scholarly journals Crowdfunding for unmet medical needs: the case of CAR-T and Gofundme

2019 ◽  
Vol 29 (Supplement_4) ◽  
Author(s):  
F Toscano ◽  
D Golinelli ◽  
S Guicciardi ◽  
E O’Donnell ◽  
S Rossi ◽  
...  
Keyword(s):  
Scientific Evidence ◽  
The United States ◽  
Innovative Therapy ◽  
Medical Needs ◽  
Car T ◽  
Good Expression ◽  
Experimental Treatments ◽  
The Uk ◽  
New Treatments ◽  
Unmet Medical Needs

Abstract Background Crowdfunding (CF) is the practice of financing a project by raising money from a large number of people. Many patients use online CF platforms to fund medical needs, whether unmet by their healthcare service - therefore to be sought elsewhere -, or when unable to afford them. CAR-T (Chimeric antigen receptor T-cells) is an innovative therapy approved to treat several types of lymphoma and with potential to treat many other cancers. CAR-T is available in most countries through clinical trials only and its diffusion is limited by high costs. Gofundme is a CF platform ’leader in online medical fundraising’. The objective of this study is to shed some light on the phenomenon of CF for CAR-T therapy, describing the campaigns started on Gofundme. Methods We extracted data from CAR-T campaigns started in Europe and publicly available on Gofundme until March 2019. Data related to each campaign has been analyzed and reported through descriptive analyses. Results Since 2017, 26 CAR-T treatment campaigns have been started. Due to the privacy policy of the platform, we were able to retrieve data from only 12 of them; 3 campaigns were closed, thus not disclosing enough data to be included. Nine campaigns were studied: 4 from Italy, 4 from the UK, 1 from Spain. The United States were the main destination. The median campaign goal was 180k€. The number of campaign shares on social media and the percentage of goal raised seemed to be linked. Conclusions CF is unquestionably a good expression of the Internet: it channels empathy towards important causes, providing tangible help. However, when applied to healthcare - especially to experimental treatments -, several questions arise. Patients affected by treatment resistant cancers may be prone trying new treatments to keep the hope alive, even when indication is missing or scientific evidence is lacking. Policymakers should monitor healthcare related CF campaigns, both for equity and safety reasons. Key messages Policymaker should monitor healthcare related CF campaigns, both for equity and safety reasons. Healthcare related crowdfunding campaigns could be interpreted as an indicator of unmet medical needs.

scholarly journals Medical Affairs in Pharmaceutical Companies and Related Pharmaceutical Regulations in Japan

2021 ◽  
Vol 8 ◽  
Author(s):  
Hideki Maeda
Keyword(s):  
Clinical Trials ◽  
Scientific Evidence ◽  
The United States ◽  
Current Status ◽  
Pharmaceutical Companies ◽  
Medical Needs ◽  
Long Time ◽  
Post Marketing ◽  
History Of ◽  
Scientific Value

Medical affairs has received a lot of attention in recent years in Japan, but it is also often misunderstood or poorly understood in the healthcare industry in Japan. In the United States, the function of medical affairs has been established for a long time, whereas its history in Japan is relatively short. Many scandals in clinical trials occurred with inappropriate relationship between medical doctors and the sales departments of pharmaceutical companies from 2012. These incidents undermined confidence in clinical trials in Japan and triggered the enforced separation of sales departments from the conduct of post-marketing clinical trials and evidence generation. There have been growing compliance issues identified in marketing and sales practices, and off-label promotion is also becoming an issue in Japan. These issues resulted in the establishment of independent medical affairs departments from sales departments in pharmaceutical companies operating in Japan. Due to the short history of medical affairs in Japan, the roles and responsibilities vary between companies in Japan. Medical affairs departments aim to fulfill unmet medical needs through the generation of scientific evidence and to deliver scientific value to key stakeholders and patients. People working in medical affairs need to engage in scientific exchange activities with key opinion leaders independent of sales departments. Through these activities, medical affairs ensures that patients receive optimal medical care. Medical affairs in Japan is still developing, and its roles, responsibilities, and functions are improving. This article covers the history of medical affairs in Japan and the current status and future perspectives of medical affairs in Japan.

scholarly journals Linking Obesity with Colorectal Cancer: Epidemiology and Mechanistic Insights

Cancers ◽  
2020 ◽  
Vol 12 (6) ◽  
pp. 1408 ◽  
Author(s):  
Pengfei Ye ◽  
Yue Xi ◽  
Zhiying Huang ◽  
Pengfei Xu
Keyword(s):  
Colorectal Cancer ◽  
Molecular Mechanisms ◽  
The United States ◽  
Cancer Epidemiology ◽  
Medical Needs ◽  
The Third ◽  
Common Cancer ◽  
The World ◽  
Treatment Of Obesity ◽  
Unmet Medical Needs

The incidence of obesity and colorectal cancer (CRC) has risen rapidly in recent decades. More than 650 million obese and 2 billion overweight individuals are currently living in the world. CRC is the third most common cancer. Obesity is regarded as one of the key environmental risk factors for the pathogenesis of CRC. In the present review, we mainly focus on the epidemiology of obesity and CRC in the world, the United States, and China. We also summarize the molecular mechanisms linking obesity to CRC in different aspects, including nutriology, adipokines and hormones, inflammation, gut microbiota, and bile acids. The unmet medical needs for obesity-related CRC are still remarkable. Understanding the molecular basis of these associations will help develop novel therapeutic targets and approaches for the treatment of obesity-related CRC.

Perceived Discrimination and Increased Odds of Unmet Medical Needs Among US Children

2021 ◽  
pp. 002073142199708
Author(s):  
Sze Yan Liu ◽  
Roman Pabayo ◽  
Peter Muennig
Keyword(s):  
United States ◽  
Perceived Discrimination ◽  
The United States ◽  
Exact Matching ◽  
Cross Sectional Survey ◽  
Cross Sectional ◽  
Model Estimate ◽  
Medical Needs ◽  
Us Children ◽  
Unmet Medical Needs

Our study examines the association between perceived discrimination due to race and unmet medical needs among a nationally representative sample of children in the United States. We used data from the 2016-2017 National Survey of Children's Health, a population-based cross-sectional survey of randomly selected parents or guardians in the United States. We compared results from the coarsened exact matching (CEM) method and survey-weighted logistic regression to assess the robustness of the results. Using self-reported measures from caregivers, we find that ∼2.7% of US children have experienced racial discrimination with prevalence varying significantly by race. While <1% of non-Hispanic whites have experienced some measure of racism, this increases to 8.8% among non-Hispanic blacks. Perceived discrimination was associated with significantly greater odds of unmet medical needs in the adjusted, survey-weighted multivariate-adjusted model (adjusted odds ratio [OR] = 2.4 and 95% confidence interval [CI] = 1.2, 4.9) as well as in the CEM-model estimate (OR = 2.8 and 95% CI = 1.8, 4.0). Children who have experienced perceived discrimination had higher odds of unmet medical needs. Awareness of discrimination among children may help inform future intervention development that addresses unmet medical needs during childhood.

scholarly journals Unmet Medical Needs and Future Perspectives for Leiomyosarcoma Patients—A Position Paper from the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN)

Cancers ◽  
2021 ◽  
Vol 13 (4) ◽  
pp. 886
Author(s):  
Bernd Kasper ◽  
Annie Achee ◽  
Kathrin Schuster ◽  
Roger Wilson ◽  
Gerard van Oortmerssen ◽  
...  
Keyword(s):  
Metastatic Disease ◽  
Basic Research ◽  
The United States ◽  
Position Paper ◽  
Joint Effort ◽  
Medical Needs ◽  
Systemic Treatments ◽  
Research Meeting ◽  
Unmet Medical Needs ◽  
Systemic Therapies

As leiomyosarcoma patients are challenged by the development of metastatic disease, effective systemic therapies are the cornerstone of outcome. However, the overall activity of the currently available conventional systemic treatments and the prognosis of patients with advanced or metastatic disease are still poor, making the treatment of this patient group challenging. Therefore, in a joint effort together with patient networks and organizations, namely Sarcoma Patients EuroNet (SPAEN), the international network of sarcoma patients organizations, and the National LeioMyoSarcoma Foundation (NLMSF) in the United States, we aim to summarize state-of-the-art treatments for leiomyosarcoma patients in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This position paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the NLMSF and SPAEN.

scholarly journals The Unmet Medical Needs of Correctional Populations in the United States

2012 ◽  
Vol 104 (11-12) ◽  
pp. 487-492 ◽  
Author(s):  
Karen L. Cropsey ◽  
Ingrid A. Binswanger ◽  
C. Brendan Clark ◽  
Faye S. Taxman
Keyword(s):  
United States ◽  
The United States ◽  
Medical Needs ◽  
Unmet Medical Needs

Nano-antidotes for drug overdose and poisoning

2015 ◽  
Vol 7 (290) ◽  
pp. 290ps14-290ps14 ◽  
Author(s):  
Vincent Forster ◽  
Jean-Christophe Leroux
Keyword(s):  
United States ◽  
Drug Overdose ◽  
Safety Profile ◽  
Recent Progress ◽  
The United States ◽  
Proof Of Concept ◽  
Medical Needs ◽  
Higher Doses ◽  
Unmet Medical Needs

The number of intoxications from xenobiotics—natural or synthetic foreign chemicals, or substances given in higher doses than typically present in humans—has risen tremendously in the last decade, placing poisoning as the leading external cause of death in the United States. This epidemic has fostered the development of antidotal nanomedicines, which we call “nano-antidotes,” capable of efficiently neutralizing offending compounds in situ. Although prototype nano-antidotes have shown efficacy in proof-of-concept studies, the gap to clinical translation can only be filled if issues such as the clinical relevance of intoxication models and the safety profile of nano-antidotes are properly addressed. As the unmet medical needs in resuscitative care call for better treatments, this Perspective critically reviews the recent progress in antidotal medicine and emerging nanotechnologies.

An exploratory study of intangibles risk disclosure in annual reports of banking companies from the UK, US, Germany and Italy - Some descriptive insights

2013 ◽  
pp. 81-120 ◽  
Author(s):  
Susanne Durst
Keyword(s):  
Intellectual Capital ◽  
Exploratory Study ◽  
The United States ◽  
Annual Reports ◽  
Risk Disclosure ◽  
Related Risk ◽  
Potential Benefits ◽  
Report Data ◽  
Key Drivers ◽  
The Uk

Intangibles are viewed as the key drivers in most industries, and current research shows that firms voluntarily disclose information about their investments in intangibles and their potential benefits. Yet little is known of the risks relating to such resources and the disclosures firms make about such risks. In order to obtain a more balanced and complete picture of firms' activities, information about the risky side of their intangibles is also needed. This exploratory study provides some descriptive insights into intangibles-related risk disclosure in a sample of 16 large banks from the United States (US), United Kingdom (UK), Germany and Italy. Annual report data is analyzed using the three Intellectual Capital dimensions. Study findings illustrate the variety of intangibles-related risk disclosure as demonstrated by the banks involved.

‘To see oursels as ithers see us’: Reflections on Scottish Independence from the United States

2014 ◽  
Vol 23 (3) ◽  
pp. 381-388 ◽  
Author(s):  
Euan Hague ◽  
Alan Mackie
Keyword(s):  
United States ◽  
The United States ◽  
The Us ◽  
Scottish Independence ◽  
The Uk ◽  
Scottish Diaspora

The United States media have given rather little attention to the question of the Scottish referendum despite important economic, political and military links between the US and the UK/Scotland. For some in the US a ‘no’ vote would be greeted with relief given these ties: for others, a ‘yes’ vote would be acclaimed as an underdog escaping England's imperium, a narrative clearly echoing America's own founding story. This article explores commentary in the US press and media as well as reporting evidence from on-going interviews with the Scottish diaspora in the US. It concludes that there is as complex a picture of the 2014 referendum in the United States as there is in Scotland.

Science, Politics, and the Pandemic

2020 ◽  
Vol 119 (820) ◽  
pp. 303-309
Author(s):  
J. Nicholas Ziegler
Keyword(s):  
United States ◽  
Biomedical Research ◽  
Scale Up ◽  
Political Leadership ◽  
The United States ◽  
Scientific Advice ◽  
The United Kingdom ◽  
Effective Policy ◽  
Scientific Expertise ◽  
The Uk

Comparing the virus responses in Germany, the United Kingdom, and the United States shows that in order for scientific expertise to result in effective policy, rational political leadership is required. Each of these three countries is known for advanced biomedical research, yet their experiences in the COVID-19 pandemic diverged widely. Germany’s political leadership carefully followed scientific advice and organized public–private partnerships to scale up testing, resulting in relatively low infection levels. The UK and US political responses were far more erratic and less informed by scientific advice—and proved much less effective.

The Special Perils of Being Old and Sick in Prison

2020 ◽  
Vol 32 (5) ◽  
pp. 276-284
Author(s):  
William J. Jefferson
Keyword(s):  
United States ◽  
Health Care ◽  
Medical Care ◽  
Health Care Providers ◽  
The United States ◽  
United States Constitution ◽  
Care Providers ◽  
Medical Practitioners ◽  
Medical Needs ◽  
Access To Medical Care

The United States Supreme Court declared in 1976 that deliberate indifference to the serious medical needs of prisoners constitutes the unnecessary and wanton infliction of pain…proscribed by the Eighth Amendment. It matters not whether the indifference is manifested by prison doctors in their response to the prisoner’s needs or by prison guards intentionally denying or delaying access to medical care or intentionally interfering with treatment once prescribed—adequate prisoner medical care is required by the United States Constitution. My incarceration for four years at the Oakdale Satellite Prison Camp, a chronic health care level camp, gives me the perspective to challenge the generally promoted claim of the Bureau of Federal Prisons that it provides decent medical care by competent and caring medical practitioners to chronically unhealthy elderly prisoners. The same observation, to a slightly lesser extent, could be made with respect to deficiencies in the delivery of health care to prisoners of all ages, as it is all significantly deficient in access, competencies, courtesies and treatments extended by prison health care providers at every level of care, without regard to age. However, the frailer the prisoner, the more dangerous these health care deficiencies are to his health and, therefore, I believe, warrant separate attention. This paper uses first-hand experiences of elderly prisoners to dismantle the tale that prisoner healthcare meets constitutional standards.

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