 39% for regular approval (RA). Median development times for AA and RA were 7.41 and 7.50 years, respectively. Randomization, number of patients, and end point in pivotal studies were identified as key potential factors that influenced the decision to require PMR/PMCs for both confirmatory and clinical safety studies. Conclusion: Robustness of the pivotal study design was identified as one of the key factors for the decision by the FDA to require PMR/PMCs—in particular, significant PMR/PMCs, such as those for confirmatory studies. That is, the FDA approved products with surrogate markers and smaller studies but required PMR/PMCs to fully prove the risk-benefit profile in the postmarketing period.


Esculapio ◽  
2021 ◽  
Vol 17 (1) ◽  
pp. 3-4
Author(s):  
Sonikpreet Aulakh ◽  
Asher Chanan Khan

COVID-19 pandemic has exposed vulnerabilities all across the global healthcare systems including those within the United States. A systematic evaluation of these soft spots has been crucial in order to reengineer the healthcare system for enhanced competences and superior quality of care. One area that has been undoubtedly affected is the diagnosis and management of neoplastic diseases. The healthcare system in the US witnessed an instantaneous implementation of a “social distancing” strategy, which was implemented in an effort to flatten the infectivity “curve”. This required an urgent modification in the general administration of healthcare delivery, independent of COVID-19 infection status of a patient. For the non-COVID patients, it meant a shift from in-person to a virtual administration platform.''(Royce et al., 2020) Neither the healthcare providers, nor the patients, or the hospital management were adequately prepared for this sudden transition. Various healthcare services offered through these healthcare systems were required to be triaged based upon patients' assessment of needs into either emergent, urgent or routine/non- urgent. Patients seeking services that fell in the non- urgent/routine clinical visits were encouraged to stay home until the pandemic simmered down/resolved. This strategy was erroneously predicated on a rather short anticipated duration of the pandemic. As expected, cancer screening visits were deemed non- urgent and thus most healthcare facilities in and outside the US suspended these services, inadvertently compromising the timely diagnosis of neoplastic disorders.


Sign in / Sign up

Export Citation Format

Share Document